The biotech sector has been in the spotlight in the past week with pipeline and regulatory updates.
Recap of the Week’s Most Important Stories
:
Good news for bluebird bio
:
bluebird
BLUE
received a string of good news, which saw its share price surge significantly. The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (“CTGTAC”) has unanimously recommended
approval
of its gene therapy candidate betibeglogene autotemcel (beti-cel) to treat beta-thalassemia, a severe genetic blood disorder. The committee voted 13-0, unanimously recommending that the benefits of treatment with beti-cel outweigh the risks. A final decision by the FDA is expected by Aug 19, 2022. A tentative approval will make beti-cel the first potentially curative gene therapy option for people with beta-thalassemia who require regular red blood cell transfusions.
Earlier, CTGTAC unanimously recommended
approval
of its gene therapy candidate elivaldogene autotemcel (eli-cel) to treat cerebral adrenoleukodystrophy (CALD), a rare neurodegenerative disease. CTGTAC voted 15-0, recommending that the benefits of treatment with eli-cel outweigh the risks.
bluebird bio carries a Zacks Rank #3 (Hold) at present. You can see
the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here
.
Rigel Down on Pipeline Update
:
Rigel Pharmaceuticals, Inc
.
RIGL
slumped
significantly after the company announced that its late-stage study of fostamatinib was not successful. The phase III clinical trial, a global, multi-center, randomized, double-blind, placebo-controlled trial, FORWARD, is evaluating fostamatinib in patients with warm autoimmune hemolytic anemia (wAIHA). However, the study did not demonstrate statistical significance in the primary efficacy endpoint of durable hemoglobin response in the overall study population. Patients treated with fostamatinib had a favorable durable hemoglobin response compared to placebo, as shown in a post-hoc regional analysis of U.S., Canadian, Australian and Western European trial sites. However, in the Eastern European trial sites, patients did not show any such response.
Rigel’s fostamatinib is already approved in the United States under the brand name Tavalisse for the treatment of adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.
Valneva Tanks on Regulatory Update
:
Valneva SE
VALN
shares were
down
after the company provided an update on its European inactivated whole-virus COVID-19 vaccine candidate VLA200.Valneva has proposed a remediation plan after receiving the European Commission’s (“EC”) notice of intent to terminate the Advance Purchase Agreement (APA) for the vaccine last month.
The APA provides the EC with the right to terminate the agreement if, by Apr 30, 2022, VLA2001 did not receive marketing authorization from the European Medicines Agency (“EMA”). Valneva has 30 days from May 13, 2022, to obtain a marketing authorization or propose an acceptable remediation plan based on the terms of the APA. Valneva has stated that the preliminary, unofficial volume indications received from the EC would not be sufficient to ensure the sustainability of the company’s COVID-19 vaccine program even though some member states have confirmed their interest in having an inactivated, adjuvanted whole-virus vaccine solution in their portfolio. Hence, the EC is likely to terminate the agreement as Valneva will not be able to enter into an amendment to the APA that could allow for a reduced order.
Cogent Up on Study Data
:
Cogent Biosciences, Inc
.
COGT
announced positive initial data from its ongoing phase II APEX study evaluating the selective KIT D816V inhibitor bezuclastinib in patients with advanced systemic mastocytosis (AdvSM), a severe, debilitating hematologic disorder. Initial data from the study included 11 patients who were evaluated for signs of clinical activity. All patients treated with bezuclastinib achieved ≥50% reduction in serum tryptase, with a median decrease of 89%, regardless of prior KIT D816V inhibitor treatment. All bone marrow biopsy-assessed patients achieved ≥50% bone marrow mast cell reduction and decreased blood KIT D816V variant allele fraction (VAF). Bezuclastinib demonstrated favorable initial safety and tolerability profile with no reported periorbital or peripheral edema, cognitive effects or intracranial bleeding events. Shares of Cogent surged on the update.
Performance
Medical – Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology Index has lost 9.83% in the past five trading sessions. Among the biotech giants, Moderna has lost 16.36% during the period. Over the past six months, shares of Moderna have lost 56.14%. (See the last biotech stock roundup here:
Biotech Stock Roundup: BMYs Acquisition, REGN to Buy Libtayo & More
)
Image Source: Zacks Investment Research
What’s Next in Biotech?
Stay tuned for more pipeline and regulatory updates.
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