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Archive for the ‘Stem Cell Negative’ Category

Bioprinted therapeutic offers novel solution for treating Type 1 Diabetes – Canada NewsWire

Tuesday, September 22nd, 2020

Current treatments for diabetes are costlywith diabetes-related healthcare costs in Canada expected to increase to over $16.9 billion by 2020.They carry costs for patients as well, frequently in the form of negative side effects. One conventional method of treating T1D is the daily administration of insulin to manage blood sugar levels. This treatment is different from the control of a pancreatic cell, meaning patients are more likely to experience the debilitating consequences of improper glucose management. These can include damage to the eyes, nerves, kidneys, heart, and later life health complications.

To regain the benefits of working pancreatic cells, a more elegant approach is to simply replace those cells by transplanting insulin-producing pancreatic tissue into a T1D patient. However, this is not a viable long-term solution. A finite supply of donor working pancreatic tissue available to transplant, coupled with the requirement for the patient to administer life-long anti-rejection drugs that can themselves lead to significant health complications, means that an alternative is needed. An ideal solution is treating T1D with insulin made by pancreatic cells in the patient's own body, like a transplant but in a way that hides those cells from the patient's immune system so anti-rejection drugs are not needed.

In a collaborative project with Dr. Timothy Kieffer of the University of British Columbia (UBC), Vancouver-based company Aspect Biosystems is producing a bioprinted therapeutic of pancreatic cells surrounded by a protective layer that can be implanted in T1D patients. This bioprinted therapeutic will contain genetically modified stem-cell derived pancreatic cells that will take over the production of insulin and thereby regulate blood sugar levels naturally. This could serve as an effective and scalable therapy for individuals with T1D, allowing them to move away from both daily insulin injections and long-term treatment with anti-rejection drugs.

"This generous and strategic support from Genome BC allows us to strengthen our existing collaboration with Dr. Kieffer, a world leader in the development of stem cell-derived pancreatic beta cells for clinical use," said Dr. Sam Wadsworth, Chief Scientific Officer, Aspect Biosystems. "By working together, we look forward to developing a bioprinted pancreatic therapeutic that could significantly improve the quality of life for millions of people globally."

This collaboration, supported through Genome BC's GeneSolve program, represents a novel technological and genomics-based approach that will circumvent the shortcomings of existing methods. "This therapeutic design thoughtfully addresses the risks that current treatments pose to patients and incorporates genomics tools to test the system and make it safer," says Dr. Pascal Spothelfer, President and CEO, Genome BC. "It could represent a big step forward for patients and for the healthcare system."

About Genome British Columbia:

Genome BC is a not-for-profit organization supporting world-class genomics research and innovation to grow globally competitive life sciences sectors and deliver sustainable benefits for British Columbia, Canada and beyond. The organization's initiatives are improving the lives of British Columbians by advancing health care in addition to addressing environmental and natural resource challenges. In addition to scientific programming, Genome BC works to integrate genomics into society by supporting responsible research and innovation and foster an understanding and appreciation of the life sciences among educators, students and the public. http://www.genomebc.ca

About Aspect Biosystems:

Aspect Biosystems is a privately held biotechnology company combining the power of microfluidics and 3D bioprinting to fuel medical research and the development of bioprinted therapeutics. By adopting Aspect's microfluidic 3D bioprinting platform and collaborating within Aspect's network, researchers worldwide are accelerating the development and commercialization of 3D bioprinted tissues. In addition, Aspect is advancing its internal regenerative medicine programs focused on metabolic diseases and musculoskeletal injuries and disorders and partnering with key industry players to bring bioprinted therapeutics to the clinic. Learn more at http://www.aspectbiosystems.com

SOURCE Genome British Columbia

For further information: Jennifer Boon, Communications Manager, Sectors, Genome BC, Mobile: 778.327.8374, Email: [emailprotected], @genomebc #genomebc

http://www.genomebc.ca

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Oncologie Announces New Data and Analyses from Clinical Programs and Name Change to OncXerna Therapeutics – GlobeNewswire

Tuesday, September 22nd, 2020

OncXernas RNA-based biomarker platform successfully identified responders versus non-responders in trials with late-stage cancer patients

Interim results from a Phase 2 trial of bavituximab with KEYTRUDA (pembrolizumab) demonstrates a 19% overall response rate (ORR)and 43% (3/7) ORR from an exploratory analysis in a biomarker-driven subgroup of advanced gastric cancer patients

OncXernas RNA-based biomarker panel predicts enhanced response in a Phase 1b trial of navicixizumab in late-stage ovarian cancer patients. Patients in thebiomarker positive panel achieved a 70% ORR, and excludedall who had progressive disease, compared with a 31% ORR for patients in the biomarker negativepanel

WALTHAM, Mass., Sept. 18, 2020 (GLOBE NEWSWIRE) -- Oncologie, Inc., a precision medicine company using an innovative RNA-based biomarker platform to predict patient responses for potentially first-in-class targeted oncology therapies, today announced new data and analyses from its lead clinical programs, bavituximab and navicixizumab. On the basis of these positive data, the company also announced its rebranding to OncXerna Therapeutics, Inc., a change that reinforces the companys focus on using its RNA-based approach to guide novel, targeted treatments to specific people with cancer.

With a deep understanding of the tumor microenvironment biology at the RNA-level through our novel biomarker panel, we aim to dramatically improve clinical outcomes by matching patients to therapies with a mechanism of action that targets that specific biology, said Laura Benjamin, Ph.D., President and Chief Executive Officer at OncXerna Therapeutics. Todays results demonstrate a clear ability of our first panel to distinguish responders versus non-responders in our bavituximab and navicixizumab programs, and we are excited to deploy this approach in the next prospectively-defined trials that could support registration.

Interim results from Phase 2 (ONCG100) trial of bavituximab and KEYTRUDA

Trial design and background:

The Phase 2 (ONCG100) trial is a multicenter, open-label, single-arm global trial designed to assess the safety, tolerability, and antitumor activity of the investigational agent bavituximab, a chimeric monoclonal antibody that targets phosphatidylserine, in combination with KEYTRUDA, Mercks anti-PD-1 therapy, in patients with advanced gastric and gastroesophageal cancer who have progressed on or after at least one prior standard therapy. Bavituximab previously demonstrated clinical activity in a post-hoc subset analysis in patients with non-small cell lung cancer (NSCLC) who were given a PD-1 inhibitor following bavituximab treatment, suggesting that a treatment combination of bavituximab and a PD-1 inhibitor could generate similar activity in a prospective clinical trial. In addition to measuring safety and antitumor activity in this trial, OncXerna is deploying its proprietary RNA biomarker platform (TME Panel-1) to identify patients based on their response to treatment and the dominant biology of their tumor microenvironment with the potential to dramatically improve outcomes in the next, prospectively designed trial.

Approximately 80 patients in the U.S., United Kingdom, South Korea and Taiwan are planned for enrollment in two separate groups of patients: Checkpoint inhibitor-nave and checkpoint inhibitor-relapsed. The trial is continuing to enroll both groups with planned updates from all patients during the first half of 2021.

Interim results:

Interim results provided today, from the first 36 patients enrolled and with a post-baseline scan in the checkpoint inhibitor-nave group, include the following:

Next steps:

These data are being presented at the European Society for Molecular Oncology (ESMO) Virtual Congress 2020 taking place September 19-21, 2020.

OncXerna plans to conduct additional clinical trials designed to prospectively enrich for TME Panel-1 biomarker positive patients, as well as to explore additional solid tumor types.

OncXerna biomarker analysis from Phase 1b trial evaluating navicixizumab in ovarian cancer

Previously announced data and background:

OncXernas navicixizumab is a bispecific antibody designed to inhibit both Delta-like ligand 4 (DLL4) in the Notch cancer stem cell pathway as well as vascular endothelial growth factor (VEGF). Interim data from a Phase 1b dose escalation and expansion trial of navicixizumab plus paclitaxel in 44 platinum-resistant ovarian cancer patients who had failed more than two prior therapies and/or received prior Avastin (bevacizumab) therapy were presented virtually at the 2020 Society of Gynecologic Oncology (SGO) Annual Meeting in May 2020. Treatment with navicixizumab and paclitaxel demonstrated an ORR of 43%in all patients, and 64% and 33% in bevacizumab-nave, and bevacizumab pre-treated patients, respectively. Treatment-related adverse events were manageable and included hypertension (58%), headache (29%), fatigue (26%) and pulmonary hypertension (18%).

Updated biomarker analyses and results:

Using its RNA-based biomarker TME Panel-1, OncXerna recently analyzed patient tissue samples obtained from 28 of the 44 patients from the Phase 1b trial. Results from this analysis revealed the following:

Next steps:

As a result of these analyses, OncXerna plans to conduct additional clinical trials designed to prospectively enrich for TME Panel-1 biomarker positive patients with ovarian cancer who are platinum-resistant and Avastin-experienced to support registration, as well as to explore additional solid tumor types.

About Bavituximab

Bavituximab is an investigational antibody that reverses immune suppression by inhibiting phosphatidylserine (PS) signaling and is currently in Phase 2 clinical trials to treat a specific subset of patients with advanced gastric cancer to improve their response to anti-PD-1 treatment. The mechanism of action of bavituximab is to block tumor immune suppression signaling from PS to multiple immune cell receptor families (e.g., TIMs and TAMs). The dominant biology targeted by bavituximab may be relevant for patients with many types of solid tumors whose immune systems are too suppressed to benefit from currently available immune oncology therapies. Our clinical trials currently combine bavituximab with KEYTRUDA to test the hypothesis that relieving immunosuppression can enhance responses to checkpoint inhibitors. Bavituximab is an investigational agent that has not been licensed or approved anywhere globally, and it has not been demonstrated to be safe or effective for any use, including for the treatment of advanced gastric cancer.

About Navicixizumab

Navicixizumab is an investigational anti-DLL4/VEGF bispecific antibody that has demonstrated antitumor activity in patients who have progressed on Avastin (bevacizumab) in a Phase 1a/b clinical trial. The U.S. Food and Drug Administration granted Fast Track designation to navicixizumab for the treatment of high-grade ovarian, primary peritoneal or fallopian tube cancer in patients who have received at least three prior therapies and/or prior treatment with Avastin. OncXerna is targeting patients whose dominant tumor biology is driven by angiogenesis with a focus beyond VEGF to include broader anti-angiogenic pathways. Navicixizumab is an investigational agent that has not been licensed or approved anywhere globally, and it has not been demonstrated to be safe or effective for any use, including for the treatment of advanced ovarian cancer.

About OncXerna Therapeutics

OncXerna is aiming to deliver next-generation precision medicine for a larger group of cancer patients by leveraging the companys deep understanding of how to prospectively identify patients based on the dominant, RNA-based biology of their tumor microenvironments. This allows OncXerna to pair those patients with OncXernas clinical-stage therapies and known mechanism of action that directly address these biologies, to dramatically improve patient outcomes. For more information on OncXerna, please visit oncxerna.com/

About OncXernas RNA-based Biomarker Platform

Existing precision medicines target only approximately 10% of cancersthose with gene mutations or oncogenic drivers for a small number of genes. Using its proprietary biomarker platform, OncXerna is leveraging the companys deep understanding of tumor biology at the RNA level to identify the dominant biology underlying a patients cancer. OncXernas first biomarker panel is specific to the tumor microenvironment (TME Panel-1). Initial results from TME Panel-1 reveal 4 different dominant biologies, demonstrating the presence of specific patient subgroups and their predictive value in responding to treatment. OncXerna is further optimizing the biomarker platforms tumor microenvironment panel through multiple research collaborations, including a collaboration with Moffitt Cancer Center.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Investor and Media Contact:

Ashley R. RobinsonLifeSci Partners, LLCarr@lifesciadvisors.com

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How the single product brand trend could be an environmental home run for the beauty industry – GlobalCosmeticsNews

Tuesday, September 22nd, 2020

As beauty professionals, Id hazard a guess that were all well acquainted, and more than a little embarrassed, about our industrys negative association with environmental issues. As one of the worlds most prolific offenders of plastic waste, we are, despite ongoing and progressive initiatives and packaging developments, one of the worst culprits of single-use products destined for landfill.

So, what about the effect of the increasing popularity of single-product beauty launches? Rather than single use, although they are creeping into the mix, the recent upsurge in single product skincare regimes is becoming a popular marketing tactic for brands and also creating a new breed of skincare founders capitalizing on the popularity of the new trend. In terms of skincare, it seems that maximalism is out, and minimalism is in.

Augustinus Bader kickstarted the movement with the launch of his stem cell moisturizer, The Cream, last year. The launch came without bells and whistles, no add on items and the range was notable for its minimalist offering just one product. The skincare guru has seemingly paved the way. Founders of cult beauty brand Summer Fridays got into game in 2018 with one product the Jet Lag Mask (although I wonder how thats faring in the current climate). While theyve since expanded to a core product range of six, the mask was the hero, and only, sell-out offering for some time.

But what effect does this new skincare approach have in terms of sustainability in comparison to its multi-step predecessor? The more is more, previously much-loved, approach to skincare promotes shelves packed with products aimed at 12-step programs to create perfect skin. This generates, to put it simply, a mass of trash. Multiple bottles of what are likely potions and lotions more a marketers dream, and an environmentalists nightmare, than a skin care holy grail. But would we use less of each bottle, and therefore limit the turnover of throw-away packaging?

Meanwhile, while the latest en vogue trend on the block, single product skincare, has been lauded as a minimalist approach to perfect skin. While some could argue that using one product will promote a much higher usage, therefore a faster rotation of treasure to trash, if having to choose between the two youd obviously lean towards the manufacturing of fewer bottles, tubes and, ultimately,waste. Less is more, as they say. And with the nature of the shopper being to increasingly seek out the new, consumers across all target market groups baby boomers to gen z are also desperately searching for efficacious products that also fulfil their desire to be a green buyer.

Craigs Resurfacing Compoundseems to have hit the nail on the head. Sold out in 48 hours, the serum was two-years in the making and is said to cut beauty regimes in half with its tantalizing mix of ingredients she pulled out the big guns; retinol, antioxidants, glycolic acid and lactic acids it also comes encased in a full recyclable bottle.

With COVID-19 teaching the world to slow down and take a breath, the same could be said for our skincare routines. As stated by Vogue, a more considered approach to beauty is both needed and being lapped up by consumers. The upsurge in single-product launches is seemingly capitalizing on a desire for less is more and causing a marketing furore to boot. Less production, less waste, and, put simply, a swift U-turn from the maximalist approach of old. Get the packaging right recycledandrecyclable if you please and the industry could be on to a winner both commercially and environmentally.

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$14.6M Grant to Explore a Therapy to Control HIV Without Meds – POZ

Tuesday, September 15th, 2020

In nearly 40 years of the HIV epidemic, only two people have likely been cured of the virus. Both scenarios resulted from stem cell transplants needed to fight blood cancers such as leukemia. Inspired by these two cases, a team of scientists is studying a multipronged way to potentially control HIV without medication. It involves two different genetic alterations of immune cells and with a safer method of stem cell transplants, also referred to as bone marrow transplants, a procedure that is generally toxic and dangerous.

The research is being funded by a five-year $14.6 million grant from the National Institutes of Health. The scientists coleading the preclinical studies are Paula Cannon, PhD, a distinguished professor of molecular microbiology and immunology at the Keck School of Medicine of the University of Southern California, and Hans-Peter Kiem, MD, PhD, who directs the stem cell and gene therapy program at the Fred Hutchinson Cancer Research Center, also known as Fred Hutch. According to a Keck School of Medicine press release, the two other main partners are David Scadden, MD, a bone marrow transplant specialist and professor at Harvard University and the Harvard Stem Cell Institute, and the biotechnology company Magenta Therapeutics.

In the HIV cure scenariosinvolving the so-called Berlin and London patientsboth men received stem cell transplants from donors with a natural genetic mutation that made them resistant to HIV. Specifically, their genes resulted in immune cells that lack CCR5 receptors on their surface (HIV latches onto these receptors to infect cells). Unfortunately, this method isnt viable for the nearly 38 million people worldwide living with HIV. Not only is it expensive, toxic and riskyit involves wiping out the patients immune system and replacing it with the new immune cellsbut it also requires matched donors who are CCR5 negative. According to the press release, about 1% of the population have this mutation.

