Biohaven’s Phase III SMA miss: was it the target or the trial design?

Biohaven Ltd. (NASDAQ:BHVN) is changing direction after spinal muscular atrophy (SMA) candidate taldefgrobep alpha failed to meet the primary endpoint as adjunctive therapy in the Phase III RESILIENCE study. In addition to analyzing data in biomarker-defined patient populations, the company is accelerating development for obesity.

Biohaven reported that taldefgrobep, an inhibitor of active myostatin that also blocks activin A and other targets, led to a potentially clinically meaningful but not statistically significant improvement in 206 ambulatory and non-ambulatory SMA patients, as measured by the Motor Function Measurement-32 scale (MFM-32), compared with placebo...