Capricor Therapeutics announced less-than-stellar Q4 2021 and full-year 2021 financial results today. In addition to disappointing financial reports, Capricor said it stopped efforts on its COVID-19 vaccine to focus on other exosomal therapeutics and its Duchenne muscular dystrophy cell therapy program.
Capricor, a biotech company that develops exosome-based therapeutics for a broad range of diseases, has had generally positive results from its therapies. In August 2021, Capricor announced it was working with the U.S. Army to publish a study on the use of cardiosphere-derived exosomes to mitigate kidney damage and promote new blood vessel formation after acute trauma. The treatment also served as an anti-shock medication.
However, Capricor’s exosome mRNA COVID-19 vaccine has not been as successful. The U.S. Food and Drug Administration accepted Capricor’s investigational new drug (IND) application in August 2020 for a Phase II clinical trial. The trial used Capricor’s lead therapeutic candidate, CAP-1002, an exosomal allogeneic cell therapy with immunomodulatory properties. Capricor was already using CAP-1002 in trials for Duchenne muscular dystrophy and thought the properties could help with COVID-19 symptoms as well.
The mRNA-based CAP-1002 therapy seemed promising at first. In February 2021, Capricor received a non-exclusive license to intellectual property with Johns Hopkins University to gather data for a serology test platform for COVID-19. The hope was that the immunomodulatory properties could help calm cytokine storms, a side effect of several other COVID-19 vaccines. By November 2020, Capricor announced positive preclinical data on the vaccine.
But somewhere along the line, development stalled. As of Dec. 2021, Capricor was still working on its Phase II INSPIRE clinical trial to assess CAP-1002 as a COVID-19 treatment. Capricor expected to release topline data from the study in Q1 2022, but instead, it sidelined the effort.
“Based on the current availability of vaccines for COVID-19, we have decided to hold off on further development of our exosome-mRNA vaccine,” Linda Marban, Ph.D., Capricor’s chief executive officer, said in the financial update.
The good clinical news from the Q4/full-year 2021 report was still about CAP-1002. The treatment has produced consistently positive results in treating Duchenne muscular dystrophy. In Sept. 2021, Capricor shared positive final data from its Phase II HOPE-2 clinical trial. In patients diagnosed with Duchenne muscular dystrophy and treated with CAP-1002, muscle degeneration slowed significantly and patients retained upper limb function for longer. The pro-immune response from CAP-1002 also benefited patients with regenerated skeletal and muscle cells and anti-fibrotic properties.
Craig McDonald, MD, the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of the Department of Physical Medicine and Rehabilitation, called the study “groundbreaking” and “extremely exciting” for patients.
The Q4/full-year 2021 report said that the upcoming HOPE-3 trial had received $30 million in additional funding, with the potential of another $705 million coming from an exclusive partnership for commercialization and distribution of CAP-1002 with Kyoto-based Nippon Shinyaku that was established in January 2022.
Other critical aspects of the report included dismal financial updates. In 2021, Capricor reported a $20 million net loss, partially due to increased operating expenses. By cutting costs associated with the COVID-19 vaccine, Capricor hopes to focus funding on its Duchenne muscular dystrophy studies. If the plan succeeds, the company said “the cash and cash equivalents will be sufficient to cover expenses and capital requirements for at least two years” to continue its work.
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