Scholar Rock (SRRK) Canaccord Genuity's 46th Annual Growth Conference summary

Regulatory milestones and manufacturing strategy

• Apitegromab is approaching a PDUFA date of September 30th, with high anticipation for FDA approval as the first muscle-targeted therapy for SMA.

• Manufacturing challenges at Catalent Indiana led to a dual-path BLA resubmission, including a second fill-finish facility, ensuring regulatory flexibility.

• The second facility has a strong inspection record, and the FDA is reviewing all required data ahead of schedule, making a pre-approval inspection unlikely.

• Labeling discussions with the FDA are nearly finalized, with strong alignment and minimal outstanding issues.

• Commercial inventory from the second facility is robust and ready for launch, surpassing previous supply levels.

European regulatory outlook

• The EMA application initially listed only Catalent Indiana, and the agency awaited the outcome of FDA inspections before proceeding.

• With the recent OAI classification for Catalent, the company will now engage with EMA to add the second facility and clarify next steps.

• Mutual recognition of FDA clearance for the second facility could expedite the European approval process.

• The German commercial team is prepared for launch, with contingency plans in place for potential delays.

Commercial readiness and market expectations

• The commercial team is experienced, with leaders from successful rare disease launches, and has been preparing for over a year.

• Physician interest in apitegromab has grown, with 42% of KOLs now likely to offer it to all eligible patients, up from 23% a year ago.

• Education efforts have increased awareness of the need for muscle-targeted therapies in SMA, with 75% of neurologists recognizing its importance.

• Access and reimbursement strategies are in place, including a patient support program and anticipation of payer policy development post-launch.

• Pricing is expected to align with rare disease standards, supported by robust clinical data showing motor function gains over placebo.