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

Strategic overview and leadership vision

• Focus on supporting marketed products and driving a robust, innovative pipeline for near- and long-term growth.

• Emphasis on strong financial position enabling investment in R&D and expansion.

• Leadership highlights unique interplay between marketed products and pipeline as a differentiator.

• Recent leadership transition brings renewed focus on benefit-risk balance and patient outcomes.

Pipeline updates and clinical data

• SRP-1001 (FSHD): Early phase I data show strong PK, dose-proportional muscle delivery, and >90% DUX4 knockdown, outperforming competitors.

• Upcoming data will include multiple ascending dose cohorts and circulating biomarkers, with higher dose data expected next year.

• SRP-1003 (DM1): Early data show good PK, muscle exposure, and target engagement; higher dose cohorts and more data expected later this year and early next year.

• Both programs expect independent data readouts in the second half of the year, with DM1 likely first.

Competitive landscape and study design

• FSHD and DM1 programs aim for higher knockdown than competitors, with focus on translating molecular effects to clinical outcomes.

• Lessons from competitor trials (e.g., Novartis in DM1) inform endpoint selection and study design.

• Ongoing evaluation of functional endpoints and natural history data to optimize phase III development.