Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.
Shares of San Diego-based Capricor Therapeutics Inc. (NASDAQ: CAPR) closed Dec. 3 at $29.96, up $23.60, or 371%, as investors cheered top-line data from the pivotal phase III Hope-3 trial testing cell therapy deramiocel in Duchenne muscular dystrophy.
The winding regulatory road for the BLA to Capricor Therapeutics Inc.’s cell therapy for Duchenne muscular dystrophy has more clarity. Out of a recent type A meeting between Capricor and the U.S. FDA, prompted by a complete response letter in July regarding lead asset CAP-1002 (deramiocel), the two are in agreement about a path to potential approval.
After July’s first-ever release of more than 200 complete response letters (CRLs) by the U.S. FDA, the agency now says it will release letters shortly after sponsors receive them. In addition, the FDA released a new batch of 89 CRLs from 2024 to now that are tied to pending or withdrawn applications.
After about a two-week absence as the U.S. FDA’s CBER director, Vinay Prasad’s return overall prompted a mild reaction on Wall Street for some stocks tied to companies developing cell and gene therapies.
The news that Vinay Prasad has stepped down as CBER director at the U.S. FDA had some biotech stocks literally jumping in joy as the market opened July 30. Meanwhile, Prasad’s decisions regarding vaccine development, as well as actions by Makary and HHS Secretary Robert Kennedy, are coming under fire.
The realignment within the U.S. FDA continued with reports of the removal of two high level executives. When asked by BioWorld if the Center for Biologics Evaluation and Research’s (CBER) Office of Therapeutic Products director and deputy director had been forced out and if so, why, an HHS spokesperson responded on background with a single sentence: “Center directors deserve to be supported by managers that are aligned with aggressive goals to expeditiously advance therapeutics for rare diseases using the gold standard of science.”
The appointment May 6 of Vinay Prasad as the head of the U.S. FDA’s Center for Biologics Evaluation and Research (CBER) “bodes poorly” for Sarepta Therapeutics Inc.’s development-stage pipeline, said Wainwright analyst Mitchell Kapoor – and Wall Street reflected as much, as the stock (NASDAQ:SRPT) ended that day down 26.6% vs. an XBI drop of 6.6% – this ahead of the after-hours earnings disclosure that pushed the Cambridge, Mass.-based firm down even farther by more than another 20%, with the XBI unchanged.
Capricor Therapeutics Inc. just wrapped up a visit with the U.S. FDA and is prepping to file a BLA in October for its Duchenne muscular dystrophy treatment. Linda Marbán, the company’s CEO, is the guest on the newest BioWorld Insider podcast and she talks about deramiocel (CAP-1002), the company’s allogeneic cardiac-derived cell therapy, for treating the rare disease and how the FDA has made strong efforts in helping lay the groundwork for deramiocel.
Capricor Therapeutics Inc. has expanded its commercialization and distribution deal with Nippon Shinyaku Co. Ltd. to include the EU and the U.K. for deramiocel, Capricor’s lead asset, in treating Duchenne muscular dystrophy (DMD). In the new agreement, Capricor will receive an up-front payment of $20 million. Capricor will handle development and manufacturing duties for deramiocel while Nippon Shinyaku will be responsible for the sales and distribution.
