Biohaven Ltd. announced that its experimental drug, taldefgrobep alfa, did not statistically separate from placebo on the primary outcome at 48 weeks in the RESILIENT SMA study for spinal muscular atrophy. While clinically meaningful improvements in motor function were observed, the primary endpoint was not met.
Despite the setback in SMA, Biohaven reported signals of efficacy in clinically relevant subgroups and observed a greater reduction in total body fat mass and numerically larger increases in lean muscle mass and bone density in obesity. Biohaven intends to accelerate clinical plans for a self-administered autoinjector in adults with overweight and obesity in Q4 2024.
This development reduces competitive pressure for Scholar Rock's apitegromab in the spinal muscular atrophy market. It also highlights the growing interest in myostatin-targeting drugs for obesity, an area where Scholar Rock is also developing SRK-439.
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