Biohaven’s Taldefgrobep Alfa Misses Primary Endpoint in Phase 3 SMA Trial, Eyes FDA Discussions and Obesity Indication

Trial Outcome:
Biohaven's taldefgrobep alfa failed to meet its primary endpoint in a Phase 3 trial for spinal muscular atrophy (SMA), not showing significant improvement in motor function after 48 weeks compared to the placebo plus standard of care group[2][3][4].

Efficacy Signals:
Despite missing the primary endpoint, taldefgrobep alfa showed clinically meaningful improvements in motor function at all timepoints on the Motor Function Measurement-32 scale (MFM-32) in certain subgroups, including those related to age, ambulatory status, background therapy, and baseline myostatin levels[2].

Body Composition Changes:
The drug demonstrated significant reductions in total body fat mass and increases in lean muscle mass and bone density compared to the placebo plus standard of care group[2].

Ethnic Variability:
The study highlighted ethnic differences in response to the therapy, with Caucasian subjects showing clinically meaningful improvements, while non-Caucasian subjects had a higher placebo response and did not separate from placebo[2].

Future Plans:
Biohaven plans to engage with the FDA regarding potential next steps and will present the study data at an upcoming conference. The company also intends to explore the use of taldefgrobep alfa in obesity, given its effects on body composition[2][3][4].