Rare Disease Report

New Data Suggest Repeat Doses of CAP-1002 Enhance Exercise Capacity in DMD

APRIL 19, 2018
Mathew Shanley
According to data presented at the 11th Annual Neuromuscular Translational Research Conference in Cambridge, England, researchers found that repeat dosing of Capricor Therapeutics’ CAP-1002 results in increased exercise performance in a disease model of Duchenne muscular dystrophy (DMD), the mdx mouse.

The company has previously presented data exhibiting that the drug, in single doses, can lead to meaningful improvement in treadmill run times. In the new abstract titled, “Allogenic CDCs have a low immunogenic profile and repeat-dosing showed additional improvement when compared with single treatment in a Duchenne muscular dystrophy model,” it was reported that, while the cellular therapy is recognized by the immune system, its low immunogenic profile and immunomodulatory capabilities permit for it to be administered several times without yielding noteworthy safety issues.1

“These important findings were the foundation for the design of the HOPE-2 clinical trial we will be initiating shortly,” said Linda Marbán, Ph.D., Capricor president and chief executive officer in a press release. “The research found improvements in absolute force in soleus (leg) and in the diaphragm of a mouse model of Duchenne muscular dystrophy, the mdx mouse. Additionally, the researchers observed decreased fibrosis in skeletal and cardiac muscles after three administrations of CAP-1002.”2

In April, Capricor announced interim results from their Phase 1/2 HOPE (Halt cardiomyopathy progression in Duchenne) trial, in which it was found that found a single intracoronary dose of CAP-1002 was generally safe, well tolerated and demonstrated significant and continued signals of improvement in cardiac and skeletal muscle function in boys and young men in the advanced stages of DMD.

After presenting the results, the company was granted Rare Pediatric Disease designation for the potential treatment option in July, giving it the potential to receive a priority review voucher and fast-track a potential future therapy, should CAP-1002 be approved. Shortly thereafter, the company held a successful meeting with the FDA, in which the regulatory authority approved of plans to continue developing the drug.

Additionally, Capricor has also received orphan drug designation for CAP-1002, giving the company 7-year market exclusivity upon approval.

CAP-1002 is made largely from a unique population of cells that consist of cardiac progenitor cells, or allogeneic cardiosphere-derived cells (CDCs), and has exhibited the ability to exert potent immunomodulatory activity and stimulate cellular regeneration.

In the upcoming Phase 2, randomized, double-blind, placebo-controlled HOPE-2 clinical trial, up to 84 boys and young men with advanced stages of DMD will be enrolled to evaluate the safety and efficacy of repeat doses of CAP-1002. HOPE-2 intends to assess the consequences of repeat doses of CAP-1002.

“CAP-1002 also has the potential to work synergistically with gene and other therapies for Duchenne muscular dystrophy,” she said. “While these other therapies have the potential to restore dystrophin expression and sustain muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects. CAP-1002 may be able to work with these therapies because its primary mechanism of action is immunomodulatory, meaning it can help balance inflammation in this chronic inflammatory disease.”

  1. Moseley J, Kanagavelu S, Rachid H, et al. Allogeneic CDCs have a low immunogenic profile and repeat-dosing showed additional improvement when compared with single treatment in a Duchenne muscular dystrophy model. Presented at: 11th Annual Neuromuscular Translational Research Conference; April 19-20, 2018; Cambridge, England.
  2. Capricor Announces New Pre-Clinical Study Finds Repeat Doses of CAP-1002 Lead to Enhanced Exercise Capacity in Duchenne Muscular Dystrophy Disease Model. http://www.irdirect.net/prviewer/release/id/3056245. Accessed April 19, 2018. 

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