Biohaven Licenses a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA)

Biohaven Pharmaceutical Holding Company Ltd., announced today that it entered into a worldwide license agreement with Bristol Myers Squibb for the development and commercialization rights to taldefgrobep alfa, a novel, Phase 3-ready anti-myostatin adnectin. Taldefgrobep is the third development asset licensed to Biohaven from Bristol Myers Squibb and a clinical trial for SMA is expected to begin in 2022.

Myostatin, a natural protein that limits skeletal muscle growth, is an important process in healthy muscular development. However, in people with neuromuscular diseases, active myostatin can critically limit the growth needed to achieve developmental and functional milestones. Myostatin inhibition has potential as a therapeutic strategy for enhancing muscle mass and strength in a range of pediatric and adult neuromuscular conditions.

Taldefgrobep is a muscle-targeted experimental treatment developed for neuromuscular disease and offers the opportunity for combination therapy. Biohaven plans to initiate a Phase 3 clinical trial of taldefgrobep in SMA in 2022.

“We are happy to see such great progress with the approvals and the impact of genetic treatments for SMA, but our work is not done yet and we need to now find innovative and efficacious therapies that will work in combination to help restore muscle strength and function, especially for older and symptomatic individuals affected by SMA,” said Kenneth Hobby, President, Cure SMA.

About Taldefgrobep alfa
Taldefgrobep alfa (also known as BMS-986089) is a modified adnectin designed to specifically bind to myostatin (GDF-8). Adnectins are an established proprietary protein therapeutic class based on human fibronectin, an extracellular protein that is naturally abundant in human serum. The intrinsic properties of an adnectin align with the properties needed to make a successful drug, including high potency, specificity, stability, and favorable half-life.

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