Biohaven's spinal muscular atrophy drug fails to meet study goal
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Nov 25 (Reuters) -Biohaven BHVN.N said on Monday its experimental treatment for a rare neuromuscular disorder failed to show statistically significant difference in improving motor function in a late-stage trial, sending the drug developer's shares down 10% in premarket trading.
The treatment, taldefgrobep alpha, was being tested in patients with spinal muscular atrophy (SMA), which is the leading genetic cause of infant deaths globally. It leaves children too weak to walk, talk, swallow or even breathe.
The company said it was disappointing that the drug did not meet the main goal of the study.
However, it helped cut patients' total body fat mass and also showed numerically larger increases in lean muscle mass and bone density compared to those given placebo and the standard treatment, according to the company.
Based on the results, it plans to advance the drug as a treatment for obesity and begin a mid-stage study for the same in the fourth quarter of 2024.
The drug is designed to block the effects of the proteins myostatin and activin to prevent muscle wasting and accumulation of fat mass.
Currently, there are three approved treatments in the United States for SMA: Biogen's BIIB.O Spinraza, Novartis' NOVN.S gene therapy Zolgensma, and Roche ROG.S and PTC Therapeutics' PTCT.O oral drug, Evrysdi. However, none of these target muscle development.
A similar drug from biotech firm Scholar Rock SRRK.O last month helped significantly improve motor function measures such as standing and crawling, compared to a placebo, in individuals suffering from the rare disease.
Biohaven's study enrolled 269 ambulant and nonambulant participants with any SMA type aged four to 21 years who would receive either taldefgrobep or placebo for 48 weeks.
The company plans to discuss the results from the spinal muscular atrophy trial with the US Food and Drug Administration and present the full data at an upcoming scientific meeting.
Reporting by Mariam Sunny in Bengaluru; Editing by Arun Koyyur and Shailesh Kuber
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