Apitegromab

Scholar Rock's stock surged 362% following positive Phase III results for its SMA candidate, apitegromab, which met primary endpoint in improving motor function. The company plans to file for US and EU approval in Q1 2025. Apitegromab, a muscle-targeted treatment, demonstrated clinical proof-of-concept in SMA, with significant improvement in HFMSE scores and a well-tolerated safety profile.

Scholar Rock's apitegromab, a myostatin blocker, showed significant motor function improvement in SMA patients in Phase III SAPPHIRE trial, with a 1.8-point increase on HFMSE at 52 weeks (p-value 0.0192). The company plans regulatory submissions in Q1 2025.

Scholar Rock shares surged 319% on positive phase III data for apitegromab in spinal muscular atrophy. Astrazeneca acquired a preclinical Lp(a) disruptor from CSPC for up to $2B. The 2024 Nobel Prize in Physiology or Medicine was awarded for the discovery of microRNA. PBMs' private labels in biosimilars raise antitrust concerns. Judo Bio showcased its kidney-targeted siRNA platform at OTS. Astellas' Claus Zieler predicts gene and cell therapies will drive innovation. Novo Nordisk plans to launch Wegovy in South Korea in mid-October.

Scholar Rock's phase III Sapphire study shows apitegromab significantly improves motor function in SMA patients, causing a 362% stock surge.

Scholar Rock's apitegromab improved motor function in spinal muscular atrophy patients, showing a 1.8-point greater improvement in children ages 2 to 12 compared to placebo, with benefits starting at eight weeks and continuing over a year.

Apitegromab met primary endpoint in phase 3 SAPPHIRE trial, showing significant motor function improvement in SMA patients. Scholar Rock plans to submit a BLA to FDA and EU for marketing authorization in Q1 2025.

Scholar Rock plans to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorisation application (MAA) in Q1 2025 after apitegromab met the primary endpoint in the Phase 3 SAPPHIRE clinical trial, demonstrating statistically significant and clinically meaningful improvement in motor function in SMA patients.

Scholar Rock's apitegromab, a muscle-preserving therapy for spinal muscular atrophy, succeeded in a Phase 3 trial, showing significant motor function improvement when combined with standard SMA therapies. The company plans to seek approvals in the U.S. and Europe early next year. The drug's success has implications for ongoing research into muscle preservation and potential treatments for obesity.

Scholar Rock's stock soared 315% after a Phase 3 trial of apitegromab for spinal muscular atrophy met its main goal, showing significant motor function improvement. The company plans to submit a U.S. Biologics License Application and seek EU marketing authorization in Q1 2025.