Scholar Rock Holding Corp is advancing its pipeline, particularly apitegromab for spinal muscular atrophy (SMA), while navigating financial and developmental challenges, according to its recent Form 10-Q report.
Apitegromab's Progress in SMA Treatment
Apitegromab, a fully human monoclonal antibody, is being developed as a muscle-targeted therapy for SMA. The Phase 3 SAPPHIRE clinical trial achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in motor function. Patients receiving apitegromab showed a mean difference in change from baseline in HFMSE of 1.8 points compared to placebo. The ONYX long-term extension study, enrolling 98% of SAPPHIRE patients, is ongoing.
Regulatory Submissions and Commercial Outlook
Scholar Rock anticipates submitting a Biologics License Application (BLA) to the FDA and a marketing authorization application to the EMA for apitegromab in Q1 2025. A commercial product launch in the U.S. is expected in late 2025, followed by a European launch. Apitegromab has been granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations by the FDA, as well as PRIME designation from the EMA and Orphan Medicinal Product designation from the EC for SMA treatment.
Expanding Pipeline and Strategic Initiatives
Beyond apitegromab, Scholar Rock is developing SRK-439, an anti-myostatin monoclonal antibody, for cardiometabolic disorders, including obesity. The Phase 2 EMBRAZE trial is assessing its safety and efficacy in preserving muscle mass. SRK-181, an inhibitor of latent TGFβ1 activation, is in Phase 1 DRAGON trial for cancers resistant to checkpoint inhibitors.
Financial Overview and Capital Management
Scholar Rock reported a net loss of $64.5 million for the third quarter of 2024, an increase from $0.53 to $0.66 net loss per share compared to the same period in 2023. Total operating expenses increased by 48.3% to $64.8 million. In October 2024, the company completed a follow-on offering, raising approximately $324.4 million. This capital, along with existing resources, is expected to fund operations for at least one year after the issuance of the financial statements.
Challenges and Risks
Scholar Rock faces challenges including product development risks, reliance on third parties for clinical trials, and the need to build commercial infrastructure. The company acknowledges the lengthy and expensive nature of product development and the potential for delays or failure in obtaining regulatory approval. Intellectual property risks and stock volatility also pose concerns.
