Capricor Therapeutics (NASDAQ: CAPR) shares rose 22.15% to $9.35 on Monday, reversing a recent 61% decline as The Lancet published results from its pivotal Phase 3 HOPE-3 clinical trial for Deramiocel. The publication provides independent peer-reviewed validation of the investigational cell therapy’s efficacy in treating Duchenne muscular dystrophy (DMD), addressing earlier skepticism from the U.S. Food and Drug Administration regarding statistical significance. This external validation strengthens Capricor’s position ahead of the FDA’s Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026, for its Biologics License Application (BLA).

The study, titled "Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial," confirms that Deramiocel slowed upper limb function decline by 54% versus placebo (PUL 2.0, p=0.03). The trial enrolled 106 patients and utilized Statistical Analysis Plan version 3.0 (SAP 3.0), which Capricor has consistently maintained is the correct governing document for the data. Craig McDonald, M.D., National Principal Investigator and lead author, described the 54% slowing of disease progression as a "substantial, meaningful effect" in a population where functional decline is typically irreversible.

Linda Marbán, Ph.D., CEO of Capricor Therapeutics, stated that the publication reinforces confidence in the strength and durability of the results. She emphasized that The Lancet’s rigorous peer review process validates the same body of evidence forming the foundation of the BLA. The company argues that the FDA’s earlier briefing materials relied on an obsolete draft of the statistical plan, whereas SAP 3.0, finalized prior to unblinding, demonstrates statistically significant benefits in both skeletal and cardiac muscle function.

Market Reaction and Regulatory Outlook

The sharp recovery in share price reflects investor relief following the independent validation of Capricor’s data integrity claims. The advisory committee meeting scheduled for July 29 will critically assess whether the data supports approval. While the committee’s recommendation is non-binding, it often influences the FDA’s final decision. Capricor has posted presentation slides on its investor website to provide what it considers an accurate framing of the data, countering the agency’s initial assessment that Studies HOPE-2 and HOPE-3 lacked substantial evidence of effectiveness.

What the Numbers Show

The divergence between the FDA’s initial assessment and the published results centers on methodological interpretation. The inclusion of Cohort B in SAP 3.0 appears critical to achieving statistical significance on the primary endpoint. For investors, the key risk remains whether the FDA will accept this re-analysis during its final review. The concurrent benefits in cardiac measures lend biological support to a consistent treatment effect, suggesting that the therapy’s mechanism of action—secreting exosomes that alter macrophage expression—is effective across muscle types. If approved, Deramiocel would be the first cell therapy for DMD, potentially qualifying Capricor for a Priority Review Voucher due to its Rare Pediatric Disease Designation.

Forward-Looking Statements

Capricor cautioned that forward-looking statements regarding Deramiocel are subject to risks, including potential delays in regulatory inspections. These risks are detailed in the company’s Annual Report on Form 10-K for the year ended December 31, 2025, and its Quarterly Report on Form 10-Q for the quarter ended March 31, 2026. Deramiocel remains an investigational candidate and has not been approved for commercial use in any indication.

Capricor Therapeutics Inc. (NASDAQ: CAPR) stock fell 13% on Friday following the announcement that the U.S. Food and Drug Administration (FDA) will convene an advisory committee meeting to review its Biologics License Application (BLA) for Deramiocel. The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) is scheduled to meet on July 29, 2026. This regulatory step precedes the PDUFA target action date of August 22, 2026. The BLA seeks approval of Deramiocel, an investigational cell therapy for the treatment of Duchenne Muscular Dystrophy (DMD), supported by data from the HOPE-2 and HOPE-3 clinical trials.

The market decline reflects investor reaction to the additional regulatory hurdle. This occurs as the FDA reconsiders multiple cell and gene therapy applications, a trend noted following the tenure of Dr. Vinay Prasad. Recent industry activity includes Replimune Group Inc. aligning with the FDA on a path forward for its melanoma therapy BLA, and Regenxbio Inc. gaining clarity on potential accelerated approval for Navsunli.

Clinical Data Supporting the BLA

The application is backed by positive five-year data from the HOPE-2 Open-Label Extension (OLE) study, demonstrating durable skeletal and cardiac muscle benefits. These findings were presented at the Parent Project Muscular Dystrophy (PPMD) 2026 Annual Conference. Additionally, the BLA includes results from the HOPE-3 Phase 3 trial, a multicenter, randomized, double-blind, placebo-controlled study enrolling 106 patients with DMD.

Five-Year HOPE-2 OLE Results

In the HOPE-2 OLE study, patients receiving Deramiocel experienced a mean total-score decline of less than 5 points on the Performance of the Upper Limb (PUL 2.0) scale over five years. By comparison, a cohort-matched external comparator of standard-of-care DMD patients showed a modeled decline of roughly 2.4 points per year, projecting to approximately 12 points over five years. A separate natural-history analysis reported a decline of approximately 8.1 points over three years in non-ambulant patients.

Cardiac function, measured by left ventricular ejection fraction (LVEF) on cardiac MRI, remained stable over the five-year period. This contrasts with a modeled decline of approximately 3.2% per year observed in a propensity-matched external cardiac comparator. Deramiocel maintained a favorable safety profile, consistent with over 800 intravenous infusions administered to date across the clinical development program.

HOPE-3 Phase 3 Results

The HOPE-3 Phase 3 trial met its primary endpoint (PUL 2.0, p=0.03) and key secondary cardiac endpoint (LVEF, p=0.04), along with all other Type I error-controlled secondary endpoints. These results align with the durable skeletal and cardiac muscle benefits observed in the HOPE-2 OLE study.

Linda Marbán, Ph.D., CEO of Capricor, stated that the combined data represent the totality of clinical evidence for Deramiocel, showing durable, statistically significant benefit to both skeletal and cardiac muscle function. The July 29, 2026, advisory committee meeting will be available for live streaming.