雅虎香港財經 Biotech Stock Roundup: ALNY Up on Study Success, ALIM on Merger News & Other Updates
It was a busy week for the biotech sector with quite a few important pipeline and regulatory updates. Among these, Alnylam Pharmaceuticals ALNY soared on study data. Meanwhile, mergers and acquisitions continued to be in the spotlight.
Recap of the Week’s Most Important Stories:
Alnylam Skyrockets on Study Success: Alnylam Pharmaceuticals reported positive top-line results from the phase III HELIOS-B study evaluating Amvuttra (vutrisiran), an investigational RNAi therapeutic, for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. The stock skyrocketed in response to the success of the study.
The phase III HELIOS-B study met the primary endpoint, demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events in the overall population as well as the monotherapy population. The study also demonstrated statistically significant improvements across all secondary endpoints in both the overall and monotherapy populations. Key measures of disease progression, such as the six-minute walk test, Kansas City Cardiomyopathy Questionnaire and New York Heart Association Class, showed improvements at month 30.
Amvuttra is already approved in the United States for the treatment of adult patients with polyneuropathy of hereditary ATTR amyloidosis. It is also approved in the EU for the same indication in adult patients with stage 1 or stage 2 polyneuropathy. Alnylam is gearing up to proceed with global regulatory submissions starting later in 2024, including filing a supplemental new drug application with the FDA using a Priority Review Voucher.
Alimera Rises on Merger Deal With ANI Pharmaceuticals: Alimera Sciences, Inc. ALIM announced that it has entered into a definitive agreement with ANI Pharmaceuticals, Inc. ANIP, whereby the latter has agreed to acquire Alimera for $381 million.
Per the terms of the deal, Alimera will be acquired by ANIP for $5.50 per share in cash, and its shareholders will be entitled to a contingent value right for up to $0.50 per share if certain revenue targets are achieved in 2026 and 2027. The purchase price represents a rich premium of 75% on Alimera’s closing share price of $3.15 on Jun 21. ANIP will also clear Alimera’s debt of $72.5 million. Shares of Alimera gained on the news.
The deal is expected to be closed late in the third quarter of 2024, subject to customary closing conditions. The board of directors of ALIM and ANIP have approved the transaction.
Sarepta’s DMD Drug Label Expansion: Sarepta Therapeutics SRPT announced that the FDA has approved the expanded use of its Duchenne muscular dystrophy (DMD) gene therapy Elevidys. The drug is now approved to treat all DMD patients aged four years and older. While the FDA granted traditional approval for the therapy to treat ambulatory DMD patients (those who can still walk), it has granted accelerated approval for non-ambulatory patients.
Sarepta’s gene therapy was initially approved by the FDA last year under the accelerated pathway to treat ambulatory pediatric patients aged between four and five years with DMD. Following the FDA nod, Elevidys became the first-ever approved gene therapy for DMD.
The label expansion is mainly supported by data from the phase III EMBARK study, announced last October. Though the study failed to achieve its primary endpoint, it achieved statistical significance on all pre-specified key secondary endpoints, indicating that treatment with Elevidys modifies the course of DMD indication.
Management will still need to conduct a confirmatory study to convert the accelerated approval for non-ambulatory DMD patients to a full one. Sarepta is currently conducting the phase III ENVISION study to evaluate the safety and efficacy of gene therapy in non-ambulatory and ambulatory DMD patients. This study also satisfies the regulatory requirements for Elevidys’ approval outside the United States.
Sarepta sports a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
JAZZ Down on Study Results: Jazz Pharmaceuticals plc JAZZ announced that a mid-stage study evaluating the efficacy and safety of its investigational candidate, suvecaltamide (JZP385), in adult patients with essential tremor (ET) did not meet its primary endpoint.
Suvecaltamide is a highly selective and state-dependent modulator of T-type calcium channels, which play a role in the brain's control of muscle movement. Per the data readout from the phase IIb study (NCT05122650), treatment with suvecaltamide 30mg failed to achieve statistical significance on the primary endpoint of change from baseline to week 12 on the Essential Tremor Rating Assessment Scale modified composite outcome score compared with placebo. The study also failed to achieve statistical significance on the key secondary endpoint of the Clinical Global Impression-Severity scale, which is a measure of the severity of illness compared with placebo at week 12.
However, treatment with suvecaltamide 30mg demonstrated numeric improvements on the primary endpoint and key secondary endpoint of the ET study compared with placebo. The numeric improvements were not statistically significant.
Performance
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The Nasdaq Biotechnology Index has gained 2.93% in the past five trading sessions and Gilead’s shares have risen 9.75% during the same time frame. In the past six months, shares of REGN have rallied 25.85%. (See the last biotech stock roundup here: Biotech Stock Roundup: ITCI Up on Study Data, ZNTL, OVID Down on Updates & More News)
What's Next in Biotech?
Stay tuned for more pipeline updates.
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Alnylam Pharmaceuticals, Inc. (ALNY) : Free Stock Analysis Report
Alimera Sciences, Inc. (ALIM) : Free Stock Analysis Report
Jazz Pharmaceuticals PLC (JAZZ) : Free Stock Analysis Report
Sarepta Therapeutics, Inc. (SRPT) : Free Stock Analysis Report
ANI Pharmaceuticals, Inc. (ANIP) : Free Stock Analysis Report
