In the RESILIENT SMA study, Biohaven announced today that taldefgrobep alpha showed clinically meaningful improvements in motor function at all time points 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.
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.
Cliff Bechtold, Taldefgrobep Development Lead and President of Biohaven Ireland, commented, "SMA is a devastating rare disease and although we are disappointed that taldefgrobep did not achieve a statistically significant difference in the broad study population on the MFM-32, we are encouraged that a majority subgroup did show a treatment benefit compared to the placebo arm.”
Taldefgrobep was well-tolerated in the RESILIENT trial with 97% of subjects continuing into the optional long-term extension, which will remain open and ongoing pending discussion with the FDA. There were no taldefgrobep treatment-related SAEs.