Patients with a deadly muscle-wasting disease received good news this afternoon from a San Diego firm that has developed a therapy for the affliction financed with millions of dollars from the California stem cell and gene therapy agency.
Linda Marbán, CEO of Capricor Therapeutics, said that the firm has devised a work-around to overcome objections raised earlier this month during a critical federal hearing on whether the treatment should be approved for use outside of a clinical trial.
The move involves a fresh focus on how the treatment improves limb function of patients with Duchenne muscular dystrophy. Capricor also plans to submit additional analyses of its clinical trial data.
The announcement sent Capricor’s stock up 59 percent to $6.75 during after hours trading.
Duchenne muscular dystrophy affects about 15,000 boys and young men in this country. The affliction erodes the muscles of their bodies, including their hearts, and leads to an early death.
The California Institute for Regenerative Medicine (CIRM) has funded Capricor with $18 million, including $3.4 million for the treatment, which is known as deramcicel. If approved, it would be only the second CIRM-backed therapy to win federal approval for widespread use.
CIRM had no comment on the Capricor announcement. The $12 billion taxpayer-financed agency has said it does not comment on business developments involving grantees.
Capricor said the Food and Drug Administration (FDA) has agreed to review Capricor’s resubmission. However, it could delay final action, which had been scheduled by Aug. 22.
During a conference call with industry analysts, Marbán said that “it is worth noting” that the FDA advisors earlier this month were “directionally supportive of the clinical evidence for the primary endpoint in upper limb function.”
“We have administered approximately 1,300 intravenous infusions across our clinical program to over 200 patients with DMD in three separate clinical trials. More than 80 patients are in our collective open-label extension studies, with some receiving continuous infusions for more than five years, and the long-term safety profile is consistent and well characterized.”
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