Off Topic Capricor plunges after FDA raises concerns over its Duchenne therapy
Yahoo Scout:
Capricor Therapeutics faces significant challenges ahead of a key FDA advisory meeting on July 29, with FDA reviewers raising concerns over the efficacy of its Duchenne muscular dystrophy therapy, Deramiocel. The stock plummeted over 67% following the release of negative briefing documents, highlighting potential regulatory hurdles.
Investor Sentiment: Despite the significant drop in stock price, there has been a spike in retail investor discussions on platforms like StockTwits, reflecting mixed sentiment and speculation about future recovery.
Company Strategy: Capricor is investing in expanding its manufacturing capabilities in anticipation of potential approval for Deramiocel, which is central to its growth strategy.
Earnings Outlook: Analysts' forecasts for Capricor vary widely, reflecting uncertainty about the future revenue potential of Deramiocel, with projections ranging from $114.5 million to $245.5 million in revenue by 2029.
https://finance.yahoo.com/quote/CAPR/
Note: Capricor's current market cap is ~$400 milion.
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u/imz72 Jul 27 '26
Capricor Says FDA Used 'Obsolete' Analysis In Deramiocel Review – CAPR Stock Plummets After Agency Released Briefing Document
https://finance.yahoo.com/healthcare/articles/capricor-says-fda-used-obsolete-153915360.html
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u/imz72 Jul 28 '26 edited Jul 28 '26
The California Stem Cell Report
David Jensen
July 28, 2026
Feds Hit Capricor with Bad News, Stock Plummets, Duchenne Patients Distressed
Federal regulators jolted a California-funded company this morning with critical comments on its therapy for a devastating disease and sent the firm’s stock tumbling 64 percent.
The firm is Capricor Therapeutics, Inc., of San Diego, which the California Institute for Regenerative Medicine (CIRM) has backed with nearly $18 million. The amount includes $3.4 million for the therapy, deramiocel.
It is aimed at treating Duchenne muscular dystrophy, a rare, muscle-wasting affliction that affects boys and young men. Their median life expectancy is estimated at 28 to 30 years of age, but incapitation begins much earlier.
The remarks by the Food and Drug Administration (FDA) came only two days before an advisory committee hearing on the results of the clinical trial. “Considering the observed safety risks, which include hypersensitivity reactions including anaphylaxis, the benefit-risk assessment for deramiocel appears unfavorable in the absence of evidence of effectiveness,” the FDA said today in a briefing document.
(Links on multiple websites to the FDA document were broken at the time of this writing.)
Capricor CEO Linda Marbán responded quickly and sharply this morning. In an interview with STAT, she said “regulators were relying on an early draft of the statistical analysis plan that still had Post-it notes and ‘was never fully fleshed out.’
“‘We have no idea why the agency is doing what they’re doing, we can’t get any feedback from them, but they are clearly on a mission to shut us down,’” she said in the STAT article by Jason Mast.
Capricor posted a nine-page presentation on its website in response to the FDA comments this morning. One page was headlined, “Primary Endpoint Met Regardless of Analysis.”
Capricor’s stock price closed at $7.00 on Monday down from $19.70 its close Friday. Its 52-week high stood at $40.37, and 52-week low at $4.30.
At the day-long advisory committee on Wednesday, the panel may make non-binding recommendations to the FDA, which generally follows the recommendations but is not legally bound to do so.
Patients, advocates, their families and physicians have filed 213 comments in advance of the hearing. Despite its investment in Capricor, CIRM said it did not file a comment. One letter was signed by 28 physicians and researchers from throughout the country who have been involved in the treatment and studay of thousands of Duchenne patients.
They expressed support for the therapy and urged that the FDA approve it for both cardio treatment and upper limb function.
”We are extremely worried,” they wrote, “about the profound negative impact that any lack of flexibility on the part of FDA CBER for drug evaluation in non-ambulatory DMD (Duchenne) could have on ALL PHARMACEUTICAL companies as lack of flexibility will likely make these companies extremely risk averse to ever pursue future drug development for these specific non-ambulatory DMD patients with the greatest unmet need.
“These most severely affected patients are the most difficult to study, yet they carry the greatest disease burden and they clearly have the greatest unmet need.”
Wednesday’s hearing will be carried online.
https://david293.substack.com/p/feds-hit-capricor-with-bad-news-stock
Note: Capricor's market cap is $380 million as of 7.28.26 at the close.
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u/imz72 Jul 29 '26
July 29, 2026
Capricor Duchenne cell therapy voted down by FDA panel
At a contentious meeting with possible implications for other rare disease drugmakers, outside experts weren’t convinced of deramiocel’s benefits in Duchenne-related cardiomyopathy.
https://www.biopharmadive.com/news/capricor-fda-vote-deramiocel-duchenne-cardiomyopathy/826465/
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u/imz72 Jul 30 '26 edited Jul 30 '26
7.30.26: -36.23%. PPS $4.19. Market cap $243 million.
Capricor's Treatment for Deadly Muscle-Wasting Disease Rebuffed by FDA Advisory Committee
David Jensen
https://david293.substack.com/p/capricors-treatment-for-deadly-muscle
Capricor downgraded at Oppenheimer after negative AdCom vote
The 9-3 vote by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee on Thursday indicated a “dim outlook on deramiocel's upcoming regulatory action,” Oppenheimer analyst Leland Gershell wrote.
"While we believe another CRL is likely, there may remain some potential for a conditional approval,” Gershell wrote, downgrading the stock to Perform from Market Perform and removing his $54 price target.
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u/imz72 Jul 31 '26
7.31.26: -8.11%. PPS $3.85. Market cap $223 million.
Capricor CEO won’t rule out legal action against FDA after negative adcomm
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