CAPR Stock: Why It Substantially Increased Today

By Amit Chowdhry ● Sep 24, 2021
  • The stock price of Capricor Therapeutics Inc (NASDAQ: CAPR) increased by over 17% pre-market. This is why it happened.

The stock price of Capricor Therapeutics Inc (NASDAQ: CAPR) – a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases – increased by over 17% pre-market. Investors are responding positively to Capricor Therapeutics announcing today positive final data from the HOPE-2 clinical trial using CAP-1002 to treat patients in advanced stages of Duchenne muscular dystrophy (DMD). 

The HOPE-2 clinical trial hadmet its primary efficacy endpoint of mid-PUL v1.2 as well as various skeletal and cardiac endpoints suggesting clinically relevant slowing of disease progression. And CAP-1002 is Capricor’s cell-based therapeutic candidate whose mechanism of action is immunomodulatory, anti-fibrotic and has been shown to regenerate skeletal and cardiac muscle cells. This final data will be presented today at this year’s World Muscle Society Virtual Congress (WMS).

HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical trial of the company’s lead investigational therapy CAP-1002 in boys and young men who have DMD and are non-ambulant, the later stage of the disease process. And the trial was conducted at nine sites across the United States. 

Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. And the data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point in the intent to treat (ITT) population. 

About 80% of the patients were non-ambulant and all patients were on a stable regimen of steroids. And demographic and baseline characteristics were similar between the two treatment groups. Final data analysis demonstrated that young men in the advanced stages of DMD experienced improvements in skeletal and cardiac measurements after receiving four doses of CAP-1002 over the course of 1 year.

Subjects in the trial were evaluated using the Performance of the Upper Limb (PUL), a validated tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist & hand) function, with a conceptual framework reflecting the progression of weakness in upper limb function. CAP-1002 was generally safe and well tolerated throughout the study. With the exception of 2 hypersensitivity reactions early in the clinical trial — which were mitigated with a common pre-medication regimen, there were no serious safety signals identified by the HOPE-2 Data and Safety Monitoring Board (DSMB).

This is the second clinical trial investigating CAP-1002 showing similar results in the treatment of DMD patients. Capricor completed the HOPE-Duchenne (Phase 1/2) trial in 2019 — the results of which were published in Neurology, the medical journal of the American Academy of Neurology. And the company has initiated a technology transfer with Lonza, a leading global CMO to prepare for commercial manufacturing of CAP-1002.


“This groundbreaking study is extremely exciting as we saw statistically significant changes of CAP-1002 in both skeletal and cardiac function. For these older boys who have limited therapeutic options, these data support the belief that CAP-1002 may become an important therapeutic option and possibly slow the progression of DMD.”

— Dr. Craig McDonald, the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of the Department of Physical Medicine and Rehabilitation

“The significance of this data is vitally important to patients and the DMD community. The data suggests that CAP-1002 slowed the decline of DMD in patients for whom few options currently exist. Now that we have clarity from the FDA and based on the strength of this data set, we are poised to embark on the HOPE-3 pivotal trial once we have secured an appropriate partner that can help drive CAP-1002 forward towards commercialization. Most importantly, we are thankful to the patients and families who participated in this study so that we can demonstrate the impact of CAP-1002 in treating DMD.”

— Dr. Linda Marbán, Ph.D., Chief Executive Officer of Capricor

Disclaimer: This content is intended for informational purposes. Before making any investment, you should do your own analysis.