Welcome to your weekly digest of approaching regulatory and clinical readouts. Puma's (NYSE:PBYI) breast cancer project neratinib will go before a US panel on May 24, but Roche's (OTCQX:RHHBY) rival Perjeta could soon show clinical data that render neratinib almost irrelevant, and the recent departure of a senior member of Puma's staff has fueled rumors of a negative outcome at the adcom.
On the same day Emmaus Life Sciences will go before the same FDA panel for its sickle cell disease project Endari, currently availbale as a nutraceutical. Regulators have questioned the efficacy analysis in the past, but with few options for patients approval could still ensue.
Puma becomes the prey
In just over a week a panel will discuss Puma's neratinib for use as an adjuvant treatment in Her2-overexpressing breast cancer. The filing was based on the phase III Extenet trial, in which patients received neratinib monotherapy after a year's Herceptin.
Puma has a number of hurdles in its path, the biggest of which is Perjeta. A positive result in Roche's phase III Aphinity study indicated that Perjeta could potentially be practice-changing for breast cancer, and Puma's shares dropped 14% (Aphinity all but confirms Puma's worst nightmare, March 2, 2017).
Roche's trial gave patients Perjeta plus Herceptin for a year versus Herceptin alone. Details, including the extent of Perjeta's benefit, will appear at Asco on June 5.
Other issues for Puma include the severe diarrhea seen with neratinib, questions over neratinib's marginal benefit in Extenet trial, and large numbers of patients being censored.
This week Puma shares took another tumble on the news that its senior vice-president of regulatory affairs, Dr. Robert Charnas, would step down. Speculation that the resignation could be linked with the upcoming panel meeting, rather than the health reasons cited in the company's 8-K, caused shares to fall 16%.
Neratinib sales are forecast to reach $809m by 2022, according to sellside consensus from EvaluatePharma, but just over a year ago these sat at $2bn. Neratinib's PDUFA date is set for July; a strong benefit with Perjeta could be hard for the FDA to ignore.
Lacking options
Emmaus Life Sciences' Endari, in sickle cell disease, will also face the panel on May 24, and has a PDFUA date set for July 7.
The Californian group says that if Endari is approved it will become the first treatment for pediatric patients and the first new therapy in nearly 20 years for adult patients. However, it does not treat the underlying cause of the disease, instead aiming to reduce the frequency of the episodes of pain called sickle cell crises. It has orphan drug designation in the US and EU and US fast-track designation.
Endari is said to work by reducing oxidative damage to red blood cells. It consists of the amino acid L-glutamine as a nutritional supplement - the same active ingredient as the company's Nutrestore nutraceutical, available for short bowel syndrome in combination with recombinant human growth hormone. Endari is filed as a drug under the 505(b)(2) pathway.
The phase III trial was conducted in 230 patients aged five and over. It tested 0.3g/kg of L-glutamine twice daily for 48 weeks, up to a daily limit of 30g. The powder is mixed with water or certain non-heated foods.
The placebo comparator was the food additive maltodextrin, and two thirds of patients were on background hydroxyurea treatment, an established drug used to reduce crises. The company reported a 25% reduction in the median frequency of sickle cell crises, with Endari patients having a median of three over the 48-week trial.
Initially the company reported a p value of 0.063, above the pre-specified p value of 0.045. However, using a different analysis it said the results were "highly statistically significant" at 0.005.
The FDA had raised concerns over the primary endpoint analysis after the data were initially unblinded in 2014. Emmaus provided a report by an independent statistician explaining the significant results using the modified Ridit method, adjusting for varying strata or subgroup size, which in this case was region and hydroxyurea use.
No doubt the panel meeting will pay close attention to the statistical analyses used.
Editor's Note: This article discusses one or more securities that do not trade on a major U.S. exchange. Please be aware of the risks associated with these stocks.
