Shares of Capricor Therapeutics Inc (CAPR) soared on Tuesday after the company announced its intention to file a biologics license application (BLA) with the U.S. Food and Drug Administration (FDA) for full approval of Deramiocel, a potential treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy.
Capricor’s decision to file the BLA comes after recent constructive meetings with the FDA. Based on these discussions, the company plans to submit the application in October, aiming for full approval of Deramiocel for the treatment of DMD-cardiomyopathy by the end of the year. The filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials.
To further bolster the application, Capricor intends to combine Cohorts A and B of the Phase 3 clinical trial to serve as a post-approval study. This strategic move highlights the company’s commitment to demonstrating the long-term effectiveness and safety of Deramiocel.
“There are currently no approved therapies for DMD cardiomyopathy, which is the leading cause of death in those with Duchenne. Based on the strength of our cardiac data, combined with the FDA’s commitment to advancing therapeutics for the treatment of rare diseases, we are seeking approval for the cardiomyopathy associated with DMD and will look to expand the label for skeletal muscle myopathy post-approval,” said Linda Marbán, CEO of Capricor Therapeutics.
“Deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. Based on the totality of evidence of the safety and efficacy data deramiocel has shown, we believe this is the best path forward to potential approval.”
The potential for Deramiocel to become the first approved treatment for DMD cardiomyopathy has sent Capricor shares soaring. The stock has seen a remarkable increase of over 55% in the past week, propelled by the recent European expansion deal with Nippon Shinyaku and positive analyst sentiment. Several analysts have raised their price targets on the stock following these developments.
Here’s a summary of the analyst updates in the past week:
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HC Wainwright & Co. analyst Joseph Pantginis
: Reiterated Capricor Therapeutics with a Buy rating and maintained a price target of $40.*
Maxim Group analyst Jason McCarthy
: Reiterated Capricor Therapeutics with a Buy rating and maintained a price target of $12.*
Oppenheimer analyst Leland Gershell
: Maintained Capricor Therapeutics with an Outperform rating and raised the price target from $14 to $15.*
Cantor Fitzgerald analyst Kristen Kluska
: Reiterated Capricor Therapeutics with an Overweight rating and maintained a price target of $8.The positive news surrounding Deramiocel and the growing investor interest in the company have created a bullish sentiment around Capricor shares. The stock is up 27.1% at $7.63 at the time of this writing, according to Benzinga Pro.
If approved, Deramiocel could significantly improve the lives of patients suffering from DMD cardiomyopathy, offering hope for a brighter future.