With funding from this new grant, researchers hope to overcome these challenges in several ways. First, Cannon has already developed a gene-editing method to remove the CCR5 receptors from a patients own stem cells. She now hopes to further genetically engineer stem cells so they release antibodies that block HIV.

Our engineered cells will be good neighbors, Cannon said in the press release. They secrete these protective molecules so that other cells, even if they arent engineered to be CCR5 negative, have some chance of being protected.

Fred Hutchs Kiem will use CAR-T therapya new method of genetically modifying immune cells that is emerging out of cancer researchwith the goal of creating T cells that attack HIV-infected cells.

In addition, other scientists involved in the federal grant aim to develop less toxic methods of bone marrow transplantationfor example, by reducing the amount of chemotherapy required and speeding up the process of creating the new immune system.

The research finding could translate to other illnesses, such as cancer, sickle cell anemia and autoimmune disorders.

A home run would be that we completely cure people of HIV, Cannon said. What Id be fine with is the idea that somebody no longer needs to take anti-HIV drugs every day because their immune system is keeping the virus under control so that it no longer causes health problems and, importantly, they cant transmit it to anybody else.

For the latest on the cure cases, see Famed London Man Probably Cured of HIV from earlier this year. And in related news, see $14M Federal Grant to Research CAR-T Gene Therapy to Cure HIV.

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Takeda opens cell therapy manufacturing facility tucked right in its Boston R&D hub – Endpoints News

Tuesday, September 15th, 2020

When Takeda unveiled its translational cell therapy engine early last year, the Japanese pharma made it clear the crew, under Novartis vet Stefan Wildt, is going all the way: clinical expertise, bioengineering chops, world-class collaborations, plus chemistry, manufacturing and control.

The final piece of the puzzle has now fallen in place, with the opening of a 24,000 square-foot cell therapy manufacturing facility at its R&D headquarters in Boston.

In that early space, having it situated in proximity to our teams is quite powerful, Chris Arendt, head of the oncology therapeutic area unit, told Endpoints News. When you think about it, the process defines very much the medicine and the cell therapy space

Designed to produce clinical-grade material from discovery through pivotal Phase IIb trials, the site will support five ongoing pacts. They include pluripotent stem cell work with Kyoto University Nobel laureate Shinya Yamanaka, gamma delta T cell research with Adrian Hayday and his biotech, armored CAR-Ts with Koji Tamada at Noile-Immune Biotech, next-gen CARs with Memorial Sloan Ketterings Michel Sadelain, and finally CAR-NK with Katy Rezvani at MD Anderson.

With three programs now in the clinic, Takeda is now picking two more to test in humans in 2021, Arendt said. While each research partnership has taken on its own bespoke approach to manufacturing up to now, the new facility will provide a central spot to lock down the process development as close to the final product as possible.

At the forefront are TAK-007, an allogeneic CD19-targeted CAR-NK being tested in Phase I/II for relapsed or refractory non-Hodgkins lymphoma; TAK-940, 19(T2)28z1xx CAR-T cells featuring a next-gen signaling domain from MSK; and TAK-102, a cytokine and chemokine armored CAR-T directed at GPC3-expressing previously treated solid tumors. The latter two are in first-in-human trials.

Having a dedicated facility scales the operations up so that the team can simultaneously advance multiple programs, he added.

Before the Covid-19 pandemic sucked out all the oxygen in the room, the booming cell therapy markets demand for physical infrastructure captured considerable attention. Gileads Kite constructed its own viral vector manufacturing center in order to leave no stone unturned. Contract manufacturers like Catalent were snapping up space, and even Deerfield got into the game with a splashy, $1.1 billion entrance.

The way hes built the team now grown to well over 150 scientists the learnings from any one program can be quickly applied to the whole portfolio, Wildt noted.

We wanted to place the engine team at that sweet spot between late-stage discovery and rapidly putting forward innovative ideas and concepts into clinical translation, he said. It was just a concept a few years ago. And now we can partner with hospitals and patients and really see hopefully we can be successful on their behalf.

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Robots can now store energy like we store fat, and they dont even need to eat pizza – SYFY WIRE

Tuesday, September 8th, 2020

More and more robo-tech is evolving in an ironic turn that goes against many of the cold metal worlds in science fiction, where flesh has been taken over by machine. Maybe the most unexpected biomimetic literally mimicking biological creatures technology is a robot battery that stores energy like fat.

Sorry, Baymax, but robots will actually lose weight with a new "fat battery" (tech throughout history has often shed bulk with each upgrade).University of Michigan professor Nicholas Kotov a professor of chemical engineering, materials science and engineering, and macromolecular science and engineering has developed a zinc battery that could really level up the function of everything from nurse and server droids to delivery drones to micro- and nanobots. Smaller but much more energy-dense than the typical lithium ion battery, it can power a robot just as biological fat reserves can keep us going.

Such multifunctional batteries require mechanical strength, toughness, and stiffness.They should also be resilient against delamination, Kotov, who recently co-authored a study published in Science Robotics, told SYFY WIRE. Combining high-energy density with new mechanical properties was made possibleby designing new material for an ion-conducting membrane separating cathode and anode.This is how the batteries acquire these mechanical properties.The membrane between them was also designed with inspiration from biology; its nanofibrous structure is nearly identical to cartilage.

Kotov and his team demonstrated the power of these batteries (below) by creating a worm that resembles a robo-maggot and an epic scorpion that could possibly take down Scorponok in a Transformers death match.

Lithium ion batteries have nothing on these. Hydroxide ions passed on by a zinc electrode at the cathode (positive end) travel through that artificial cartilage to reach the anode (negative end) and transfer to the next battery. They are inflammable, unlike the electrolytes that can catch fire in lithium ion batteries. Aramid nanofibers in the cartilage are extraordinarily tough and can actually be upcycled from used body armor, while the gel component of it is water-based. Never mind that the battery itself is mostly nontoxic and costs much less to manufacture than that lithium ion one in your smartphone.

Kotov heads a lab with a focus on developing biomimetic nanostructures, and his research team wanted to figure out a more efficient way of storing energy in robots. Bots can be held back by the weight, cost, and overall restrictiveness of the usual batteries. Living organisms store energy very differently from batteries. Humans and other creatures have fat cells distributed all over the body, holding on to reserves of lipids that are burned when there is not enough energy intake to sustain them. There is no fat storage sac, which is basically what a battery is to a robot. The biomorphic batteries developed in Kotovs lab translate biology into technology.

Fat gets too much of a negative rap. Besides being an instant energy reserve, it protects internal organs, influences hormone and stem cell production, and functions as built-in thermal insulation.

These biological concepts can be put in the foundation of the batteries for robots, said Kotov. This is exactly what we have done in this project.We distributed the batteries over the entire body of the robot and made them multifunctional.Besides storing energy, our batteries also protect robot organs.

The only downside of the battery is that it can only keep up high capacity for about 100 cycles, compared to a lithium ion batterys average of 500. Zinc is the culprit because it ends up forming spikes that mean doom for the artificial cartilage membrane and the life of the battery. At least there should be no problem recycling and replacing the materials. Eventually, fat batteries could replace the entire exterior of a robot for an unreal 72 times more power capacity than one lithium ion battery. But would that impact the size of a robot? And with that, where it would be able to go and what tasks it would be capable of? Not according to Kotov.

Robots with biomorphic fat-like batteries do not need to be enormous, he said. In fact they are slimmer, lighter, and more agile than the original ones because we removed the bulky cumbersome part.The potential drawback is that this energy storage becomes more complex, but is the tradeoff for greater efficiency and range.

So these robots are more likely to deliver your pizza than eat it, and they wont even let it get cold.

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Magenta Therapeutics Named Co-Recipient of Grant from the National Institutes of Health to Explore Use of Novel Targeted Conditioning Agents with Gene…

Tuesday, September 8th, 2020

NIH grant funds an interdisciplinary effort among researchers from University of Southern California; University of Washington and Fred Hutchinson Cancer Research Center; Harvard University and Massachusetts General Hospital; the Ragon Institute; and Magenta Therapeutics

Magenta will utilize its tool CD45 and CD117 antibody-drug conjugate (ADC) conditioning agents, as well as its stem cell biology platform to identify the optimal strategy for curative immune system transplant in patients with HIV

Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today announced it is part of a multi-project, broad-based research effort awarded a five-year, $14.6 million U19 grant from the National Institutes of Health (NIH) to explore gene- and cell-based approaches to advance research into curing HIV.

This cross-institutional research program brings together leaders in the fields of gene editing, HIV and stem cell transplant. The team, which includes researchers from the University of Southern California, the University of Washington, the Fred Hutchinson Cancer Research Center, Harvard University, Massachusetts General Hospital; the Ragon Institute and Magenta Therapeutics, will explore novel hematopoietic stem and progenitor cell (HSPC) engineering and transplantation approaches aimed at achieving complete remission of HIV-1 infection.

"We are excited to collaborate with our colleagues in this important multi-institution research team to help advance gene editing approaches with our novel targeted antibody drug conjugate (ADC) conditioning platform to one day be able to cure patients living with HIV," said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta. "These studies leverage our proprietary stem cell biology pipeline and ADC platform to provide important insights into which conditioning strategy is best suited to aim for HIV."

Story continues

Magenta will utilize its conditioning technology to optimize cell dose in animal models and determine whether targeted conditioning and gene-modified HSPC transplant enables disease control.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "will," "could", "should," "expects," "intends," "plans," "anticipates," "believes," "estimates," "predicts," "projects," "seeks," "endeavor," "potential," "continue" or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption "Risk Factors" in Magentas Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200902005248/en/

Contacts

Magenta Therapeutics: Lyndsey Scull, Director, Corporate Communications202-213-7086lscull@magentatx.com

Dan Budwick1ABdan@1abmedia.com

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Sterile Filtration Market To Reach USD 8.48 Billion By 2027 | CAGR: 7.7% | Reports And Data – PRNewswire

Friday, August 28th, 2020

NEW YORK, Aug. 27, 2020 /PRNewswire/ --The GlobalSterile Filtration Marketis expected to reach USD 8.48 Billion by 2027, according to a new report by Reports and Data. Sterile filtration finds usage in the removal of contaminants and particulates from fluids comprising media with or without buffers, serum, reagents, biologic or proteinaceous samples, or other types of fluids. Filtration through a pore size of 0.2 m is essential to get a sterile filtrate by filtering particles and germs from fluids (liquids and gases) to prevent them from contaminating the end-products. As per the GMP guidelines and the guidelines by the (FDA), producers are required to perform a filter integrity test at the pre and post-production cycle. The test confirms that the filter is completely functional and that no undesirable components got through it.

Biopharmaceuticals products normally cannot be terminally sterilized, and thus it is crucial to use sterile grade filters in aseptic processing. Application of heat sterilization or any other process in biopharmaceutical drug products results in unwanted degradation of the product. Sterilizing membrane filtration frequently necessitated reducing the levels of bioburden within process streams to prevent the potential formation of biofilm. Further, to ascertain that the sterile filtered products uphold the pure form, a growing number of firms, especially the firms in the pharmaceutical sector, are deploying disposable process solutions to store or process the subsequent filtrate.

Request free sample of this research report at: https://www.reportsanddata.com/sample-enquiry-form/3464

The growing use of sterile filtration in the food & beverage industry, especially in breweries, is playing an instrumental role in driving market growth. Recent researches uphold the use of sterile filtration as the most appropriate method for brewers for controlling microbial hazards. Even though beer is alcoholic, acidic, anaerobic, and comprises hop compounds that ply the role of preservatives, certain microorganisms can survive in the chemical environment and thrive on rich nutrients present in beer. These kinds of microorganisms may result in beer spoilage forming a haze or sedimentation, a rancid/sour flavor, and over-carbonation, thus requiring the need for sterile filtration.

COVID-19 Impact Analysis

As global economies are experiencing the negative impact of the Covid-19 pandemic, organizations are suffering losses, among various other challenges. Nevertheless, firms in the pharmaceutical industry are of immense importance in combatting the pandemic and are witnessing positive growth in the contagious disease landscape with the race for treatment approval therapy gaining momentum.

Biopharmaceutical companies are playing a significant role in human response to the COVID-19 pandemic. Various leading biotech companies are studying the genome to prepare a feasible vaccine for its treatment. Growing investments in R&D activities for making the vaccine are fuelling the growth of the sterile filtration market.

To identify the key trends in the industry, click on the link below: https://www.reportsanddata.com/report-detail/sterile-filtration-market

Further key findings from the report suggest

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For the purpose of this report, Reports and Data has segmented the Global Sterile Filtration Market on the basis of type, membrane type, application, end-user, and region:

TypeOutlook (Revenue, USD Million; 2017-2027)

Membrane TypeOutlook (Revenue, USD Million; 2017-2027)

ApplicationOutlook (Revenue, USD Million; 2017-2027)

End-UserOutlook (Revenue, USD Million; 2017-2027)

Regional Outlook (Revenue, USD Million;2017-2027)

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Laboratory Filtration Market- Filtration is a technique that is used to separate solids from liquids or solution by interposing a filter medium through which solutions or liquids can pass.

Virus Filtration Market - increasing emphasis and growing investment in R&D activities in the biotechnology sector, there has been an elevated demand for virus filtration.

Gene Expression Market - Gene expression is the method that refers to the process of measuring the activity of genes in order to comprehend the cellular functions.

In vivo CRO Market - Shifting of preference of pharmaceutical industries toward the outsourcing clinical and preclinical trials to focus on their core business, increasing frequency of outsourcing R&D activities.

Protein Engineering Market- Protein engineering is an emerging field that involves synthesis of new proteins as well as amendment in the existing protein structures that ultimately helps to achieve desired functions.

3D Cell Culture Market- The growth is mainly contributed by the government and non-government investments for cancer research & development, coupled with large scale end users for stem cell research.

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Sterile Filtration Market To Reach USD 8.48 Billion By 2027 | CAGR: 7.7% | Reports And Data - PRNewswire

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Burger Reviews BTK Inhibitors and Beyond in Frontline CLL – Targeted Oncology

Friday, August 28th, 2020

During a virtual Case Based Peer Perspectives event, Jan A. Burger, MD, PhD, professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center in Houston, TX, discussed testing and the treatment options for chronic lymphocytic leukemia (CLL), based on the a case of 71-year-old female patient.

Targeted OncologyTM: What testing would you order to confirm diagnosis if you saw this patient in the clinic?

BURGER: We need to establish the diagnosis by flow cytometry and then we would do, at a minimum, FISH cytogenetics and, ideally, the mutational status. Cytogenetics can change, butmutational status usually doesnt change. If thats been established somewhere outside [of your clinic], then you dont have to repeat that test.

Its important to repeat cytogenetics if you talk about the relapse setting. But here, were treating in the frontline setting, and she was tested. She was found to be IGHV unmutated and [positivefor] del(11q). That, traditionally, has been regarded as a higher-risk disease status because these patients respond OK to standard chemotherapy, but they have short remissions and survival times with FCR [fludarabine, cyclophosphamide, and rituximab (Rituxan)], BR [bendamustine plus rituximab], and those kinds of regimens compared with low-risk patients, such as those [who are positive for] deletion 13q and have IGHV mutated disease.

In terms of these sequences, when you see a patient with lymphocytosis, you send for flow cytometry, and part of the flow cytometry panel can test for additional markers, CD38 and ZAP-70. We have it [at MD Anderson], but Im not sure if there are any outside routine flow cytometry labs reporting CD38 positivity or negativity or ZAP-70. These markers used to be very popular 10years or so ago when IGHV-mutation status was not so commonly done and was more complicated to get. Nowadays, theres a shift with sending a sample directly for IGHV-mutation testing.

If you have that and the routine CLL FISH panel, then you have a good amount of information about your patient to say this is high-, low-, or an intermediate-risk disease. I think the main purpose for doing these is, first, to identify [patients with] high-risk disease who have a deletion 17p [del(17p)] or TP53 mutations. If its a young patient, you want to know that to [be able to] avoid chemotherapy. If its a young patient, [you may want to] send those patients for evaluation for stem cell transplant. For some patients, that is still something that eventually needs to be considered for those with del(17p).

