Regulatory and pipeline updates are back in focus this week in the biotech sector. While Celgene (CELG - Free Report) completed enrollment in trial on CAR T cell therapy, Vertex (VRTX - Free Report) announced positive data on CF candidate.
Recap of the Week’s Top Stories:
Celgene Completes Enrollment In Trial: Celgene and partner bluebird bio, Inc. (BLUE - Free Report) completed enrollment for the KarMMa pivotal study on lead investigational anti-BCMA CAR T cell therapy, bb2121. The candidate is being evaluated for patients with relapsed and refractory multiple myeloma. KarMMa is a pivotal, open-label, single-arm, multi-center phase II study, which evaluated the efficacy and safety of bb2121 in patients with relapsed and refractory multiple myeloma.
bb2121 was granted Breakthrough Therapy Designation (BTD) by the FDA and PRIority Medicines (PRIME) eligibility by the European Medicines Agency in Nov 2017. The designation was based on preliminary clinical data from the phase I CRB-401 study. The FDA action date for the bb2121 NDA is anticipated in 2020.
Celgene currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank stocks here.
Zafgen Plunges on Clinical Hold On Trial: Zafgen, Inc. (ZFGN - Free Report) shares plunged significantly after the FDA placed a clinical hold on the Investigational New Drug Application (IND) for its first U.S. clinical trial of ZGN-1061. ZGN-1061, the company’s second-generation, investigational MetAP2 inhibitor, is currently being evaluated for the treatment of type 2 diabetes. The company received a letter from the FDA last week mentioning the clinical hold, citing the possibility of cardiovascular (CV) safety risk as seen in the company’s prior compound. The agency has outlined multiple potential paths for moving forward, including nonclinical or clinical options, to address these concerns in the ongoing development of the candidate. As a result, Zafgen plans to assess these options and request a Type A meeting with the FDA to discuss further actions that can be taken regarding the program.
Zafgen now expects the cash runway to extend through calendar year 2020, given the delay of additional clinical development for the candidate.
Spark’s Luxturna Gets EC Nod: Spark Therapeutics, Inc. (ONCE - Free Report) announced that the European Commission (“EC”) has approved the marketing authorization application (“MAA”) for its gene therapy, Luxturna. The MAA sought approval for the therapy in adult as well as pediatric patients as a one-time treatment for vision loss due to inherited retinal dystrophy caused by a genetic mutation in both copies of the RPE65-gene and who have enough viable retinal cells. The RPE65 mutations, a progressive disease, can lead to total blindness. The EC decision was based on positive CHMP opinion. Data from a phase I clinical trial, its follow-up trial, and the first randomized, controlled phase III gene therapy trial for an inherited disease supported the decision.
Data from the phase III trial showed that vision improvement was recorded as early as 30 days following the treatment. Under the terms of the agreement, Spark Therapeutics received an upfront payment of $105 million and is eligible to receive additional $65 million in milestone payments from Novartis.
Vertex Reported Positive Data on CF Drug: Vertex Pharmaceuticals announced that two phase III studies evaluating its next-generation CFTR corrector, VX-659 in combination with tezacaftor and Kalydeco (ivacaftor) met the primary endpoint of improvement in lung function in patients suffering from cystic fibrosis ("CF"). The improvement in lung function was measured as mean absolute improvements in percent predicted forced expiratory volume in one second, or ppFEV1. While one study is evaluating the triple combination regimen in patients with one F508del mutation and one minimal function mutation, the other study is evaluating patients with two F508del mutations. In the study evaluating patients with one F508del mutation and one minimal function mutation, the interim data showed that the triple combination regimen led to a mean absolute improvement in ppFEV1 of 14.0 percentage points from baseline through week four of treatment compared with placebo. The second study yielded a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week four when VX-659 was added in patients already receiving tezacaftor and ivacaftor compared to placebo plus tezacaftor and ivacaftor.
Enrollment in the VX-445 phase III studies is now complete with top-line data expected to be presented in the first quarter of 2019. A regulatory submission in the United States for one of the two regimes is planned for mid-2019.
The NASDAQ Biotechnology index gained 22.1% in the last four trading sessions. Among the major biotech stocks, Vertex lost 6.2% in the last five trading sessions. Over the past six months, shares of Biogen have rallied 15% while Celgene has dropped 10.6%. (See the last biotech stock roundup here: Biotech Stock Roundup: GILD's Drug Wins Nod in China, Arena Up On Licensing Deal)
What's Next in Biotech?
Stay tuned for more regulatory and pipeline updates.
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