Scholar Rock (SRRK) Shares Soar over 320% on SMA Topline Results from Phase 3 SAPPHIRE Trial

The late-stage biopharmaceutical company Scholar Rock (SRRK) who focuses on treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases  has provided topline results from its Phase 3 SAPPHIRE clinical trial that has been evaluating the efficacy and safety of apitegromab, an investigational muscle-targeted therapy, in patients with SMA.

The study achieved its primary endpoint show significant and clinically meaningful improvement. Treatment with apitegromab was well-tolerated across all age groups. There were no clinically relevant differences in the adverse event profile by dose. The FDA has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations. The EMA has granted Priority Medicines and Orphan Medical Product designations to apitegromab for the treatment of SMA. Scholar Rock intends to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorisation application (MAA) in Q1 2025.

“We are grateful to the families and investigators who participated in our trials. The positive Phase 3 SAPPHIRE trial, along with over 4 years of TOPAZ clinical trial data, clearly demonstrate the potentially transformative benefit of apitegromab to drive clinically meaningful improvements in motor function as measured by HFMSE in a broad SMA population, where motor function would normally be expected to generally decline over time,” said Jing Marantz, M.D., Ph.D., Chief Medical Officer at Scholar Rock. “We look forward to submitting our applications to the FDA and the EMA in Q1 2025.”