Capricor Therapeutics (NASDAQ:CAPR) is awaiting a Nov. 22 Food and Drug Administration decision on Deramiocel for Duchenne muscular dystrophy, as the company prepares for a potential launch while reporting a wider third-quarter loss.
CEO Linda Marbán said on the company’s Nov. 10 earnings call that the FDA review is ongoing and that Capricor has submitted all requested information. The company’s proposed refined indication is for treating upper limb impairment in patients with Duchenne who are older than nine.
The FDA extended its target action date after agreeing to review additional information in a major amendment to Capricor’s biologics license application. The August submission included two-year open-label extension data from the HOPE-3 trial, additional analyses and a combined efficacy summary from the HOPE-2 and HOPE-3 trials, Marbán said.
Company Points to Trial and Follow-Up Data
Marbán said the one-year randomized HOPE-3 trial met its primary endpoint, showing a statistically significant slowing of upper limb disease progression. The difference between the Deramiocel and placebo groups was 1.1 points on the Performance of the Upper Limb 2.0 scale, with a p-value of 0.029. She said the trial was designed to detect a difference of approximately one point, a threshold described as clinically meaningful by Duchenne physicians and the FDA.
In the second year, patients who had received placebo were eligible to switch to Deramiocel. Marbán said those patients’ average decline slowed from about two points in their placebo year to about 0.5 point after starting treatment. She described the open-label results as descriptive, but said they were consistent with the randomized data.
Capricor also submitted more than 900 re-analyses of the HOPE-3 data to address questions about the robustness of the findings, including the handling of missing data and which patients were included. Marbán said every analysis favored Deramiocel and that the large majority were at or near statistical significance.
In the combined HOPE-2 and HOPE-3 efficacy analysis, patients receiving Deramiocel lost less than one point of upper limb function over 12 months, compared with about two points for placebo recipients, Marbán said. The combined difference was 1.29 points, with a p-value of 0.0087.
HOPE-3 also examined cardiac outcomes. Marbán said the difference in ejection fraction—a measure of heart pumping function—did not reach statistical significance across all patients. Among patients with existing cardiomyopathy, a prespecified subgroup, ejection fraction improved by approximately 2.8 percentage points relative to placebo, she said.
Asked whether earlier treatment could delay the onset of cardiomyopathy, Marbán said longer-term evaluation would be needed. She also declined to disclose whether cardiac data might appear in a potential drug label.
Launch Planning and Distributor Dispute
Capricor is preparing for a possible launch independently while it pursues a contractual dispute with Nippon Shinyaku and NS Pharma over the Deramiocel distribution agreement. Marbán said an arbitration hearing is expected to begin in mid-2027 and that the company does not anticipate the proceedings will prevent it from bringing the treatment to patients if approved.
“We are going independently in preparation for launch and commercialization of Deramiocel without the participation of NS Pharma at this point in any way,” Marbán said. She added that the arbitration panel could influence how the matter proceeds.
Capricor’s San Diego manufacturing facility is operational and ready to supply an initial launch, according to Marbán. Its first-floor clean room can support approximately 250 patients or 1,000 doses a year. The company is building out a second floor, with validation targeted for the first half of 2027; at full capacity, the facility is expected to support 2,000 to 2,500 patients, or up to 10,000 doses annually.
Marbán said Capricor does not anticipate further FDA inspections before the target action date. Asked about a July inspection, she said the company had submitted the required documents and had received no further follow-up questions, but that the FDA would determine how to manage the information.
Loss Widens as Spending Rises
Chief Financial Officer AJ Bergmann said Capricor’s third-quarter net loss widened to approximately $42.7 million, or $0.73 per share, from $24.6 million, or $0.54 per share, a year earlier. The company recognized no revenue in either third quarter.
Operating expenses rose to approximately $44.7 million from $26.3 million, primarily because of pre-commercial activities and manufacturing scale-up. For the first nine months of 2026, the net loss was approximately $117.3 million, compared with $74.9 million a year earlier.
Capricor ended September with approximately $198.5 million in cash equivalents, restricted cash and marketable securities. Bergmann said the company believes its cash equivalents and marketable securities are sufficient to fund operations for at least the next 12 months under its current plan. Marbán said Capricor reduced its workforce by approximately 10% during the quarter and slowed pipeline work not directly tied to Deramiocel.
Asked what Capricor would do if the FDA rejected the application and requested another trial, Marbán said the company would evaluate the agency’s feedback before deciding whether further investment was warranted. She said management is primarily focused on the possibility of approval.
About Capricor Therapeutics (NASDAQ:CAPR)
Capricor Therapeutics, Inc. is a biotechnology company focused on developing cell and exosome-based therapies for serious diseases. The company’s research and development programs are designed to address conditions involving muscle degeneration, inflammation and tissue damage. Capricor’s lead investigational product is deramiocel, also known as CAP-1002, an allogeneic cardiac-derived cell therapy being developed for Duchenne muscular dystrophy. The company has also developed an exosome-based technology platform intended to support the delivery of therapeutic proteins, nucleic acids and other biological materials.
