On Monday, Biohaven Ltd. (NYSE:BHVN) updated the taldefgrobep alfa development programs in Spinal Muscular Atrophy (SMA) and obesity.
In the RESILIENT SMA study, taldefgrobep 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 48 weeks compared to the placebo+standard of care (SOC) group.
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Signals of efficacy were observed in clinically relevant and biomarker-defined subgroups including those related to age, ambulatory status, background therapy, and presence of myostatin at baseline.
An unexpectedly large subgroup (35%) of subjects had no measurable levels of myostatin at baseline and had imbalances in some genetic factors (SMN2 copy number, race) across treatment arms.
Analyses of prespecified subgroups by race and ethnicity demonstrated that the largest study population (87% Caucasian; n=180) showed a 2.2-point change-from-baseline improvement with taldefgrobep treatment on the MFM-32 at Week 48 compared to a 1.1-point change-from-baseline improvement in the corresponding placebo+SOC group (p < 0.039).
Non-Caucasian subjects (n=26) had a higher-than-expected placebo response and did not separate from the placebo on the MFM-32 at Week 48 (p=0.24).
In the overall study population, taldefgrobep demonstrated a greater reduction in the percent change in total body fat mass compared to the placebo+SOC arm (p=0.008).
The taldefgrobep arm also showed numerically larger increases in lean muscle mass and bone density compared to the placebo+SOC arm.
Biohaven intends to accelerate taldefgrobep clinical plans in 4Q24, advancing a self-administered autoinjector in adults living with overweight and obesity.
In reaction to the trial update, Scholar Rock Holding (NASDAQ:SRRK) stock is trading higher. For the unversed, Scholar Rock is developing apitegromab, an investigational, fully human monoclonal antibody that inhibits myostatin activation for SMA.
In October, Scholar Rock's Phase 3 SAPPHIRE trial of apitegromab for SMA achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement for apitegromab versus placebo in motor function.
Price Action: SRRK stock is up 33.2% at $39.85, and BHVN stock is up 0.91% at $46.01 at last check Monday.
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Illustration of Phrama lab worker created with MidJourney.
