StemCell Therapy

bluebird on the Go with Multiple Positive Trial Data

bluebird bio (BLUE) announced data from its different trials, including Phase 1/2 clinical trial for sickle cell disease and from a Phase 3 clinical trial for transfusion-dependent beta-thalassemia patients.

Ongoing Phase 1/2 clinical trial HGB-206 aims to assess the potential of LentiGlobin in treating adolescents and adults with sickle cell disease. As on the data cutoff date, 37 patients had been treated. Group C showed promising results as all the patients included in the cohort stopped regular blood transfusions. These patients also remained transfusion-free for the time period of three months after the treatment.

Sixteen patients who were followed up for minimum six months maintained the median levels of gene therapy-derived anti-sickling hemoglobin. It also contributed to minimum 40 percent of total hemoglobin. 14 patients with previous background of acute chest syndrome (ACS) and vaso-occlusive crisis (VOC) who have been observed for a minimum time period of six months showed a 99.5 percent drop in the annualized rate of ACSs and VOCs.

Its ongoing Phase 3 clinical trials aimed to test the prospects of Zynteglo, formerly known as LentiGlobin, in treating adolescent and adult patients suffering from a range of genotypes of transfusion-dependent beta-thalassemia. Phase 3 of the Northstar-2 study involved 23 patients who were treated and observed for a median time period of 19.4 months. 89.5 percent of evaluable participants numbering 19 showed transfusion independence. Earlier, the 17 needed a median of 17.5 transfusions each year.

The Northstar-3 study involved 15 patients with genotypes of different variations. These patients were treated and then observed for a median time period of 14.4 months. 75.0% (n=6/8) reported to be transfusion independent. 84.6% (n=11/13) with a minimum of seven months' follow-up did not require a transfusion in more than seven months. Earlier, 11 of the patients needed a median of 18.5 transfusions every year.

bluebirds gene therapy involves extracting a patients stem cell and then altering it with new genetic instructions. Such altered stem cell is then infused back into the patients body. David Davidson, M.D., chief medical officer, bluebird bio, said, Vaso-occlusive crises (VOCs) are the painful, life-threatening episodes that are the primary clinical manifestation of sickle cell disease. The nearly complete elimination of VOCs that we saw in this study is impressive and demonstrates the potential of LentiGlobin for SCD as a treatment for this serious disease.

The Northstar-2 study showed three and two serious occurrences of veno-occlusive liver disease and thrombocytopenia respectively. Northstar-3 showed two serious occurrences of pyrexia. However, there was no reporting of any death, graft failure or leukemia. Zynteglo is already approved in the European Union, and the company is looking to start treating patients in Germany in the near future. It also plans to apply for the FDA approval in mid-2021.

For its Phase 1/2 trial in sickle cell anemia, an earlier report had shown significant improvement in VOCs and ACS after the gene therapy. Pursuant to these results, the company consulted with the FDA to alter the primary endpoint of the trial from improvement in hemoglobin measurements to the elimination of VOCs.

BeiGene (BGNE) announced new data from its trials aimed at assessing the potential of BTK inhibitor Brukinsa (zanubrutinib) and PD-1 inhibitor tislelizumab in treating various blood cancers. Brukinsa is a small molecule inhibitor of Brutons tyrosine kinase. It is currently being assessed as a monotherapy and in combination with other therapies of treating a wide range of B-cell malignancies.

For Phase 1/2 study of Brukinsa in patients suffering from B-cell malignancies, the overall response rate was 80 percent, while complete response rate was 15 percent. Further, the partial response rate for the trial was 65 percent. The median time to response was measured at 2.8 months. The progression-free survival rate and overall survival rate at 24 months were 59.4 percent and 83.2 percent respectively. Zanubrutinib was found to be well-tolerated in patients with R/R MZL, however, all patients experienced at least one adverse event.

The company also presented data from a Phase 2 trial of zanubrutinib with rituximab in patients suffering from R/R NHL. The trial had 41 participants, including 20 patients suffering from non-GCB DLBCL who previously received standard anthracycline rituximab-based treatment. For patients with R/R non-GCB DLBCL, the ORR was 35 percent and the CR and PR rates were 5 percent and 30 percent, respectively. For patients with R/R FL, the OR was 56.3 percent, with CR and PR at 19 percent and 38 percent, respectively.

Brukinsa is already approved in the United States for treating adult patients suffering from mantle cell lymphoma who have been given minimum one prior therapy. It was given the approval under accelerated approval pathway on the basis of its overall response rate. The drug is approved in China for treating MCL in adult patients who have received at least one prior therapy and CLL or SLL in adult patients who have received at least one prior therapy. However, the drug is not approved outside of the United States and China.

TG Therapeutics (TGTX) reported positive interim data from its Phase 1 parallel dose-escalation study of TG-1701. The drug candidate is being tested as a monotherapy and in combination with U2 for treating relapsed/refractory B-cell malignancies.

The data pertained to 82 patients suffering from the condition. Sixty-nine patients were given single agent TG-1701, out of which 25 were included in the monotherapy dose escalation cohort of the study and were given the drug candidate in 100mg to 400mg dosage once a day daily. The remaining 44 patients were included in the monotherapy dose expansion arm and were administered 200mg of the drug candidate. The TG-1701 plus U2 dose escalation portion of the study treated an additional 13 patients.

For the monotherapy dose escalation cohort with 25 patients, the drug candidate showed partial response for all the dose levels in CLL, MCL, WM and SLL. For the monotherapy dose expansion cohort, the overall response rate was recorded at 92 percent in CLL patients. MCL patients and WM patients showed 33 percent and 86 percent ORR respectively. The combination of the drug candidate with U2 showed positive clinical activity with a 77 percent ORR for all disease types. It included complete response rate in three patients. The company continues with dose escalation.

TG-1701 showed a positive preliminary safety profile for all dose levels. No patient had to discontinue the treatment, but 3 percent of the patients had to reduce the dose due to the occurrence of treatment-related adverse events. The company also provided data pertaining Phase I/Ib study aiming to assess the potential of ibrutinib in blend with umbralisib for patients with relapsed/refractory CLL or MCL.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, weve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Original post:
bluebird On The Go, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

This entry was posted on June 18, 2020 at 8:45 am and is filed under Gene therapy. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |