Capricor Therapeutics Inc. (NASDAQ:CAPR) has announced that it will present proprietary data from its double-blind, randomized phase 2 HOPE-2 clinical study evaluating its experimental treatment, CAP-102, in young men and boys having Duchene muscular dystrophy.
Capricor to present HOPE-2 study results at the 2020 International Muscle Society Congress
Craig McDonald, the Chair CINRG Duchenne Natural History study and the lead investigator in the HOPE-2 study and its management, will discuss the results. The results will be presented in an e-poster at the 2020 virtual International World Muscle Society Congress. Duchenne muscular dystrophy is a genetic condition characterized by chronic inflammation and progressive weakness in skeletal, respiratory, and heart muscles. Usually, DMD patients will lose the ability to walk in the teenage years and die due to respiratory or cardiac complications by the age of 30.
Capricor CEO Linda Marban said that DMD is a rare disease with thousands of patients currently experiencing the disease’s immobilizing effects nationally. Linda said that the company’s lead asset, CAP-1002, can exert immunomodulatory activity, and the Capricor is committed to its goal of establishing a treatment path and alleviating DMD symptoms for enhanced quality of life for young adults and boys. She also said that the company discusses the program with the FDA to ascertain the next steps and potential approval pathways.
Capricor reported positive HOPE-2 study results
In May this year, the company reported positive topline results of the 12-month HOPE-2 clinical study evaluating CAP-1002 in treating patients with advanced DMD. According to the data, there was an improvement in cardiac, respiratory, and upper limb function with p values of less than 0.05. The 12-month results from the HOPE-2 study demonstrated statistically significant improvement in PUL 2.0 in patients treated with CAP-1002 with a median change of 2.4 over control patients.
Apart from steroids, preserving muscle function in DMD patients is uncommon, and those on placebo in the study declined consistently with natural history. However, there was stable and enhanced muscle function in the treated group through the 12-month treatment period.