Market reactionShares rose 14% on Nov 3. Yahoo Finance: “CAPR Stock Rises As Clinical Trial Investigators Write To FDA Urging Approval For Deramiocel”
Capricor Therapeutics is developing deramiocel, a cell therapy for Duchenne muscular dystrophy, a rare inherited muscle disease; it is under FDA review and not approved.
Deramiocel slowed upper-limb decline 54% versus placebo in Phase 3 HOPE-3 (p=0.029), after a 2025 rejection. In July, FDA advisers voted 9 to 3 that the evidence did not support effectiveness in Duchenne heart disease. Capricor then refocused the application on upper limb function and added two-year open-label data. FDA extended the decision date to November 22, 2026. Cash of $237.9 million funds at least 12 months.
Thesis checklist
2 risks need attention
Why it is in this state
The evidence, what would weaken it and the source filing
Why it is in this state
The evidence, what would weaken it and the source filing
Why it is in this state
The evidence, what would weaken it and the source filing
Why it is in this state
The evidence, what would weaken it and the source filing
Next decision point
Tests: FDA approves deramiocel
Does FDA approve deramiocel for upper limb function in Duchenne muscular dystrophy?
Strong, inconclusive and weak outcomes
What to look for in the result and how each would move the checklist
Price and material events
Select an event marker to read the update below. Marker colors show Thesis status.
FDA reviews deramiocel for upper limb function in Duchenne; decision due November 22
Phase 3 data in Lancet, one CRL, a 9-3 adcomm loss, and a reshaped upper-limb filing: approval hinges on whether FDA accepts the refined indication
Phase 3 data in hand but adcomm voted 3-9 against; FDA reviewing amended filing ahead of PDUFA — approval or a second CRL determines everything
Phase 3 data in hand but adcomm voted 9-3 against effectiveness; PDUFA approval would vindicate the resubmission, a second CRL ends it
About this history: every entry shows when the Thesis moved and what it said. The reasoning behind each move (the evidence, the analysis and the source filing) and the earnings write-ups are part of a subscription.
Dates, trials and completed outcomes are open to everyone. What each upcoming milestone hinges on is part of a subscription.
Drug pages: Deramiocel
Disease areas: Neuromuscular diseases, Infectious diseases
Catalyst calendar · Upcoming FDA decisions · Thesis changes
Competitive landscape
4 peers tracked
Cash: Market-data provider estimate; measurement period unavailable.
Debt: Market-data provider estimate; a complete comparable filing balance is unavailable.
Quarterly burn: Estimated from trailing free cash flow; measurement period unavailable.
Net cash and estimated runway require verified matching measurement periods. Missing debt components are not assumed to be zero.
Runway: No underlying guidance available.