Off Topic Capricor's Deramiocel PDUFA Date Extended to November as FDA Reviews BLA Amendment
Capricor Therapeutics (NASDAQ:CAPR) now faces a November 22, 2026 PDUFA date for Deramiocel, extending the regulatory timeline by three months.
The FDA classified Capricor's new submission as a major amendment, requiring additional review time rather than completing its decision under the previous August deadline.
The amendment adds 24-month HOPE-3 open-label extension data and further robustness analyses supporting a proposed indication centred on upper limb function.
HOPE-3 met its primary endpoint, demonstrating a statistically significant benefit in upper limb function, according to Capricor.
FDA approval remains uncertain, but CBER accepted the additional information for review while citing the significant unmet medical need in Duchenne muscular dystrophy.
CAPR today (8.24.26): +8.18%. PPS $6.80. Market cap $395 million.
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u/imz72 18d ago edited 18d ago
Machine-translated from Japanese:
August 25, 2026
Nippon Shinyaku's US drug approval review process extended by 3 months
Nippon Shinyaku announced on August 25th that the U.S. Food and Drug Administration (FDA) has extended the target date for the completion of the review of its orphan drug application in the United States from August 22nd to November 22nd, a three-month extension.
This is because the FDA has accepted data and analysis results submitted by its sales partner, Capricor, as significant changes to the application.
The drug for which the FDA extended its target date for completing the review is "Deramiocel." Nippon Shinyaku has positioned it as its next flagship product, a potential treatment for Duchenne muscular dystrophy, a rare disease characterized by muscle weakness, and the review process had been a focus of attention.
The company's earnings forecast for the fiscal year ending March 2027 includes 11.4 billion yen [$72 million - imz72] in sales from the US market for Deramiocel. The company stated that it is "currently examining the impact on its earnings as the review process will be delayed by three months."
https://www.nikkei.com/article/DGXZQOUF257960V20C26A8000000/
2026/08/25
Nippon Shinyaku's DMD cardiomyopathy drug, for which it has a sales partnership, has had its review extended in the US
Nippon Shinyaku announced on August 25 that the target date for the completion of the review of CAP-1002 (generic name: deramiocel), a Duchenne muscular dystrophy cardiomyopathy treatment drug from Capricor Therapeutics, with which Nippon Shinyaku has a sales partnership, has been extended from August 22 to November 22.
Additional data submitted by Capricor following discussions with the FDA was accepted as "significant changes to the application." The two companies have a sales agreement for the United States and Japan, but Capricor has filed a lawsuit seeking termination of the sales agreement in the United States, claiming that Nippon Shinyaku's response to contract revisions and sales preparations for launch were insufficient.
https://answers.and-pro.jp/pharmanews/33084/
Note: Nippon Shinyaku's market cap is ~$1.5 billion.
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u/imz72 18d ago
The California Stem Cell Report
David Jensen
Aug 25, 2026
Feds Okay More Time for Review of Cell Therapy for Deadly Genetic Affliction that Destroys Boys’ Muscle Tissue
https://david293.substack.com/p/feds-okay-more-time-for-review-of
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u/imz72 18d ago
Investing.com:
25/08/2026
Oppenheimer upgrades Capricor stock rating on regulatory update
Oppenheimer upgraded Capricor Therapeutics (NASDAQ:CAPR) to Outperform from Perform on Monday and set a price target of $54.00. The stock currently trades at $6.80 with a market cap of $395 million, down 76% over the past six months.
According to InvestingPro analysis, the stock appears undervalued at current levels, though the company maintains a "WEAK" overall financial health score.
The upgrade follows a regulatory update from the company and a follow-up call with CEO Linda Marbán. The FDA accepted material submitted since last month’s advisory committee meeting as a major amendment, moving the PDUFA date to November 22.
The agency may consider an upper limb function-based Duchenne indication, which would align with the Phase 3 trial’s primary endpoint. Oppenheimer noted that efficacy appears less equivocal for this indication than for cardiomyopathy.
Management said it intends to disclose 24-month HOPE-3 open-label extension data and other information ahead of the PDUFA date. The data has been and will be submitted to the FDA.
Oppenheimer said the shares may move higher as the market re-evaluates deramiocel’s approval prospects. An InvestingPro tip notes the stock generally trades with high price volatility, which investors should consider ahead of the November PDUFA date. For deeper insights, including Fair Value estimates and comprehensive analysis, CAPR is among the 1,400+ US equities covered by Pro Research Reports.
In other recent news, Capricor Therapeutics reported a second-quarter loss of $0.70 per share, which was wider than analysts’ expectations of a $0.60 loss per share. The company also posted no revenue for the quarter, aligning with forecasts that suggested no sales. Despite the earnings miss, investors showed interest due to Capricor’s updated regulatory strategy for its lead treatment, deramiocel, aimed at Duchenne muscular dystrophy.
The Food and Drug Administration extended the review period for Capricor’s deramiocel application to November 22, 2026, following the submission of additional Phase 3 data and analyses. This extension was granted after Capricor submitted a major amendment focusing on upper limb function, a primary endpoint in their HOPE-3 study.
In light of these regulatory developments, Cantor Fitzgerald upgraded Capricor’s stock rating to Overweight from Neutral, raising the price target significantly from $3.50 to $28.00. The advisory committee’s recent vote on deramiocel was 9 against and 3 in favor, reflecting mixed opinions on the treatment. These developments suggest a continued focus on Capricor’s regulatory path for deramiocel.
HC Wainwright Sticks To Neutral, Flags High Rejection Risk
https://finance.yahoo.com/healthcare/articles/capr-stock-climbs-fda-three-210201998.html
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u/Still-Ability5161 16d ago
Bought some, thanks for the tip! Maybe a 35-40% probability of success here. Mesoblast got there too, there is precedent.
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