Scholar Rock (SRRK) investor relations material

Regulatory and clinical milestones

• Apitegromab, a first-in-class myostatin inhibitor, is nearing its FDA PDUFA date with strong phase III results in spinal muscular atrophy (SMA), showing significant motor function improvement in a broad patient population.

• The BLA was resubmitted with both Catalent Indiana and an alternate fill-finish facility; after a poor inspection at Catalent, only the alternate facility remains in the BLA under FDA guidance.

• Label discussions with the FDA have been collaborative, with expectations for a broad label covering SMA patients aged two and older, based on positive exchanges prior to the previous PDUFA date.

• The company is preparing for global expansion, with a JNDA filing in Japan by year-end and plans to resubmit the MAA in Europe after addressing manufacturing site changes.

• No other muscle-targeted therapies are in clinical development for SMA, positioning apitegromab as a unique offering with a significant competitive moat.

Manufacturing and supply chain

• Transitioned commercial supply to a globally recognized alternate fill-finish facility with validated capacity and regulatory approvals in the US, Europe, and Japan.

• Catalent Indiana was removed as a commercial supplier, but its data remains in the BLA, and product from that site continues to be used in long-term clinical trials.

• The alternate facility has scaled up manufacturing, with commercial vials ready for launch pending approval.

• Ongoing dialogue with the FDA and positive CMC and clinical reviews support confidence in timely approval.

Market dynamics and launch strategy

• Demand is expected from both pediatric and adult SMA patients, with strong support from the community and physicians for a muscle-targeted therapy.

• Surveys show increasing physician willingness to offer apitegromab, with awareness and education efforts benefiting from the launch delay.

• The therapy is positioned as complementary to existing SMN-targeted treatments, addressing the muscle component of SMA and enabling combination approaches.

• High compliance rates and long-term retention in clinical trials underscore patient need and satisfaction.