It was a regular week in the biotech sector with a focus on pipeline and regulatory updates. While smaller biotechs were in focus on regulatory updates, bigwigs like Bristol Myers (BMY Quick QuoteBMY - Free Report) and Moderna (MRNA Quick QuoteMRNA - Free Report) gained the spotlight on drug label expansion and pipeline data, respectively.

Recap of the Week’s Most Important Stories:

Updates from BMY:  Bristol Myers announced that the FDA approved a label expansion for chimeric antigen receptor (CAR) T cell immunotherapy Abecma. The therapy is now approved for the treatment of adult patients with relapsed or refractory multiple myeloma after two or more prior lines of therapy, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 monoclonal antibody.

It is to be noted that Abecma is being jointly developed and commercialized in the United States by Bristol Myers Squibb and 2seventy bio TSVT. Outside the United States, BMY assumes the sole responsibility for Abecma’s manufacturing and commercialization. The latest approval, granted on Apr 4, was based on results from the KarMMa-3 trial, wherein Abecma tripled progression-free survival compared to standard regimens, with a 51% reduction in risk of disease progression or death and a well-established safety profile.

The expanded approval brings this personalized CAR T cell therapy to more patients with relapsed or refractory multiple myeloma earlier in their treatment journey as a one-time infusion offering meaningful treatment-free intervals when responding to the therapy.

An approval was mostly expected as the FDA Oncologic Drugs Advisory Committee recently voted in favor (8:3) of Abecma’s benefit/risk profile for patients with triple-class exposed relapsed or refractory multiple myeloma. BMY and TSVT were expecting a decision in December but the FDA delayed and stated that the ODAC will meet to review data supporting the application for Abecma.

Abecma is already approved in the United States for adult patients with triple-class exposed relapsed or refractory multiple myeloma after four or more prior lines of therapy. Abecma was recently approved in Japan, Switzerland and the European Union for adult patients with triple-class exposed relapsed and/or refractory multiple myeloma after two prior lines of therapy.

BMY also announced data from the cohorts of the KRYSTAL-1 study, evaluating the lung cancer drug Krazati (adagrasib) in combination with Erbitux (cetuximab), for the treatment of patients with previously treated KRASG12C-mutated locally advanced or metastatic colorectal cancer (CRC).

The combination of Krazati and cetuximab demonstrated clinically meaningful activity as a targeted treatment option for CRC patients.  The combination demonstrated an objective response rate of 34% with a median follow-up of 11.9 months in 94 patients. The median progression-free survival and the median overall survival was 6.9 months and 15.9 months, respectively, in pre-treated patients with KRASG12C-mutated locally advanced or metastatic CRC.

BMY currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Moderna Up on Vaccine Data: Moderna announced encouraging data from a portion of an early-stage study on mRNA-4157/V940, its investigational individualized neoantigen therapy (INT), in certain patients with head and neck cancer. Its shares gained on the news.

The INT is being developed in collaboration with Merck.  This portion of the study evaluated the combination of mRNA-4157 and Merck’s blockbuster immuno-oncology drug Keytruda in patients with human papillomavirus negative (HPV-) head and neck squamous cell carcinoma (HNSCC).

Data from the study showed that treatment with the combination not only achieved positive clinical responses in HPV- HNSCC patients but also showed evidence of activation of immune responses. The combined therapy was also found to be safe and well-tolerated by study participants.

Per management, treatment with the combination achieved an objective response rate in six out of 22 patients, including two complete responses and four partial ones. The disease control rate was 63.6%. These results were presented at the American Society of Cataract and Refractive Surgery, earlier this week.

Amylyx Withdraws Drug, Reduces Jobs: Amylyx Pharmaceuticals, Inc. (AMLX Quick QuoteAMLX - Free Report) announced that it will remove its amyotrophic lateral sclerosis (ALS) drug Relyvrio (sodium phenylbutyrate and taurursodiol, also known as AMX0035) from the U.S. markets and it has already started a process with the FDA on the same. The drug, approved under the brand name Albrioza in Canada, will also be removed from Canadian markets.

While the formal decision to remove the drug from these markets has come recently, the company was contemplating the same following the failure of the late-stage PHOENIX study last month, which led to a massive crash in the company’s shares. PHOENIX did not meet its primary endpoint of reaching statistical significance as measured by a change from baseline in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score at week 48. The study also failed to achieve statistical significance in secondary endpoints.

Consequently, Relyvrio/Albrioza will no longer be available for new patients. Nevertheless, the patients currently on therapy in the United States and Canada who, in consultation with their physician, wish to continue can be transitioned to a free drug program.

Meanwhile, the PHOENIX Open Label Extension is ongoing. Amylyx will now focus on advancing two key programs investigating its lead candidate, AMX0035, in Wolfram syndrome and progressive supranuclear palsy and AMX0114, an antisense oligonucleotide targeting calpain-2, in ALS.

Concurrently, Amylyx announced a restructuring plan, whereby it will reduce its workforce by approximately 70% and decrease financial commitments outside its priority areas. Amylyx expects to have a cash runway into 2026 by undertaking this plan.

Regulatory Update From Syros: Syros Pharmaceuticals (SYRS Quick QuoteSYRS - Free Report) announced that the FDA has granted Fast Track Designation to tamibarotene for the treatment of newly diagnosed acute myeloid leukemia (AML). The designation was granted to tamibarotene, in combination with azacitidine and venetoclax, for the treatment of newly diagnosed AML with RARA overexpression, as detected by an FDA-approved test in adults aged more than 75 years or who have comorbidities that preclude the use of intensive induction chemotherapy. Shares were up on the news.

The Fast Track designation allows therapeutic candidates for priority review and accelerated approval if supported by clinical data.

SYRS is currently developing tamibarotene, an oral, selective, retinoic acid receptor alpha or RARα agonist in combination studies as a frontline treatment of AML and higher-risk myelodysplastic syndrome (HR-MDS). The FDA also granted a Fast Track Designation to tamibarotene for treating HR-MDS.

Performance

The Nasdaq Biotechnology Index has gained 0.41% in the past five trading sessions and Moderna’s shares have increased 7.72% during the same time frame. Over the past six months, shares of VRTX have risen 13.87%. (See the last biotech stock roundup here: Biotech Stock Roundup: IRON, VERV Down on Study Updates, BMYs Drug News & More)

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What's Next in Biotech?

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