To hear Capricor Therapeutics’ chief executive Linda Marbán tell it, the company started its phase II trial of its Duchenne muscular dystrophy candidate CAP-1002 merely hoping for a suggestion of a trend towards efficacy. The statistically significant interim data the group reported today was apparently entirely unexpected, and drove its shares up 124%, though the group's market cap remains a tiny $25m. CAP-1002 is a cell therapy based on cardiosphere-derived cells taken from donor hearts, which Capricor believes can help patients’ muscles regenerate. The Hope-2 results are particularly interesting as the trial enrolled older, more severely affected DMD patients, a group in whom gene therapies might face challenges since very large doses would be required. As positive as the Hope-2 data are, the trial is too small for the group to request accelerated approval from the FDA, Ms Marbán said on a call today. Only seven patients received CAP-1002 at the trial’s dose of 150 million cells per intravenous infusion every three months, with 10 in the placebo arm. The trial will continue until the 12-month time point, and if final results are good enough the company just might be able to file on them.
|Six-month Hope-2 interim data|
|Change in score on the mid-level performance of the upper limb PUL 2.0||-0.2 (1.17)||-0.8 (0.75)||0.0389|
|Change in tip to tip pinch strength||3.3 (2.88)||-0.3 (1.51)||0.0674|
|Change in grip strength||0.8 (4.54)||-2.2 (1.83)||0.0389|
|Source: company communications.|