Capricor’s DMD Therapy Shows 76% Slower Upper-Limb Decline, Analyst Expects CRL In November

 

• Patients who received placebo for one year and then switched to Deramiocel saw their rate of upper-limb decline slow by 76%.

• The FDA had extended its decision date from August 22 to November 22.

• Roth said another FDA panel focused specifically on upper-limb preservation is possible.

Capricor Therapeutics (CAPR) was on investors' radar after the biotech firm presented new long-term data suggesting its Duchenne muscular dystrophy therapy (DMD) may slow the loss of upper-limb function, the critical part of its case ahead of the U.S. Food and Drug Administration decision in November.

At the Annual Congress of the World Muscle Society (WMS) last week, Capricor showcased data from the ongoing Phase 3 HOPE-3 open-label extension. Patients who received placebo for one year and then switched to Deramiocel saw their rate of upper-limb decline slow by 76% in the following year.

Why Upper Limb Function Is Particularly Important

DMD is a rare genetic disease that causes muscles to become progressively weaker over time. Upper-limb function is particularly important in DMD as arm strength becomes critical for everyday activities such as eating and controlling a wheelchair.

"Upper limb function is critical to quality of life for young men with DMD because it is directly tied to their independence. Their arms are how they feed themselves, control their wheelchairs and use their phones, and how they stay independent. Time is muscle, and slowing that loss means more time doing the things that matter most to them," said Craig McDonald, Principal Investigator of the HOPE-3 trial at UC Davis Health.

FDA Review Shifted Toward Upper-Limb Benefit 

The new data take on greater importance after an FDA advisory committee voted 9-3 against the strength of evidence supporting Deramiocel for DMD-related cardiomyopathy in July. Panelists questioned the cardiac data and Capricor's statistical analysis, although some advisers viewed the upper-limb results more favorably.

Capricor subsequently submitted the 24-month extension data and additional analyses, with its proposed indication more focused on preserving upper-limb function. The FDA then extended the PDUFA deadline from August 22 to November 22, giving the agency additional time to review the new package.

HC Wainwright Expects FDA CRL

H.C. Wainwright said Capricor's 24-month extension data were encouraging, but they did not remove the statistical uncertainty from the original Hope-3 trial. The firm still expects the FDA is more likely to issue a Complete Response Letter (CRL) on November 22.

Roth Capital also said the new data strengthen the case for Deramiocel's upper-limb benefit, but noted that the treatment effect was modest early on and the extension study was not randomized. Roth said another FDA panel focused specifically on upper-limb preservation is possible.

https://finance.yahoo.com/healthcare/articles/capricor-dmd-therapy-shows-76-155902409.html