As Capricor Therapeutics awaits a pivotal decision for its embattled Duchenne muscular dystrophy cell therapy deramiocel, the biotech revealed it cut around 10% of its workforce.
Capricor had 231 fulltime employees as of Dec. 31, 2025, according to an SEC filing, meaning the layoffs may have affected around 23 individuals.
The move was made “to align our organization with our near-term priorities,” the biotech said in its third quarter earnings report on Tuesday.
Capricor is nearing a possible end to a 16-month regulatory saga. Deramiocel was rejected by the FDA in July 2025 after a planned advisory committee meeting was canceled. Then, in December last year, the company claimed that the Phase 3 HOPE-3 trial hit both its primary endpoint, showing statistically significant benefits in upper-limb function, and the secondary endpoint of slowed decline in cardiac function, as measured by left ventricular ejection fraction.
Six months after receiving a surprise rejection due to what the FDA called “lack of substantial evidence of effectiveness,” Capricor’s cell therapy deramiocel showed significant benefits in upper-limb function and slowed decline in cardiac function in a Phase III trial.
December 3, 2025
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However, the FDA questioned the therapy’s success on these endpoints, and an adcomm in July did not go well.
After much discussion on the FDA’s decision to base its review on an outdated statistical analysis plan, the agency’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 9-3 against approval of deramiocel for Duchenne muscular dystrophy (DMD) cardiomyopathy. Discussion on the primary upper limb function endpoint was more positive, and Capricor went back to the FDA in August with additional data and a refined indication focused on upper limb function. This pushed the FDA’s target action date from Aug. 22—a deadline that had already passed when Capricor announced the delay—to Nov. 22.
Capricor has also slowed programs not directly related to deramiocel, including the biotech’s exosome-based platform, in order to prioritize the DMD cell therapy “pending further regulatory clarity,” according to its third quarter earnings report on Tuesday.
This decision is unlikely to please Kaos Capital. The firm, which in an August letter to other company stakeholders described itself as a “significant and growing shareholder” of Capricor, urged a greater focus on the company’s efforts outside of deramiocel. Other Kaos demands included “an immediate meeting, board change, and capital-preservation plan.”
Less than two weeks away from a highly anticipated FDA ruling on its cell therapy for Duchenne muscular dystrophy, Capricor Therapeutics has reported increased investment in its San Diego plant to support commercial production.
November 11, 2026
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Capricor also revealed on Tuesday that it has initiated regulatory engagement in Europe and Japan for deramiocel. The company is also considering expanding the therapy into younger patients with DMD and Becker muscular dystrophy, a similar but less severe form of the disease, “with next steps to be informed by the U.S. regulatory outcome for Deramiocel,” according to the earnings report.
Capricor last month presented open label extension data from HOPE-3 showing that patients who transferred to the cell therapy after a year on placebo saw upper limb decline slow by 76% compared to their prior year data. These data were included in the August amendment. And on Monday, Capricor revealed that an abstract outlining the therapy’s cardiac results from HOPE-3 and presented at the American Heart Association (AHA) conference this month received AHA’s Outstanding Research Award in Pediatric Cardiology.
Compared to placebo, treatment with deramiocel slowed the progression of heart muscle scarring as measured by cardiac MRI, Capricor CEO Linda Marbán said on Tuesday’s earnings call. While the difference in ejection fraction across all patients did not reach statistical significance, in patients with existing cardiomyopathy this metric improved by approximately 2.8 percentage points relative to placebo, Marbán noted.
There are currently no therapies available specifically targeting cardiomyopathy, which is the leading cause of death for patients with DMD, Marbán told BioSpace in August last year.
“Put simply, the data tell a consistent story,” Marbán said on the call. “One year, two years, two randomized trials and a decade of clinical experience all points in the same direction. We do not believe that this is a case that rests solely on unmet medical need, secondary endpoints or regulatory flexibility.”
Capricor Therapeutics met with the FDA last week for a type A meeting, during which CEO Linda Marbán aimed to explain to the regulator that it got it wrong. Capricor plans to resubmit the application based on deramiocel’s existing dataset.
August 20, 2025
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