(MENAFN- AsiaNet News)

• Capricor plans to resubmit its Duchenne therapy application in 2026 with new cardiac efficacy data.
• FDA signaled flexibility that could speed approval if results are strong.
• Retail traders called the company's RMAT status a potential“secret weapon.”

Capricor Therapeutics shares rose over 3% in after-hours trading after the company said during its third-quarter conference call that it is rebuilding its approval bid for its Duchenne muscular dystrophy therapy, deramiocel, following an FDA rejection that requested additional safety, efficacy, and manufacturing data.

The company said it plans to resubmit its Biologics License Application (BLA) in 2026, after the FDA indicated it would review the totality of data from its study rather than rely solely on skeletal muscle endpoints. The approach allows Capricor to highlight cardiac efficacy, a key determinant of disease progression in Duchenne-associated cardiomyopathy.

FDA Meeting Opens Door To Flexible Review

While the Complete Response Letter (CRL) issued in July was unexpected, Capricor said on the call that Capricor was“well-positioned” to respond using data from its ongoing late-stage clinical trial, which is expected to report results within weeks.

During an FDA Type A meeting in August, the agency said those trial results could be used to address the issues cited in the CRL. Capricor also asked the FDA to keep its current BLA open and maintain the indication for Duchenne-associated cardiomyopathy.

To advance that path, Capricor proposed designating left ventricular ejection fraction (LVEF), a cardiac function measure, as the primary efficacy endpoint. While the FDA did not allow the formal change, it agreed to exercise regulatory flexibility in reviewing the data, indicating that strong cardiac results could support approval.

Manufacturing Update

Capricor said its San Diego GMP facility, which produces the commercial-scale version of deramiocel, has passed its pre-license inspection (PLI) and that all chemistry, manufacturing, and controls (CMC) concerns noted in the CRL have been resolved.

The company plans a Type 2 resubmission, triggering a six-month FDA review window, and said a strong efficacy signal could accelerate approval as early as 2026.

If approved before Sept. 30, 2026, Capricor would qualify for a Priority Review Voucher (PRV) under its Rare Pediatric Disease Designation, potentially adding non-dilutive value. The company ended the third quarter with $98.6 million in cash, providing runway into late 2026, and remains eligible for an $80 million milestone from NS Pharma upon approval.

Stocktwits Traders Call RMAT Capricor's 'Secret Weapon'

On Stocktwits, retail sentiment for Capricor was 'extremely bullish' amid 'extremely high' message volume.

CAPR sentiment and message volume as of November 10 | Source: Stocktwits

Some users pointed to Capricor's Regenerative Medicine Advanced Therapy (RMAT) designation as a potential catalyst. One user said the RMAT pathway was the company's“secret weapon,” noting that Duchenne's fatal nature and unmet medical need could allow the FDA to grant accelerated approval based on surrogate endpoints such as cardiac function improvements rather than long-term survival data.

Another user said the“best takeaway” from Capricor's earnings call was the expectation of a“surprise data drop” within one to three weeks, adding that they were optimistic about positive top-line results from the study.

A third user said the FDA's stated willingness to show flexibility could let Capricor“turn on the RMAT cheat code,” meaning the company could leverage its designation to pursue accelerated approval if left ventricular ejection fraction (LVEF) data are strong even when skeletal endpoints fall slightly short. The user described this as a potential“fallback” that could bring deramiocel to market months faster.

Capricor's stock has declined 60% so far in 2025.

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