Capricor Presents Additional Positive Data from Ongoing HOPE-2 Study of CAP-1002 in Duchenne Muscular Dystrophy at World Muscle Society: Data Demonstrates Improved PUL 2.0 Performance at 6 Months

--Company Exploring Potential for Accelerated Approval under RMAT Designation, an Expedited Program for Regenerative Therapies--

--Company to Host Conference Call Today at 5:30 AM PT / 8:30 AM ET--

LOS ANGELES, Oct. 07, 2019 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company, in its presentation at the 24th Annual International Congress of the World Muscle Society, presented additional six-month interim data from HOPE-2, its ongoing Phase 2 clinical trial of its lead investigational product, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD). CAP-1002 is Capricor’s cell-based therapeutic candidate whose mechanism of action is immunomodulatory, anti-fibrotic and has been shown to generate new muscle cells.

Data from the pre-specified interim analysis demonstrated that teens and young men in the advanced stages of DMD saw improvements in skeletal, pulmonary, and cardiac measurements after receiving multiple doses of CAP-1002. Specifically, patients showed improvements in the Performance of the Upper Limb (PUL), a tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist & hand) function, with a conceptual framework reflecting the progression of weakness in ambulant and non-ambulant patients. As previously reported, additional independent tests assessing grip strength and tip to tip pinch strength also showed positive results.   

The U.S. Food and Drug Administration (FDA) has suggested the use of the updated PUL 2.0 version as the primary efficacy endpoint in support of a Biologics License Application (BLA). The PUL scale was designed to measure upper limb motor performance across the spectrum of severity of DMD and is specifically focused on patients that are unable to perform the six-minute walk test.

“We are very pleased that the interim analysis from this double-blind placebo-controlled study, has demonstrated meaningful improvements across three clinically relevant endpoints in older patients with limited remaining treatment options,” said Linda Marban, PhD, Chief Executive Officer of Capricor. “We continue to explore ways to utilize our FDA-granted Regenerative Medicine Advanced Therapy (RMAT) designation to pursue accelerated approval of CAP-1002, and we believe the agency’s written guidance on the matter supports our efforts to introduce this potentially groundbreaking therapy to physicians and patients as quickly as possible. We look forward to meeting with FDA later this year to further define our path to commercialization.”

For the interim analysis, data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 3- and 6-month time-point in the intent to treat (ITT) population. Approximately 80% of the patients were non-ambulant and all patients were on a stable regimen of steroids. Demographic and baseline characteristics were similar between the two treatment groups.

The late breaking podium presentation presented the pre-specified top-line, 6-month results from the HOPE-2 clinical trial of CAP-1002. By expanding the skeletal assessment beyond the mid-level and evaluating patients’ PUL “scores” at the upper and distal dimensions, Capricor’s CAP-1002 showed meaningful improvements, as shown in the summary table below. 

Skeletal Assessments at 3 and 6-month time-points (PUL 2.0)

Time-point3 months6 months
Treatment CAP-1002
Shoulder + Mid + Distal Level0.5 (1.69)-1.2 (1.69)0.0549-0.3 (0.52)-2.3 (1.49)0.0299
Mid + Distal Level 
0.4 (1.30)-0.4 (0.70)0.10350.2 (1.47)-1.4 (0.92)0.0177
0.1 (0.99)-0.4 (0.52)0.2202-0.2 (1.17)-1.1 (0.99)0.0612

Mean Change from baseline (standard deviation) shown.
ITT (intent to treat) population shown
Comparisons treated vs. placebo using mixed model repeated ANOVA with covariates

To reduce the risk of future adverse events, Capricor initiated a commonly used pre-medication strategy including oral steroids and antihistamines to prevent or mitigate potential allergic reactions during the administration. Since the initiation of the pre-treatment regimen, approximately 40 infusions of investigational drug (CAP-1002 or placebo) have been administered to HOPE-2 patients with only one serious adverse event reported that required an overnight observation of the patient.

“The results from this trial to date are very promising in that the cells appear to positively impact skeletal, pulmonary and cardiac assessments in older DMD patients who have few, if any, remaining treatment options. We are eager to meet with the FDA to discuss the next steps for this promising program,” said Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial and UC Davis professor, Chair of its Department of Physical Medicine and Rehabilitation and Professor of Pediatrics.

The FDA has granted Capricor’s CAP-1002 RMAT and Orphan Drug Designation, for which the FDA has also granted a Rare Pediatric Disease Designation. The Rare Pediatric Disease Designation, as well as the Orphan Drug Designation previously granted, covers the broad treatment of DMD. If Capricor were to receive market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher.

Capricor expects to announce further updates on its clinical development strategy for CAP-1002 in DMD later this year, including an update on guidance it receives from the FDA.

Conference Call and Webcast

Capricor will host a conference call and webcast with slides today, October 7, 2019, at 8:30 a.m. ET to discuss the updated interim results of the HOPE-2 clinical trial. To participate in the conference call, please dial 866-717-4562 (domestic) or 210-874-7812 (international) and reference the access code: 8074349.

To participate via a webcast, please visit: The webcast will be archived for approximately 30 days and will be available at

About HOPE-2

HOPE-2 is a randomized, double-blind, placebo-controlled, Phase II clinical trial of the company’s lead investigational therapy, CAP-1002 in steroid-treated boys and young men who are in advanced stages of DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of progenitor cell that has been shown in pre-clinical and clinical studies to exert potent immuno-modulatory activity, and is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 150 human subjects across several clinical trials.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. The HOPE-Duchenne trial was funded in part by the California Institute for Regenerative Medicine. For more information, visit
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Cautionary Note Regarding Forward-Looking Statements

Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings, and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission on March 29, 2019, and as amended by its Amendment No. 1 to Annual Report on Form 10-K/A filed with the Securities and Exchange Commission on April 1, 2019, in its Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2019, as filed with the Securities and Exchange Commission on August 8, 2019, and in its Registration Statement on Form S-3 as filed with the Securities and Exchange Commission on October 24, 2018, and as amended by its Amendment No. 1 to Form S-3 filed with the Securities and Exchange Commission on July 17, 2019, together with prospectus supplements thereto. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

CAP-1002 is an Investigational New Drug and is not approved for any indications. CAP-2003 has not yet been approved for clinical investigation.

For more information, please contact:

AJ Bergmann, Chief Financial Officer