January 21, 2026 3:03pm

Another Opinion: Is FDA’s No-New-Trial Stance on Deramiocel Reshaping the Investment Case for Capricor Therapeutics (CAPR)? https://finance.yahoo.com/news/fda-no-trial-stance-deramiocel-111135647.html I am NOT the only DISSENTER!  s Capricor Therapeutics (CAPR) Still Attractive After A 72% One Year Share Price Jump. https://finance.yahoo.com/news/capricor-therapeutics-capr-still-attractive-052444220.html

FDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review process. CAPR expects to submit updates to the BLA in February 2026 to support continued FDA review. As previously disclosed, CAPR provided topline results from its P3 HOPE-3 clinical study to the FDA in late 2025. Rejected for more data.

For all my … <some say> … RANTINGS, I say TRUTH …  timing will be everything until the FDA “speaks;” the higher CAPR rose from 12/2’s $6.36, the steeper the fall!

STICKING to my CONVICTION that Capricor Therapeutics (CAPR) is overbought and whose valuation is disconnected from fundamentals.

The FDA has formally requested the full HOPE-3 clinical study report (CSR) and supporting data to address the Complete Response Letter (CRL). The FDA did not request any additional clinical studies or new patient data as part of this request.

Preparation of the HOPE-3 will address the items outlined in the CRL and support continued review of the BLA, including the assignment of a new Prescription Drug User Fee Act (PDUFA) target action date.

  • Don't be swayed by incremental positive swings, a blunderbuss effect will cause a swarf lacking in subtlety

Doubts still abound re: regulatory fragility and marginal HOPE-3 results and valuation concerns remain a key hang-up!

  • CAPR remains a structurally challenged, chronically unprofitable company whose valuation is disconnected from fundamentals.
  • It has begun; timing will be everything!
  • The higher CAPR rises, the steeper the fall!

 

Several companies are developing gene therapies for DMD:

Including Solid Biosciences (SLDB), which is evaluating SGT-003 for DMD. During the 1H/ 2026, SLDB plans to engage in multiple discussions with the FDA to align on the design of a P3 confirmatory study and the evidence required to support a potential accelerated approval pathway for SGT-003, with an update anticipated by mid-2026. In addition, SLDB expects to report data from the P1/2 INSPIRE study on DMD in mid-2026.

At present, Sarepta Therapeutics (SRPT) markets 3 FDA-approved exon skipping drugs, EXONDYS 51 (eteplirsen), VYONDYS 53 (golodirsen) and AMONDYS 45 (casimersen), which are phosphorodiamidate morpholino oligomers, or PMOs, approved for the treatment of patients with DMD who are amenable to exon 51, exon 53 and exon 45 skipping.

  • In 2023, SRPT obtained the FDA’s accelerated approval of its Elevidys, an adeno-associated virus-based gene therapy for the treatment of ambulatory pediatric patients aged 4-5 years with DMD with a confirmed mutation in the DMD gene.

 

Opinions expressed are those of the author and are subject to change, and not intended to be a forecast of future events, a guarantee of future results, nor investment advice. Whether information or intelligence is good, bad, or somewhere in between; I put into context what is relevant and useful for investors.  All investments are subject to risks. Investors should consider investment objectives.

Henry McCusker, the editor, and publisher of RegMed Investors does not hold or have positions in securities referred to in this publication. Regulation Analyst Certification (Reg AC):

The research analyst primarily responsible for the content of this report certifies the following under Reg AC: I hereby certify that all views expressed in this report accurately reflect my personal views about the subject company or companies and it’s or their securities. I also certify that no part of my compensation was, is or will be, directly or indirectly, related to the specific recommendations or views expressed in this report.