Capricor Therapeutics has notched a key win in its efforts to bring its Duchenne muscular dystrophy cell therapy, deramiocel, to market, hitting the primary endpoint in a phase 3 trial.
In 105 boys and young men enrolled in the HOPE-3 trial, patients given deramiocel every three months for a year had 54% slower deterioration in upper limb function compared to placebo, Capricor announced in a Dec. 3 release, achieving the study’s main goal.
In the key secondary endpoint of left ventricular ejection fraction, a measure of heart function, deramiocel bested placebo with a 91% slowing of disease progression in 83 patients, Capricor said.
Both measures were statistically significant.
Treatment reflected a safety profile similar to past studies of deramiocel, the biotech added. The cell therapy consists of donor-derived cardiac cells called cardiosphere-derived cells, which are meant to reprogram macrophages to combat inflammation.
Capricor’s stock soared on the news, shooting up from $6.36 per share at Tuesday's market close to a high of $40.37. As of 10:50 a.m. ET on Dec. 3, the share price has settled at $25.56, an increase from yesterday of about 300%.
San Diego-based Capricor submitted a biologics license application for deramiocel last year on the back of data from a phase 2 study and open-label extension, which similarly showed that the therapy improved patients’ upper limb and heart function.
But despite the FDA previously okaying Capricor’s study designs, the company said, the drug regulator rejected deramiocel in July, and later made its complete response letter (CRL) public in September as part of a new initiative to release CRLs as they are issued to sponsors.
“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 complete response letter received earlier this year, consistent with prior FDA guidance that HOPE-3 results should be sufficient to support regulatory approval,” Capricor CEO Linda Marbán, Ph.D., said in the release.
Capricor now plans to submit its response to the CRL, with the topline HOPE-3 data included, according to the release.
In rejecting deramiocel, the FDA said that the phase 2 trial “failed to demonstrate efficacy for its prespecified primary efficacy endpoint.” Capricor responded that the endpoint actually was met if the proper statistical test was used to analyze the data.