Why Capricor May Be the Next Big Name in Duchenne Muscular Dystrophy

Health and Healthcare

Published: July 15, 2019 11:05 am

Last Updated: January 11, 2020 7:43 am

Capricor Therapeutics Inc. (NASDAQ: CAPR) shares more than doubled on Monday after the firm announced interim analysis for its Duchenne muscular dystrophy (DMD) midstage trial. Overall, there were significant results across several independent clinical measures. Specifically, these results are coming from the Phase 2 clinical trial of the company’s lead investigational therapy, CAP-1002.

In the interim analysis, top-line data from a total of 17 patients was analyzed in the per-protocol population (10 placebo and seven treated) at the three-month point and 12 patients (six placebo and six treated) were analyzed at the six-month point.

Ultimately, the results showed a statistically significant outcome relative to placebo controls in Performance of the Upper Limb 2.0 at six months, with supportive, positive treatment effects also seen in some independent skeletal and pulmonary assessments. Positive trends, although not statistically significant, were observed in other skeletal, pulmonary and cardiac measures.

Previously, the U.S. Food and Drug Administration (FDA) granted Capricor RMAT and Orphan Drug Designation. Additionally, the FDA had granted a Rare Pediatric Disease Designation to CAP-1002.

Looking forward, upon receiving market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher.

Linda Marbán, president and CEO of Capricor, commented:

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 randomized double-blind, placebo-controlled study in DMD that has shown statistically significant functional improvement in steroid treated boys.

Shares of Capricor were last seen up just over 100% to $6.65. The 52-week range is $2.50 to $15.80, and the consensus price target is $35.00.