Capricor Therapeutics Inc. (CAPR:NASDAQ) decided on a path toward approval of its cell therapy, Deramiocel, in Duchenne Muscular Dystrophy (DMD) after a Type A meeting with the U.S. Food and Drug Administration (FDA), reported Alliance Global Partners Analyst James Molloy in a Sept. 26 research note.
"In our view, the company has been in need of guidance from the agency on a go-forward plan to get Deramiocel across the finish line," Molloy wrote. "That clarity was provided to the market yesterday, and we feel it is a positive outcome for Capricor."
145% Implied Return
Alliance Global maintained its US$16 per share price target on the California-based biotech, trading at the time of Molloy's report at about US$6.52 per share, the analyst noted. From this price, the return to target is 145%.
Capricor Therapeutics remains a Buy. It has 45.72 million shares outstanding, a market cap of US$349.2 million (US$349.2M) and a 52-week range of US$5.68–23.40 per share.
FDA Meeting Takeaways
Molloy presented the key points made in Capricor's Type A meeting with the FDA. One, instead of a new biologics license application (BLA), the biotech may submit a major amendment to the currently open BLA. This will comprise the upcoming data from HOPE-3, a placebo-controlled, blinded study of 104 boys with Duchenne. This approach could translate into a shorter lag time between amendment submission and possible approval, the analyst pointed out.
Two, the primary endpoint for HOPE-3 will remain performance of upper limb (PUL), and the secondary endpoint will be left ventricular ejection fraction (LVEF). This is instead of switching the primary endpoint to LVEF from PUL. This approach could support a dual label of improvement in both skeletal and cardiac muscle function, noted Molloy. This would be the first dual label for any DMD treatment and the first DMD treatment with a label for cardiomyopathy.
"In the event that the topline skeletal endpoint is not statistically significantly improved, but LVEF is, we still think there is a similar path to approval for Deramiocel," Molloy wrote. "Cardiomyopathy is now the leading cause of death in DMD patients."
Three, according to the FDA, Capricor resolved all of the CMC (chemistry, manufacturing and controls) issues mentioned in the agency's previous Complete Response Letter.
At this point, after the Type A meeting with the FDA and a clear path forward, Capricor "is in a conventionally strong position in biotech," the analyst wrote. The life sciences firm passed a prelicense inspection, ameliorated CMC issues, has positive data on its asset and has more confirmatory Phase 3 data on the way.
What to Watch For
Next steps for Capricor regarding Deramiocel could catalyze its share price. By YE25, Capricor will unblind and analyze the topline mature data from HOPE-3.
"We expect the upcoming HOPE-3 LVEF and PUL data to continue to show Deramiocel's clinical efficacy and safety in this patient population," Molloy wrote.
By early Q1/26, the company will release the HOPE-3 data and file the major amendment to their existing BLA. The FDA could return a PDUFA date for Deramiocel as early as March-April 2026, noted the analyst.
The best outcome, according to Molloy, would be approval under a dual label for the improvement of skeletal muscle and cardiac muscle function in DMD patients.
"We are bullish on Deramiocel's chances for approval," the analyst wrote. "We continue to project that Capricor will receive a priority review voucher upon approval (worth about US$160M)."
Molloy pointed out that Capricor has sufficient funds, about US$120M in cash, to commercially launch Deramiocel.
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Disclosures for Alliance Global Partners, Capricor Therapeutics Inc., September 26, 2025
Disclosures "Firm" used in the this section of the report entitled "Disclosures" refers to A.G.P. / Alliance Global Partners or Euro Pacific Capital, a division of A.G.P. / Alliance Global Partners. The Firm expects to receive or intends to seek compensation for investment banking services from all companies under research coverage within the next three months. The Firm or its officers, employees or affiliates, other than the research analyst authoring this report and his/her supervisor, may execute transactions in securities mentioned in this report that may not be consistent with the report’s conclusions. Sources referenced in this report: The information and statistics in this report have been obtained from sources we believe are reliable but we do not warrant their accurance or completeness. Matthew Venezia: The analyst has long positions in the shares of Johnson & Johnson which is not covered by AGP Alliance Regulation Analyst Certification ("Reg AC") — The views expressed in this report (which include the actual rating assigned to the company as well as the analytical substance and tone of the report) accurately reflect the personal views of the analyst(s) covering the subject securities. An analyst's sector is the universe of companies for which the analyst provides research coverage. Accordingly, the rating assigned to a particular stock represents solely the analyst's view of how that stock will perform over the next 12 months relative to the analyst's sector average. Furthermore, in accordance with FINRA Rules 2711, 2241, and their amendments related to disclosure of conflicts of interest, the analyst preparing this report certifies: • The analyst or member of the analyst's household does not have a financial interest in the company that is the subject of this report, including a position in the debt or equity of the company, without limitation, whether it consists of any option, right, warrant, future, long or short position. • The analyst or member of the analyst's household does not serve as officer, director or advisory board member of the company that is the subject of this report. • The analyst has not received any compensation from the subject company or from investment banking revenues, directly or indirectly, for preparing this report. • The report discloses all material conflicts of interest related to the analyst, the member firm, and the subject company that are known at the time of publishing this report.
Ratings Buy: Expected to materially outperform sector average over 12 months and indicates total return of at least 10% over the next 12 months. Neutral: Returns expected to be in line with sector average over 12 months and indicates total return between negative 10% and 10% over the next 12 months. Sell: Returns expected to be materially below sector average over 12 months and indicates total price decline of at least 10% over the next 12 months. Not Rated: We have not established a rating on the stock. Under Review: The rating will be updated soon pending information disclosed from a near-term news event. Volatility Index 1 (Low): Little to no sharp movement in stock price in a 12 month period 2 (Low to medium): Modest changes in stock price in a 12 month period 3 (Medium): Average fluctuation in stock price in a 12 month period 4 (Medium to High): Higher than average changes in stock price in a 12 month period 5 (High): Extremely sharp movements in stock price in a 12 month period All financial information is taken from company disclosures and presentations (including Form 10Q, 10K and 8K filings and other public announcements), unless otherwise noted. Any prices or quotations contained herein are indicative only and are not a commitment by A.G.P. / Alliance Global Partners to trade at any price. If A.G.P. / Alliance Global Partners acts in a principal capacity with respect to the instruments mentioned herein it will be disclosed in the previous section of this report entitled “Disclosures.” In the event that A.G.P. / Alliance Global Partners does act in a principal capacity, the commentary is therefore not independent from the proprietary interests of A.G.P. / Alliance Global Partners, which interests may conflict with your interests. Opinions expressed herein may differ from the opinions expressed by other divisions and/or business units of A.G.P. / Alliance Global Partners. The Firm does not undertake any obligation to update this material. This material is current as of the indicated date and as of the time it was sent to you. This material was prepared from information believed to be reliable, but A.G.P. / Alliance Global Partners makes no representations or warranties as to its accuracy or completeness. This communication and the information contained herein is neither an offer to buy or sell nor a solicitation of an offer to buy or sell any security or instrument or to participate in any particular trading strategy. This report should not be used as a complete analysis of the company, industry or security discussed in the report. Additional information is available upon request. Any opinions or estimates in this report are subject to change without notice. An investment in the stock may involve risks and uncertainties that could cause actual results to differ materially from the forward-looking statements. Additionally, an investment in the stock may involve a high degree of risk and may not be suitable for all investors. No part of this report may be reproduced without the express written permission of A.G.P. / Alliance Global Partners, member FINRA/SIPC. Copyright 2025.
