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July 8, 2022

Biohaven enrols first subject in Phase III spinal muscle atrophy trial

The trial will analyse the safety and efficacy of taldefgrobep as an adjunctive treatment for SMA.

Biohaven Pharmaceutical has enrolled the first subject in Phase III RESILIENT clinical trial of taldefgrobep alfa (BMS-986089) in spinal muscle atrophy (SMA).

A modified adnectin, taldefgrobep alfa can attach to myostatin (GDF-8). 

Taldefgrobep is an investigational, muscle-targeted, fully human anti-myostatin recombinant protein.

It reduces free myostatin and serves as an Activin 2b receptor antagonist with the myostatin-taldefgrobep complex. 

The international, randomised, double-blind, placebo-controlled trial will analyse the safety and efficacy of taldefgrobep as an adjunctive treatment in subjects who are currently receiving a stable dose of nusinersen or risdiplam or have received onasemnogene abeparvovec-xioi previously versus placebo. 

Nealy 180 subjects will be enrolled in the trial.

The enrolment of patients in the trial is irrespective of their ambulatory status or classification of SMA. 

Assessing the efficacy of taldefgrobep alfa versus placebo in change from baseline in the 32-item Motor Function Measure (MFM-32) total score to week 48 is the primary outcome measure of the trial.

Biohaven Pharmaceutical Neurology senior vice-president Irfan Qureshi said: “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.”

A rare genetic neurodegenerative ailment usually diagnosed in young children, SMA is characterised by motor neuron loss, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness. 

Inadequate survival of motor neuron (SMN) protein production leads to the underlying pathology of SMA. SMN protein is vital for the survival of motor neurons.

In March this year, the company enrolled the first subject in a Phase II/III trial of oral zavegepant as the preventive treatment for migraine.

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