Read more here:
Panel Woes For Puma And Statistical Uncertainties For Emmaus - Seeking Alpha
- Biotechnology Research Among Science Returned to Earth - ISS National Lab - May 5th, 2024
- Program to prepare next generation of biotech experts receives renewed funding - The University of Rhode Island - May 5th, 2024
- Designing drugs with reversible activity - Nature.com - May 5th, 2024
- ScienceLIVE helps answer 'What the Heck is Biotech?' for career-minded adults - UMass Medical School - May 5th, 2024
- Boston Micro Fabrication Launches BMF Biotechnology Inc. to Advance Use of 3D BioChips in Pharmaceutical and ... - Morningstar - May 5th, 2024
- Time to leverage intellectual property to drive innovation in seed and biotechnology sectors: Experts - The Times of India - May 5th, 2024
- Vir Biotechnology to Participate in the BofA Securities Healthcare Conference 2024 - Business Wire - May 5th, 2024
- WuXi Biologics withdraws from 2024 BIO convention amid worsening geopolitical tensions - FiercePharma - May 5th, 2024
- Boston Micro Fabrication Launches BMF Biotechnology Inc. to Advance Use of 3D BioChips in Pharmaceutical and ... - Business Wire - May 5th, 2024
- U.S. Biotechnology Market Size to Increase USD 1.79 Trillion by 2033 - BioSpace - April 16th, 2024
- 6 biotechnology universities in the USA developing young talent - Labiotech.eu - April 16th, 2024
- Lee Hsien Loong presided over efforts to retool Singapore's export-driven economy by focusing on advanced ... - NBC Right Now - April 16th, 2024
- UNITY Biotechnology, Inc. Reports Fourth Quarter and Full Year 2023 Financial Results and Business Updates - GlobeNewswire - April 16th, 2024
- Lee Hsien Loong presided over efforts to retool Singapore's export-driven economy by focusing on advanced ... - pdclarion.com - April 16th, 2024
- BIO's top lobbyist heads out the door - FiercePharma - April 16th, 2024
- BIO's top lobbyist exits, signaling reorganization - STAT - STAT - April 16th, 2024
- Biotech Rebound Could Boost This ETF - ETF Trends - April 16th, 2024
- US National Security, China and Biotechnology in 2024 - BioSpace - April 8th, 2024
- How to Invest in Biotechnology (Updated 2024) - Nasdaq - April 8th, 2024
- Self-pigmenting textiles grown from cellulose-producing bacteria with engineered tyrosinase expression - Nature.com - April 8th, 2024
- Genetically encoding colors and images into bioengineered microbial materials - Nature.com - April 8th, 2024
- Ambition and urgency: Biotechnology and Biomanufacturing in the EU - EURACTIV - April 8th, 2024
- Scientists create plastic-free vegan leather that dyes itself, grown from bacteria - Phys.org - April 8th, 2024
- For national security, the U.S. must maintain its biotech dominance - STAT - April 8th, 2024
- LLY, REGN, BHVN: Which Biotech Stock Offers the Most Upside? - Yahoo Finance - April 8th, 2024
- Is biotechnology a good career? Here are the pros and cons - Labiotech.eu - March 18th, 2024
- Saving Biology With Blue Biotechnology - The Maritime Executive - March 18th, 2024
- 30 Biggest Biotechnology Companies in the World - Yahoo Finance - March 18th, 2024
- WuXi out of biotech trade group, U.S. and China relations to blame - STAT - March 18th, 2024
- Can Biotechnology Save the Fashion Industry? - EARTH.ORG - March 18th, 2024
- Huber high-precision temperature control solutions for the pharmaceutical industry and biotechnology - Labmate Online - March 18th, 2024
- The twelve hottest biotech companies in the Boston area - Labiotech.eu - March 18th, 2024
- Vancouver readies for its position as a global pharma capital as B.C.'s biotech boom takes off - The Globe and Mail - March 10th, 2024
- Here's Why We're A Bit Worried About Unity Biotechnology's (NASDAQ:UBX) Cash Burn Situation - Simply Wall St - March 10th, 2024
- The Top 25 Biotechnology Companies of 2024 - The Healthcare Technology Report. - March 10th, 2024
- Near-cognate tRNAs increase the efficiency and precision of pseudouridine-mediated readthrough of premature ... - Nature.com - March 10th, 2024
- MAIA Biotechnology and Nationwide Children's Hospital Announce Presentation of THIO's Potency in Pediatric Brain ... - Business Wire - March 10th, 2024
- Global Pharmaceuticals and Biotechnology Distribution Deals Analysis Report and Directory 2024: Breakdown of ... - PR Newswire - March 10th, 2024
- Pharmaceuticals and Biotechnology Co-promotion and Co-marketing Partnering Trends Report 2024: Analysis of ... - PR Newswire - March 10th, 2024
- INTERNATIONAL BIOTECHNOLOGY TRUST has a healthy attitude towards paying investors - This is Money - March 10th, 2024
- Where Does Capricor Therapeutics Inc (CAPR) Stock Fall in the Biotechnology Field After It Has Risen 24.73% This Week? - InvestorsObserver - March 10th, 2024
