Capricor Therapeutics announces long-term benefits

Capricor Therapeutics announces long-term benefits
Capricor Therapeutics announces long-term benefits

-Upper limb performance (PUL v2.0) results continue to show a slowing of disease progression in late-stage DMD patients-

-Improvements in several cardiac endpoints demonstrate stabilization of cardiac function after three years of treatment-

-Pre-BLA meeting with FDA planned for Q3 2024 to discuss options to accelerate BLA submission-

-Results will be presented on June 29 at the PPMD ​​Annual Conference in the session entitled “Approaches to Changing Progression”-

SAN DIEGO, June 28, 2024 (GLOBE NEWSWIRE) – Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing breakthrough cell- and exosome-based therapeutics to treat rare diseases, today announced additional positive 3-year safety and efficacy results from its ongoing HOPE-2 Open Label Extension (OLE) study of its lead product deramiocel (CAP-1002) for the treatment of Duchenne muscular dystrophy (DMD).

Data from the HOPE-2 OLE trial showed improvements in several cardiac parameters, including left ventricular ejection fraction (LVEF) as well as indexed volumes (left ventricular end-systolic volume (LVESV) and left ventricular end-diastolic volume (LVEDV)). These are parameters of cardiac function considered highly relevant for predicting long-term outcomes. In addition, greater improvements in cardiac function were observed in patients who had higher ejection fractions (>45%) at the start of the randomized HOPE-2 trial. Published data support the need for early intervention to maintain function and potentially slow the progression of cardiomyopathy, one of the leading causes of death in patients with DMD. There is currently no approved treatment specifically for DMD cardiomyopathy, highlighting the need for additional therapies to treat DMD. In addition, as previously reported, patients demonstrated a statistically significant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score compared to an external comparison dataset of similar DMD patients. The HOPE-2 OLE study continues to demonstrate a favorable safety profile for long-term treatment with deramiocel. These data will be presented at this year’s Parent Project Muscular Dystrophy (PPMD) The annual conference will be held on June 29, 2024 in Orlando, Florida.

Results of the 3-year HOPE-2-OLE study

Primary endpoint
Skeletal muscles (function of the upper extremities)
3-year point in time
Change from baseline*
Delta change
Upper Extremity Performance (PUL v2.0) Deramiocel (n=12) External comparator (n=32)
-4.1 points -7.8 points +3.7 points
p< 0.001

External PUL comparison data provided by Cincinnati Children’s Hospital Medical Center (CCHMC)
*Baseline refers to the start of the HOPE-2 OLE study, changes in mean values ​​are shown

Secondary endpoints
Heart function
3-year point in time
Change from baseline*
Deramiocel (n=10**) Deramicel (n=8)
Patients with >45% LVEF at the end of HOPE-2
Left ventricular ejection fraction (LVEF %)
A positive value indicates an improvement
+1.2% +3.0%
Indexed disks
Negative value means improvement
LV end-systolic volume, indexed (ml/m2 ) -2.4 -5.1
LV end-diastolic volume, indexed (ml/m2) -5.7 -10.0

*Baseline is the start of the HOPE-2 study, changes are shown in median values; 3-year time point for HOPE-2 OLE corresponds to 5 years after HOPE-2 baseline
All cardiac outcomes were measured using magnetic resonance imaging (cMRI).
**10 of 12 participants were able to receive an MRI

“The open-label study results are tremendously important for DMD patients as they demonstrated sustained skeletal and cardiac benefits after 3 years of continuous treatment with deramiocel, underscoring the potential long-term benefits this therapy can provide to patients with DMD,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “Based on our HOPE-2 OLE data and previous clinical results, we plan to discuss options with the U.S. Food and Drug Administration (FDA) to expedite our Biologics License Application (BLA) submission. We continue to work closely with the FDA to make deramiocel available to patients as quickly as possible and look forward to sharing further updates as they become available. We thank patients, their families and the Duchenne community at large for their continued collaboration with us as we develop this promising therapy.”

