Earnings

Capricor Extends Deramiocel Review to November 22

The Tip Desk · November 12, 2026 · 4 min read

The company posted a $42.7 million third-quarter net loss, or $0.73 a share, as cash, cash equivalents, restricted cash and marketable securities stood at about $198.5 million.

Capricor Therapeutics (CAPR), a biotechnology company developing cell and exosome-based therapeutics for rare diseases, said the Food and Drug Administration classified an August amendment to its Deramiocel biologics license application as a major amendment and extended the review to a PDUFA target action date of November 22, 2026.

The amendment added two-year open-label extension data from HOPE-3, the randomized, placebo-controlled Phase 3 trial, and proposed a refined indication focused on upper limb impairment in patients older than 9 years, the population studied in HOPE-3 and the focus of its primary endpoint. Chief Executive Officer Linda Marbán said the company submitted the extension data and additional robustness analyses in August and an integrated summary of efficacy across HOPE-2 and HOPE-3.

In October, Capricor presented the two-year HOPE-3 open-label extension as a late-breaking poster at the 31st Annual World Muscle Society Congress. Patients who switched from placebo to Deramiocel after year one declined 1.56 points less on the Performance of the Upper Limb 2.0 scale than they had in their year on placebo, which the company described as a 76% slowing in this open-label, cross-phase comparison. Marbán said patients who switched from placebo to Deramiocel saw their upper limb decline slow by 76% compared with their year on placebo.

HOPE-3 cardiac results received the American Heart Association Outstanding Research Award in Pediatric Cardiology. On November 7, Jonathan Soslow of Vanderbilt University Medical Center presented results showing slower progression of myocardial fibrosis with Deramiocel versus placebo and, in patients with cardiomyopathy, left ventricular ejection fraction favoring Deramiocel. The full HOPE-3 dataset had been published in The Lancet in July, reporting a statistically significant slowing of upper limb disease progression with Deramiocel as measured by PUL 2.0 (p=0.029).

As part of the BLA review, the FDA conducted a Bioresearch Monitoring inspection in July 2026 and issued a Form 483 with one observation. The company said it has submitted its response and is awaiting FDA feedback. Marbán said the company appreciates the FDA’s engagement and is encouraged by the Agency’s current focus on rare disease, including its recently launched Rare Disease Framework Initiative.

There was no revenue recognized for the first three quarters of 2026 or 2025. Total operating expenses for the third quarter of 2026 were approximately $44.7 million, compared with approximately $26.3 million for the third quarter of 2025. The company said the increase was primarily driven by pre-commercial activities and manufacturing scale-up. Net loss widened to approximately $42.7 million, or $0.73 a share, from approximately $24.6 million, or $0.54 a share, a year earlier.

Cash, cash equivalents, restricted cash and marketable securities totaled approximately $198.5 million as of September 30, 2026, compared with approximately $318.1 million as of December 31, 2025. From January 1, 2026, through November 10, 2026, the company said it had not sold any shares under its at-the-market program. In the third quarter, Capricor implemented a workforce reduction affecting approximately 10% of its employees to reduce operating expenses and align resources with its priorities.

The company said available cash, cash equivalents and marketable securities are sufficient to fund operating capital requirements for at least the next twelve months. It expects to provide additional guidance on its longer-term financial outlook following greater regulatory clarity. That outlook excludes any potential revenue from product sales, the potential monetization of a Priority Review Voucher, if received, and other non-operating sources of capital.

The San Diego GMP facility is positioned to support an initial commercial launch, if Deramiocel is approved. A second-floor cleanroom expansion is underway, with validation targeted for the first half of 2027. Capricor has initiated regulatory engagement in Europe and Japan for Deramiocel. Programs not directly related to Deramiocel, including the company’s exosome-based platform, have been slowed to prioritize Deramiocel pending further regulatory clarity.

Capricor continues to pursue its contractual dispute with Nippon Shinyaku and its U.S. subsidiary, NS Pharma, regarding the Deramiocel distribution agreement. The company withdrew its motion for a preliminary injunction, without prejudice, to resolve the dispute in arbitration following the FDA’s decision. It expects the arbitration hearing to commence in mid-2027.

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