- In the RESILIENT SMA study, taldefgrobep alpha showed clinically meaningful improvements in motor function at all timepoints on the Motor Function Measurement-32 scale (MFM-32), but the treatment arm did not statistically separate on the primary outcome at Week 48 compared to the placebo+standard of care (SOC) 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 (87% Caucasian; n=180) showed clinically meaningful improvements on the MFM-32 at all timepoints, including Week 48, compared to the corresponding placebo+SOC group (p < 0.05). Additional analyses of these subjects (n=123) who had measurable baseline myostatin (the pharmacological target of taldefgrobep) showed an improved efficacy signal within this myostatin-positive population (p=0.02).
- Biohaven 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 (p=0.008) as measured by dual energy x-ray absorptiometry (DXA). 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 (i.e., fat mass, lean muscle mass, and bone density), 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 (SAEs).
Evaluation of additional RESILIENT clinical and biomarker data is ongoing, and Biohaven plans to engage with FDA regarding these emerging data to discuss a path forward. Full topline data will be presented at an upcoming scientific meeting.
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