Mechanistic Potential of Taldefgrobep Alfa in Spinal Muscular Atrophy: Lindsey Lee Lair, MD


The vice president of clinical development at Biohaven shed light on a newly initiated phase 3 study evaluating a promising agent for patients with all types of spinal muscular atrophy. [WATCH TIME: 2 minutes]

WATCH TIME: 2 minutes

"Taldefgropeb alfa is an antimyostatin that targets the muscle and has a dual activity, so it suppresses myostatin levels but also blocks receptor signaling. It already has an established safety and tolerability profile.”

After decades without an approved disease-modifying agent for spinal muscular atrophy (SMA), industry leaders finally broke through with the first approved therapy, nusinersen (Spinraza; Biogen), in 2016. Since then, the FDA has cleared 2 additional therapies, including one gene replacement approach, to help treat the disease. Now, a new agent, taldefgrobep alfa (Biohaven), is set to be evaluated in a large-scale phase 3 trial after years of preclinical work and studies in patients with another neuromuscular disorder, Duchenne muscular dystrophy.1

The placebo-controlled, double-blind trial is expected to enroll approximately 180 patients with SMA who are already taking a stable dose of nusinersen or risdiplam (Evrysdi; Novartis), or have a history of treatment with onasemnogene abeparvovec-xioxi (Zolgensma; Novartis). Previously known as BMS-986089, taldefgrobep alfa is a fully human antimyostatin recombinant protein that lowers free myostatin and acts an activin 2b receptor antagonist with the myostatin-taldefgrobep complex. Originally developed by Bristol Myers Squibb, Biohaven acquired the worldwide rights to the phase 3 neuromuscular program in February 2022, while allowing Bristol Myers Squibb to benefit off potential regulatory approval milestone payments and tiered, sales-based royalties as part of the agreement.

NeurologyLive® recently sat down with Lindsey Lee Lair, MD, vice president, Clinical Development, Biohaven, to learn more about the study, its unique design, and why taldefgrobep alfa stands as a promising candidate. She also provided insight on the mechanistic action of the agent and how it could be used in coordination with other previously approved therapies.

1. Biohaven enrolls first patient in phase 3 trial of taldefgrobep alfa in spinal muscular atrophy (SMA). News release. Biohaven Pharmaceuticals. July 7, 2022. Accessed July 28, 2022.
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