CAPR Stock Surges 67% After-Hours — Is There Still A Path Forward For Deramiocel?

CEO Marbán said Capricor remains focused on delivering the therapy to patients and is working closely with the FDA on a path forward centered on improvements in upper-limb skeletal muscle function demonstrated in trials.

• All pipeline work not related to deramiocel is on hold right now, Capricor said.
• The FDA has expressed its willingness to accept the company’s amended application, the company said. 
• The company ended the quarter with around $238 million in cash, which it views as sufficient to fund operations for at least the next 12 months.

Shares of Capricor Therapeutics (CAPR) soared 67% in after-hours trading on Thursday after the company outlined a clearer regulatory path for its drug candidate deramiocel after a negative advisory committee vote last month.

Focus Remains On Getting Deramiocel To Patients

On July 29, the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee reviewed Capricor’s Biologics License Application for Deramiocel in Duchenne muscular dystrophy. It voted on whether the available data for Deramiocel provided substantial evidence of effectiveness for treating cardiomyopathy associated with DMD. The vote was 3 in favor and 9 against.

CEO Linda Marbán noted the outcome was “not the outcome we had planned for, and we are of course disappointed.” The committee was not asked to vote on whether the drug’s positive impact on upper-limb function, as shown in the late-stage HOPE-3 trial, was sufficient to approve the drug, or on its overall benefit-risk profile, the company noted.

Marbán said Capricor remains focused on delivering the therapy to patients and is working closely with the FDA on a path forward centered on improvements in upper-limb skeletal muscle function demonstrated in trials. Following discussions with the FDA, the company plans to submit an amendment to its application that includes 24-month open-label extension data from HOPE-3, along with additional analyses.

“FDA has indicated it is willing to review this amendment and upon receipt to extend the PDUFA action date accordingly,” Marbán said. The current target action date is August 22.

Capricor is finalizing the timing of the submission, the company said, while adding that manufacturing remains ready for an initial launch if approved. The company is continuing commercial readiness activities at a slower pace until further regulatory clarity.

All pipeline work not related to deramiocel is also on hold right now, Capricor said. However, Capricor has begun early discussions with regulators in Europe and Japan about eventually obtaining approval for Deramiocel there, though it will only move forward depending on what happens with the U.S. FDA process.

Capricor’s Q2 Financials

The company ended the quarter with around $238 million in cash, which it views as sufficient to fund operations for at least the next 12 months.

For the second quarter, the company reported a wider-than-expected net loss of about $0.70 per share, with higher spending on research and development activities.

How Did CAPR Retail Traders React?

On Stocktwits, retail sentiment around CAPR stock jumped from ‘bearish’ to ‘neutral’ over the past 24 hours, while message volume remained ‘low.’

A Stocktwits user voiced optimism about the eventual approval of deramiocel, which could take the company to a $40 billion market cap, and dismissed the company’s Q2 loss.

Another user dismissed the company’s update.

CAPR stock has fallen 85% year-to-date. 

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