Capricor Hosts Call to Discuss Heart Complications of DMD, Its CAP-1002 Cell Therapy

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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Capricor Therapeutics will host a conference call and webcast with a cardiac expert to discuss heart complications associated with Duchenne muscular dystrophy (DMD) and will also provide an update on the development of CAP-1002, its lead cell therapy candidate for people with DMD.

The event, at 1:30 p.m. EDT on April 15, will feature Michael Taylor, MD, PhD, a pediatric cardiologist at the Cincinnati Children’s Hospital Medical Center and director of Advanced Imaging Innovation and cardiac magnetic resonance at the center’s Heart Institute.

Details on how to join the call are available here, and registration to watch the webcast is here.

During the call, Taylor will give a presentation focused on cardiac complications of DMD, as well as its progression, and treatment options. He will be available at the end of his presentation to answer questions.

CAP-1002 is made up of heart progenitor cells harvested from a healthy donor, called cardiosphere-derived cells, that have regenerative properties and the ability to reduce inflammation and tissue scarring. The therapy is currently being investigated for DMD due to its potential to modify the body’s immune system in a way to promote heart repair.

A Phase 1/2 trial (NCT02485938) called HOPE showed that when administered directly into the heart, treatment with CAP-1002 was able to improve heart muscle function and reduce tissue scarring in people with DMD.

CAP-1002 is being evaluated in a Phase 2 trial (NCT03406780) called HOPE-2, which is assessing the therapy’s safety and efficacy in boys with DMD. During the trial, boys are randomly assigned to receive either CAP-1002 or a placebo, both administered by an intravenous infusion (directly into the bloodstream), every three months, for a period of one year.

Early data from HOPE-2 showed that compared to a placebo, CAP-1002 significantly improved boys’ grip strength, arm muscle function, and lung function within a period of three to six months. Mild improvements in heart function and tip-to-tip pinch strength were also reported in boys receiving CAP-1002.

“We plan to announce final 12-month data from our HOPE-2 study by the middle of the second quarter as all patient visits have now been completed,” Linda Marbán, PhD, CEO of Capricor, said in a press release. “Based on the positive results we saw at six months, we are hopeful that the data will remain strong at 12 months after active patients will have received up to four doses of CAP-1002 over the course of one year.”

“Although we recognize the challenges we all face in the midst of the international COVID-19 pandemic, we remain committed to our goal of advancing our therapeutic for patients with DMD and look forward to working with the FDA after receipt of this data with the goal of bringing this therapeutic to patients as quickly as possible,” she added.