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.