FDA Rejects CAPR's Cell Therapy BLA for Genetic Disorder, Stock Tanks
DMD is a serious genetic disorder showing progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years.
Per the CRL, the FDA informed the company that it had concluded review of the BLA but could not approve the application in the current form. The regulatory body specifically noted that the application failed to meet the statutory requirement for substantial evidence of effectiveness and highlighted the need for additional clinical data.
The CRL also highlighted certain unresolved issues within the Chemistry, Manufacturing and Controls ('CMC') section of the application. Management believes that most of these concerns have already been addressed in its prior communications with the FDA. However, the FDA did not review these materials, as they were submitted too close to the issuance of the CRL.
Year to date, shares of Capricor have plunged 44.7% compared with the industry's decline of 0.9%.
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CRL to CAPR's BLA Comes as a Major Surprise
The investor community seemed surprised by the CRL, especially after the FDA accepted and granted a priority review to the BLA for deramiocel in March. A decision was due on Aug. 31, 2025. More so, the FDA also did not identify any review issues with the BLA.
In a regulatory update provided last month, the company also confirmed that the FDA indicated an Advisory Committee meeting would not be required for the application.
The BLA was based on data from its phase II HOPE-2 and an open-label extension study compared with natural history data.
A potential approval could have made deramiocel the first therapy to treat DMD cardiomyopathy. However, the latest CRL is likely to delay the approval for deramiocel, which could have given Capricor its first approved product.
The company plans to hold further discussions with the FDA to determine the next steps of development for deramiocel.
Capricor Therapeutics, Inc. Price
Capricor Therapeutics, Inc. price | Capricor Therapeutics, Inc. Quote
CAPR's Zacks Rank & Stocks to Consider
Capricor currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the biotech sector are Arvinas ARVN, Alkermes ALKS and BioXcel Therapeutics BTAI, each sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today's Zacks #1 Rank stocks here.
In the past 60 days, Arvinas' 2025 loss per share estimates have improved from $1.60 to $1.51. Loss per share estimates for 2026 have narrowed from $3.28 to $2.98 during the same period. ARVN stock has plunged 59.3% year to date.
Arvinas' earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 82.09%.
In the past 60 days, estimates for Alkermes' earnings per share have increased from $1.74 to $1.79 for 2025. During the same time, earnings per share estimates for 2026 have risen from $1.79 to $1.91. Year to date, shares of ALKS have gained 5.3%.
Alkermes' earnings beat estimates in one of the trailing four quarters, while missing the same on the remaining three occasions, the negative average surprise being 8.24%.
In the past 60 days, BioXcel Therapeutics' 2025 loss per share estimates have improved from $16.44 to $8.60. Loss per share estimates for 2026 have narrowed from $23.76 to $7.02 during the same period. BTAI stock has declined 67.1% year to date.
BioXcel Therapeutics' earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 42.84%.
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