Biohaven provides updates on taldefgrobep alfa development programs

In the RESILIENT SMA study, taldefgrobep alpha showed clinically meaningful improvements in motor function at all timepoints on the Motor Function Measurement-32 scale, but the treatment arm did not statistically separate on the primary outcome at Week 48 compared to the placebo+standard of care group. Efficacy signals were observed in clinically relevant and biomarker-defined subgroups including those related to age, ambulatory status, background therapy, and baseline myostatin level. Analyses of prespecified subgroups by race and ethnicity demonstrated that the largest study population showed clinically meaningful improvements on the MFM-32 at all timepoints, including Week 48, compared to the corresponding placebo+SOC group. Additional analyses of these subjects who had measurable baseline myostatin showed an improved efficacy signal within this myostatin-positive population. Biohaven (BHVN) plans to engage the FDA regarding potential next steps forward and will present the study data at an upcoming conference. The optional long-term extension phase of the trial will remain ongoing pending further data analysis as well as regulatory discussions. Prespecified outcome measures in the overall study population analyzing the change from baseline in body composition at Week 48 demonstrated a greater reduction in the percent change in total body fat mass in the taldefgrobep arm compared to the placebo+SOC arm as measured by dual energy x-ray absorptiometry. The taldefgrobep arm also showed numerically larger increases in lean muscle mass and bone density compared to the placebo+SOC arm. Given the overall strength and consistency of the taldefgrobep-associated changes in body composition, Biohaven plans to rapidly advance taldefgrobep into a placebo-controlled Phase 2 obesity study in 4Q2024 using a user-friendly, self-administered autoinjector. Taldefgrobep demonstrated robust target engagement in the RESILIENT study, reducing myostatin levels below detection in all treated subjects over 48 weeks. Taldefgrobep was well-tolerated in the RESILIENT study with 97% of subjects continuing into the optional long-term extension. There were no taldefgrobep treatment-related serious adverse events