Sarepta Therapeutics (SRPT) TD Cowen 46th Annual Health Care Conference summary

Commercial and financial outlook

• 2026 net product revenue is projected at $1.2–$1.4 billion, with PMO revenue consensus at $900 million and ELEVIDYS revenue expected between $300–$500 million, depending on run rates and potential label expansion.

• Revenue guidance is based on current start forms and a six-month turnaround time, with a conservative approach factoring in possible cancellations and seasonal illness.

• Salesforce expansion is underway, doubling the number of reps, with contract reps focusing on peripheral sites and patient finding.

• Q1 is expected to be slightly down due to anticipated cancellations and sickness, with a 15% decrease factored into guidance.

• Margins for PMO products have remained stable, and price competition is not expected to be a major factor due to the small patient population.

Product development and clinical updates

• Three-year EMBARK data for ELEVIDYS has been well received by physicians, showing consistent efficacy and safety with real-world experience.

• New referral sites are emerging post-EMBARK data, indicating growing adoption.

• Cohort 8 sirolimus pretreatment study in nonambulant patients is progressing, with five sites in the process of opening and the primary endpoint focused on ALI rate, defined by GGT levels.

• Top-line data for Cohort 8 is expected by year-end, with a goal to reduce ALI risk by at least 30%.

• The pathway to restoring the non-ambulant indication in the label is not yet defined and will depend on forthcoming data and agency discussions.

Pipeline and research initiatives

• Arrowhead assets for DM1 and FSHD are on track for interim data in Q1, focusing on dose response curves and muscle concentration using the TRiM siRNA platform.

• Dosing regimens include single doses with biopsies at 30 and 90 days; full top-line data for all cohorts is expected by year-end.

• Pivotal trial starts for FSHD and DM1 are gated by commercial manufacturing readiness, likely pushing next trials into 2027.

• Ongoing assay validation work is expected to resolve without further delays, with higher dose cohort data forthcoming.

• Early-stage work on 3rd generation PPMOs using the TRiM platform is underway, with potential updates in 2027.