Sarepta Therapeutics (SRPT) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Key achievements and strategic milestones

• Achieved a broad label for ELEVIDYS in June, enabling treatment for a wide range of Duchenne muscular dystrophy patients, including non-ambulatory individuals.

• ELEVIDYS launch has outperformed previous gene therapy launches, with significant revenue growth and strong payer engagement.

• Over 75 infusion sites are operational across the U.S., providing robust national coverage.

• Organization is now profitable and expects to be sustainably cash flow positive within the next few quarters.

• 2025 revenue guidance set at $3 billion, with approximately two-thirds expected from ELEVIDYS.

Market access, reimbursement, and patient journey

• Payer policies are evolving, with most granting broad access to label; typical time from start form to infusion is three to five months, expected to stabilize at three to four months.

• Strong track record in overcoming payer objections and winning appeals, ensuring high therapy access rates.

• Significant number of start forms are from non-ambulatory patients, reflecting broad label utilization.

• Patient age range is broad, with ongoing efforts to lower the minimum age through additional data and newborn screening expansion.

• Four states have adopted newborn screening, with plans to expand further.

Manufacturing, supply, and future scalability

• Current supply and manufacturing capacity is strong, with no immediate need for expansion.

• Transitioning to suspension manufacturing to improve yields, lower costs, and enable global scale; GMP material and bridging study targeted for 2025, with full implementation by early 2027.

• Product quality and scale-up data for suspension manufacturing are positive, supporting future growth.