Scholar Rock (SRRK) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Key regulatory and clinical milestones

• FDA decision on apitegromab expected by September 30, following resolution of manufacturing issues and successful transition to an alternate fill-finish facility.

• MAA resubmission to the EMA is pending guidance, with all documentation prepared and ready for expedited review once allowed.

• PMDA submission in Japan planned by year-end, enabled by agreement to file without a local clinical trial.

• Ongoing phase II studies: OPAL (SMA patients under two years post-gene therapy) and FORGE (FSHD patients), with robust enrollment and initial dosing underway.

• Initial data from SRK-439, a novel myostatin antibody, expected by year-end or early 2027.

Manufacturing and regulatory strategy

• Alternate fill-finish facility established and validated, replacing Catalent Indiana due to compliance issues.

• FDA and EMA have both inspected and approved the alternate facility for regulatory filings.

• FDA labeling discussions are ongoing, with the final label based on prior agreements and recent feedback.

• Safety database updated with long-term extension and expanded access program data, supporting regulatory submissions.

Commercialization and market opportunity

• Apitegromab targets a global SMA population of 35,000, with 7,000–10,000 in the U.S. and significant presence in Europe and Japan.

• Market for SMN-targeted therapies exceeds $5 billion, projected to reach $7 billion in 4–5 years.

• European operations launched in Switzerland and Germany; Japanese office opening in Tokyo in October.

• Anticipated strong initial demand, with patient and physician enthusiasm for muscle-targeted therapy.

• Expectation of reimbursement and access hurdles in the first 3–5 quarters, but long-term outlook is for multi-billion dollar annual sales.