Capricor Therapeutics, Inc. CAPR announced that CAP-1002 has been granted orphan drug status by the FDA for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). Shares were up 11.28% on the news.
The FDA usually grants orphan drug status to drugs or biologics that are being developed for the treatment of rare diseases/conditions affecting less than 200,000 people in the U.S. at any given time. This designation may allow the product to enjoy a seven-year period of marketing exclusivity in the U.S., upon approval. Incentives that come with orphan drug status include tax credits as well as waiver of certain administrative fees.
According to the U.S. National Library of Medicine, DMD occurs in about 1 out of every 3,600 male infants.
We note that CAP-1002 is also being evaluated in other studies for the treatment of post myocardial infarction (phase II – ALLSTAR) and advanced heart failure (DYNAMIC). The company expects to report top-line results from the DYNAMIC and ALLSTAR studies later this year and by the end of first-quarter of 2017 respectively.
The company expects to file an investigational new drug application with the FDA for CAP-1002 in the near future.
Apart from CAP-1002, another candidate in Capricor’s pipeline is Cenderitide. The candidate is in a phase II study for ambulatory heart failure patients with top-line results expected in the second half of 2015.
We note that Capricor is focused on the development of therapeutics for the prevention and treatment of heart disease. According to the American Heart Association, heart failure is the leading cause of hospitalization, accounting for more than 1 million hospital admissions in the U.S. annually.
We are encouraged by Capricor’s efforts on developing heart failure treatments keeping in mind the patient population and expect investor focus to remain on further pipeline updates.
Capricor is a Zacks Rank #2 (Buy) stock. Some better-ranked stocks in the health care sector include CorMedix, Inc. CRMD, AVEO Pharmaceuticals, Inc. AVEO and Biogen BIIB. All three sport a Zacks Rank #1 (Strong Buy).
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