Positive 6-month results for CAP-1002 in Duchenne muscular dystrophy

After plunging back in May on disappointing results with CAP-1002 in adults who have experienced a large heart attack with residual cardiac dysfunction, Capricor Therapeutics (Nasdaq: CAPR) saw its shares leap 21% to $3.97 pre-market today after it presented more encouraging data on the compound in Duchenne muscular dystrophy (DMD).

At the 22nd Annual International Congress of the World Muscle Society, Capricor reported that teens and young men in the advanced stages of DMD experienced meaningful improvements in cardiac and upper limb function after a single dose of its lead investigational product, CAP-1002. DMD is a rare, life-threatening genetic disorder for which treatment options are limited.

The late breaking abstract and results describe the first six months of follow-up data from the randomized 12-month Phase I/II HOPE clinical trial of CAP-1002. CAP-1002 is a cell-based therapeutic candidate and consists of allogeneic cardiosphere-derived cells, whose mechanism of action is immunomodulatory and anti-fibrotic, and which have been shown to generate new muscle cells in preclinical models.