Capricor Therapeutics Nears Regulatory Milestone with Key FDA Submission

Biotechnology firm Capricor Therapeutics is approaching a pivotal moment for its lead candidate, Deramiocel. The company is preparing to submit the complete clinical study report from its Phase 3 HOPE-3 trial to the U.S. Food and Drug Administration (FDA) this month. This action represents a critical step in addressing previous regulatory feedback and advancing the therapy for Duchenne Muscular Dystrophy (DMD) toward a potential approval.

Capricor enters this important period with a strengthened balance sheet. Following a $150 million equity offering, the company’s proforma cash position stood at approximately $335 million as of February 5, 2026. Management has indicated these funds are sufficient to support operations for the next two to three years, covering the anticipated timeline for a potential U.S. commercial launch.

The immediate market focus is on the formal submission of the HOPE-3 report, expected in February 2026. Subsequently, investors will gain further insight into the company’s commercial preparations with the release of full-year 2025 financial results in mid-March 2026. Should the FDA accept the application for review, a final approval decision under the PDUFA framework could arrive in the second half of 2026.

Addressing FDA Requirements and Clinical Data

The upcoming submission is a direct response to a Complete Response Letter (CRL) issued by the FDA in July 2025. Following a regulatory update in January, Capricor’s management confirmed that while the agency requested comprehensive data and reports, it did not require new clinical trials or additional patient enrollment for a resubmission. The company interprets this as a standard procedural step following the initial review of topline results.

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The clinical data underpinning this effort, released in December 2025, showed Deramiocel successfully met both its primary and key secondary endpoints in the HOPE-3 study. The therapy demonstrated statistically significant improvements in skeletal muscle and cardiac function. The cardiac data is considered particularly consequential, as heart complications remain the leading cause of mortality for individuals with DMD.

Commercial Strategy and Pipeline Development

In preparation for a potential market entry, Capricor has established a commercialization partnership for Deramiocel. The company has a collaboration with NS Pharma covering distribution in the United States and Japan, which includes milestone payments and revenue sharing.

Alongside its lead program, Capricor continues to develop its StealthX exosome platform. A Phase 1 study for a vaccine candidate, conducted in partnership with the National Institutes of Health (NIH), is currently underway. This diversified approach provides an additional avenue for long-term growth beyond the DMD program.

The coming weeks will be decisive for Capricor as it seeks to resolve the FDA’s prior concerns and set Deramiocel on a clear path toward becoming an approved treatment for DMD patients.