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It was a busy week for the biotech sector as usual, with lots of regulatory and pipeline updates, before the first-quarter earnings season kicks in. Among these, biotech giant Bristol Myers Squibb BMY was down after a late-stage study on cardiovascular drug Camzyos failed. On the other hand, Verve Therapeutics VERV skyrocketed on positive data from a cholesterol study.
Recap of the Week’s Most Important Stories:
BMY Stock Down on Camzyos Study Data
Shares of Bristol Myers Squibb declined after the company announced that the late-stage ODYSSEY-HCM study evaluating cardiovascular drug Camzyos (mavacamten) failed.
The phase III study was evaluating Camzyos for the treatment of adult patients with symptomatic New York Heart Association (“NYHA”) class II-III non-obstructive hypertrophic cardiomyopathy (nHCM).
The dual-primary endpoints for the trial were to examine changes from baseline in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-23 CSS) and peak oxygen consumption (pVO2) at week 48. However, the study did not meet its dual primary endpoints.
Camzyos, a selective, reversible, allosteric inhibitor of cardiac myosin, is already approved for the treatment of adults with symptomatic NYHA class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms. ODYSSEY-HCM tested the hypothesis that a cardiac myosin inhibitor would improve measures of feel and function for patients with nHCM.
While the results of the study were disappointing, no new safety signals were observed.
Bristol Myers currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
VERV Stock Surges on Study Data
Shares of Verve Therapeutics surged after the company announced positive initial data from an early-stage study of its investigational candidate, VERVE-102, which is being developed for reducing cholesterol levels.
The patient population enrolled in the phase Ib Heart-2 study comprised individuals with heterozygous familial hypercholesterolemia and/or premature coronary artery disease — two groups that require substantial and sustained reductions in low-density lipoprotein cholesterol (LDL-C) levels.
The data readout includes 14 patients from the first three cohorts who received doses of VERVE-102 at 0.3 mg/kg, 0.45 mg/kg, or 0.6 mg/kg, respectively, with each participant having at least 28 days of follow-up as of the data cutoff date of March 13, 2025.
Verve Therapeutics reported that a single infusion of VERVE-102 resulted in dose-dependent reductions in blood PCSK9 protein levels and LDL-C in the Heart-2 study, with an average LDL-C decrease of 53% and a maximum reduction of 69% observed among four participants in the 0.6 mg/kg dose cohort.
In cohort 1 of the study, patients receiving 0.3 mg/kg of the candidate had an LDL-C reduction of 21% and a PCSK9 reduction of 46%. Patients receiving 0.45 mg/kg achieved an LDL-C reduction of 41% and a PCSK9 reduction of 53%. In cohort 3 (0.6 mg/kg dose), the LDL-C reduction was 53%, and the PCSK9 reduction was 60%.
Additionally, Verve Therapeutics reported that VERVE-102 was well-tolerated across all dose levels.
IRWD Slumps on Regulatory Update
Ironwood Pharmaceuticals IRWD announced regulatory updates on the next-generation GLP-2 analog, apraglutide, which is being developed for treating short bowel syndrome (SBS) with intestinal failure (IF).
Ironwood initiated the rolling new drug application (“NDA”) submission to the FDA for apraglutide to treat SBS patients who are dependent on parenteral support in January 2025.
The NDA filing was initially expected to be completed in the third quarter of 2025.
The phase III STARS study evaluated apraglutide for reducing parenteral support dependency in adult patients with SBS-IF. The open-label extension study, STARS Extend, also evaluated apraglutide for the given indication.
While gearing up for the NDA filing for apraglutide, pharmacokinetic analysis showed that the exposure and dose delivered in the STARS study were lower than planned due to dose preparation and administration.
However, following the latest discussion with the FDA, a confirmatory phase III study will now be required for approval of the candidate. This, in turn, is likely to extend the timeline for the NDA filing completion and also potentially delay the approval for apraglutide.
Shares of IRWD plunged on this update.
Biogen and Eisai Get EC Approval for AD Drug
Biogen BIIB and partner Eisai announced that the European Commission (EC) has granted marketing authorization to amyloid-beta (Aβ) monoclonal antibody Leqembi (lecanemab) for Alzheimer’s disease (AD).
Lecanemab is indicated for the treatment of adult patients with a clinical diagnosis of mild cognitive impairment and mild dementia due to early AD who are apolipoprotein E ε4 (ApoE ε4*) non-carriers or heterozygotes with confirmed amyloid pathology.
Leqembi is already approved in the United States, Japan and other countries.
Eisai is leading lecanemab’s development and regulatory submissions globally, and Biogen and Eisai are co-commercializing and co-promoting the product.
Performance
The Nasdaq Biotechnology Index has lost 7.47% in the past five trading sessions and MRNA’s shares have lost 6.49%. In the past six months, shares of MRNA have plunged 54.21%. (See the last biotech stock roundup here: Biotech Stock Roundup: PCRX Up on Patent News, EWTX Down on Study Data & More)
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What's Next in Biotech?
Stay tuned for more pipeline updates.
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