Biohaven Pharmaceutical Holding Company Ltd. announced 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 Spinal Muscular Atrophy (SMA) is expected to begin in 2022.
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. As a leader in innovative trials addressing neurodegenerative diseases, Biohaven plans to initiate a Phase 3 clinical trial of taldefgrobep in SMA in 2022. SMA is a rare, progressively debilitating motor neuron disease in which development and growth of muscle mass are compromised, resulting in progressive weakness and muscle atrophy, reduced motor function, impaired quality of life and often death.
Vlad Coric, M.D., Chief Executive Officer and Chairman of the Board of Biohaven stated, “We are extremely excited about the potential for taldefgrobep to improve the lives of patients and families affected by neuromuscular diseases. 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.”
Dr. Coric added, “The in-licensing of this late-stage asset demonstrates Biohaven’s commitment to addressing patients’ needs, and particularly those suffering from rare neurodegenerative and neuromuscular disorders.”
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.
John Tilton, Chief Commercial Officer, Rare Diseases at Biohaven added, “The addition of taldefgrobep to Biohaven’s portfolio of clinical-stage rare disease therapies aligns with our mission to make a meaningful difference in the lives of patients and families affected by devastating conditions with high unmet medical need. We look forward to partnering with the entire SMA community in bringing this potential treatment – and further hope – to people living with 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.
About Spinal Muscular Atrophy (SMA)
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 U.S., SMA affects approximately 1 in 11,000 births, and about 1 in every 50 Americans is a genetic carrier.
Biohaven is a commercial-stage biopharmaceutical company with a portfolio of innovative, best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases, including rare disorders. Biohaven’s Neuroinnovation™ portfolio includes FDA-approved NURTEC® ODT (rimegepant) for the acute treatment of migraine and a broad pipeline of late-stage product candidates across three distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine; glutamate modulation for obsessive-compulsive disorder, Alzheimer’s disease, and spinocerebellar ataxia; and MPO inhibition for amyotrophic lateral sclerosis.