Previewing Some Thoughts Ahead of the Deramiocel AdCom and PDUFA This Summer

Research evidence excerpt

Previewing Some Thoughts Ahead of the Deramiocel AdCom and PDUFA This Summer

July 6, 2026

●We believe the meeting could provide valuable insight into the

FDA's thinking ahead of the PDUFA, potentially serving as the more

significant catalyst for the stock.

●Further, firsthand perspectives from patients/caregivers and treating

physicians could help the Committee and FDA contextualize the clinical

data within the significant unmet need in DMD. We've heard several

of these testimonies first hand and believe they are quite supportive.

●Deramiocel's ability to preserve both skeletal muscle function and

cardiac function distinguishes it from many existing DMD therapies,

and we expect physicians to view the cardiac benefits as particularly

compelling.

●There is precedent of AdComs being positive for DMD therapies,

including some who did not have successful Phase 3 data, where we'd

also argue the benefits were less clear.

●We do recognize the FDA calling for an AdCom may mean they aren't

fully confident in a decision yet, but we'd also highlight that rare

disease groups have been asking the FDA to conduct these meetings

(link to Cantor note).

Does the Litigation with NS Pharma Affect Commercialization Outlook?

●The ongoing litigation with NS Pharma could be a potential barrier to

commercialization since the dispute centers on U.S. distribution rights

and launch economics.

●The latest on the case was that the judge ruled in CAPR's favor by

remanding the case back to New Jersey state court.

●This is important because it allows CAPR to resume its request for

expedited consideration of its preliminary injunction.

●Because neither party benefits from delaying commercialization, we

believe both have an incentive to resolve the dispute quickly or