Capricor Therapeutics (NASDAQ:CAPR) rallied 14.6% in premarket trading on Wednesday after the company reported encouraging topline data from its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel for Duchenne muscular dystrophy (DMD).
The placebo-controlled, double-blind study reached its primary goal, showing a 54% reduction in the rate of upper-limb functional decline (PUL v2.0) compared with placebo (p=0.029). The therapy also hit a key secondary endpoint tied to cardiac health, delivering a 91% slowing in deterioration of left ventricular ejection fraction (LVEF) (p=0.041).
The year-long trial enrolled 106 patients across 20 clinical sites in the U.S., with treatments administered once every three months. The average patient age was about 15, and more than three-quarters had cardiomyopathy at baseline.
“HOPE-3 delivered strong and definitive evidence that Deramiocel can meaningfully improve the course of Duchenne muscular dystrophy, demonstrating statistically significant improvements in both skeletal and cardiac function,” said CEO Linda Marbán.
Capricor intends to leverage these results to address a previous Complete Response Letter from the FDA and believes the new data will form a strong foundation for potential approval.
Still, the announcement drew skepticism from some corners of the market. Notably, investor Martin Shkreli — who is short the stock — questioned the findings, saying: “I think CAPR failed. it doesn’t say prespecified primary endpoint. they’re taking the primary endpoint SCALE and doing post-hoc analyses on it! 1.2 point improvement on 42 point scale is a 3% improvement vs. placebo — that can’t be stat sig”
Capricor reiterated that Deramiocel remains well-tolerated, with no new safety signals identified. The company expects to present full results at upcoming medical conferences and pursue publication in peer-reviewed journals.