What systemic therapy would you offer?

If you have treated with ibrutinib [Imbruvica] and youre comfortable with that, I dont think at this time there is a strong reason to change. In selected patients, it might be reasonable to tryswitching them from one [agent] to the other. But right now, for this patient, consensus says a BTK [Bruton tyrosine kinase] inhibitor is a good treatment.

Both ibrutinib and acalabrutinib [Calquence] can be used as single agents or in combination with CD20 antibodies. Weve done a clinical study with ibrutinib where patients were randomized to receive monotherapy versus a combination with rituximab, and the outcome was virtually identicalwhere patients had the exact same progression-free survival [PFS] with a single agentversus the combination with a CD20 antibody.1

CD20 antibodies with BTK inhibitors dont seem to add benefit in terms of survival if you go with the long-term BTK inhibitor treatment and if youre not planning to stop your treatment atsome point. What they do is they get patients into remission faster and you clear the disease faster if you add a CD20 antibody, but then you stop after 6 months. You continue your BTKinhibitor, and patients do great 2, 3, and 4 years later. Then, you dont see any effect in terms of longer-term PFS or overall survival [OS] from the addition of the CD20 antibody.

What data support the use of single-agent ibrutinib in patients with untreated CLL?

There are data from the RESONATE-2 study [NCT01722487], which randomized patients between ibrutinib and chlorambucil. This study was designed at the time when chlorambucil monotherapy was still the standard of care. Patients were randomized 1:1, and patients with del(17p) were excluded.2

What is nice about this study is that we have a long follow-up now.3 At the 5-year follow-up, you see this major difference in terms of PFS [HR, 0.146; 95% CI, 0.098-0.218]. There is also anoverall survival benefit [HR, 0.450; 95% CI, 0.266-0.761].

What [we saw was] that patients with del(11q) seemed to have a better PFS than those patients who lack del(11q) when they are treated with ibrutinib. Patients with del(11q) who are treated with chemotherapy do not do as well as those who lack this cytogenetic abnormality. The same is true here for [IGHV] mutational status.

The PCYC-1102-CA study [NCT01105247] opened around 2010, and we now have 7 to 8 years of follow-up. If you use a BTK inhibitor in the frontline setting, you can expect that most patients are going to do well for an extended period of time. At 5, 6, and 7 years or longer, 70% to 80% of patients are still in remission and have not died.4

Another randomized study that created some waves [is the E1912 study (NCT02048813)]. Weve been big proponents of FCR, which was the comparator arm [of this trial] versus ibrutinib. Patients receive either 6 cycles FCR or continuous ibrutinib [with rituximab] for the first 6 cycles.5

That study showed that compared with FCR, there was a significant increase in PFS [HR, 0.39; 95% CI, 0.26-0.57; P <.0001] but also in OS benefit from the BTK inhibitorcontaining regimen[HR, 0.34; 95% CI, 0.15-0.79; P = .009].

Would you say ibrutinib is the standard of care for treatment of CLL in the frontline setting?

Ibrutinib monotherapy, I would say, is the standard of care, but ibrutinib plus rituximab can be used. Some of you use it and, based on the data we just saw, the FDA has now officiallyapproved it.6 It doesnt mean you must use rituximab.

What other ibrutinib combinations are available?

The ALLIANCE trial [NCT01886872] had a single-agent ibrutinib arm versus ibrutinib plus rituximab versus bendamustine plus rituximab.7 When you have patients randomized to receive ibrutinib/rituximab versus ibrutinib as a single agent, the [Kaplan-Meier survival] curves are basically identical, and thats what we got as well in a slightly diff erent patient population, mostlyrelapsed patients. In terms of PFS, rituximab doesnt seem to add very much when you go with continuous ibrutinib treatment. You see the difference for bendamustine/rituximab, with whichpatients have significantly shorter PFS.

I think the theme is the same over and over again with these randomized studies. With the new targeted agents, such as the BTK inhibitors and venetoclax [Venclexta], we see the samepattern. The new agents are doing better than our traditional chemoimmunotherapy.

ILLUMINATE [NCT02264574] is the study comparing ibrutinib/obinutuzumab [Gazyva] with another chemoimmunotherapy regimen, which has been somewhat popular for older populations, more frail patients for whom you dont want to use FCR or BR. You traditionally use chlorambucil alone and then more recentlyits combined with CD20 antibodies. The patients were randomized to either [ibrutinib/obinutuzumab] versus chlorambucil/obinutuzumab treatment.8

The results show a major PFS benefits for patients on the BTK inhibitor [HR, 0.23; 95% CI, 0.15-0.37; P < .0001]. There was a big difference for genetically high-risk patients [HR, 0.15; P <.0001] or patients who had bulky disease.

What other BTK inhibitors would you consider here?

Now were going to the second-generation BTK inhibitor, acalabrutinib [Calquence], which is somewhat more selective and doesnt inhibit some other kinases that ibrutinib does. Its a newBTK inhibitor with not as much long-term follow-up data available.

[In the phase 3 ELEVATE TN trial (NCT02475681)], you have 3 arms: single-agent acalabrutinib, acalabrutinib combined with obinutuzumab, and the comparator arm of chlorambucil/obinutuzumab. 9 If you give that to treatment-nave patients, those receiving BTK inhibitor alone or with the CD20 antibody do well. Its debatable if the PFS difference is significant, but clearly, the BTK inhibitortreated patients do much better than those receivingchlorambucil plus obinutuzumab.

[If you look at the] subgroups of patients benefitting from the BTK inhibitor treatment versus obinutuzumab/chlorambucil, it basically shows that all subgroups have benefit. Some may be alittle more than others...but I think particularly patients that we traditionally called high risk are the ones who benefit the most from new agents. Theres less difference if you go into the lowriskpatient populations.

Are there data supporting the use of a BCL2 inhibition?

The other frontline option involves venetoclax, and thats coming from this CLL14 trial [NCT02242942]. Patients were receiving venetoclax/obinutuzumab or chlorambucil/obinutuzumab, and this is a finite treatment for 12 months. These are patients who were older and who have some comorbidities. Deletion(17p) was not excluded.10

There is a major difference in PFS favoring the new targeted agent venetoclax. Now its approved for the frontline treatment of selected patients,11 but you can also see in comparison to theBTK inhibitors [that] the follow-up is relatively short of 3 years.

With venetoclax, you get more complete remissions and some of these remissions are MRD [minimal residual disease] negative. As long as these differences are not translating into a survival benefit, those are just numbers.

Would you recommend venetoclax after the first line?

I dont think theres a reason to make that change [from BTK inhibitors] because venetoclax has its own issues in terms of how its used and adverse effects [AEs]. For that questionmaybe [we ask [is] venetoclax better in terms of outcome than a BTK inhibitor?

Its difficult to be better than the BTK inhibitor in the frontline CLL setting, and you need a very long follow-up to show any differences if there are any.

A substantial number of patients [treated with venetoclax] receive MRD-negative remissions with this combination. MRD negativity doesnt mean patients are cured. There is drop off in PFS, so MRD negativity doesnt mean those patients will survive and never need treatment again. Most likely, those patients eventually will lose MRD and eventually have disease progression and need treatment again. I think for those studies based on frontline venetoclax for 12 months, we just have to stay tuned and wait for what the long-term outcome is going to be.

What are the AEs of venetoclax?

You see more AEs that are reminiscent of chemotherapy days, where patients get more cytopenia. Its well established that venetoclax is myelosuppressive. Certainly, neutropenia can be seen, and less frequently, thrombocytopenia and anemia. If you treat a patient with venetoclax with or without a CD20 antibody, then you have to prepare for some patients having issueswith neutropenia and some who cannot be fully dose-escalated because of those cytopenias.

If the patient was younger, would you treat differently?

My answer would be no. I dont see any difference. This patient was 71 years old. We wouldnt use chemoimmunotherapy.

Somebody voted no. I think thats interesting because its something Im interested in [finding out about]. Im wondering if we have to accept treating patients with BTK inhibitorsfor very long periods or if we can maybe try it at least as an alternative treatment just for a certain period of time until we have the best response. Then, some patients maybe stop. I think thats interesting for a clinical trial.

Outside of clinical trials, Im not so sure. We have no data. But if you have a low-risk patient and you want to stop after 2 years and just see what happens, you need to tell the patient we dontknow whats going to happen and you have to watch that patient more closely. If its a patient with del(17p), a high-risk patient who was very symptomatic, I wouldnt do that. But in low-risk patients, I think its an interesting question and not totally unreasonable.

Over time, we will find new solutions. Everybodys working on transitioning BTK inhibitorsto limited-duration treatments for many reasons. Its not the optimal situation to have patients onkinase inhibitors for 5, 10, or 20 years. Right now, its a long-term treatment until we have better treatments.

References:

1. Burger JA, Sivina M, Jain N, et al. Randomized trial of ibrutinib vs ibrutinib plus rituximab in patients with chronic lymphocytic leukemia. Blood. 2019;133(10):1011-1019. doi:10.1182/blood-2018-10-879429

2. Burger JA, Tedeschi A, Barr PM, et al. Ibrutinib as initial therapy for patients with chronic lymphocytic leukemia. N Engl J Med. 2015;373(25):2425-2437. doi:10.1056/NEJMoa1509388

3. Burger JA, Barr PM, Robak T, et al. Long-term effi cacy and safety of fi rst-line ibrutinib treatment for patients with CLL/SLL: 5 years of follow-up from the phase 3 RESONATE-2 study. Leukemia. 2020;34(3):787-798. doi:10.1038/s41375-019-0602-x

4. Byrd JC, Furman RR, Coutre SE, et al. Ibrutinib treatment for fi rst-line and relapsed/ refractory chronic lymphocytic leukemia: fi nal analysis of the pivotal phase Ib/II PCYC- 1102 study. Clin Cancer Res. Published online March 24, 2020. doi:10.1158/1078-0432.CCR-19-2856

5. Shanafelt TD, Wang XV, Kay NE, et al. Ibrutinib and rituximab provides superior clinical outcome compared to FCR in younger patients with chronic lymphocytic leukemia (CLL): extended follow-up from the E1912 Trial. Blood. 2019;134(suppl 1):33. doi:10.1182/blood-2019-126824

6. FDA approves ibrutinib plus rituximab for chronic lymphocytic leukemia. News release. FDA. April 21, 2020. Accessed July 27, 2020. https://bit.ly/3jV1hGW

7. Woyach JA, Ruppert AS, Heerema NA, et al. Ibrutinib regimens versus chemoimmunotherapy in older patients with untreated CLL. N Engl J Med. 2018;379(26):2517-2528. doi:10.1056/NEJMoa1812836

8. Moreno C, Greil R, Demirkan F, et al. Ibrutinib plus obinutuzumab versus chlorambucil plus obinutuzumab in fi rst-line treatment of chronic lymphocytic leukaemia (iLLUMINATE): a multicentre, randomised, open-label, phase 3 trial. Lancet Oncol. 2019;20(1):43-56. Published correction appears in Lancet Oncol. 2019;20(1):e10.doi:10.1016/S1470-2045(18)30788-5

9. Sharman JP, Egyed M, Jurczak W, et al. Acalabrutinib with or without obinutuzumab versus chlorambucil and obinutuzmab for treatment-naive chronic lymphocytic leukaemia (ELEVATE TN): a randomised, controlled, phase 3 trial. Lancet. 2020;395(10232):1278-1291. Published correction appears in Lancet. 2020;395(10238):1694. doi:10.1016/S0140-6736(20)30262-2

10. Fischer K, Al-Sawaf O, Bahlo J, et al. Venetoclax and obinutuzumab in patients with CLL and coexisting conditions. N Engl J Med. 2019;380(23):2225-2236. doi:10.1056/NEJMoa1815281

11. FDA approves venetoclax for CLL and SLL. News release. FDA. May 15, 2019.Accessed July 27, 2020. https://bit.ly/3jLnEOU

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CytoDyn Reaches Enrollment of 195 Patients in its Phase 3 Trial for COVID-19 Patients with Severe-to-Critical Symptoms – GlobeNewswire

Friday, August 28th, 2020

Interim analysis to commence after 28 days; results anticipated by mid-October

VANCOUVER, Washington, Aug. 25, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today the Company has reached the requisite number of enrolled patients in its Phase 3 trial for COVID-19 patients with severe-to-critical symptoms to perform an interim analysis following the 28-day phase of the trial.

This Phase 3 trial is a two-arm, randomized, double blind, placebo controlled, adaptive design multicenter study to evaluate the safety and efficacy of leronlimab in patients with severe-to-critical symptoms of respiratory illness caused by COVID-19. Patients are randomized to receive weekly doses of 700 mg leronlimab or placebo, administered via weekly subcutaneous injection for two weeks. The study has three phases lasting 28 days: Screening Period, Treatment Period, and Follow-Up Period. The primary outcome measured in this study is: all-cause mortality at Day 28. Secondary outcomes measured are: (1) all-cause mortality at Day 14, (2) change in clinical status of subject at Day 14, (3) change in clinical status of subject at Day 28, and (4) change from baseline in Sequential Organ Failure Assessment (SOFA) score at Day 14. Recently, the Data Safety Monitoring Committee (DSMC) completed its first safety review of patients in the Phase 3 trial and reported it saw no cause to modify the study. The DSMC reviewed safety data from 149 of the 169 patients enrolled at the time of their review. The DSMC did not raise any concerns regarding safety and recommended the trial continue as planned.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, stated, We are very thankful for the many clinicians and their staff who have worked tirelessly to advance enrollment this quickly and for their care of these seriously ill patients. We are eager to perform an interim analysis of the data and remain optimistic the interim results will be consistent with those experienced by patients who received leronlimab through multiple EINDs (over 60) previously authorized by the FDA. And, in the event we are successful, we are well positioned with our distribution partner to accelerate distribution of leronlimab to patients throughout the U.S.

About Coronavirus Disease 2019CytoDyn completed its Phase 2 clinical trial (CD10) for COVID-19, a randomized clinical trial for mild-to-moderate patients in the U.S. Enrollment continues in its Phase 3 randomized clinical trial for the severe-to-critically ill COVID-19 population in several hospitals throughout the country.

SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 is believed to typically transmit person-to-person through respiratory droplets. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140)The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for critical illnesses. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer.Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells.The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH.

CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. The FDA has agreed to provide written responses to the Companys questions concerning its recent Biologics License Application by September 4, 2020, in lieu of a Type A teleconference meeting for this HIV combination therapy.

CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years.

CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors: Michael MulhollandOffice: 360.980.8524, ext. 102Mobile: 503.341.3514mmulholland@cytodyn.com

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CytoDyn Reaches Enrollment of 195 Patients in its Phase 3 Trial for COVID-19 Patients with Severe-to-Critical Symptoms - GlobeNewswire

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Tiger Woods, Rory McIlroy and more — This isn’t the best time for some of the PGA Tour’s biggest names – ESPN

Friday, August 28th, 2020

NORTON, Mass. - What the first week of the FedEx Cup playoffs revealed was it is not the best time to be one of the biggest names in golf.

With the exception of Dustin Johnson, the runaway winner at The Northern Trust, the first event of the PGA Tour's FedEx Cup playoffs, some of the sport's brightest stars have looked a bit lost.

Sure, Tiger Woods closed with a 66, including a four-birdie run to start his final round, but all it did was mask three uneven days that had him in a spot he never wants to be in -- playing early in the morning on the weekend of a tournament as nothing more than an afterthought.

Rory McIlroy, who played poorly enough to be Woods' early morning playing partner on the weekend, had no other choice but to make a joke or two about his own mediocrity lately when it was all over. More telling was that he feels at times he is simply going through the motions.

Brooks Koepka didn't even make it to the first tee, withdrawing before the event began because his bad knee isn't getting any better.

Jordan Spieth and Phil Mickelson didn't make the cut, placing them outside the top 70, which is required to gain entry into next week's BMW Championship, the second stop of the FedEx Cup playoffs.

