Capricor’s share price rockets up by 150% on Phase 2 Duchenne muscular dystrophy success

pharmafile | July 16, 2019 | News story | Manufacturing and Production, Research and Development CAP-1002, Capricor Therapeutics, Duchenne Muscular Dystrophy, pharma 

Capricor Therapeutics has unveiled impactful new Phase 2 data for its investigational allogeneic cardiosphere-derived cell therapy CAP-1002 in the treatment of Duchenne muscular dystrophy, a progressive genetic disorder which brings chronic inflammation of skeletal, heart and respiratory muscles.

The company’s share price rocketed by as much as 150% on the publishing of the new data.

The findings were pulled from a pre-specified analysis of six months of Phase 2 efficacy data. The 17 examined patients – around 80% of whom were non-ambulant – had received either CAP-1002 or placebo intravenously, and then assessed for skeletal muscle function using the Performance of the Upper Limb (PUL) 1.2 and 2.0 tools. Results measured with the 1.2 version were positive and consistent with previous studies, while additional independent tests showed statistically significant improvements in grip strength and positive results in tip to tip pinch strength.

Additionally, a statistically significant improvements was observed in inspiratory flow reserve after 3 months, and positive trends were observed in improvements to cardiac muscle function, including thickening of the systolic wall and cardiac mass, with CAP-1002 compared to placebo.

“I am incredibly pleased with the outcome of the interim analysis as it has demonstrated the biologic activity of CAP-1002 that has resulted in changes of clinically relevant outcomes including the upper limb, the hand and diaphragmatic function,” explained Dr Craig McDonald, principal investigator for the HOPE-2 clinical trial and UC Davis Professor and Chair of its Department of Physical Medicine and Rehabilitation. “For these older boys who have no further therapeutic options, these data support the hope that CAP-1002 may one day become an important therapeutic option and possibly slow the advancement of the disease.”

Linda Marbán, President and CEO of Capricor, also remarked on the data: “We are extremely pleased and it is truly extraordinary that even in such as small sample size, we achieved statistically significant improvements in several clinically relevant parameters. In these older patients, functional improvement in the upper limb is highly meaningful for their quality of life. To our knowledge, this is the first randomised double-blind, placebo-controlled study in DMD that has shown statistically significant functional improvement in steroid treated boys.”

Matt Fellows

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