What is Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy (DMD) is a rare genetic condition that is ultimately fatal. It causes muscles to weaken and usually results in heart failure before age 30. The disease affects approximately 200,000 males worldwide. There are few effective therapies for DMD and to this day there is no cure.
However, Capricor Therapeutics has been working on the development of a new potential option for patients.
CAP-1002 works to exert immuno-modulatory activity. Researchers believe it could promote cellular regeneration in patients with DMD. Specifically, researchers at Capricor believe it could be an effective treatment for older individuals living with the disease. Older DMD patients are frequently not eligible for clinical trials that are examining gene therapy as a treatment for the disorder. But Capricor’s trial of CAP-1002 is specifically looking to enroll 40-50 older patients.
Their trial is called HOPE-2. It is a Phase II trial which is double-blind, randomized, and placebo-controlled. Currently, 17 patients are enrolled. Originally, Capricor was planning on enrolling 84 patients. However, after talking with the FDA they decided to limit enrollment to a number which would still provide clinically meaningful results, but would accelerate the time to the trials completion. Ultimately, a faster trial means the faster a new treatment could potentially be in the hands of patients.
You may have heard that HOPE-2 was put on hold after a patient in the trial suffered anaphylaxis after treatment. The hold was initiated in December of 2018 and the company took a step back to evaluate how to prevent this reaction for other participants. Further evaluation has indicated that the patient most likely was allergic to an inactive ingredient or some other component of the product. But moving forward, Capricor plans to use a pre-medication strategy to ensure no future patients undergo such a reaction.
After evaluating Capricor’s new approach the FDA and the Data and Safety Monitoring Review Board have approved the resumption of HOPE-2. While a date is not set in stone yet for when it will re-initiate, things are certainly on the upswing for this trial.
Collaboration with the FDA
Capricor Therapeutics has already received Regenerative Medicine Therapy designation for CAP-1002. As part of this designation, the company is granted expedited review of the product.
Collaboration with the FDA has been an essential part of their research progression. Just recently, Capricor had their a comprehensive multidisciplinary meeting with the FDA and they have reported that the conversation was positive. If HOPE-2 continues to show efficacy of CAP-1002, the FDA has announced it can serve as the registration study. Additionally, the FDA has reiterated their support of HOPE-2’s primary endpoint which examines patient’s ability to use the muscles in their arms and hands.
If HOPE-2 continues to show that CAP-1002 has a positive effect for Duchenne muscular dystrophy it could become a new therapeutic option for this population of patients who so desperately needs alternatives. Stay tuned to hear updates on this trial, and the future of CAP-1002 as a potential treatment for DMD.
You can read more about Capricor’s work on HOPE-2 here.