On August 13, Capricor Therapeutics (NASDAQ:CAPR) held its second-quarter earnings call, just over two weeks after an FDA advisory committee voted 3 to 9 against the company's bid to treat cardiomyopathy in ****** nne muscular dystrophy patients on July 29. That rejection forced a strategic pivot. Management now plans to amend its Biologics License Application to pursue a narrower upper limb skeletal muscle indication, the same measure that carried the HOPE-3 trial's primary endpoint. The shift extends the regulatory timeline, but it also puts the spotlight back on the data that held up best.
Deramiocel's case still rests on real numbers. The HOPE-3 primary endpoint showed a statistically significant slowing of upper limb disease progression, with a 4.5% mean difference favoring the drug and a p-value of 0.029. During a separate advisory committee discussion, feedback on upper limb function was described as directionally supportive of that evidence. The cardiomyopathy subgroup's ejection fraction data held too, showing a 2.8 percentage point treatment difference at a p-value of 0.02, unchanged from the original ****** ysis.
Safety data spans more than 1,300 intravenous infusions across over 200 patients in three clinical trials, with more than 80 patients enrolled in open-label extension studies, some receiving infusions for over five years. The full HOPE-3 data set was published in The Lancet in July following independent peer review. The FDA has indicated it will review the coming BLA amendment and extend the PDUFA date accordingly, and Capricor has already opened regulatory conversations in Europe and ****** an while its manufacturing facility in San Diego remains operational for a potential launch.
The financial picture moved the other direction. Cash and marketable securities fell to $237.9 million as of June 30, 2026, down from $318.1 million at the end of 2025. Second quarter net loss widened to $40.7 million, or $0.70 per share, compared to $25.9 million, or $0.57 per share, a year earlier, as operating expenses climbed to $42.9 million from $27.7 million. Research and development spending rose to $28.9 million and general and administrative costs jumped to $14.1 million from $5.7 million. The company booked zero revenue again.
Complicating matters, the left ventricular ejection fraction result across all patients was revised under the prespecified statistical model from a 2.4 percentage point difference at p=0.04 down to 1.8 percentage points at p=0.09, weakening a key secondary measure. An FDA Bioresearch Monitoring inspection in July produced a Form 483 with one observation. Non-Deramiocel pipeline work remains on hold, and the company's arbitration with NS Pharma over their distribution agreement isn't expected to begin until fall 2026.
#limb
Deramiocel's case still rests on real numbers. The HOPE-3 primary endpoint showed a statistically significant slowing of upper limb disease progression, with a 4.5% mean difference favoring the drug and a p-value of 0.029. During a separate advisory committee discussion, feedback on upper limb function was described as directionally supportive of that evidence. The cardiomyopathy subgroup's ejection fraction data held too, showing a 2.8 percentage point treatment difference at a p-value of 0.02, unchanged from the original ****** ysis.
Safety data spans more than 1,300 intravenous infusions across over 200 patients in three clinical trials, with more than 80 patients enrolled in open-label extension studies, some receiving infusions for over five years. The full HOPE-3 data set was published in The Lancet in July following independent peer review. The FDA has indicated it will review the coming BLA amendment and extend the PDUFA date accordingly, and Capricor has already opened regulatory conversations in Europe and ****** an while its manufacturing facility in San Diego remains operational for a potential launch.
The financial picture moved the other direction. Cash and marketable securities fell to $237.9 million as of June 30, 2026, down from $318.1 million at the end of 2025. Second quarter net loss widened to $40.7 million, or $0.70 per share, compared to $25.9 million, or $0.57 per share, a year earlier, as operating expenses climbed to $42.9 million from $27.7 million. Research and development spending rose to $28.9 million and general and administrative costs jumped to $14.1 million from $5.7 million. The company booked zero revenue again.
Complicating matters, the left ventricular ejection fraction result across all patients was revised under the prespecified statistical model from a 2.4 percentage point difference at p=0.04 down to 1.8 percentage points at p=0.09, weakening a key secondary measure. An FDA Bioresearch Monitoring inspection in July produced a Form 483 with one observation. Non-Deramiocel pipeline work remains on hold, and the company's arbitration with NS Pharma over their distribution agreement isn't expected to begin until fall 2026.
#limb
5 days ago
