Sarepta Therapeutics (SRPT) Morgan Stanley 24th Annual Global Healthcare Conference summary

Strategic focus and financial position

• Emphasis on supporting marketed products and leveraging a strong balance sheet to drive innovation and pipeline growth.

• Near-term strategy centers on stabilizing and expanding commercial operations, particularly for ELEVIDYS and the PMO franchise.

• Long-term growth expected from advancing a differentiated siRNA pipeline targeting neuromuscular diseases.

• Belief in significant untapped value based on cash flows and pipeline potential.

Pipeline updates and clinical data

• SRP-1001 (FSHD): Phase I single ascending dose data showed strong PK, dose-proportional muscle delivery, and >90% DUX4 knockdown, outperforming competitors.

• Upcoming data will include multiple ascending dose cohorts and higher doses, with circulating biomarkers to be reported.

• SRP-1003 (DM1): Early data demonstrated good PK, muscle exposure, and target engagement; multiple ascending dose data expected later this year.

• Focus on achieving higher DMPK knockdown than competitors, with further data and higher dose cohorts coming early next year.

Clinical endpoints and competitive landscape

• Functional endpoints (e.g., reachable workspace, Timed Up and Go) are being evaluated, but larger trials are needed for robust signals.

• Lessons from competitor trials (e.g., Novartis in DM1) inform endpoint selection and study design, with an emphasis on maximizing knockdown and learning from prior studies.

• Both FSHD and DM1 multiple ascending dose data readouts are expected in the second half of the year, likely as separate releases.