He admitted Sunday that life without fans has been a difficult adjustment. He has always fed off the energy of the galleries that engulfed him -- and used it as an advantage over opponents who were shell-shocked by all the people and all the commotion.

But the silence seems to be a secondary problem for Woods. The inconsistency in his game remains the central issue. At The Northern Trust, he struggled at times with his putting; during Saturday's ugly 2-over 73, he missed six putts inside 10 feet. Until Sunday, he was again off with his short irons, regularly leaving lengthy birdie putts instead of medium-to-short range opportunities. His average distance from the hole on approach was just under 37 feet, which doesn't provide a ton of realistic birdie chances.

"Every day is different," he said. "That's golf. Shot-to-shot is different. That's the ebb and flow of playing golf."

He has played three events since golf's return from a three-month shutdown because of the coronavirus pandemic. He was a non-factor at both the Memorial and PGA Championship, finished tied for 37th and 40th. This week, at the Northern Trust, he had a strong opening round, then faded on Friday and Saturday. He's committed to this week's BMW Championship but hasn't earned a spot in the season-ending Tour Championship. And in this new, condensed schedule, the U.S. Open, the year's second major, is lurking right around the corner, set to begin Sept. 17 at ultra-difficult Winged Foot.

"This is going to be a long haul," he said.

Sure, the state of McIlroy's game is unsettling.

"If you need anyone to shoot even to 2-under for a week, I'm your man," he joked after finishing the week at, you guessed it, 2 under. "That's sort of what I've done the last few weeks. Everything is just a little off."

More concerning than his game might be his mindset.

"This is going to sound really bad, but I feel like the last few weeks, I've just been going through the motions," he said. "I want to get an intensity and some sort of fire, but I just haven't been able to. And look, that's partly to do with the atmosphere and partly to do with how I'm playing. I'm not inspiring myself, and I'm trying to get inspiration from outside sources to get something going."

On Saturday, with Woods, he started his round birdie/triple bogey/birdie. As he walked to the fourth tee he chuckled, "Yeah, 3-8-2 is a good area code." He was asked, in this time of playing tournaments without crowds and largely in silence, what the difference is now between a birdie and triple bogey?

"Honestly, not that much," he admitted. "It's not that much. Sometimes I come off a green and make a birdie, and I'm sort of -- you know, you're sort of laughing coming off a hole that you've just messed up, and you make a birdie, and it's sort of almost a more negative emotion in some ways. It's weird. It's very strange."

With two more playoff events and two more majors still on the 2020 calendar, how does he fix it?

"Do you go on YouTube and look at past successes do you give yourself something to sort of think about, a mantra as you go around?" he wondered. "There's different ways to do it. You know, what's always sort of done well with me is a bet or a game or a play for something. That sometimes gets something out of me. Maybe that's the strategy. I do that in practice rounds sometimes with [caddie and close friend] Harry [Diamond]. We make a lot of bets, and I try to shoot scores and try to win dinner or whatever it is. Maybe that's it. We're playing in the FedEx Cup [playoffs]. There's a lot of incentive here to play well. It's just trying to get it out of you."

McIlroy was on a roll before golf stopped in March. In the six events he played before the sport started at the Players Championship, McIlroy had not finished worse than a tie for fifth, and that included a win at the WCG-HSBC Champions. Since the return, has just one top-30 finish, a tie for 11th at the Travelers Championship.

"Yeah, it's just been a bit of a struggle," he said.

On Friday, as his round and season concluded, Jordan Spieth walked quickly off the 18th green at TPC Boston, never picking his head up as he headed toward the clubhouse. He had missed his second cut in six events, this one following a T-71 finish at the PGA Championship and a T-72 at the Wyndham Championship. He's had just one top-20 since a T-10 at the Charles Schwab Challenge, the first event after the break.

I'm a little uncertain. Like it feels pretty good, not great, but good enough to be able to compete," he said after the Wyndham Championship.

The missed cut at The Northern Trust meant he would not qualify for next week's BMW Championship, marking the first time in his career he did not advance to the second stage of the FedEx Cup playoffs. He hasn't made it to the season-ending Tour Championship since 2017. He hasn't won an event since the 2017 Open at Royal Birkdale, a stretch of 69 events.

"I'll keep trucking, I'll keep working hard," he said.

As long as his knee is up to it, the next time we'll see Koepka is at the U.S. Open, where he will try to win that major for the third time in four years. It's been an up-and-down year for the four-time major winner, who had a stem cell procedure on his left knee after last year's Tour Championship then re-aggravated when he slipped during an event in South Korea in October, which required him to sit out until January.

Since then, he's had just two top-10s, missed four cuts and had to withdraw from two events, one because of injury, one because his caddie, Ricky Elliott, tested positive for COVID-19.

Earlier this month, in search of his third consecutive PGA Championship, he entered the final round tied for fourth, just 2 shots off the lead. He faded on Sunday, shooting 74 to tie for 29th. The next week he missed the cut at the Wyndham Championship.

"Brooks is one of the biggest names in the game. We've all seen his physical struggles since last year," McIlroy said earlier this week. "It's maybe never a good time, but it's a better time than any other time to get it right. Take a few weeks off, try to get himself ready for the U.S. Open, and then the Masters coming up [in November]. I think it's smart on his part to do that and hopefully comes back healthy and comes back ready to play."

Like Spieth, Mickelson missed the cut and didn't qualify for the BMW Championship. So to get some work in before returning to what the site of one of his most painful major championship disappointments -- Winged Foot, site of this year's U.S. Open -- he needed to find a place to play.

Enter the PGA Tour Champions, the over-50 circuit that has an event this week in the Ozarks.

"I've been playing well and I want to play," he said. "I wish I was playing in Chicago next week but excited to play my first Champions event."

Mickelson, who turned 50 in June, has one top-20 finish and two missed cuts since golf's return. He had been reluctant to head over to the Champions Tour, making it clear he thinks he can still play -- and win -- on the PGA Tour.

"When I stop hitting bombs, I'll play the Champions Tour, but I'm hitting some crazy bombs right now," he joked in January.

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Tiger Woods, Rory McIlroy and more -- This isn't the best time for some of the PGA Tour's biggest names - ESPN

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G1 Therapeutics Announces Acceptance and Priority Review of NDA for Trilaciclib for Patients with Small Cell Lung Cancer – GlobeNewswire

Saturday, August 22nd, 2020

- PDUFA action date of February 15, 2021 assigned by U.S. Food and Drug Administration- Priority Review for trilaciclib is based on positive data from three randomized clinical trials showing robust myelopreservation benefits- G1 launching expanded access program (EAP) for patients with small cell lung cancer in the U.S.

RESEARCH TRIANGLE PARK, N.C., Aug. 17, 2020 (GLOBE NEWSWIRE) -- G1 Therapeutics, Inc. (Nasdaq: GTHX), a clinical-stage oncology company, today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for trilaciclib for small cell lung cancer (SCLC) patients being treated with chemotherapy and granted Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of February 15, 2021. Trilaciclib is a first-in-class investigational therapy designed to preserve bone marrow and immune system function during chemotherapy and improve patient outcomes.

There are currently no available therapies to protect patients from chemotherapy-induced toxicities before they occur, said Raj Malik, M.D., Chief Medical Officer and Senior Vice President, R&D. If approved, trilaciclib would be the first proactively administered myelopreservation therapy that is intended to make chemotherapy safer and reduce the need for rescue interventions, such as growth factor administrations and blood transfusions.

The FDA grants Priority Review to applications for potential therapies that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.The trilaciclib NDA was supported by compelling myelopreservation data from three randomized, double-blind, placebo-controlled clinical trials in which trilaciclib was administered prior to chemotherapy treatment in patients with SCLC. Trilaciclibhas been granted Breakthrough Therapy Designation by the FDA.In the NDA acceptance letter, the FDA also stated that it is currently not planning to hold an advisory committee meeting to discuss this application.

While undergoing chemotherapy, many patients experience significant myelosuppression, become fatigued and susceptible to infection, and often require transfusions and growth factor administrations, said Jared Weiss, M.D., Lineberger Comprehensive Cancer Center,University of North Carolina Chapel Hill, NC. Preventing bone marrow damage proactively is an opportunity to improve the quality of life of patients receiving chemotherapy for small cell lung cancer and reduce costly rescue interventions.

Myelosuppression is the result of damage to bone marrow stem cells and is one of the most common side effects of chemotherapy. Myelosuppression can lead to serious conditions such as anemia, neutropenia or thrombocytopenia, which have broad ranging clinical, patient experience and economic impacts on ongoing cancer treatment and overall outcomes. In clinical trials, trilaciclib significantly reduced chemotherapy-induced myelosuppression, and patients receiving trilaciclib experienced fewer dose delays/reductions, infections, hospitalizations, and need for rescue therapies compared to patients receiving chemotherapy alone.

Expanded Access ProgramG1 is making trilaciclib available to SCLC patients in the U.S., who are unable to enter clinical trials and for whom there are no appropriate alternative treatments while the trilaciclib NDA is under regulatory review, pursuant to FDAs expanded access program (EAP). To facilitate needed access through the EAP, G1 is collaborating with Bionical Emas, a global specialist clinical research organization (CRO). For more information about the EAP access to trilaciclib, email patient.access.us@Bionical-emas.com.

Complications from myelosuppression have been a long-standing challenge when treating patients with SCLC, said Dr. Malik. Establishing an expanded access program provides qualified patients in serious need with access to trilaciclib while the NDA is under review.

Trilaciclib in Small Cell Lung CancerTrilaciclib is a first-in-class investigational therapy designed to improve outcomes for people with cancer treated with chemotherapy. In 2019, trilaciclib received FDA Breakthrough Therapy Designation, and, in June 2020, G1 submitted the NDA based on myelopreservation data from three randomized, double-blind, placebo-controlled clinical trials in which trilaciclib was administered prior to chemotherapy in patients with small cell lung cancer (SCLC). In August 2020, G1 received FDA Priority Review with the Prescription Drug User Fee Act (PDUFA) date of February 15, 2021.

In June 2020, G1 announced a co-promotion agreement with Boehringer Ingelheim for trilaciclib in small cell lung cancer in the U.S. and Puerto Rico. If approved, G1 will lead marketing, market access and medical engagement initiatives for trilaciclib. The Boehringer Ingelheim oncology commercial team, well-established in lung cancer, will lead sales force engagement initiatives.G1 will book revenue and retain development and commercialization rights to trilaciclib and pay Boehringer Ingelheim a promotional fee based on net sales. The three-year agreement does not extend to additional indications that G1 is evaluating for trilaciclib. Press release details of the G1/ Boehringer Ingelheim agreement can be found here.

Evaluating Trilaciclib in Other CancersIn a randomized trial of women with metastatic triple-negative breast cancer, preliminary data showed that trilaciclib improved overall survival when administered in combination with chemotherapy compared with chemotherapy alone. The company plans to present final overall survival data from this trial in the fourth quarter of 2020. Trilaciclib is being evaluated in neoadjuvant breast cancer as part of the I-SPY 2 TRIAL, and the company expects to initiate a Phase 3 trial in patients treated with chemotherapy for colorectal cancer in the fourth quarter of 2020.

About G1 TherapeuticsG1 Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development and delivery of next generation therapies that improve the lives of those affected by cancer. The company is developing and advancing two novel therapies: trilaciclib is a first-in-class therapy designed to improve outcomes for patients being treated with chemotherapy; rintodestrant is a potential best-in-class oral selective estrogen receptor degrader (SERD) for the treatment of ER+ breast cancer. In 2020, the company out-licensed global development and commercialization rights to its differentiated oral CDK4/6 inhibitor, lerociclib.

G1 Therapeutics is based in Research Triangle Park, N.C. For additional information, please visit http://www.g1therapeutics.com and follow us on Twitter @G1Therapeutics.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements in this press release include, but are not limited to, those relating to the therapeutic potential of trilaciclib, rintodestrant and lerociclib, the timing of marketing applications in the U.S. and Europe for trilaciclib in SCLC, trilaciclibs possibility to improve patient outcomes across multiple indications, rintodestrants potential to be best-in-class oral SERD, lerociclibs differentiated safety and tolerability profile over other marketed CDK4/6 inhibitors, our reliance on partners to develop and commercial licensed products, and the impact of pandemics such as COVID-19 (coronavirus), are based on the companys expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Factors that may cause the companys actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in the companys filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein and include, but are not limited to, the companys ability to complete clinical trials for, obtain approvals for and commercialize any of its product candidates; the companys initial success in ongoing clinical trials may not be indicative of results obtained when these trials are completed or in later stage trials; the inherent uncertainties associated with developing new products or technologies and operating as a development-stage company; and market conditions. Except as required by law, the company assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

Contacts:Investors:Jeff MacdonaldG1 Therapeutics, Inc.Senior Director, Investor Relations & Corporate Communications919-907-1944jmacdonald@g1therapeutics.comMedia:Christine RogersG1 Therapeutics, Inc.Associate Director, Corporate Communications984-365-2819crogers@g1therapeutics.com

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G1 Therapeutics Announces Acceptance and Priority Review of NDA for Trilaciclib for Patients with Small Cell Lung Cancer - GlobeNewswire

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CytoDyn Will Attempt to Duplicate Berlin and London Patients’ HIV Cure by Using Leronlimab During Bone Marrow Transplant for 5 HIV Patients Who also…

Tuesday, August 18th, 2020

HIV monotherapy trials update: 215 patients completed almost one year of monotherapy. Only some were allowed to continue in extension arm; five patients reached almost 6 years. Twenty-five reached 2 to 4 years and 20 patients are 1 to 2 years

VANCOUVER, Washington, Aug. 17, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company"), a late-stage biotechnology company gives full update on all of its HIV programs.

HIV CureThe HIV co-receptor CCR5 has proven to be a key molecule in mediating HIV remission. The only two individuals functionally cured of HIV, one from London and the other from Berlin, received allogeneic stem cell transplantations from CCR5-deficient donors. However, because it is extremely rare to find a stem cell donor who lacks CCR5 and meets stringent MHC matching criteria, such an approach is unfeasible to cure HIV on a larger scale. CytoDyn believes its CCR5 blocking antibody, leronlimab, could be used in the setting of allogeneic stem cell transplantation to functionally convert a stem cell graft from a wildtype CCR5 stem cell donor into one from a CCR5 deficient donor, and thereby functionally cure the recipient of HIV.

CytoDyn plans to test this theory in a pilot clinical trial of five HIV patients with cancer who require bone marrow transplantation. Leronlimab will be used during the peri-transplant period to mimic a CCR5 deficient donor in order to achieve HIV cure.

HIV PrEPAs presented at the AIDS 2020 Virtual Conference, a pre-clinical study in the macaque model of HIV sexual transmission demonstrated leronlimab can prevent infection by blocking HIVs access to the CCR5 co-receptor. This protection is similar to that seen in individuals naturally CCR5 deficient and forms the rationale for use in HIV cure. CytoDyn believes leronlimab could be a once-a-month self-injectable, subcutaneous treatment for HIV PrEP and is in discussions with potential organizations to fund its next trial in HIV PrEP.

MonotherapySignificantly, for the first time documented, of the 49 HIV patients who stopped their HIV medications and used leronlimab as a monotherapy, 25 have been in monotherapy trial for two to four years and five patients for nearly or over six years. Monotherapy was successful for some of these patients by switching from 350 mg to a higher dose of 525 mg or 700 mg. The number of participants in the extension groups was limited due to costs.

The Company will submit manuscripts for two publications in regards to its findings.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, stated, We now have four paths forward for use of leronlimab in the HIV indication for different populations. The first path is a combination therapy where we successfully completed a Phase 3 trial with statistically significant p value for our primary endpoint. CytoDyn is awaiting a Type A meeting with the FDA for this treatment. Second is our monotherapy; we will discuss the potential approval path for label expansion at the time of our Type A meeting. Third is our PrEP study to examine the use of leronlimab for once-a-month self-injection for HIV prevention. Our fourth path is an HIV-Cure, where 5 patients will be put to test to duplicate the Berlin and London patients HIV functional cure.

About Leronlimab (PRO 140)The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for critical illnesses.

