Scholar Rock (SRRK) Barclays 28th Annual Global Healthcare Conference summary

Company overview and strategic focus

• Celebrating its 50th anniversary, the company leverages innovative antibody technology to target difficult growth factors, notably myostatin, with significant success in spinal muscular atrophy (SMA).

• Achieved the first positive pivotal trial for myostatin inhibition in SMA, with regulatory approvals and launches anticipated in the U.S. and Europe this year.

• Plans to build a 50-country operating platform, expanding into Asia-Pacific and Latin America, with ongoing pipeline advancement.

Financial and operational updates

• Secured a $550 million debt instrument, with $100 million to be drawn by quarter-end, supporting commercial readiness and pipeline development.

• Ended the year with $368 million in cash, excluding the additional $100 million to be drawn.

• Expects to monetize a rare pediatric disease priority review voucher upon approval, not yet factored into the financial runway.

Pipeline and clinical development

• Advancing phase II OPAL study for SMA in infants/toddlers and a phase II FORGE study for FSHD, with the latter enrolling 60 patients in a randomized, double-blind, placebo-controlled trial.

• Developing a subcutaneous version of apitegromab, with phase I data showing comparable pharmacodynamics to IV formulation; regulatory path to be determined post-IV approval.

• SRK-439, a high-affinity myostatin inhibitor, is in phase I with data expected later this year.