Biohaven's Taldefgrobep Alfa Shows Promise in Obesity Despite Failing SMA Phase III Trial

Biohaven's investigational drug, taldefgrobep alfa, did not achieve its primary goal in a Phase III trial for spinal muscular atrophy (SMA), as it failed to significantly improve motor function compared to a placebo on the Motor Function Measurement-32 (MFM-32) scale[1][2]. The trial showed a significant placebo effect among non-Caucasian participants, potentially due to genetic polymorphisms, which Biohaven is considering as a future biomarker for patient selection[2]. Despite the setback, Biohaven remains hopeful and plans to discuss subsequent steps with the FDA, pointing out certain efficacy signals in specific subgroups, notably among Caucasian patients where some MFM-32 score improvements were noted[1]. While the drug did not meet its primary endpoint in SMA, it did show a promising reduction in body fat mass, paving the way for its exploration in obesity treatment[1].