The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer.Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells.The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH.

CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. The Company has requested a Type A meeting with the FDA to discuss the FDAs request for additional information in order to resubmit its Biologics License Application for this HIV combination therapy.

CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years.

CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors:Michael MulhollandOffice: 360.980.8524, ext. 102Mobile: 503.341.3514mmulholland@cytodyn.com

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CytoDyn Will Attempt to Duplicate Berlin and London Patients' HIV Cure by Using Leronlimab During Bone Marrow Transplant for 5 HIV Patients Who also...

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What do we need to know about our bone health during this pandemic – Times of India

Tuesday, August 18th, 2020

As the on-going COVID-19 pandemic continues we are facing a huge healthcare crisis. Globally the pandemic has accelerated or rather decelerated the entire human population into the confines. Work from home, social shielding and discreet outdoor ventures has not only disrupted our emotional well-being but has also drastically affected our physical health. As people are confined to their homes with reduced physical activity there is rapid bone resorption (loss) as muscles and bones are not getting adequate stimulation. Also lack of exposure to sun during the pandemic has critically affected vitamin D levels in our body. People are frequently feeling tired with lack of energy and strength. Everyone needs to be cautious about the health of their bones as much as their other needs. Bones support us and allow us to be mobile. Bone health is always a priority and we always tend to overlook it. Bone density problem is a silent manifestation and could lead to a major medical issue over a period of time. While osteoporosis onsets with age among men & women, women face the brunt a little earlier, like from their 30s. Well-versed with the situation and to avoid unnecessary bones issues, let us now take a look at some measures that can take care of your bones during the pandemic.Eat a well-balanced diet rich in calcium and vitamin DGood sources of calcium include low-fat dairy products, green leafy veggies and dry fruits. Good sources of vitamin D include fortified cereals, egg yolks, saltwater fish, liver and milk. Calcium and vitamin D work together to protect your bones - calcium helps to build and maintain bones; while vitamin D helps your body to effectively absorb calcium.

Get exposure to sunlight to make enough vitamin DRegular sun exposure is the most natural way to get enough vitamin D. The sun's ultraviolet B (UVB) rays hit cholesterol in the skin cells, providing the energy for vitamin D synthesis. Vitamin D has a significant role in calcium homeostasis and metabolism.

As per pan-India study the best time to get exposed to the sun is between 11 am and 1 pm since the wavelength of ultraviolet B (UVB) rays is 290-320nm during this period which is essential for skin to make vitamin D.

Get plenty of physical activityLike muscles, bones become stronger with exercise. The best exercises for healthy bones are strength-building and weight-bearing exercise like walking, climbing stairs, lifting weights and dancing. Try to get 30 minutes of exercise each day.

Strength-building and weight-bearing exercise provides stimulation to bone cells and helps to increased bone mineral density and bone size thus reduced the risk of osteoporosis.

Live a healthy lifestyle

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What do we need to know about our bone health during this pandemic - Times of India

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Cellect Biotechnology Ltd ADR (NASDAQ:APOP) Receives an Approval of a Pivotal Patent for Stem Cells Activation from the European Patent Office – BP…

Saturday, August 15th, 2020

Every successful medicinal activity requires mass production of quality stem cells. This is what avails regenerative medicine to the public the need occasioned the founding of Cellect Biotechnology Ltd ADR (NASDAQ:APOP). In 2011. The companys vision was to bring to reality regenerative treatments, which would accelerate the production of stem cell-based treatments.

Cellect has spent 15 years in research, thanks to its ApoGraft methodology, which is long-sought-after because it engages an inexpensive process. This is what has birthed seven families of patents. The company just received a pivotal patent for Stem Cells Activation from the European Patent Office. This patent, which will expire on October 7, 2034, is the most significant event for the company seeking to strengthen its Intellectual Property (IP) portfolio.

Over the past few years, the need to strengthen its IP has been a core component of the companys business strategy. According to the CEO Dr. Shai Yarkoni, they wanted to tap into the clinical and economic benefits, which come with the patent. The primary benefit and, as the company has demonstrated previously, is the increased safety and efficacy of the cell product. This is enhanced by the development of a robust and user-friendly process.

While the extensive issued patents cover the accurate elimination (negative selection) of the mature cells and therefor enables SAFETY, this patent covers the increased ACTIVITY (positive selection=efficacy) of the regenerative capacity of the stem and progenitor cells Yarkoni explained.

Cellect Biotechnology has 65 patents in nine patent families. Out of these, 45 of them have been allowed patent, 18 are waiting for examination while the remaining two are the Patent Cooperation Treaty (PCT) applications. The Biotech aim is to expand and protect global IP. This will provide researchers and clinical community tools to isolate stem cells in quantity and quality rapidly. The final results will automatically be a wide variety of applications in regenerative medicine.

Meanwhile, the company has also been consulting and discussing widely with global leaders. Before long, it will be able to take control of innovation in the development of cell therapies.

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Cellect Biotechnology Ltd ADR (NASDAQ:APOP) Receives an Approval of a Pivotal Patent for Stem Cells Activation from the European Patent Office - BP...

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Coronavirus Drug and Treatment Tracker – The New York Times

Saturday, August 15th, 2020

The Covid-19 pandemic is one of the greatest challenges modern medicine has ever faced. Doctors and scientists are scrambling to find treatments and drugs that can save the lives of infected people and perhaps even prevent them from getting sick in the first place.

Below is an updated list of 20 of the most-talked-about treatments for the coronavirus. While some are accumulating evidence that theyre effective, most are still at early stages of research. We also included a warning about a few that are just bunk.

We are following 20 coronavirus treatments for effectiveness and safety:

Tentative or

mixed evidence

We are following 20 coronavirus treatments

for effectiveness and safety:

Tentative or

mixed evidence

We are following 20 coronavirus treatments

for effectiveness and safety:

There is no cure yet for Covid-19. And even the most promising treatments to date only help certain groups of patients and await validation from further trials. The F.D.A. has not fully licensed any treatment specifically for the coronavirus. Although it has granted emergency use authorization to some treatments, their effectiveness against Covid-19 has yet to be demonstrated in large-scale, randomized clinical trials.

This list provides a snapshot of the latest research on the coronavirus, but does not constitute medical endorsements. Always consult your doctor about treatments for Covid-19.

New additions and recent updates:

Added ivermectin, a drug typically used against parasitic worms that is being increasingly prescribed in Latin America. Aug. 10

Updated descriptions for several treatments. Aug. 10

We will update and expand the list as new evidence emerges. For details on evaluating treatments, see the N.I.H. Covid-19 Treatment Guidelines. For the current status of vaccine development, see our Coronavirus Vaccine Tracker.

WIDELY USED: These treatments have been used widely by doctors and nurses to treat patients hospitalized for diseases that affect the respiratory system, including Covid-19.

PROMISING EVIDENCE: Early evidence from studies on patients suggests effectiveness, but more research is needed. This category includes treatments that have shown improvements in morbidity, mortality and recovery in at least one randomized controlled trial, in which some people get a treatment and others get a placebo.

TENTATIVE OR MIXED EVIDENCE: Some treatments show promising results in cells or animals, which need to be confirmed in people. Others have yielded encouraging results in retrospective studies in humans, which look at existing datasets rather than starting a new trial. Some treatments have produced different results in different experiments, raising the need for larger, more rigorously designed studies to clear up the confusion.

NOT PROMISING: Early evidence suggests that these treatments do not work.

PSEUDOSCIENCE OR FRAUD: These are not treatments that researchers have ever considered using for Covid-19. Experts have warned against trying them, because they do not help against the disease and can instead be dangerous. Some people have even been arrested for their false promises of a Covid-19 cure.

EVIDENCE IN CELLS, ANIMALS or HUMANS: These labels indicate where the evidence for a treatment comes from. Researchers often start out with experiments on cells and then move onto animals. Many of those animal experiments often fail; if they dont, researchers may consider moving on to research on humans, such as retrospective studies or randomized clinical trials. In some cases, scientists are testing out treatments that were developed for other diseases, allowing them to move directly to human trials for Covid-19.

All treatmentsWidely usedPromisingTentative or mixedNot promisingPseudoscience

Antivirals can stop viruses such as H.I.V. and hepatitis C from hijacking our cells. Scientists are searching for antivirals that work against the new coronavirus.

PROMISING EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSEMERGENCY USE AUTHORIZATIONRemdesivirRemdesivir, made by Gilead Sciences, was the first drug to get emergency authorization from the F.D.A. for use on Covid-19. It stops viruses from replicating by inserting itself into new viral genes. Remdesivir was originally tested as an antiviral against Ebola and Hepatitis C, only to deliver lackluster results. But preliminary data from trials that began this spring suggested the drug can reduce the recovery time of people hospitalized with Covid-19 from 15 to 11 days. (The study defined recovery as either discharge from the hospital or hospitalization for infection-control purposes only.) These early results did not show any effect on mortality, though retrospective data released in July hints that the drug might reduce death rates among those who are very ill.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSFavipiravirOriginally designed to beat back influenza, favipiravir blocks a viruss ability to copy its genetic material. A small study in March indicated the drug might help purge the coronavirus from the airway, but results from larger, well-designed clinical trials are still pending.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSMK-4482Another antiviral originally designed to fight the flu, MK-4482 (previously known as EIDD-2801) has had promising results against the new coronavirus in studies in cells and on animals. Merck, which has been running clinical trials on the drug this summer, has announced it will launch a large Phase III trial in September.Updated Aug. 6

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS Recombinant ACE-2To enter cells, the coronavirus must first unlock them a feat it accomplishes by latching onto a human protein called ACE-2. Scientists have created artificial ACE-2 proteins which might be able to act as decoys, luring the coronavirus away from vulnerable cells. Recombinant ACE-2 proteins have shown promising results in experiments on cells, but not yet in animals or people.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS AND HUMANS IvermectinFor decades, ivermectin has served as a potent drug to treat parasitic worms. Doctors use it against river blindness and other diseases, while veterinarians give dogs a different formulation to cure heartworm. Studies on cells have suggested ivermectin might also kill viruses. But scientists have yet to find evidence in animal studies or human trials that it can treat viral diseases. As a result, Ivermectin is not approved to use as an antiviral.

In April, Australian researchers reported that the drug blocked coronaviruses in cell cultures, but they used a dosage that was so high it might have dangerous side effects in people. The FDA immediately issued a warning against taking pet medications to treat or prevent Covid-19. These animal drugs can cause serious harm in people, the agency warned.

Since then a number of clinical trials have been launched to see if a safe dose of ivermectin can fight Covid-19. In Singapore, for example, the National University Hospital is running a 5,000-person trial to see if it can prevent people from getting infected. As of now, theres no firm evidence that it works. Nevertheless ivermectin is being prescribed increasingly often in Latin America, much to the distress of disease experts.Updated Aug. 10

NOT PROMISING EVIDENCE IN CELLS AND HUMANS Lopinavir and ritonavirTwenty years ago, the F.D.A. approved this combination of drugs to treat H.I.V. Recently, researchers tried them out on the new coronavirus and found that they stopped the virus from replicating. But clinical trials in patients proved disappointing. In early July, the World Health Organization suspended trials on patients hospitalized for Covid-19. They didnt rule out studies to see if the drugs could help patients not sick enough to be hospitalized, or to prevent people exposed to the new coronavirus from falling ill. The drug could also still have a role to play in certain combination treatments.

NOT PROMISING EVIDENCE IN CELLS, ANIMALS AND HUMANSHydroxychloroquine and chloroquineGerman chemists synthesized chloroquine in the 1930s as a drug against malaria. A less toxic version, called hydroxychloroquine, was invented in 1946, and later was approved for other diseases such as lupus and rheumatoid arthritis. At the start of the Covid-19 pandemic, researchers discovered that both drugs could stop the coronavirus from replicating in cells.

Since then, theyve had a tumultuous ride. A few small studies on patients offered some hope that hydroxychloroquine could treat Covid-19. The World Health Organization launched a randomized clinical trial in March to see if it was indeed safe and effective for Covid-19, as did Novartis and a number of universities. Meanwhile, President Trump repeatedly promoted hydroxychloroquine at press conferences, touting it as a game changer, and even took it himself. The F.D.A. temporarily granted hydroxychloroquine emergency authorization for use in Covid-19 patients which a whistleblower later claimed was the result of political pressure. In the wake of the drugs newfound publicity, demand spiked, resulting in shortages for people who rely on hydroxychloroquine as a treatment for other diseases.

But more detailed studies proved disappointing. A study on monkeys found that hydroxychloroquine didnt prevent the animals from getting infected and didnt clear the virus once they got sick. Randomized clinical trials found that hydroxychloroquine didnt help people with Covid-19 get better or prevent healthy people from contracting the coronavirus. Another randomized clinical trial found that giving hydroxychloroquine to people right after being diagnosed with Covid-19 didnt reduce the severity of their disease. (One large-scale study that concluded the drug was harmful as well was later retracted.) The World Health Organization, the National Institutes of Health and Novartis have since halted trials investigating hydroxychloroquine as a treatment for Covid-19, and the F.D.A. revoked its emergency approval. The F.D.A. now warns that the drug can cause a host of serious side effects to the heart and other organs when used to treat Covid-19.

In July, researchers at Henry Ford hospital in Detroit published a study finding that hydroxychloroquine was associated with a reduction in mortality in Covid-19 patients. President Trump praised the study on Twitter, but experts raised doubts about it. The study was not a randomized controlled trial, in which some people got a placebo instead of hydroxychloroquine. The studys results might not be due to the drug killing the virus. Instead, doctors may have given the drug to people who were less sick, and thus more likely to recover anyway.

Despite negative results, a number of hydroxychloroquine trials have continued, although most are small, testing a few dozen or a few hundred patients. A recent analysis by STAT and Applied XL found more than 180 ongoing clinical trials testing hydroxychloroquine or chloroquine, for treating or preventing Covid-19. Although its clear the drugs are no panacea, its theoretically possible they could provide some benefit in combination with other treatments, or when given in early stages of the disease. Only well-designed trials can determine if thats the case.Updated Aug. 10

Most people who get Covid-19 successfully fight off the virus with a strong immune response. Drugs might help people who cant mount an adequate defense.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS AND HUMANS Convalescent plasmaA century ago, doctors filtered plasma from the blood of recovered flu patients. So-called convalescent plasma, rich with antibodies, helped people sick with flu fight their illness. Now researchers are trying out this strategy on Covid-19. In May, the F.D.A. designated convalescent plasma an investigational product. That means that despite not yet being shown as safe and effective, plasma can be used in clinical trials and given to some patients who are seriously ill with Covid-19. Tens of thousands of patients in the U.S. have received plasma through a program launched by the Mayo Clinic and the federal government.

The Trump administration has praised convalescent plasma, despite the lack of evidence yet that it works. The first wave of trials have been small and the results have been mixed. Large randomized clinical trials are underway, but theyve struggled to enroll enough participants, some of whom worry they will receive a placebo instead of the treatment itself.

Experts say that its vital to complete these trials to determine if convalescent plasma is safe and effective. If these trials are successful, it could serve as an important stopgap measure until more potent therapies become widely available.Updated Aug. 10

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSMonoclonal antibodiesConvalescent plasma from people who recover from Covid-19 contains a mix of different antibodies. Some of the molecules can attack the coronavirus, but many are directed at other pathogens. Researchers have sifted through this slurry to find the most potent antibodies against Covid-19. They have then manufactured synthetic copies of these molecules, known as monoclonal antibodies. Researchers have begun investigating them as a treatment for Covid-19, either individually or in cocktails.

Monoclonal antibodies were first developed as a therapy in the 1970s, and since then the F.D.A. has approved them for 79 diseases, ranging from cancer to AIDS. Since the start of the pandemic, researchers have found dozens of monoclonal antibodies that show promise against Covid-19 in preclinical studies on cells and animals. Companies like Eli Lilly and Regeneron recently began clinical trials studying monoclonal antibodies. Several other firms, as well as teams at universities, are slated to enter the race soon as well.Updated Aug. 10

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN CELLS, ANIMALS AND HUMANSInterferonsInterferons are molecules our cells naturally produce in response to viruses. They have profound effects on the immune system, rousing it to attack the invaders, while also reining it in to avoid damaging the bodys own tissues. Injecting synthetic interferons is now a standard treatment for a number of immune disorders. Rebif, for example, is prescribed for multiple sclerosis.

