San Diego, March 9, 2026, 07:36 PDT
Capricor Therapeutics jumped 7.6% to $30.01 early Monday after the company scheduled its fourth-quarter and full-year results for March 12, putting deramiocel—the company’s experimental treatment for Duchenne muscular dystrophy—back in focus. Duchenne is a genetic disorder that causes progressive muscle loss. Capricor plans to publish results after Thursday’s close and will hold its conference call at 4:30 p.m. ET.
Timing is key here: Capricor is working to get U.S. approval back on track for deramiocel in Duchenne-related cardiomyopathy after the FDA rejected it in July. The company plans to showcase full Phase 3 results at the Muscular Dystrophy Association meeting on March 11, and as of February, said it had already submitted the complete HOPE-3 study report as part of its biologics license application, or BLA—the formal step for approval.
The conference abstract indicated deramiocel hit both its primary skeletal-muscle and cardiac endpoints in the 106-patient HOPE-3 trial. The drug’s placebo-adjusted improvement landed at 4.55% on the Performance of Upper Limb test, used to gauge arm and hand movement, while left ventricular ejection fraction ticked up by 2.4 percentage points—reflecting better cardiac output. No imbalance in severe or serious adverse events was noted, according to the abstract.
Craig McDonald, the national principal investigator for the trial at UC Davis, called the nearly 54% reduction in skeletal-muscle decline “extraordinary in Duchenne,” according to a company statement released Dec. 3. Jonathan Soslow, a pediatric cardiologist at Vanderbilt, said the cardiac results mark a “significant advance” in a disease where heart issues are the leading cause of death. Capricor Therapeutics, Inc.
On Feb. 24, Chief Executive Linda Marbán called the late-breaking slot at the MDA meeting a sign of the “strength” of the evidence, adding that Capricor was still zeroed in on “a potential approval decision.” The company’s oral presentation will go up at 2:45 p.m. ET in Orlando. Capricor Therapeutics, Inc.
The FDA shot down Capricor’s therapy in July with a complete response letter, citing insufficient evidence for effectiveness and saying part of the manufacturing file wasn’t reviewable. Capricor, in January, noted the agency had come back requesting the full HOPE-3 report plus backup data—no demand for an additional trial or new patient information. Marbán called that move “expected and appropriate.” Reuters
The tone on Wall Street took a different turn after that. B. Riley Securities analyst Madison El-Saadi, commenting on the December topline results, called the HOPE-3 trial’s efficacy signal “unambiguous” across all endpoints and said he was anticipating approval. Reuters
The financials are in focus Thursday, not just the pipeline. Capricor finished September with roughly $98.6 million in cash, equivalents and marketable securities—management says that should keep things going into the fourth quarter of 2026. Third-quarter revenue? Zero, after running through all prior milestone payments from Nippon Shinyaku.
Capricor is taking a cell-therapy approach in Duchenne, where safety concerns still loom large. Sarepta Therapeutics’ Elevidys stands as the sole gene therapy approved for Duchenne in the U.S., but last November, the FDA tightened its label and slapped on a boxed warning—the agency’s toughest safety notice—after fatal liver injuries occurred in non-ambulatory patients, those unable to walk independently.
Even so, Capricor faces unresolved hurdles. Its pipeline remains empty of any approved therapies, there’s no word yet on a fresh FDA decision timeline, and officials haven’t determined if the HOPE-3 data and manufacturing tweaks will actually address the concerns behind last summer’s setback.
The upcoming markers are packed in tight. Capricor will give its MDA presentation on March 11, with the results call following a day later at 4:30 p.m. ET.
