Searching for a cure, Improving lives

Capricor present positive results at World Muscle Society

Posted on: October 4th, 2017 | 0 comments

Capricor presented at the 22nd Annual International Congress of the World Muscle Society, and reported that teens and young men in the advanced stages of Duchenne muscular dystrophy experienced meaningful improvements in cardiac and upper limb function after a single dose of Capricor’s lead investigational product, CAP-1002.

The late breaking abstract and results presented at the late breaking poster session describe the first six months of follow-up data from the randomized 12-month Phase I/II HOPE clinical trial of CAP-1002. CAP-1002 is a cell-based therapeutic candidate and consists of allogeneic cardiosphere-derived cells, whose mechanism of action is immunomodulatory and anti-fibrotic, and which have been shown to generate new muscle cells in preclinical models.

“These findings are especially significant because the patients in the HOPE trial were preteens or young men who were in advanced stages of Duchenne muscle disease,” said Ron Victor, M.D., professor of medicine and Burns and Allen Chair in Cardiology Research at Cedars-Sinai Heart Institute and a principal investigator for the HOPE Trial. “Most other studies in DMD have focused on pediatric patients in earlier stages of the disease. To see such positive results in a clinical trial with just one dose of CAP-1002 sets the stage for the next step of evaluating multiple doses of this innovative cellular therapy in a larger trial.”

About the HOPE Trial

The HOPE Trial was a randomized, open label trial of 25 males living with Duchenne, of ages 12 to 25 years (mean 17.8). For 17 of them, the disease had progressed to the point of wheelchair dependence for mobility. Cardiomyopathy, or heart disease, secondary to Duchenne was an eligibility criterion and was evidenced by scar in four or more left ventricular segments. All participants had been receiving chronic corticosteroid therapy at entry. Thirteen received a single dose of CAP-1002, while the others received the standard of care, and all participants were to be followed for 12 months. CAP-1002 was administered by infusion into each of the three main coronary arteries for a total dose of 75 million cells.


“[The] exciting findings propel us into our next phase of development of CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s president and chief executive officer. “Subject to regulatory approvals, we expect to initiate the randomized, double-blind, placebo-controlled HOPE-2 clinical trial of intravenous (IV), repeat-dose CAP-1002 in the first quarter of 2018. The primary efficacy endpoint will be based on the PUL, and the HOPE-2 Trial may potentially serve as a registration study. We also look forward to presenting 12-month follow-up results from the HOPE Trial at a major medical conference later this quarter.”

Leave a Reply

Your email address will not be published. Required fields are marked *


Support Action Duchenne with a monthly or one off donation



View our events

View All Events


Join the DMD registry for access to clinical trials


Action Duchenne launches new Research Strategy for Duchenne muscular dystrophy

November 17th, 2017

  Action Duchenne, a leading UK-wide patient and parent-led organisation for Duchenne Muscular Dystrophy, is

Tell me more

Long-term microdystrophin pre-clinical work, moving towards a potential therapy restores muscle function in canines

July 26th, 2017

Promising microdystrophin pre-clinical work, moving towards a potential therapy has shown to restore muscle function

Tell me more

FDA new approach would reduce number of patients treated with placebo

December 11th, 2017

The U.S. Food and Drug Administration (FDA) has published its recommendation for a new approach

Tell me more

Results announced from survey ‘Juggling care and daily life’

December 11th, 2017

The 17th October 2017 was an important date for the rare disease community.  The highlights of the

Tell me more

Duchenne – How do you measure a life?

Living with Duchenne – an animation

Taking charge of Duchenne

wordpress counter

Stay informed with regular updates from Action Duchenne