As part of its strategy to attack our bodies, the coronavirus appears to tamp down interferon. That finding has encouraged researchers to see whether a boost of interferon might help people weather Covid-19, particularly early in infection. Early studies, including experiments in cells and mice, have yielded encouraging results that have led to clinical trials.

An open-label study in China suggested that the molecules could help prevent healthy people from getting infected. On July 20, the British pharmaceutical company Synairgen announced that an inhaled form of interferon called SNG001 lowered the risk of severe Covid-19 in infected patients in a small clinical trial. The full data have not yet been released to the public, or published in a scientific journal. On August 6, the National Institute of Allergy and Infectious Diseases launched a Phase III trial on a combination of Rebif and the antiviral remdesivir, with results expected by fall 2020.Updated Aug. 10

The most severe symptoms of Covid-19 are the result of the immune systems overreaction to the virus. Scientists are testing drugs that can rein in its attack.

PROMISING EVIDENCE EVIDENCE IN HUMANS DexamethasoneThis cheap and widely available steroid blunts many types of immune responses. Doctors have long used it to treat allergies, asthma and inflammation. In June, it became the first drug shown to reduce Covid-19 deaths. That study of more than 6,000 people, which in July was published in the New England Journal of Medicine, found that dexamethasone reduced deaths by one-third in patients on ventilators, and by one-fifth in patients on oxygen. It may be less likely to help and may even harm patients who are at an earlier stage of Covid-19 infections, however. In its Covid-19 treatment guidelines, the National Institutes of Health recommends only using dexamethasone in patients with COVID-19 who are on a ventilator or are receiving supplemental oxygen.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN HUMANS Cytokine InhibitorsThe body produces signaling molecules called cytokines to fight off diseases. But manufactured in excess, cytokines can trigger the immune system to wildly overreact to infections, in a process sometimes called a cytokine storm. Researchers have created a number of drugs to halt cytokine storms, and they have proven effective against arthritis and other inflammatory disorders. Some turn off the supply of molecules that launch the production of the cytokines themselves. Others block the receptors on immune cells to which cytokines would normally bind. A few block the cellular messages they send. Depending on how the drugs are formulated, they can block one cytokine at a time, or muffle signals from several at once.

Against the coronavirus, several of these drugs have offered modest help in some trials, but faltered in others. Drug companies Regeneron and Roche drug both recently announced that two drugs called sarilumab and tocilizumab, which both target the cytokine IL-6, did not appear to benefit patients in Phase 3 clinical trials. Many other trials remain underway, several of which combine cytokine inhibitors with other treatments.Updated Aug. 10

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN HUMANS EMERGENCY USE AUTHORIZATIONBlood filtration systemsThe F.D.A. has granted emergency use authorization to several devices that filter cytokines from the blood in an attempt to cool cytokine storms. One machine, called Cytosorb, can reportedly purify a patients entire blood supply about 70 times in a 24-hour period. A small study in March suggested that Cytosorb had helped dozens of severely ill Covid-19 patients in Europe and China, but it was not a randomized clinical trial that could conclusively demonstrate it was effective. A number of studies on blood filtration systems are underway, but experts caution that these devices carry some risks. For example, such filters could remove beneficial components of blood as well, such as vitamins or medications.Updated Aug. 10

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN HUMANS Stem cellsCertain kinds of stem cells can secrete anti-inflammatory molecules. Over the years, researchers have tried to use them as a treatment for cytokine storms, and now dozens of clinical trials are under way to see if they can help patients with Covid-19. But these stem cell treatments havent worked well in the past, and its not clear yet if theyll work against the coronavirus.

Doctors and nurses often administer other supportive treatments to help patients with Covid-19.

WIDELY USEDProne positioningThe simple act of flipping Covid-19 patients onto their bellies opens up the lungs. The maneuver has become commonplace in hospitals around the world since the start of the pandemic. It might help some individuals avoid the need for ventilators entirely. The treatments benefits continue to be tested in a range of clinical trials.

WIDELY USEDEMERGENCY USE AUTHORIZATIONVentilators and other respiratory support devicesDevices that help people breathe are an essential tool in the fight against deadly respiratory illnesses. Some patients do well if they get an extra supply of oxygen through the nose or via a mask connected to an oxygen machine. Patients in severe respiratory distress may need to have a ventilator breathe for them until their lungs heal. Doctors are divided about how long to treat patients with noninvasive oxygen before deciding whether or not they need a ventilator. Not all Covid-19 patients who go on ventilators survive, but the devices are thought to be lifesaving in many cases.

TENTATIVE OR MIXED EVIDENCE EVIDENCE IN HUMANS AnticoagulantsThe coronavirus can invade cells in the lining of blood vessels, leading to tiny clots that can cause strokes and other serious harm. Anticoagulants are commonly used for other conditions, such as heart disease, to slow the formation of clots, and doctors sometimes use them on patients with Covid-19 who have clots. Many clinical trials teasing out this relationship are now underway. Some of these trials are looking at whether giving anticoagulants before any sign of clotting is beneficial.

False claims about Covid-19 cures abound. The F.D.A. maintains a list of more than 80 fraudulent Covid-19 products, and the W.H.O. debunks many myths about the disease.

WARNING: DO NOT DO THISDrinking or injecting bleach and disinfectantsIn April, President Trump suggested that disinfectants such as alcohol or bleach might be effective against the coronavirus if directly injected into the body. His comments were immediately refuted by health professionals and researchers around the world as well as the makers of Lysol and Clorox. Ingesting disinfectant would not only be ineffective against the virus, but also hazardous possibly even deadly. In July, Federal prosecutors charged four Florida men with marketing bleach as a cure for COVID-19.

WARNING: NO EVIDENCEUV lightPresident Trump also speculated about hitting the body with ultraviolet or just very powerful light. Researchers have used UV light to sterilize surfaces, including killing viruses, in carefully managed laboratories. But UV light would not be able to purge the virus from within a sick persons body. This kind of radiation can also damage the skin. Most skin cancers are a result of exposure to the UV rays naturally present in sunlight.

WARNING: NO EVIDENCESilverThe F.D.A. has threatened legal action against a host of people claiming silver-based products are safe and effective against Covid-19 including televangelist Jim Bakker and InfoWars host Alex Jones. Several metals do have natural antimicrobial properties. But products made from them have not been shown to prevent or treat the coronavirus.

Note: After additional discussions with experts we have adjusted several labels on the tracker. The Strong evidence label has been removed until further research identifies treatments that consistently benefit groups of patients infected by the coronavirus. In its place, Promising evidence will be used for drugs such as remdesivir and dexamethasone that have shown promise in at least one randomized controlled trial, and Widely used for treatments such as proning and ventilators that are often used with severely ill patients, including those with Covid-19. And we may reintroduce the Ineffective label when ongoing clinical trials repeatedly end with disappointing results.

Sources: National Library of Medicine; National Institutes of Health; William Amarquaye, University of South Florida; Paul Bieniasz, Rockefeller University; Jeremy Faust, Brigham & Womens Hospital; Matt Frieman, University of Maryland School of Medicine; Noah Haber, Stanford University; Swapnil Hiremath, University of Ottawa; Akiko Iwasaki, Yale University; Paul Knoepfler, University of California, Davis; Elena Massarotti, Brigham and Womens Hospital; John Moore and Douglas Nixon, Weill Cornell Medical College; Erica Ollman Saphire, La Jolla Institute for Immunology; Regina Rabinovich, Harvard T.H. Chan School of Public Health; Ilan Schwartz, University of Alberta; Phyllis Tien, University of California, San Francisco.

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Coronavirus Drug and Treatment Tracker - The New York Times

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Statement By Premier Fahie On COVID-19 Update – Phase II – Government of the Virgin Islands

Saturday, August 15th, 2020

STATEMENT BY PREMIER AND MINISTER OF FINANCEHONOURABLE ANDREW A. FAHIE

COVID-19 UPDATE- PHASE II

Friday, 14th August, 2020

I say good day and Gods Blessings to all the people of the Virgin Islands.

We thank the Lord our God for his continued blessings in our lives and in our Territory.

The world continues to grapple with the realities of COVID-19 and the BVI, is not immune.

I appreciate what we continue to do together in our little corner of the world to keep ourselves and each other safe.

So far we have only had nine confirmed cases, including one death with 8 recoveriesmost of the cases being classified as imported or related.

Together, we have been able to stem the tide, but we are not out of the woods.

The problem of COVID-19 has not yet been solved.

It remains a real, deadly, imminent threat to all persons in all countries throughout the world.

We continue to see countries rushing forward with haste to open their borders, refusing to close their borders, or disobeying the recommended hygiene and safety protocols, only for them to hastily retreat as they experience a surge of infections in their population.

Can I tell you that right now, the only measures that are scientifically proven to keep us all safe are social distancing, wearing of appropriate masks or shield, and washing our hands regularly?

These measures are simple for us to do, so let us continue to do them.

In all our actions, your Government is being guided by the law, international protocols and international best practice, with guidance from God Almighty.

In particular, we are being guided by the provisions of the Public Health Ordinance, the Quarantine Act and the Infectious Diseases (Notification) Act, all of which are designed to treat with circumstances such as these, and which prescribes measures that should be taken whenever there is the risk of an outbreak of disease.

All of our precautionary measures for protecting our people from the public health threat of COVID-19 are soundly grounded in the law of the land. And, your Government is being very cautious in dealing with this very fluid situation because another round of lockdown can cripple our economy to the point that we may not be able to bounce back.

Indeed, we understand and empathise with the inconvenience and hardship that COVID-19 is causing to everyone; however we must be cognisant of the double-threat to public health and the economy.

In the BVI, we cannot afford another lockdown. It will not be healthy for our economy, economically, socially and healthwise.

We are not immuned to the trickled effect of COVID-19, which is a health issue.

That is why even with the controlled re-entry of Virgin Islands, Belongers, permanent residence and Naturalisaed Overseas Territories citizens, since 2 June 2020 and the full internal re-opening of businesses, we have been moving cautiously, and in a phased way so that we can realistically manage our environment and surroundings in the midst of the New Regular of living and working with COVID-19.

There have been several views from many quarters trying to pressure the Government to open up quickly and to open up now.

But, I want to echo something that the Chief Medical Officer Dr. Irad Potter said to Members of Cabinet, which I think was the most profound statement that caused all of us to pause, he said and I quote,

We must take into consideration what risk we can take. We must take into consideration what risk we can manage. And, we must take into consideration what we can and cannot allow. End of quote.

Your Government intends to concentrate on the areas that united us as a people because only through unity will we get through this pandemic, successfully.

To date we have been able to manage and monitor the process to the point where on behalf of the Cabinet of the Virgin Islands, I can now announce that we are now moving into Phase two of our Restricted Border Re-opening Plan.

We are satisfied that the different Government agencies have the necessary protocols in place as we advance into Phase two of the restricted border controlled process.

We have had Special meetings of the Cabinet on 13th August and we had to continue the meeting on 14th August. These are not easy decisions, as checks and balances are important.

But having been satisfied that we have properly ventilated and learn from lessons in Phase one to strengthen Phase two, on behalf of Cabinet, I wish to make the following announcements:

1. We decided that the Ministry of Health and Social Development instruct the Attorney General's Chambers to draft a new Immigration and Prohibition Order for the period from 15th August, 2020 to 31st August, 2020;

People of the Virgin Islands Cabinet also advised that the National Security Council should not extend the Curfew Order, which expired on 13th August, 2020, thereby re-opening the Territory internally for 24-hours a day, seven days a week.

However, all businesses and individuals must adhere to all of the approved social measures; and Cabinet also advised that the National Security Council will impose restrictions on the movement of vessels within Territorial waters in a manner prescribed by Order.

Let me say here.

We are not out of the woods with COVID-19. People are coming in, in phase one and two.

We must continue to remain vigilant.

We cannot leave any stone unturned with COVID-19.

That is why the social distancing measures must apply. That is why adherence to the health protocol is a must.

This is our opportunity to test the strength of our system, our protocol, our preventative measures and our commitment to a healthy and safe Virgin Islands.

If we all follow the rules, all will be well.

I want to take this time to remind our bikers those on the scoters, all our bikers, that safe riding save lives. Please ensure that you have a scooter licence, ensure that your motorbikes are licensed and insured, always wear a helmet, know your scooters limits, watch your speed, keep your distance from other vehicles, use both brakes, avoid blind spots, always use signals. Our young generation is important to the future of this Territory. When our youth damage themselves, they damage their future potential in the Virgin Islands. Remember Your Life Matters!

Our responsibility as a Government is to keep the people of the Virgin Islands healthy and safe as much is humanly possible during this COVID-19 era and at all times.

I want to thank everyone who continues to help us to be safe during this COVID-19 era.

My Government, Cabinet Members, National Security Council, Attorney General Chambers, Cabinet Office, all Members of the House of Assembly, the Health Emergency Operations Centre, the Government public officers at varying levels, the Joint Information Cell/Government Information Service, industry partners and members of the public for all coming together and doing their part in this fight against COVID-19.

In these Virgin Islands, each of us has a role to play to help our economy and to keep us safe.

Remember we are all in this together, but I must warn again that we are not out of the woods as far as COVID-19 is concerned.

Remember that COVID-19 is not playing around with us and we must not play around with COVID-19

Now that the curfew is lifted, more than ever, and the borders remain closed, with limited entry, we must hold each other accountable to get through this experience together.

This phase is our greatest test and I know with Gods help we will pass this test.

Please be advised that the main point of entry remains the TB Lettsome International Airport.

So, let us continue to work together in the oneness of purpose.

Remember, God is with us, and He is opening doors for us so that we can have a Virgin Islands that generations would be proud of.

As leader of Government business, we will continue to keep you the people informed of our efforts to continue to combat COVID-19 as this remains a fluid situation. We will continue to make adjustments, so that the people of the Virgin Islands will remain safe while we balance our economy starting with the rebooting and the revitalization of our internal economy.

May God continue to bless these beautiful Virgin Islands and the people of the Virgin Islands.

I thank you.

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Statement By Premier Fahie On COVID-19 Update - Phase II - Government of the Virgin Islands

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HPU Students, Faculty and Staff Recognized for Research and Innovation – High Point University

Saturday, August 15th, 2020

HIGH POINT, N.C., Aug. 14, 2020 Members of the High Point University community frequently conduct, publish and share research and creative works in a variety of ways. Below is a recap of recent research initiatives.

HPU Student, Alumna and Faculty Research Featured in National Scientific Journal

Casey Garr, HPU alumna; Candyce Sturgeon, HPU rising senior; Dr. Veronica Segarra, HPU assistant professor of biology; and Noah Franks, student at Penn Griffin School of the Arts in High Point, North Carolina; recently conducted research that was published in Autophagy, a national scientific journal.

The study, titled, Autophagy as an on-ramp to scientific discovery, examines HPUs Cell Art Collaborative program to gain understanding around how the recruitment of highly creative students into STEM fields through connections to art can be a first step in defining a specialized career path that leads to a valuable and unique contribution to science.

In addition to providing experiential learning opportunities for students at HPU to conduct hands-on research and co-author peer-reviewed articles, the Cell Art Collaborative program encourages students in the local community to explore careers that incorporate both science and art, says Segarra. This initiative continues to facilitate conversations around STEAM-based learning environments for educators to take advantage of a wider range of student talents and interests, preparing them to go forth into society as the creative thinkers and problem solvers the world needs.

HPU Students Research Featured in CBE: Life Sciences Education Journal

Clara Primus, a rising junior majoring in biology and Bonner Leader at HPU, recently collaborated with prominent scientists at the Mayo Clinic, University of California Davis and Northwestern to conduct research that was published in CBE: Life Sciences Education, a quarterly journal published by the American Society for Cell Biology. The article, titled, Scientific Societies Fostering Inclusive Scientific Environments through Travel Awards: Current Practices and Recommendations, examines how scientific societies can contribute to a diverse and inclusive workforce.