- Biotechnology should be high on the agenda at the Munich Security Conference - The Hill - February 18th, 2024
- Boston is best in the world at biotech. Beijing wants to do it better - WBUR News - February 18th, 2024
- Multiomics Are The Next Step In Biotechnology - Securities.io - February 18th, 2024
- SPARKing academic technologies across the valley of death - Nature.com - February 18th, 2024
- Is Nurix Therapeutics Inc (NRIX) Stock at the Top of the Biotechnology Industry? - InvestorsObserver - February 18th, 2024
- Addressing the Threat to U.S. Biotechnology: Concerns Over Genetic Data Security - Medriva - February 18th, 2024
- Where Does SpringWorks Therapeutics Inc (SWTX) Stock Fall in the Biotechnology Field After It Has Gained 12.41% This Week? - InvestorsObserver - February 18th, 2024
- Should Biotechnology Stock Axsome Therapeutics Inc (AXSM) Be in Your Portfolio Wednesday? - InvestorsObserver - February 18th, 2024
- Where Does Candel Therapeutics Inc (CADL) Stock Fall in the Biotechnology Field After It Has Gained 22.38% This Week? - InvestorsObserver - February 18th, 2024
- Is Tempest Therapeutics Inc (TPST) a Leader in the Biotechnology Industry? - InvestorsObserver - February 18th, 2024
- Is Immunome Inc (IMNM) a Good Buy in the Biotechnology Industry? - InvestorsObserver - February 18th, 2024
- Is Larimar Therapeutics Inc (LRMR) a Leader in the Biotechnology Industry? - InvestorsObserver - February 18th, 2024
- US lawmakers, visiting Cambridge, warn of a rising Chinese threat to US biotech - The Boston Globe - February 18th, 2024
- Should Biotechnology Stock Viking Therapeutics Inc (VKTX) Be in Your Portfolio Monday? - InvestorsObserver - February 18th, 2024
- How the push to limit Chinese biotech could threaten U.S. edge - Axios - February 18th, 2024
- What is Biotechnology? Types and Applications - Iberdrola - January 25th, 2024
- BioAro Inc to Host Inaugural Longevity Innovation Forum in Dubai, UAE - Yahoo Finance - January 25th, 2024
- Is Akebia Therapeutics Inc (AKBA) a Bad Choice in Biotechnology Wednesday? - InvestorsObserver - January 25th, 2024
- Agricultural Biotechnology Market Projected to Reach USD 64.7 billion, with Advancing CAGR of 9.5% by 2031 ... - Yahoo Finance - January 25th, 2024
- Where Does Immunitybio Inc (IBRX) Stock Fall in the Biotechnology Field After It Is Lower By -9.32% This Week? - InvestorsObserver - January 25th, 2024
- Is Lyra Therapeutics Inc (LYRA) Stock at the Top of the Biotechnology Industry? - InvestorsObserver - January 25th, 2024
- ZWI Therapeutics Announces Closing of $10M Series A Financing - FinSMEs - January 25th, 2024
- Breaking Barriers: Tiamat Sciences Shatters Norms with All-Female Executive Team - Yahoo Finance - January 25th, 2024
- Can Sana Biotechnology Inc (SANA) Stock Rise to the Top of Healthcare Sector Monday? - InvestorsObserver - January 25th, 2024
- Is Cabaletta Bio Inc (CABA) a Leader in the Biotechnology Industry? - InvestorsObserver - January 25th, 2024
- Should You Buy Sana Biotechnology Inc (SANA) Stock After it Has Fallen 15.41% in a Week? - InvestorsObserver - January 25th, 2024
- Why Investors and Media are Calling Biotech Showcase The Place to Be to Start the Year - Business Wire - January 25th, 2024
- Is IGC Pharma Inc (IGC) Stock at the Top of the Biotechnology Industry? - InvestorsObserver - January 25th, 2024
- Is Summit Therapeutics Inc (SMMT) a Leader in the Biotechnology Industry? - InvestorsObserver - January 25th, 2024
- Biotech Stocks: The Top 5 To Watch As Shares Trend Down - Investor's Business Daily - January 25th, 2024
- Where Does Tscan Therapeutics Inc (TCRX) Stock Fall in the Biotechnology Field After It Is Lower By -9.81% This Week? - InvestorsObserver - January 25th, 2024
- HRMY: Are These 3 Biotech Stocks Worth Buying? - StockNews.com - January 25th, 2024
- Global Preparative Chromatography Market is projected to reach US$ 2,139.4 Million by 2034 Amid Growing ... - Yahoo Finance - January 25th, 2024
- Is Immunome Inc (IMNM) a Leader in the Biotechnology Industry? - InvestorsObserver - January 25th, 2024
- Should Biotechnology Stock Mink Therapeutics Inc (INKT) Be in Your Portfolio Monday? - InvestorsObserver - May 9th, 2023
- Where Does Ambrx Biopharma Inc - ADR (AMAM) Stock Fall in the Biotechnology Field After It Has Risen 22.18% This Week? - InvestorsObserver - May 9th, 2023
- Should Biotechnology Stock Dermata Therapeutics Inc (DRMA) Be in Your Portfolio Thursday? - InvestorsObserver - May 9th, 2023
- Should Biotechnology Stock Tempest Therapeutics Inc (TPST) Be in Your Portfolio Wednesday? - InvestorsObserver - May 9th, 2023
- Biotechnology - Applications of biotechnology | Britannica - May 1st, 2023