Trial Tuesday: Same Trial, Two Verdicts, As Deramiocel Meets The FDA Panel

Tomorrow morning, a US Food and Drug Administration advisory panel sits down to answer a simple question: does one trial prove that deramiocel works?

The Cellular, Tissue, and Gene Therapies Advisory Committee meets July 29, with the FDA's final verdict on Capricor’s deramiocel due by August 22. Going in, the two sides are telling almost opposite stories about the same clinical trial.

Let's dig into the gap in this week's Trial Tuesday.

The treatment, from Capricor Therapeutics, is an allogeneic, cardiosphere-derived cell therapy for cardiomyopathy, the heart-muscle disease that develops in people with Duchenne muscular dystrophy (DMD). DMD is a rare, X-linked genetic condition that mostly affects boys and steadily breaks down muscle, including the heart. Several DMD treatments already exist, but none have been studied for their effects on cardiac progression, so their impact on the heart is still unknown. This is why the FDA calls this an area of high unmet need.

Here's the strange part: the FDA and Capricor are looking at the same Phase III study – HOPE-3, a randomized, placebo-controlled trial of about 104 boys – and reaching contradictory conclusions about whether the therapy is efficacious.

In its pre-meeting briefing, the agency's central complaint is procedural. The FDA says Capricor rewrote the trial's Statistical Analysis Plan (SAP) repeatedly after the blinded portion of the study ended, and that the final version was locked in just a day before the data were unblinded – changing, among other things, how missing data were handled. Because the results shift depending on which SAP version is used, the FDA argues the "winning" analyses should be treated as exploratory rather than confirmatory.