The research compares and contrasts the broad approaches that scientific societies within the National Science Foundation-funded Alliance to Catalyze Change for Equity in STEM Success (ACCESS) use to implement and assess their travel award programs for underrepresented minority (URM) trainees. Findings will improve collaboration and better position scientific societies to begin addressing some of these questions and learning from each other.

The recommendations included in this research shed light on how even scientific societies can be allies in furthering inclusion efforts, said Primus. Ive spent nearly two years studying equity and diversity, and I hope that I can take the knowledge Ive learned from all of my research to educate my peers at HPU.

HPU Exercise Science Professor Publishes Statement for the American Heart Association

Dr. Colin Carriker, assistant professor of exercise science in HPUs Congdon School of Health Sciences, recently co-authored an American Heart Association (AHA) scientific statement on medicinal and recreational cannabis use published in Circulation.

The statement critically reviews the use of medicinal and recreational cannabis from a clinical but also a policy and public health perspective by evaluating its safety and efficacy profile, particularly in relation to cardiovascular health. The purpose of this scientific statement was to explore the evidence and science pertaining to medical marijuana, recreational cannabis and cardiovascular health to provide physicians and health care providers with the information available to date. While cannabis may have some therapeutic benefits, these do not appear to be cardiovascular in nature. Health care providers would benefit from increased knowledge, education and training pertaining to various cannabis products and health implications, including recognition that cannabis use may, in fact, exacerbate cardiovascular events or other health problems. In this regard, the negative health implications of cannabis should be formally and consistently emphasized in policy, while aligning with the American Heart Associations commitment to minimizing the smoking and vaping of any products and banning cannabis use for youth.

It was an honor to work alongside such a high-quality team of researchers, says Carriker. I want to especially thank our committee chairs, Dr. Robert L. Page II and Dr. Larry A. Allen, as their extraordinary leadership and organization were integral components in the completion and publication of this AHA scientific statement. We publish these statements to counterbalance and debunk misinformation because the public requires high-quality information about cannabis from reputable organizations such as the American Heart Association.

Carriker is the advocacy ambassador for the American Heart Associations Council on Lifestyle and Cardiometabolic Health and served as a member of the writing committee tasked with writing this AHA Scientific Statement initiated by the AHAs Council on Clinical Cardiology.

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BeyondSpring Initiates Expanded Access Program with Plinabulin for Patients Suffering from CIN in the US – BioSpace

Tuesday, August 11th, 2020

- NCCN Guideline Updates Highlight Need for Maximum CIN Prevention and Resource Allocation for COVID-19 Patients -

- First Patient Dosed in the U.S. Avoided Grade 4 Neutropenia in Cycle 2 with Plinabulin and Pegfilgrastim, Despite Experiencing Grade 4 Neutropenia in Cycle 1 with Pegfilgrastim Alone -

NEW YORK, Aug. 11, 2020 (GLOBE NEWSWIRE) -- BeyondSpring Inc.(the Company or BeyondSpring) (NASDAQ: BYSI), a global biopharmaceutical company focused on the development of innovative immuno-oncology cancer therapies, today announced that the Company has initiated an Expanded Access Program (EAP) to enable doctors across the U.S. to use BeyondSprings late-stage asset, Plinabulin, to prevent cancer patients chemotherapy-induced neutropenia (CIN), both alone and in combination with G-CSFs (the current standard of care), during the COVID-19 pandemic. Dr. Emad Ibrahim enrolled the first patient at Redlands Community Hospital in California on July 28, 2020.

In response to COVID-19, the National Comprehensive Cancer Network (NCCN) recently updated its treatment guidelines for the prophylaxis of CIN, with the objective of preserving hospital and ER resources for COVID-19 patients and maximizing protection for cancer patients against CIN development. This is designed to help necessitate healthcare interactions, and avoidance of hospital / ER visits will also minimize cancer patients risk of contracting COVID-19. In light of these NCCN guideline updates, BeyondSpring initiated an Expanded Access Program to enable the use of Plinabulin by oncologists to better protect cancer patients against CIN with the use of myelosuppressive chemotherapies under the current COVID-19 challenges.

Dr. Emad Ibrahim enrolled the first patient under this EAP at Redlands Community Hospital in California:

The recent updates to the NCCN guidelines aim to protect cancer patients from developing CIN in the most effective way possible and enable the healthcare system to reserve precious resources for COVID-19 patients, said Ramon Mohanlal, BeyondSprings Chief Medical Officer and Executive Vice President, Research and Development. In our CIN studies, Plinabulin, in combination with Pegfilgrastim, provided superior protection against CIN, compared to the standard of care alone. The observation in this first EAP patient who completely avoided Grade 4 CIN when given Plinabulin and Pegfilgrastim is a significant achievement for us. At BeyondSpring, we strive to play our part in serving patients and healthcare providers to the highest degree while working through the many challenges imposed by COVID-19.

Preventing CIN during chemotherapy is extremely important, as this will enable cancer patients to receive the full regimen of chemotherapy and achieve treatment goals. The onset of CIN is the No. 1 reason for treatment modifications, such as downgrading the strength of chemotherapy or stopping chemotherapy altogether. When a patient develops CIN, the treating physician is required to delay the next round of chemotherapy until a patients white blood cell count recovers. These changes can have a profoundly negative impact on patient outcomes.

For more information on BeyondSprings Plinabulin Expanded Access Program, please visit http://www.beyondspringpharma.com/EAP/. Supplies may be limited.

If you are a physician in the U.S. who would like to request Plinabulin EAP access for your patient, please email expandedaccess@beyondspringpharma.com.

About BeyondSpringHeadquartered in New York, BeyondSpring is a global, clinical-stage biopharmaceutical company focused on developing innovative immuno-oncology cancer therapies to improve clinical outcomes for patients with high unmet medical needs. BeyondSprings first-in-class lead immune asset, Plinabulin, is a potent antigen-presenting cell (APC) inducer. It is currently in two Phase 3 clinical trials for two severely unmet medical needs indications: one is for the prevention of chemotherapy-induced neutropenia (CIN), the most frequent cause for a chemotherapy regimen doses decrease, delay, downgrade or discontinuation, which can lead to suboptimal clinical outcomes. The other is for non-small cell lung cancer (NSCLC) treatment in EGFR wild-type patients. As a pipeline drug, Plinabulin is in various I/O combination studies to boost PD-1 / PD-L1 antibody anti-cancer effects. In addition to Plinabulin, BeyondSprings extensive pipeline includes three pre-clinical immuno-oncology assets and a drug discovery platform dubbed molecular glue that uses the protein degradation pathway.

About PlinabulinPlinabulin, BeyondSprings lead asset, is a differentiated immune and stem cell modulator. Plinabulin is currently in late-stage clinical development to increase overall survival in cancer patients, as well as to alleviate chemotherapy-induced neutropenia (CIN). The durable anticancer benefits of Plinabulin have been associated with its effect as a potent antigen-presenting cell (APC) inducer (through dendritic cell maturation) and T-cell activation (Chem andCell Reports, 2019). Plinabulins CIN data highlights the ability to boost the number of hematopoietic stem / progenitor cells (HSPCs), or lineage-/cKit+/Sca1+ (LSK) cells in mice. Effects on HSPCs could explain the ability of Plinabulin to not only treat CIN but also to reduce chemotherapy-induced thrombocytopenia and increase circulating CD34+ cells in patients.

Cautionary Note Regarding Forward-Looking StatementsThis press release includes forward-looking statements that are not historical facts. Words such as "will," "expect," "anticipate," "plan," "believe," "design," "may," "future," "estimate," "predict," "objective," "goal," or variations thereof and variations of such words and similar expressions are intended to identify such forward-looking statements. Forward-looking statements are based on BeyondSpring's current knowledge and its present beliefs and expectations regarding possible future events and are subject to risks, uncertainties and assumptions. Actual results and the timing of events could differ materially from those anticipated in these forward-looking statements as a result of several factors including, but not limited to, difficulties raising the anticipated amount needed to finance the Company's future operations on terms acceptable to the Company, if at all, unexpected results of clinical trials, delays or denial in regulatory approval process, results that do not meet our expectations regarding the potential safety, the ultimate efficacy or clinical utility of our product candidates, increased competition in the market, and other risks described in BeyondSprings most recent Form 20-F on file with the U.S. Securities and Exchange Commission. All forward-looking statements made herein speak only as of the date of this release and BeyondSpring undertakes no obligation to update publicly such forward-looking statements to reflect subsequent events or circumstances, except as otherwise required by law.

Media ContactsCaitlin Kasunich / Raquel ConaKCSA Strategic Communications212.896.1241 / 212.896.1276ckasunich@kcsa.com / rcona@kcsa.com

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CELLECTAR BIOSCIENCES : Management’s Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) – marketscreener.com

Tuesday, August 11th, 2020

Overview

We are a clinical stage biopharmaceutical company focused on the discovery,development and commercialization of drugs for the treatment of cancer. We aredeveloping proprietary drugs independently and through research and developmentcollaborations. Our core objective is to leverage our proprietary phospholipiddrug conjugate (PDC) delivery platform to develop PDCs that are designed tospecifically target cancer cells and deliver improved efficacy and better safetyas a result of fewer off-target effects. Our PDC platform possesses thepotential for the discovery and development of the next generation ofcancer-targeting treatments, and we plan to develop PDCs both independently andthrough research and development collaborations. The COVID-19 pandemic hascreated uncertainties in the expected timelines for clinical stagebiopharmaceutical companies such as us, and because of such uncertainties, it isdifficult for us to accurately predict expected outcomes at this time. We havenot yet experienced any significant impacts as a result of the pandemic and havecontinued to enroll patients in our clinical trials. However, COVID-19 mayimpact our future ability to recruit patients for clinical trials, obtainadequate supply of CLR 131 and obtain additional financing.

Our lead PDC therapeutic, CLR 131 is a small-molecule PDC designed to providetargeted delivery of iodine-131 directly to cancer cells, while limitingexposure to healthy cells. We believe this profile differentiates CLR 131 frommany traditional on-market treatment options. CLR 131 is the company's leadproduct candidate and is currently being evaluated in a Phase 2 study inrelapsed/refractory (r/r) B-cell malignancies, including multiple myeloma (MM),chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),lymphoplasmacytic lymphoma/Waldenstrom's macroglobulinemia (LPL/WM), marginalzone lymphoma (MZL), mantle cell lymphoma (MCL), and diffuse large B-celllymphoma (DLBCL).CLR 131 is also being evaluated in a Phase 1 dose escalationstudy in pediatric solid tumors and lymphoma. The U.S. Food and DrugAdministration ("FDA") granted CLR 131 Fast Track Designation for both r/r MMand r/r DLBCL and Orphan Drug Designation (ODD) of MM, LPL/WM, neuroblastoma,rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma. CLR 131 was also grantedRare Pediatric Disease Designation (RPDD) for the treatment of neuroblastoma,rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma. Most recently, the EuropeanCommission granted an ODD for r/r MM.

Our product pipeline also includes one preclinical PDC chemotherapeutic program(CLR 1900) and several partnered PDC assets. The CLR 1900 Series is beingtargeted for solid tumors with a payload that inhibits mitosis (cell division) avalidated pathway for treating cancers.

We have leveraged our PDC platform to establish four collaborations featuringfive unique payloads and mechanisms of action. Through research and developmentcollaborations, our strategy is to generate near-term capital, supplementinternal resources, gain access to novel molecules or payloads, accelerateproduct candidate development and broaden our proprietary and partnered productpipelines.

Our PDC platform provides selective delivery of a diverse range of oncologicpayloads to cancerous cells, whether a hematologic cancer or solid tumor, aprimary tumor, or a metastatic tumor and cancer stem cells. The PDC platform'smechanism of entry does not rely upon specific cell surface epitopes or antigensas are required by other targeted delivery platforms. Our PDC platform takesadvantage of a metabolic pathway utilized by all tumor cell types in all stagesof the tumor cycle. Tumor cells modify specific regions on the cell surface as aresult of the utilization of this metabolic pathway. Our PDCs bind to theseregions and directly enter the intracellular compartment. This mechanism allowsthe PDC molecules to accumulate over time, which enhances drug efficacy, and toavoid the specialized highly acidic cellular compartment known as lysosomes,which allows a PDC to deliver molecules that previously could not be delivered.Additionally, molecules targeting specific cell surface epitopes face challengesin completely eliminating a tumor because the targeted antigens are limited inthe total number on the cell surface, have longer cycling time frominternalization to being present on the cell surface again and available forbinding and are not present on all of the tumor cells in any cancer. This meansa subpopulation of tumor cells always exist that cannot be targeted by therapiestargeting specific surface epitopes. In addition to the benefits provided by themechanism of entry, PDCs offer the ability to conjugate payload molecules innumerous ways, thereby increasing the types of molecules selectively deliveredvia the PDC.

The PDC platform features include the capacity to link with almost any molecule,provide a significant increase in targeted oncologic payload delivery and theability to target all types of tumor cells. As a result, we believe that we cangenerate PDCs to treat a broad range of cancers with the potential to improvethe therapeutic index of oncologic drug payloads, enhance or maintain efficacywhile also reducing adverse events by minimizing drug delivery to healthy cells,and increasing delivery to cancerous cells and cancer stem cells.

We employ a drug discovery and development approach that allows us toefficiently design, research and advance drug candidates. Our iterative processallows us to rapidly and systematically produce multiple generations ofincrementally improved targeted drug candidates.

In June 2020, the European Medicines Agency (EMA) granted us Small andMedium-Sized Enterprise status by the EMA's Micro, Small and Medium-sizedEnterprise office. SME status allows us to participate in significant financialincentives that include a 90% to 100% EMA fee reduction for scientific advice,clinical study protocol design, endpoints and statistical considerations,quality inspections of facilities and fee waivers for selective EMA pre andpost-authorization regulatory filings, including orphan drug and PRIMEdesignations. We are also eligible to obtain EMA certification of quality andmanufacturing data prior to review of clinical data. Other financial incentivesinclude EMA-provided translational services of all regulatory documents requiredfor market authorization, further reducing the financial burden of the marketauthorization process.

A description of our PDC product candidates follows:

Our lead PDC therapeutic, CLR 131 is a small-molecule, PDC designed to providetargeted delivery of iodine-131 directly to cancer cells, while limitingexposure to healthy cells. We believe this profile differentiates CLR 131 frommany traditional on-market treatments and treatments in development. CLR 131 iscurrently being evaluated in a Phase 2 study in r/r B-cell lymphomas, and twoPhase 1 dose-escalating clinical studies, one in r/r MM and one in r/r pediatricsolid tumors and lymphoma. The initial Investigational New Drug (IND)application was accepted by the FDA in March 2014 with multiple INDs submittedsince that time. Initiated in March 2017, the primary goal of the Phase 2 studyis to assess the compound's efficacy in a broad range of hematologic cancers.The Phase 1 study is designed to assess the compound's safety and tolerabilityin patients with r/r MM (to determine maximum tolerated dose) and was initiatedin April 2015. The FDA previously accepted our IND application for a Phase 1open-label, dose escalating study to evaluate the safety and tolerability of asingle intravenous administration of CLR 131 in up to 30 children andadolescents with cancers including neuroblastoma, sarcomas, lymphomas (includingHodgkin's lymphoma) and malignant brain tumors. This study was initiated duringthe first quarter of 2019. These cancer types were selected for clinical,regulatory and commercial rationales, including the radiosensitive nature andcontinued unmet medical need in the r/r setting, and the rare diseasedeterminations made by the FDA based upon the current definition within theOrphan Drug Act.

