Biohaven Licenses Phase 3-Ready SMA Therapy from Bristol Myers Squibb

Biohaven Pharmaceutical Holding entered 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 for the neurodegenerative condition spinal muscular atrophy.

Photo: Kenneth Hobby, president, Cure SMA

Taldefgrobep is the third development asset licensed to Biohaven from Bristol Myers Squibb and a clinical trial for spinal muscular atrophy is expected to begin in 2022.

Spinal muscular atrophy (SMA) is a rare genetic neurodegenerative disorder characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness that is often fatal and typically diagnosed in young children. The underlying pathology of SMA is caused by insufficient production of the SMN (survival of motor neuron) protein, essential for the survival of motor neurons, and is encoded by two genes, SMN1 and SMN2. In the United States, SMA affects approximately 1 in 11,000 births, and about 1 in every 50 Americans is a genetic carrier.

Myostatin is a natural protein that limits skeletal muscle growth, an important process in healthy muscular development. However, in patients 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 believe the development of innovative anti-myostatin therapies designed to enhance muscle mass and strength may represent the next frontier of neuromuscular treatments and will build on the tremendous progress made by existing motor neuron-targeted therapies,” said Vlad Coric, CEO and chairman of the board of Biohaven.

The in-licensing of taldefgrobep expands Biohaven’s portfolio of innovative, late-stage product candidates for the treatment of neurologic, neuroinflammatory, and psychiatric indications. Under the terms of the agreement, Biohaven will receive worldwide rights to taldefgrobep and Bristol Myers Squibb will be eligible for regulatory approval milestone payments, as well as tiered, sales-based royalties beginning in the high teens.

“We are happy to see such great progress with the approvals and the impact of potential 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.

Author: Rare Daily Staff