Capricor Therapeutics’ stock has soared by more than 370% after a pivotal trial of its Duchenne muscular dystrophy (DMD) cell therapy candidate met its endpoints.
The Phase III HOPE-3 trial (NCT05126758) enrolled 106 patients with ambulatory and non-ambulatory DMD who received either intravenous deramiocel at 150 million cells per infusion or placebo every three months for a 12-month period.
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In the performance of upper limb (PUL v2.0) total score, there was a 54% difference in the slowing of progression between patients treated with deramiocel versus a placebo. In left ventricular ejection fraction (LVEF), the difference in the slowing of progression was 91%.
The cell therapy maintained a favourable safety and tolerability profile consistent with prior clinical experience.
Dr Craig McDonald, the trial’s principal investigator and distinguished professor of physical medicine and rehabilitation and paediatrics at UC Davis Health, said: “A nearly 54% slowing of skeletal muscle disease progression is extraordinary in DMD and directly linked to maintaining independence and quality of life in the most severely affected patients with greatest unmet need.
“The preservation of function reflected in PUL v2.0 translates into real, practical benefits for boys and young men living with this disease, and the effect of deramiocel on cardiomyopathy will potentially translate to improved long-term survival.”
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By GlobalDataNotably, the fact that non ambulatory patients have benefited from the treatment could put Capricor in a good position after a label update for Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl) removed it as a treatment option for the non-ambulatory population.
Sarepta is seeking a solution to this by enrolling a new cohort into the Endeavour trial, in which non-ambulatory patients will receive sirolimus as well as the gene therapy to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF). The Endeavour trial was previously linked with two deaths after the gene therapy’s dosing in non-ambulatory patients.
Capricor’s bounce back
Capricor was trading at $6.36 at market close on 2 December, with data announced before market open the following day. After the market opened, the stock hit a high of $40.27, but this settled down to a close of $29.96 – a 371.07% rise between each close period.
High share prices will be a huge relief for Capricor after it received a complete response letter (CRL) from the US Food and Drug Administration (FDA) in July 2025, which was published without the company’s knowledge in September 2025.
The allogenic cardiosphere-based cell therapy was rejected by the FDA because the HOPE-2 (NCT05126758) and its open-label extension portion “failed to demonstrate efficacy for its prespecified primary … and secondary endpoints”. However, Capricor rebutted this claim in September 2025, noting that the primary endpoint data was analysed with a non-parametric test. This indicated that it needed transforming to reach a statistically significant p value of 0.014.
In announcing the HOPE-3 trial meeting its endpoints, Capricor’s CEO Dr Linda Marbán said the new data showcases deramiocel’s strong clinical profile.
Marbán said: “These results reinforce the durable benefits seen in HOPE-2 and its open-label extension, which has continued for over 48 months, and highlight the strength, consistency, and reproducibility of deramiocel’s clinical profile after more than a decade of rigorous clinical development.
“We believe these pivotal study results, in addition to the evidence from the HOPE-2 and HOPE-2 OLE studies, position us to address the clinical issues in the CRL received earlier this year, consistent with prior FDA guidance that HOPE-3 results should be sufficient to support regulatory approval.”
Capricor said that more detailed HOPE-3 results will be submitted for presentation at a future scientific meeting and for publication in a peer-reviewed journal.
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