The PPMD ​​Annual Conference is the largest annual international event focusing on the latest research, clinical trials and care initiatives for DMD. Now in its 30th year,th Each year, the meeting brings together researchers, caregivers and patients who want to share ideas and drive change in the fight against DMD. For more information or to register, please click here. Capricor plans to make its presentation available in the publications section of the company’s website after the official conference presentation.

About the HOPE-2 Open Label Extension (OLE) Study

HOPE-2 was a randomized, double-blind, placebo-controlled Phase 2 clinical trial of Capricor’s lead investigational therapy deramiocel in boys and young men with DMD. Study patients were treated with either deramiocel (150 million cells per infusion) or placebo intravenously every 3 months. After 12 months, data from a total of 20 patients (12 placebo and 8 treated) were analyzed and results published in The Lancet. After completion of the HOPE-2 study, all patients withdrew treatment for approximately 392 days (mean, range (239, 567)), known as the gap period. Subsequently, all eligible patients who wished to continue treatment entered the HOPE-2 OLE study, where they will receive deramiocel (150 million cells per infusion) every 3 months. The HOPE-2 OLE study previously met its primary endpoint at one year on the PUL v2.0 (p=0.02). The HOPE-2-OLE study is currently in its fourth year and participants’ safety, cardiac health and function continue to be monitored.

About Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, cardiac, and respiratory muscles, resulting in death on average at about age 30. DMD is estimated to occur in about one in 3,500 male newborns, and the number of patients in the United States is estimated to be about 15,000 to 20,000. The pathophysiology of DMD is due to impaired production of functional dystrophin, which normally functions as a structural protein in muscle. Lack of functional dystrophin in muscle cells leads to significant cellular damage and eventual muscle cell death and fibrotic replacement. Treatment options are limited, and there is no cure.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate deramiocel (CAP-1002), an allogeneic cardiac cell therapy. Extensive preclinical and clinical studies have demonstrated that deramiocel has immunomodulatory, antifibrotic and regenerative effects specifically tailored to dystrophinopathies and cardiac diseases. Deramiocel is currently in Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also leveraging the power of our exosome technology and using our proprietary StealthX™ platform in preclinical development with a focus on vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a wide range of diseases. At Capricor, we are committed to pushing the boundaries of what is possible and forging a path to transformative treatments for those in need. For more information, visit Also follow Capricor on Facebook, Instagram and Twitter.

Cautionary note regarding forward-looking statements

Statements in this press release regarding efficacy, safety and intended uses of Capricor’s product candidates; initiation, conduct, scope, timing and results of discovery efforts and clinical trials; pace of clinical trial enrollment; plans regarding regulatory filings, future research and clinical trials; regulatory developments relating to products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; schedules of regulatory meetings; statements regarding our financial outlook; the ability to achieve product milestones and receive milestone payments from commercial partners; plans regarding current and future collaboration activities and ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future revenue sources and projections; expectations regarding the expected use of proceeds from the recently completed offerings and the expected impact of the offerings; and any other statements regarding the future expectations, beliefs, objectives, plans or prospects of Capricor’s management team are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements that are not historical facts (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “targets,” “will,” “would” and similar expressions) should also be considered forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated in such forward-looking statements. For more information about these and other risks that may affect Capricor’s business, see Capricor’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed with the Securities and Exchange Commission on March 11, 2024, and our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, filed with the Securities and Exchange Commission on May 14, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor undertakes no obligation to update these forward-looking statements.

Capricor has entered into an agreement for the exclusive marketing and distribution of Deramiocel (CAP-1002) for DMD in the USA and Japan with Nippon Shinyaku Co., Ltd. (US subsidiary: NS PharmaInc.), subject to regulatory approval. Deramiocel is an investigational new drug and is not approved for any indication. None of Capricor’s exosome-based candidates have been approved for clinical investigation.

For more information, please contact:

Company contact Capricor:
AJ Bergmann, Chief Financial Officer
[email protected]

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