Capricor Shares Jump on Positive Phase 3 Muscular Dystrophy Drug Data
Pivotal HOPE-3 trial for cell therapy deramiocel meets primary and key cardiac endpoints, paving the way for a renewed FDA submission.
Shares of Capricor Therapeutics Inc. (NASDAQ: CAPR) jumped in pre-market trading Wednesday after the biotechnology firm announced positive pivotal data from a late-stage trial of its lead drug candidate for Duchenne muscular dystrophy (DMD), a severe genetic disorder.
The company reported that its Phase 3 HOPE-3 study for deramiocel (CAP-1002) met its primary endpoint, demonstrating a statistically significant slowing of disease progression in the upper limbs of patients. Crucially, the trial also met a key secondary endpoint related to cardiac function, a major area of concern for those with DMD.
The positive results represent a major clinical victory for Capricor and position the company to resubmit its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA). In a company announcement released early Wednesday, Capricor highlighted the results as statistically significant and clinically meaningful.
According to the top-line data, the study showed a 54% slowing of disease progression as measured by the Performance of the Upper Limb (PUL v2.0) scale, with a p-value of 0.029. The therapy also demonstrated a 91% slowing in the decline of left ventricular ejection fraction, a measure of the heart's pumping ability, with a p-value of 0.041. DMD is characterized by progressive muscle degeneration and weakness, typically leading to severe cardiac and respiratory complications.
Investors reacted favorably to the news, which significantly de-risks the path to market for deramiocel. Capricor, which closed Tuesday with a market capitalization of approximately $304 million, is now poised to compete in the growing DMD treatment market, which is projected to reach approximately $3.42 billion in 2025.
The successful trial provides a clear path forward for Capricor to address a previous Complete Response Letter from the FDA. The company stated its intention to submit its response incorporating the new Phase 3 data, renewing hopes for an approval that could bring a new class of cell therapy to DMD patients.
Wall Street analysts have been optimistic about Capricor's prospects leading up to the data release. Prior to Wednesday's announcement, the consensus rating on the stock was a "Strong Buy," with price targets ranging from $13 to $30 per share. The positive outcome of the HOPE-3 trial is expected to reinforce this bullish sentiment and may lead to upward revisions of those targets.
The competitive landscape for DMD treatments is fierce, including gene therapies from Sarepta Therapeutics and exon-skipping drugs from PTC Therapeutics. However, Capricor's deramiocel offers a different approach as a cell therapy designed to regenerate skeletal and cardiac muscle cells, potentially offering benefits to a broad range of DMD patients, including those with advanced disease.
Looking ahead, Capricor's management will now focus on preparing its comprehensive data package for the FDA. Investors and patients will be closely watching for the agency's acceptance of the BLA filing, which would mark the next major milestone on the path to potential commercialization.