StealthX - (Project NextGen, NIAID trial)

March 12, 2026: Capricor Therapeutics (NASDAQ: CAPR) reported FY/Q4 2025 results and provided a corporate update highlighting its lead program deramiocel (CAP-1002) for Duchenne muscular dystrophy (DMD). The U.S. FDA is reviewing Capricor’s BLA seeking full approval; the agency stated the company’s response to a prior Complete Response Letter was complete to support continued review and assigned a PDUFA target action date of August 22, 2026 (Class 2 resubmission).

Clinical update: The pivotal HOPE-3 Phase 3 trial (n=106) met its primary endpoint (PUL v2.0; p=0.03) and a key secondary cardiac endpoint (LVEF; p=0.04); all Type I error-controlled secondary endpoints were statistically significant, with a safety/tolerability profile consistent with prior studies. Late-breaking HOPE-3 analyses presented at the 2026 MDA Clinical & Scientific Conference showed reduced myocardial fibrosis by cardiac MRI (LGE three-segment treatment difference vs placebo at 12 months; p=0.022), LVEF improvement vs placebo in baseline cardiomyopathy patients (+3.3 percentage points; p=0.017), and DVA “eat 10 bites” task ~83% slowing of progression vs placebo (p=0.018).

Manufacturing/commercial: The San Diego GMP facility completed an FDA pre-license inspection; Form 483 observations were addressed, and the site is operational to support a potential launch.

Other catalysts: A NIAID-sponsored Phase 1 StealthX™ exosome-based vaccine trial is ongoing; final results are expected in Q2 2026. Capricor uplisted to the Nasdaq Global Select Market on March 9, 2026. Cash and marketable securities were ~$318.1M at Dec. 31, 2025, expected to fund operations through 2027.