Biohaven Enrolls First Patient in Phase 3 Trial of Taldefgrobep alfa in Spinal Muscle Atrophy (SMA)

NEW HAVEN, CT, USA I July 7, 2022 I Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) a commercial-stage biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological and neuropsychiatric diseases, today announced that it has commenced enrollment in a Phase 3 clinical trial assessing the efficacy and safety of taldefgrobep alfa in Spinal Muscle Atrophy (SMA).

Taldefgrobep is an investigational, muscle-targeted recombinant protein with the potential to enhance muscle mass and strength in people living SMA when used in combination with other approved treatments.    Taldefgrobep targets myostatin, a natural protein that limits skeletal muscle growth, through two mechanisms:  lowering myostatin directly and blocking key downstream signaling mechanisms. Myostatin inhibition is a potential therapeutic strategy for children and adults with a range of neuromuscular conditions for whom active myostatin can limit the skeletal muscle growth needed to achieve developmental and functional milestones.

Irfan Qureshi, M.D., Senior Vice President, Neurology at Biohaven, stated, "This is an important milestone for the taldefgrobep program and for people living with SMA. There have been great strides in advancing therapeutics in this challenging disease. Yet, there remains a significant unmet need to address the residual weakness and functional impairments, such as difficulty walking, that are caused by the disease. We are thankful for the SMA community that has welcomed and worked with us to develop this patient-focused study."

RESILIENT, is a Phase 3 placebo-controlled, double-blind trial designed to evaluate the efficacy and safety of taldefgrobep as an adjunctive therapy for participants who are already taking a stable dose of nusinersen or risdiplam or have a history of treatment with onasemnogene abeparvovec-xioi, compared to placebo.  The study is not restricted nor limited to patients based on ambulatory status or classification of SMA. 

Han Phan, MD, Director of Clinical Research, Rare Disease Research, said, "We are happy to see progress being made with additional novel therapies focused on neuromuscular health.  Despite current therapies, a high unmet need for safe and effective supportive treatments for SMA remains, as many patients still experience significant weakness and reduced levels of functioning. Additional research is needed to help improve the quality of life for people living with SMA".

Biohaven expects to enroll approximately 180 patients in this randomized, double-blind, placebo-controlled global trial. Additional details about the trial can be found at SMATrials.com or  https://clinicaltrials.gov/ (NCT05337553).

About Taldefgrobep alfa

Taldefgrobep alfa (also known as BMS-986089) is a modified adnectin designed to specifically bind to myostatin (GDF-8). Taldefgrobep is a fully human anti-myostatin recombinant protein that lowers free myostatin and acts as an Activin 2b receptor antagonist with the myostatin-taldefgrobep complex.  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.

About Biohaven

Biohaven is a global 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 and preventive treatment of migraine (EMA-approved as Vydura®  for the acute treatment of migraine with or without aura, and prophylaxis of episodic migraine in adults who have at least four migraine attacks per month) and a broad pipeline of late-stage product candidates across five distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine and other CGRP-mediated diseases; glutamate modulation for obsessive-compulsive disorder and spinocerebellar ataxia; myeloperoxidase (MPO) inhibition for amyotrophic lateral sclerosis; Kv7 ion channel activators for focal epilepsy and neuronal hyperexcitability, and myostatin inhibition for neuromuscular diseases. More information about Biohaven is available at www.biohavenpharma.com.

SOURCE: Biohaven Pharmaceutical Holding

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