Sarepta Advances Non-Ambulant DMD Gene Therapy Study with FDA-Approved Cohort

What happened

Sarepta Therapeutics announced FDA approval to begin Cohort 8 of the ENDEAVOR study, evaluating an enhanced immunosuppression regimen for its ELEVIDYS gene therapy in non-ambulant Duchenne patients. This addresses a key uncertainty from the DeepValue report, where non-ambulatory access has been constrained due to safety concerns like the boxed warning and temporary dosing pause. The cohort aims to assess if better immunosuppression can mitigate risks, potentially enabling broader use in this population. However, this is only an investigational step, and any safety issues or poor outcomes could delay or restrict approval, maintaining execution risk. Overall, it represents a cautious move to manage ELEVIDYS's safety profile while progressing toward potential label expansion.

Implication

If Cohort 8 demonstrates that enhanced immunosuppression safely enables non-ambulant use, it could lead to FDA label expansion, boosting ELEVIDYS revenue and supporting a thesis upgrade. This would address a major swing factor in Sarepta's investment case, reducing uncertainty around growth and access constraints. However, failure or new safety events might prolong restrictions, reinforcing the HOLD stance and potentially driving stock volatility. Near-term, investor sentiment may improve, but financial impact depends on timely, positive data and regulatory alignment. Long-term, successful risk management could strengthen Sarepta's moat, though high execution risks from manufacturing and EU challenges persist.

Thesis delta

The approval of Cohort 8 signals incremental progress in tackling non-ambulant safety risks, slightly reducing near-term downside but not yet justifying a thesis shift. A move to BUY would require confirmed efficacy and broader access restoration from this study. Until then, the HOLD stance remains appropriate given ongoing uncertainties.