Capricor files cell therapy for DMD cardiomyopathy with FDA
Capricor Therapeutics has completed its US marketing application for deramiocel, a cell therapy the US biotech hopes will become the first approved treatment for cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
The filing is viewed as the first stage for Capricor in its commercialisation strategy for deramiocel, as it waits for clinical data that could extend the use of the cell therapy into the treatment of the skeletal muscle-wasting (myopathy) associated with DMD.
The completion of the FDA filing prompts a $10 million milestone payment to Capricor from Japan's Nippon Shinyaku, which licensed US rights to deramiocel in 2022 and added in European rights in a second deal towards the end of 2024. It also has rights to the therapy in Japan.
Capricor's chief executive, Linda Marban, said the competition of the filing is a "pivotal step" for the San Diego-based company and "the culmination of a body of work that has been focused on bringing this potentially transformational therapy to those patients in need."
Capricor revealed three-year data from the HOPE-2 open-label extension (OLE) study in June that revealed improvements in multiple measures of cardiac function, including left ventricular ejection fraction (LVEF), as well as improvements in upper limb performance scores compared to a matched cohort of untreated DMD patients.
Deramiocel (formerly CAP-1002) consists of donor cardiosphere-derived cells (CDCs) given by intravenous infusion once every three months that are thought to modulate the immune system and regenerate skeletal and cardiac muscle cells.
The cells are not thought to engraft into muscle, but exert their effects by secreting various anti-inflammatory and regenerative factors.
If approved, Capricor could be in line to receive a priority review voucher from the FDA, which can be used to shorten the regulatory review of new therapies and can be worth upwards of $100 million if sold on to a third party.
The company has previously said that it sees deramiocel as an add-on therapy to current chronic treatments for DMD, including corticosteroids and exon-skipping therapies.
Deramiocel is approaching the market as new treatments for DMD are starting to emerge. Last year, Sarepta – which leads the market for exon-skipping DMD drugs – got FDA approval for Elevidys (delandistrogene moxeparvovec), the first gene therapy for the muscle-wasting disease. The label for the gene therapy was extended last year, while Pfizer abandoned the development of rival gene therapy fordadistrogene movaparvovec.
CAP-1002 was previously partnered by Johnson & Johnson in a 2014 deal as a potential treatment for myocardial infarction, but J&J returned rights to the drug after it failed a phase 2 trial.
Image by un-perfekt from Pixabay
