Capricor Therapeutics (CAPR.O) shares more than doubled in premarket trading on Friday after the company said the U.S. health regulator is willing to review new data on its experimental Duchenne muscular dystrophy drug.
The stock had crashed last month after an FDA advisory panel voted nine-to-three against the effectiveness data of the drug, deramiocel.
The panel's vote aligned with concerns of the FDA staff that Capricor had changed how it measured the trial's main outcomes, and questioned whether enrolled patients even had Duchenne-related cardiomyopathy, given their normal heart function at baseline.
Capricor Chief Executive Linda Marban said, late on Thursday, the company will submit an amendment with new 24-month data focused on improving upper limb muscle function, the main goal of its late-stage study.
Some panel members had been more receptive to the treatment's arm and hand function data than its contested heart benefits.
The FDA has said it is willing to review this new information and will push back its decision deadline once it gets the data, Marban said. The agency's decision was expected by August 22.
"We are continuing to work closely with FDA on a potential path forward," Marban said, adding that the study's main muscle-function results remain "significant both statistically and clinically."
Shares of Capricor surged 118% to $9.18 before the bell, though still down sharply from $19.70 before last month's FDA setbacks.
The U.S. Department of Health and Human Services, which oversees the FDA, did not immediately respond to a Reuters request for comment on the amended submission and potential new review timeline.
Cantor analyst Kristen Kluska called the FDA's willingness to review the new muscle data a "potentially positive surprise", noting the agency would have little reason to extend the deadline if it had already decided against approval.
Capricor, which had $237.9 million in cash at the end of June, said it is pacing its commercial launch spending while it awaits regulatory clarity.
