Capricor Therapeutics has placed a voluntary hold on its Phase II clinical trial for a Duchene Muscular Dystrophy treatment on hold after a patient experienced a severe allergic reaction during dosing.
Capricor Therapeutics has placed a voluntary hold on its Phase II clinical trial for a Duchene Muscular Dystrophy treatment on hold after a patient experienced a severe allergic reaction during dosing.
First reported by Reuters, Capricor announced the voluntary hold in a filing with the U.S. Securities and Exchange Commission on Dec. 21. The Los Angeles-based Capricor said the patient experienced the reaction during the infusion process. The patient was part of a blinded test, so it is unclear if the individual was dosed with Capricor’s experimental treatment, CAP-1002, or a control. In its filing, Capricor said the patient responded well to medical treatment and at this time, is asymptomatic.
Capricor said it notified the FDA of the reaction and is working with the regulatory agency on a mitigation plan. The company’s filing did not indicate how long the hold will be placed on the trial, dubbed HOPE-2. For the trial, the company had anticipated enrolling approximately 84 boys with DMD who have seen their ability to walk diminish due to the loss of muscle function that occurs with the disease. Capricor believes that if the primary endpoint is reached, the HOPE-2 Trial could serve as a potential registration trial.
Capricor’s CAP-1002 consists of allogeneic cardiosphere-derived cells, which contain cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and stimulate cellular regeneration, the company said. Last year, Capricor announced six-month data from the Phase I/II DMD trial assessing CAP-1002. In that trial, the asset demonstrated statistically significant improvement compared to control. Patients treated with CAP-1002 demonstrated statistically-significant improvement in certain measures of upper limb function compared to control. CAP-1002 was generally safe and well-tolerated over the initial six-month follow-up period.
DMD is a genetic disorder that is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 boys born worldwide, about 200,000 people. The condition is universally fatal, and death usually occurs before the age of 30. Currently, there is only one approved DMD treatment in the United States, Sarepta Therapeutics’ Exondys 51, which skips the exon 51 mutation in DMD patients.
CAP-1002 is designed to release exosomes that are immunomodulatory. The exomes are then designed to apply anti-inflammatory, anti-fibrotic, and anti-apoptotic effects on the patients. Last summer, following a meeting with the FDA, Capricor Chief Executive Officer Linda Marban said by ameliorating the myocyte damage induced by dystrophin mutations, CAP-1002 has demonstrated the ability “to preserve and improve the structure and function of dystrophic skeletal muscle.” Marban said CAP-1002’s mechanism of action supports its potential to be a standalone therapy as well as an adjunct to dystrophin-modulating agents.
While it is unknown if the allergic reaction is a result of dosing with CAP-1002, this would not be the first setback that drug has experienced. Last year, CAP-1002 failed in a mid-stage trial for the treatment of adults who have experienced a large heart attack with residual cardiac dysfunction. That failure forced the company to make some cuts, including layoffs.
