Capricor Therapeutics Inc. (NASDAQ:CAPR) stock is trading lower on Monday.
The company provided an update ahead of the company’s upcoming FDA Advisory Committee meeting for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD).
The date for the Advisory Committee meeting is July 29.
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FDA Briefing Document Questions Deramiocel’s Efficacy
In the FDA briefing document, the staff noted that the submitted data from Study HOPE-3, considered together with the results from Study HOPE-2, does not provide substantial evidence of effectiveness for deramiocel in DMD.
“Overall, Study HOPE-3 demonstrates small and variable changes in measures of upper limb skeletal muscle function and in several cardiac imaging parameters that did not reach statistical significance and are, thus, difficult to interpret.”
The results from Study HOPE-2 and HOPE-2-OLE did not demonstrate a
statistically significant treatment difference between deramiocel and placebo on upper limb skeletal muscle decline or cardiac imaging findings (LVEF, LGE, and others) on cardiac MRI.
HOPE-3 Trial Missed Its Primary Endpoint, FDA Says
Study HOPE-3 was designed and statistically powered to demonstrate a treatment difference between deramiocel and placebo on the change from baseline to month 12 in PUL 2.0 total score.
The results demonstrate that this pre-specified primary efficacy endpoint was not met.
Similarly, no statistically significant difference was observed between deramiocel and placebo on the change from baseline in LVEF at month 12.
Changes in other cardiac imaging parameters (LV volumes, LGE) showed small and variable changes of unclear clinical significance.
The results on cardiac imaging parameters taken together are difficult to interpret and do not appear to provide conclusive evidence of a favorable effect of deramiocel on slowing the decline of cardiac function in the studied population.
Company Says It Worked Closely With FDA
The company on Monday commented, “Our results are governed by the final analysis plan, SAP version 3.0, which was finalized prior to unblinding. It is critical to understand that the post-hoc analyses in the FDA’s briefing materials rely on SAP version 1.1, an unsigned incomplete internal draft which became obsolete with the addition of cohort B and did not include content specifically requested by FDA.”
The Phase 3 HOPE-3 results demonstrate a statistically significant benefit on the primary endpoint, PUL 2.0, with supportive benefits in cardiac function,” said Linda Marbán, CEO of Capricor Therapeutics.
The FDA document also noted that the company did not incorporate certain previous FDA comments.
However, on Monday, Capricor said it has engaged fully and transparently with the FDA throughout the review process for our Biologics License Application for Deramiocel and has been responsive to every request from the FDA.
CAPR Stock Price Activity: Capricor Therapeutics shares were down 61.12% at $7.66 at the time of publication on Monday, according to Benzinga Pro data.
Photo: Pavel Kapysh from Shutterstock
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