The biotech sector was in focus with normal pipeline, regulatory and collaboration updates. Data readouts at the European Society for Medical Oncology (ESMO) Virtual Congress 2020 also grabbed headlines.
Recap of the Week’s Most Important Stories:
Bristol-Myers & bluebird’s CAR T Drug Gets Priority Review: Bristol Myers Squibb BMY and partner bluebird bio, Inc. BLUE announced that the FDA has accepted their Biologics License Application (BLA) for pipeline candidate, idecabtagene vicleucel (ide-cel; bb2121), for Priority Review.
Idecabtagene vicleucel is an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, which is being evaluated for the treatment of adult patients with multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of Mar 27, 2021.
Earlier, Bristol Myers Squibb announced initial results from the phase III CheckMate -577 trial, wherein adjuvant treatment with Opdivo (nivolumab) showed a statistically significant and clinically meaningful improvement in disease-free survival (DFS) compared to placebo in patients with esophageal or gastroesophageal junction (GEJ) cancer following neoadjuvant chemoradiation therapy (CRT) and tumor resection.
Separately, the company also reported primary results from the phase III study CheckMate -649 at the ESMP Virtual Congress 2020. Results showed that Opdivo (nivolumab) plus chemotherapy showed a statistically significant and clinically meaningful improvement in the overall survival (OS) and progression-free survival (PFS) of patients with unresectable advanced or metastatic gastric cancer, GEJ cancer or esophageal adenocarcinoma compared to treatment with chemotherapy alone.
Meanwhile, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Opdivo plus Yervoy (ipilimumab) with two cycles of platinum-based chemotherapy for the first-line treatment of metastatic non-small cell lung cancer (NSCLC) in adults whose tumors have no sensitizing EGFR mutation or ALK translocation.
Vaccinex Plunges on Study Data: Shares of clinical-stage biotechnology company, Vaccinex, Inc. VCNX, plunged after it announced disappointing data on lead clinical candidate, pepinemab, in patients with early manifest and prodromal Huntington’s disease (HD).
Top-line results from the early manifest treatment arm (Cohort B1, N=179) of the phase II double-blind, placebo-controlled SIGNAL trial showed that the study did not meet pre-specified co-primary endpoints — a family of two cognitive assessments from the Huntington’s Disease Cognitive Assessment Battery and Clinical Global Impression of Change (CGIC). Nevertheless, results support continued development in Alzheimer’s disease and mid-stage Huntington’s disease patients with greater cognitive deficits.
Separately, the company announced that it has entered into a clinical collaboration agreement with Merck to evaluate the combination of pepinemab and the latter’s anti-PD-1 therapy, Keytruda (pembrolizumab), for the treatment of patients with advanced, recurrent or metastatic head and neck squamous cell carcinoma (HNSCC).
Blueprint Gains on Positive Study Data: Shares of Blueprint Medicines Corporation BPMC were up after it announced positive top-line data from the phase I EXPLORER study and the phase II PATHFINDER study, which evaluated its lead drug, Ayvakit (avapritinib), for treating patients with advanced systemic mastocytosis (SM), a rare, debilitating disease. Data from both studies showed that treatment with Ayvakit led to profound reductions in mast cell burden, high overall response and complete remission rates, as well as durable clinical benefit, including prolonged median overall survival (OS) in the patient population. The drug was generally well-tolerated and reinforced its safety profile at the once-daily 200 mg dose. Blueprint plans to submit a supplemental new drug application (sNDA) to the FDA for Ayvakit for the treatment of advanced SM in the fourth quarter of 2020.
Arrowhead Surges on Phase II Data: Arrowhead Pharmaceuticals Inc. ARWR announced positive interim 24-week liver biopsy results in four subjects from the open-label phase II study of ARO-AAT, its second-generation investigational RNA interference (RNAi) therapeutic being developed as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD). Data from AROAAT2002 showed evidence of a meaningful pharmacodynamic effect by ARO-AAT, leading to improvements in relevant biomarkers, including substantial reductions in intra-hepatic mutant AAT protein. After 24 weeks of treatment with investigational ARO-AAT in the AROAAT2002 study, serum and total intra-hepatic Z-AAT decreased in all four patients by up to 93% and 95%, respectively.
Seattle Genetics' Padcev Reports Positive Data: Seattle Genetics, Inc. SGEN and partner Astellas Pharma Inc. announced that a phase III study of Padcev (enfortumab vedotin-ejfv) met its primary endpoint of overall survival compared to chemotherapy. The results were reviewed by an independent Data Monitoring Committee, following a planned interim analysis. The global EV-301 clinical study compared Padcev to chemotherapy in adult patients with locally advanced or metastatic urothelial cancer who were previously treated with platinum-based chemotherapy and a PD-1/L1 inhibitor. Padcev significantly improved overall survival (OS), with a 30% reduction in risk of death. Padcev also significantly improved progression-free survival (PFS), a secondary endpoint, with a 39% reduction in risk of disease progression or death.
Medical - Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology index lost 1.60% in the last five trading sessions. Among the biotech giants, Vertex gained 0.27% during this period. Over the past six months, shares of Alexion have gained 41.14%. (See the last biotech stock roundup here: Biotech Stock Roundup: GILD to Buy IMMU, SGEN Collaborates With Merck & More)
What's Next in Biotech?
Stay tuned for more pipeline updates.
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