Deramiocel

San Diego – October 19, 2026 -- The U.S. Food and Drug Administration's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) will meet on July 29, 2026, to review Capricor Therapeutics' Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), less than four weeks before the agency's August 22, 2026 PDUFA target action date.

Panel meeting precedes the August 22 PDUFA decision date

Capricor (NASDAQ: CAPR) said its BLA remains on track against the August 22 deadline. The advisory meeting will be livestreamed. The company pointed to the Federal Register and its own website for meeting details.

San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) disputed the post-hoc analyses in the FDA's briefing materials, two days before an advisory committee meets on July 29 to discuss its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD).

Capricor says FDA analyses rely on an obsolete draft analysis plan

The company said the FDA's post-hoc analyses use statistical analysis plan (SAP) version 1.1. Capricor described that version as an unsigned, incomplete internal draft that became obsolete when cohort B was added. It also said the draft lacked content the FDA had specifically requested.

Capricor said its results are governed by SAP version 3.0, which was finalized before unblinding.

San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) said The Lancet has published results from its Phase 3 HOPE-3 trial, in which Deramiocel slowed upper limb function decline by 54 percent versus placebo in Duchenne muscular dystrophy (PUL 2.0, p=0.03).

The randomized, double-blind, placebo-controlled trial enrolled 106 participants and met its primary endpoint. Capricor also reports a clinically meaningful cardiac benefit. The results rest on the company's prespecified Statistical Analysis Plan version 3.0 (SAP 3.0).

SAN DIEGO – October 19, 2026 -- The FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted 9-3, with no abstentions, that available evidence did not support the effectiveness of Capricor Therapeutics' Deramiocel for cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), leaving the company's PDUFA target action date of August 22, 2026 as the next decision point.

Non-binding vote addresses narrower indication than Capricor proposed

The committee's vote is non-binding. Capricor (NASDAQ: CAPR) said the voting question covered a narrower indication than the one it had proposed and did not include a vote on Deramiocel's overall benefit-risk profile.

Cardiomyopathy in DMD currently has no approved treatment. Capricor said heart muscle deterioration leads to cardiomyopathy and heart failure, the leading cause of death in DMD, which affects approximately 15,000 people in the United States.

San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) ended the second quarter with about $237.9 million in cash, cash equivalents and marketable securities, down from about $318.1 million at Dec. 31, 2025. The decline came as an FDA advisory committee voted 9 against and 3 in favor on whether evidence shows Deramiocel is effective for cardiomyopathy in Duchenne muscular dystrophy (DMD) patients.

Advisory panel vote is non-binding and limited to cardiomyopathy

The Cellular, Tissue and Gene Therapies Advisory Committee voted on July 29, 2026. Capricor says the panel was not asked to vote on the HOPE-3 primary endpoint or on overall benefit-risk. In a separate discussion of upper limb function, the committee's feedback was directionally supportive of the HOPE-3 evidence, according to the company. The recommendation is advisory and non-binding, and the Biologics License Application (BLA) remains under active FDA review.

San Diego – October 19, 2026 -- The U.S. Food and Drug Administration has pushed the decision date for Capricor Therapeutics' Deramiocel Biologics License Application by three months, from August 22, 2026 to November 22, 2026, after classifying the company's latest data submission as a major amendment.

Deramiocel is an investigational cell therapy for Duchenne muscular dystrophy (DMD). Capricor (NASDAQ: CAPR) disclosed the extension of the Prescription Drug User Fee Act (PDUFA) target action date on the day of release.

Capricor files 24-month HOPE-3 extension data after July advisory committee

Following the July 2026 Advisory Committee meeting, Capricor submitted a BLA amendment containing 24-month open-label extension data from its pivotal Phase 3 HOPE-3 study. The filing also includes additional robustness analyses.

San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) will present 24-month skeletal muscle and cardiac data from its Phase 3 HOPE-3 trial of Deramiocel in Duchenne muscular dystrophy (DMD) at the World Muscle Society Congress, weeks ahead of an FDA PDUFA target action date of November 22, 2026.

The 31st Annual Congress (WMS 2026) runs September 29 to October 3, 2026, in Hiroshima, Japan. Capricor will deliver one late-breaking poster and one oral presentation on Deramiocel, plus two preclinical posters from its StealthX exosome platform.

HOPE-3 24-month cohort reaches 82 of 106 randomized patients

Of the 106 patients randomized in HOPE-3, 82 reached the 24-month time point: 40 originally assigned to Deramiocel and 42 to placebo. The data build on the 12-month results reported previously.

San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) reported that Duchenne muscular dystrophy patients who switched from placebo to Deramiocel after one year slowed their upper limb decline by 76% compared with their own prior year, according to open-label extension data from the Phase 3 HOPE-3 trial. The U.S. FDA has set a PDUFA target action date of November 22, 2026, for the therapy.

Crossover analysis shows 76% slower decline on PUL 2.0

The late-breaking poster was presented at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan. It measured upper limb function with the Performance of Upper Limb (PUL 2.0) scale. Of 106 randomized patients, 98 entered the open-label extension, 49 per arm. At Month 24, 42 patients in the placebo-origin arm and 40 in the Deramiocel arm were assessed.