Scholar Rock (SRRK) investor relations material

Scientific and clinical innovation

• Achieved first-ever phase III success for a myostatin inhibitor in spinal muscular atrophy (SMA), targeting pro and latent myostatin for high selectivity and safety.

• Differentiated approach avoids off-target effects seen in prior attempts by focusing on unique myostatin forms.

• Therapy complements existing SMN-targeted drugs by addressing muscle health, not just motor neuron survival.

• Phase III SAPPHIRE trial showed clinically meaningful motor function improvements in both children and adults.

• Plans to expand into other rare neuromuscular disorders leveraging platform expertise.

Regulatory and manufacturing updates

• Initial BLA submitted in January 2025; FDA granted priority review with a September 2025 action date.

• Approval delayed due to compliance issues at Catalent, Indiana fill-finish facility; a second U.S.-based facility was rapidly established.

• BLA resubmitted and accepted with both facilities; FDA re-inspection underway, with a 90-day review window aligning with commercial drug readiness in July.

• Guidance remains for potential U.S. approval by Q3 2026, with sufficient launch supply anticipated.

• European application includes Catalent, Indiana; EMA approval expected in H2 2026, with discussions ongoing to add the second facility.

Commercial strategy and market outlook

• Significant pent-up demand in the SMA community, with nearly all patients and neurologists seeking muscle-targeted therapy.

• Launch team strengthened during regulatory delay, with robust payer engagement and access programs in place.

• Expectation of rapid uptake post-approval, though revenue ramp will depend on payer conversion timelines.

• Pricing strategy reflects rarity and severity of SMA, with confidence in reimbursement and manageable budget impact.

• Global patient pool estimated at 35,000, with U.S. and European launches prioritized.