In December 2014, the FDA granted ODD for CLR 131 for the treatment of MM.Multiple myeloma is an incurable cancer of the plasma cells and is the secondmost common form of hematologic cancers. In 2018, the FDA granted ODD and RPDDfor CLR 131 for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing'ssarcoma and osteosarcoma. The FDA may award priority review vouchers to sponsorsof rare pediatric disease products that meet its specified criteria. The keycriteria to receiving a priority review voucher is that the disease beingtreated is life-threatening and that it primarily effects individuals under theage of 18. Under this program, a sponsor who receives an approval for a drug orbiologic for a rare pediatric disease can receive a priority review voucher thatcan be redeemed to receive a priority review of a subsequent marketingapplication for a different product. Additionally, these priority reviewvouchers can be exchanged or sold to other companies for them to use thevoucher. In May 2019, the FDA granted Fast Track designation for CLR 131 for thetreatment of multiple myeloma in July 2019 for the treatment of DLBCL, inSeptember, CLR 131 received Orphan Drug Designation from the European Union forMultiple Myeloma, and in January 2020, the FDA granted Orphan Drug Designationfor CLR 131 in lymphoplasmacytic lymphoma (LPL).

Phase 2 Study in Patients with r/r select B-cell Malignancies

In February 2020, we announced positive data from our Phase 2 CLOVER-1 study inpatients with relapsed/refractory B-cell lymphomas. Relapsed/Refractory MM andnon-Hodgkin lymphoma (NHL) patients were treated with three different doses(<50mCi, ~50mCi and ~75mCi total body dose (TBD). The <50mCi total body dose wasa deliberately planned sub-therapeutic dose. CLR 131 achieved the primaryendpoint for the study. Patients with r/r MM who received the highest dose ofCLR 131 showed a 42.8% overall response rate (ORR). Those who received ~50mCiTBD had a 26.3% ORR with a combined rate of 34.5% ORR (n=33) while maintaining awell-tolerated safety profile. Patients in the studies were elderly with amedian age of 70, and heavily pre-treated, with a median of five prior lines oftreatment (range: 3 to 17), which included immunomodulatory drugs, proteasomeinhibitors and CD38 antibodies for the majority of patients. Additionally, amajority of the patients (53%) were quad refractory or greater and 44% of alltreated multiple myeloma patients were triple class refractory. 100% of allevaluable patients (n=43) achieved clinical benefit (primary outcome measure) asdefined by having stable disease or better. 85.7% of multiple myeloma patientsreceiving the higher total body dose levels of CLR 131 experienced tumorreduction. The 75mCi TBD demonstrated positive activity in both high-riskpatients and triple class refractory patients with a 50% and 33% ORR,respectively.

Patients with r/r NHL who received ~50mCi TBD and the ~75mCi TBD had a 42% and43% ORR, respectively and a combined rate of 42%. These patients were alsoheavily pre-treated, having a median of three prior lines of treatment (range, 1to 9) with the majority of patients being refractory to rituximab and/oribrutinib. The patients had a median age of 70 with a range of 51 to 86. Allpatients had bone marrow involvement with an average of 23%. In addition tothese findings, subtype assessments were completed in the r/r B-cell NHLpatients. Patients with DLBCL demonstrated a 30% ORR with one patient achievinga complete response (CR), which continues at nearly 24 months post-treatment.The ORR for CLL/SLL/MZL patients was 33%. Current data from our Phase 2 CLOVER-1clinical study show that four LPL/WM patients demonstrated 100% ORR with onepatient achieving a CR which continues at nearly 27 months post-treatment. Thismay represent an important improvement in the treatment of relapsed/refractoryLPL/WM as we believe no approved or late-stage development treatments forsecond- and third-line patients have reported a CR. LPL/WM is a rare, indolentand incurable form of NHL that is composed of a patient population in need ofnew and better treatment options.

The most frequently reported adverse events in r/r MM patients were cytopenias,which followed a predictable course and timeline. The frequency of adverseevents have not increased as doses were increased and the profile of cytopeniasremains consistent. Importantly, these cytopenias have had a predictable patternto initiation, nadir and recovery and are treatable. The most common grade ?3events at the highest dose (75mCi TBD) were hematologic toxicities includingthrombocytopenia (65%), neutropenia (41%), leukopenia (30%), anemia (24%) andlymphopenia (35%). No patients experienced cardiotoxicities, neurologicaltoxicities, infusion site reactions, peripheral neuropathy, allergic reactions,cytokine release syndrome, keratopathy, renal toxicities, or changes in liverenzymes. The safety and tolerability profile in patients with r/r NHL wassimilar to r/r MM patients except for fewer cytopenias of any grade. Based uponCLR 131 being well tolerated across all dose groups and the observed responserate, especially in difficult to treat patients such as high risk and tripleclass refractory or penta-refractory, and corroborating data showing thepotential to further improve upon current ORRs and durability of thoseresponses, the study has been expanded to test a two-cycle dosing optimizationregimen of CLR 131.

In July 2016, we were awarded a $2,000,000National Cancer Institute (NCI)Fast-Track Small Business Innovation Research grant to further advance theclinical development of CLR 131. The funds are supporting the Phase 2 studyinitiated in March 2017 to define the clinical benefits of CLR 131 in r/r MM andother niche hematologic malignancies with unmet clinical need. These nichehematologic malignancies include Chronic Lymphocytic Leukemia, Small LymphocyticLymphoma, Marginal Zone Lymphoma, Lymphoplasmacytic Lymphoma and DLBCL. Thestudy is being conducted in approximately 10 U.S. cancer centers in patientswith orphan-designated relapse or refractory hematologic cancers. The study'sprimary endpoint is clinical benefit response (CBR), with additional endpointsof ORR, progression free survival (PFS,) median Overall Survival (mOS) and othermarkers of efficacy following a single 25.0 mCi/m2 dose of CLR 131, with theoption for a second 25.0 mCi/m2dose approximately 75-180 days later. Based onthe performance results from Cohort 5 of our Phase 1 study in patients with r/rMM, reviewed below, we have modified the dosing regimen of this study to afractionated dose of 15.625 mCi/m2 administered on day 1 and day 8.

In May 2020, we announced that the FDA granted Fast Track Designation for CLR131 in LPL/WM in patients having received two prior treatment regimens or more.

Phase 1 Study in Patients with r/r Multiple Myeloma

In February 2020, we announced the successful completion of our Phase 1 doseescalation study. Data from the study demonstrated that CLR 131 was safe andtolerated at total body dose of approximately 90mCi in r/r MM. The Phase 1multicenter, open-label, dose-escalation study was designed to evaluate thesafety and tolerability of CLR 131 administered as a 30-minute I.V. infusion,either as a single bolus dose or as two fractionated doses. The r/r multiplemyeloma patients in this study received single cycle doses ranging fromapproximately 20mCi to 90mCi total body dose. To date, an independent DataMonitoring Committee determined that all doses have been safe and well-toleratedby patients.

CLR 131 in combination with dexamethasone is currently under investigation inadult patients with r/r MM. Patients must have been refractory to or relapsedfrom at least one proteasome inhibitor and at least one immunomodulatory agent.The clinical study is a standard three-plus-three dose escalation safety studyto determine the maximum tolerable dose. Multiple myeloma is an incurable cancerof the plasma cells and is the second most common form of hematologic cancers.Secondary objectives include the evaluation of therapeutic activity by assessingsurrogate efficacy markers, which include M protein, free light chain (FLC), PFSand OS. All patients have been heavily pretreated with an average of five priorlines of therapy. CLR 131 was deemed by an Independent Data Monitoring Committee(IDMC) to be safe and tolerable up to its planned maximum single, bolus dose of31.25 mCi/m2. The four single dose cohorts examined were: 12.5 mCi/m2(~25mCiTBD), 18.75 mCi/m2 (~37.5mCi TBD), 25 mCi/m2(~50mCi TBD), and 31.25mCi/m2(~62.5mCi TBD), all in combination with low dose dexamethasone (40 mgweekly). Of the five patients in the first cohort, four achieved stable diseaseand one patient progressed at Day 15 after administration and was taken off thestudy. Of the five patients admitted to the second cohort, all five achievedstable disease however one patient progressed at Day 41 after administration andwas taken off the study. Four patients were enrolled to the third cohort and allachieved stable disease. In September 2017, we announced results for cohort 4,showing that a single infusion up to 30-minutes of 31.25mCi/m2 of CLR 131 wassafe and tolerated by the three patients in the cohort. Additionally, all threepatients experienced CBR with one patient achieving a partial response (PR). Weuse the International Myeloma Working Group (IMWG) definitions of response,which involve monitoring the surrogate markers of efficacy, M protein and FLC.The IMWG defines a PR as a greater than or equal to 50% decrease in FLC levels(for patients in whom M protein is unmeasurable) or 50% or greater decrease in Mprotein. The patient experiencing a PR had an 82% reduction in FLC. This patientdid not produce M protein, had received seven prior lines of treatment includingradiation, stem cell transplantation and multiple triple combination treatmentsincluding one with daratumumab that was not tolerated. One patient experiencingstable disease attained a 44% reduction in M protein. In January 2019, weannounced that the pooled mOS data from the first four cohorts was 22.0 months.In late 2018, we modified this study to evaluate a fractionated dosing strategyto potentially increase efficacy and decrease adverse events.

Following the determination that all prior dosing cohorts were safe andtolerated, we initiated a cohort 7 utilizing a 40mCi/m2 fractionated doseadministered 20mCi/m2 (~40mCi TBD) on days 1 and day 8. Cohort 7 was the highestpre-planned dose cohort and subjects have completed the evaluation period. Finalstudy report and study close-out will be completed later this year.

In May 2019, we announced that the FDA granted Fast Track Designation for CLR131 in fourth line or later r/r MM. CLR 131 is our small-moleculeradiotherapeutic PDC designed to deliver cytotoxic radiation directly andselectively to cancer cells and cancer stem cells. It is currently beingevaluated in our ongoing CLOVER-1 Phase 2 clinical study in patients withrelapsed or refractory multiple myeloma and other select B-cell lymphomas.

Phase 1 Study in r/r Pediatric Patients with select Solid tumors, Lymphomas andMalignant Brain Tumors

In December 2017 the Division of Oncology at the FDA accepted our IND and studydesign for the Phase 1 study of CLR 131 in children and adolescents with selectrare and orphan designated cancers. This study was initiated during the firstquarter of 2019. In December 2017, we filed an IND application for r/r pediatricpatients with select solid tumors, lymphomas and malignant brain tumors. ThePhase 1 clinical study of CLR 131 is an open-label, sequential-group,dose-escalation study evaluating the safety and tolerability of intravenousadministration of CLR 131 in up to 30 children and adolescents with cancersincluding neuroblastoma, sarcomas, lymphomas (including Hodgkin's lymphoma) andmalignant brain tumors. Secondary objectives of the study are to identify therecommended Phase 2 dose of CLR 131 and to determine preliminary antitumoractivity (treatment response) of CLR 131 in children and adolescents. In 2018,the FDA granted OD and RPDD for CLR 131 for the treatment of neuroblastoma,rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma. Should any of theseindications reach approval, the RPDD would enable us to receive a priorityreview voucher. Priority review vouchers can be used by the sponsor to receivepriority review for a future New Drug Application ("NDA") or Biologic LicenseApplication ("BLA") submission, which would reduce the FDA review time from 12months to six months. Currently, these vouchers can also be transferred or soldto another entity.

Phase 1 Study in r/r Head and Neck Cancer

In August 2016, the University of Wisconsin Carbone Cancer Center ("UWCCC") wasawarded a five-year Specialized Programs of Research Excellence ("SPORE") grantof $12,000,000 from the National Cancer Institute and the National Institute ofDental and Craniofacial Research to improve treatments and outcomes for head andneck cancer, HNC, patients. HNC is the sixth most common cancer across the worldwith approximately 56,000 new patients diagnosed every year in the U.S. As a keycomponent of this grant, the UWCCC researchers completed testing of CLR 131 invarious animal HNC models and initiated the first human clinical trial enrollingup to 30 patients combining CLR 131 and external beam radiation with recurrentHNC in Q4 2019. This clinical trial was suspended due to the COVID-19 pandemicbut has now been reopened for enrolment.

We believe our PDC platform has potential to provide targeted delivery of adiverse range of oncologic payloads, as exemplified by the product candidateslisted below, that may result in improvements upon current standard of care("SOC") for the treatment of a broad range of human cancers:

Research and development expense. Research and development expense consist ofcosts incurred in identifying, developing and testing, and manufacturing productcandidates, which primarily include salaries and related expenses for personnel,cost of manufacturing materials and contract manufacturing fees paid to contractmanufacturers and contract research organizations, fees paid to medicalinstitutions for clinical trials, and costs to secure intellectual property. TheCompany analyzes its research and development expenses based on four categoriesas follows: clinical project costs, preclinical project costs, manufacturing andrelated costs, and general research and development costs that are not allocatedto the functional project costs, including personnel costs, facility costs,related overhead costs and patent costs.

General and administrative expense. General and administrative expense consistsprimarily of salaries and other related costs for personnel in executive,finance and administrative functions. Other costs include insurance, costs forpublic company activities, investor relations, directors' fees and professionalfees for legal and accounting services.

Three Months Ended June 30, 2020 and 2019

Research and Development. Research and development expense for the three monthsended June 30, 2020 was approximately $2,465,000 compared to approximately$1,810,000 for the three months ended June 30, 2019.

The following table is an approximate comparison summary of research anddevelopment costs for the three months ended June 30, 2020 and June 30, 2019:

General research and development costs 1,018,000 384,000 634,000

The overall increase in research and development expense of $655,000, or 36%,was primarily a result of increased general research and development costsresulting from increased personnel related costs and in clinical project costs.Manufacturing and related costs decreased due to a decrease in materialsproduction processes and related costs. Pre-clinical study costs were relativelyconsistent.

General and administrative. General and administrative expense for the threemonths ended June 30, 2020 was approximately $1,157,000, compared toapproximately $1,391,000 in the three months ended June 30, 2019. The decreaseof approximately $234,000, or 17%, was primarily a result of lower stock-basedcompensation expense.

Six Months Ended June 30, 2020 and 2019

Research and Development. Research and development expense for the six monthsended June 30, 2020 was approximately $5,082,000 compared to approximately$4,118,000 for the six months ended June 30, 2019.

The following table is a comparison summary of research and development costsfor the six months ended June 30, 2020 and June 30, 2019:

General research and development costs 1,779,000 914,000 865,000

The overall increase in research and development expense of approximately$964,000, or 23%, was primarily a result of increased general research anddevelopment costs resulting from increased personnel related costs and inclinical project costs. Manufacturing and related costs decreased due to adecrease in materials production processes and related costs. Pre-clinical studycosts were relatively consistent.

General and Administrative. General and administrative expense for the sixmonths ended June 30, 2020 was approximately $2,499,000, compared toapproximately $2,712,000 in the six months ended June 30, 2019. The decrease ofapproximately $213,000, or 8%, was primarily a result of lower stock-basedcompensation expense.

Liquidity and Capital Resources

As of June 30, 2020, we had cash and cash equivalents of approximately$22,450,000 compared to $10,615,000 as of December 31, 2019. This increase wasdue primarily to the approximately $18,300,000 of net proceeds received inconnection with the June 5, 2020 public offering. Net cash used in operatingactivities during the six months ended June 30, 2020 was approximately$6,562,000.

Our cash requirements have historically been for our research and developmentactivities, finance and administrative costs, capital expenditures and overallworking capital. We have experienced negative operating cash flows sinceinception and have funded our operations primarily from sales of common stockand other securities. As of June 30, 2020, we had an accumulated deficit ofapproximately $119,251,000.

We believe that the cash balance is adequate to fund our basic budgetedoperations for at least 12 months from the filing of these financial statements.However, our future results of operations involve significant risks anduncertainties. Our ability to execute our operating plan beyond that timedepends on our ability to obtain additional funding via the sale of equityand/or debt securities, a strategic transaction or otherwise. We plan toactively pursue all available financing alternatives; however, there can be noassurance that we will obtain the necessary funding. Other than theuncertainties regarding our ability to obtain additional funding, there arecurrently no known trends, demands, commitments, events or uncertainties thatare likely to materially affect our liquidity. Because we have had recurringlosses and negative cash flows from operating activities, and in light of ourexpected expenditures, the report of our independent auditors with respect tothe financial statements as of December 31, 2019 and for the year ended December31, 2019 contains an explanatory paragraph as to the potential inability tocontinue as a going concern. This opinion indicated at that time, thatsubstantial doubt existed regarding our ability to remain in business.

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