Capricor Therapeutics Announces Long-Term Benefit of Deramiocel (CAP-1002) in Both Skeletal Muscle and Cardiac Function in the HOPE-2 OLE Study in Duchenne Muscular Dystrophy
Capricor Therapeutics (NASDAQ: CAPR) announced positive long-term results from the HOPE-2 OLE study for its drug deramiocel (CAP-1002), targeting Duchenne muscular dystrophy (DMD). The study showcased improvements in both skeletal muscle and cardiac function over three years, including left ventricular ejection fraction (LVEF) and indexed volumes. Patients exhibited a statistically significant benefit in the Performance of the Upper Limb (PUL v2.0) score compared to an external comparator. A pre-BLA meeting with the FDA is scheduled for Q3 2024 to expedite the Biologics License Application (BLA) filing. The results will be presented at the PPMD Annual Conference on June 29, 2024.
- 3-year treatment showed sustained improvements in skeletal muscle and cardiac function.
- Patients had a statistically significant improvement in the PUL v2.0 score (+3.7 points, p<0.001).
- Pre-BLA meeting with FDA scheduled for Q3 2024 to expedite BLA filing.
- No approved treatment currently exists specifically for DMD cardiomyopathy, highlighting the potential market opportunity for deramiocel.
- None.
Insights
The recent long-term data regarding deramiocel (CAP-1002) provides substantial evidence that this treatment could potentially alter the progression of Duchenne muscular dystrophy (DMD). Sustained improvements in both skeletal and cardiac functions are particularly noteworthy for two reasons. Firstly, for patients with DMD, maintaining function in these areas is critical for their quality of life. The data points showing a statistically significant improvement in the Performance of the Upper Limb (PUL v2.0), as well as improved or stabilized cardiac measures, highlight a meaningful impact on disease progression. These results suggest that deramiocel could fill a significant treatment gap, particularly given the lack of approved therapies for DMD cardiomyopathy.
One key insight is the observed benefit in patients who commenced treatment with higher ejection fractions. This aligns with existing literature on the importance of early intervention in managing cardiomyopathy. For stakeholders, understanding that early treatment initiation might yield better outcomes could influence future therapeutic strategies and patient management.
Finally, the favorable safety profile over an extended period is a positive indicator for potential long-term use. Safety is a critical consideration for chronic conditions, where patients require ongoing therapy.
The announcement of the 3-year study results for deramiocel (CAP-1002) is highly significant from a financial perspective. The data supports the therapeutic's efficacy and safety, both of which are essential metrics for investor confidence. Importantly, this study's positive outcomes bolster Capricor's upcoming plans to discuss an expedited Biologics License Application (BLA) filing with the FDA. Successful FDA approval could potentially open up a new revenue stream, given the unmet need in the DMD market.
Investors should also consider the market exclusivity that might come with such an approval, potentially offering Capricor a significant competitive edge. With no approved treatments specifically for DMD cardiomyopathy, deramiocel could capture a substantial market share, further amplified by its orphan drug designation and potential for premium pricing.
In the short term, the news could catalyze a positive reaction in Capricor's stock price. However, long-term investors should keep an eye on the pre-BLA meeting outcomes and subsequent regulatory milestones, which will be critical for the commercial viability of deramiocel.
The long-term results of deramiocel have important implications not only for current stakeholders but also for the broader market landscape. The observed benefits in cardiac and skeletal muscle functions are critical differentiators in a highly specialized market with few effective treatments. Moreover, these outcomes could potentially set a new benchmark for therapeutic effectiveness in DMD management.
Capricor's strategy to present detailed study results at the PPMD Annual Conference underscores its commitment to transparency and community engagement, which are important for building trust and support among patients, caregivers and healthcare providers. This engagement will likely contribute to broader acceptance and adoption, should the therapy gain regulatory approval.
Market analysts should also note the broader implications for Capricor's research pipeline. Successful progress with deramiocel could enhance the company's reputation and credibility, potentially easing the path for future products. This can attract strategic partnerships, research funding and improve the company's overall market positioning in the biotech space focused on rare diseases.
-Results in Performance of the Upper Limb (PUL v2.0) Continue to Show Slowing of Disease Progression in Later-Stage DMD Patients-
-Improvements Seen in Multiple Cardiac Endpoints Demonstrating Stabilization of Cardiac Function Over 3 Years of Treatment-
-Pre-BLA Meeting with FDA Scheduled for Q3 2024 to Discuss Options to Expedite BLA Filing-
-Results to be Presented at PPMD Annual Conference on June 29 in Session titled “Approaches to Alter Progression”-
SAN DIEGO, June 28, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced additional positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, deramiocel (CAP-1002), for the treatment of Duchenne muscular dystrophy (DMD).
Data from the HOPE-2 OLE study demonstrated improvements in multiple cardiac measures, 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 measures of cardiac function and are considered highly relevant in terms of predicting long-term outcomes. In addition, greater improvements in cardiac function were observed in those patients that had higher ejection fractions (>
3-Year HOPE-2 OLE Study Results
| Primary endpoint Skeletal muscle (upper-limb function) | 3-year timepoint Change from baseline* | Delta change p-value | |
| Performance of upper limb (PUL v2.0) | Deramiocel (n=12) | External comparator (n=32) | |
| -4.1 points | -7.8 points | +3.7 points p< 0.001 | |
External comparator PUL data provided by Cincinnati Children’s Hospital Medical Center (CCHMC)
*Baseline is referred to as start of HOPE-2 OLE study, changes in means are shown
| Secondary endpoints Cardiac function | 3-year timepoint Change from baseline* | |||
| Deramiocel (n=10**) | Deramiocel (n=8) Patients with > | |||
| Left ventricular ejection fraction (LVEF %) Positive value indicates improvement | + | + | ||
| Indexed volumes Negative value indicates 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 referred to as start of HOPE-2 study, changes in medians are shown; 3-year timepoint on HOPE-2 OLE corresponds to 5 years post HOPE-2 Baseline
All cardiac outcomes were measured using magnetic resonance imaging (cMRI)
**10 of 12 participants were able to receive MRI
“The results of the open label study are tremendously important for DMD patients, as they showed sustained skeletal and cardiac benefits after 3 years of continuous treatment with deramiocel, which underscores the potential long-term benefits this therapy can offer patients with DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “Based on our HOPE-2 OLE data and prior clinical results, we plan to discuss with the U.S. Food and Drug Administration (FDA) options to expedite our Biologics License Application (BLA) filing. We continue to work closely with FDA with the goal of bringing deramiocel to patients as quickly as possible and look forward to sharing further updates as they become available. We thank the patients, their families and the broader Duchenne community for continuing to work with us throughout the development of this promising therapy.”
The PPMD Annual Conference is the largest annual, international event focused on the latest research, clinical trials and care initiatives for DMD. Now in its 30th year, the meeting is attended by researchers, caregivers and patients looking to interact and drive change in the fight to end DMD. For more information, or to register, please click here. Capricor plans to make its presentation available on the publications section of the Company’s website following the formal conference presentation.
About HOPE-2 Open Label Extension (OLE) Study
HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, deramiocel, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either deramiocel (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet. After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment entered the HOPE-2-OLE study where they receive deramiocel (150 million cells per infusion) every three months. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 (p=0.02). The HOPE-2-OLE study remains ongoing and into its fourth year and participants continue to be monitored for safety, cardiac and functional performance.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes 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-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter.
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; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and 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, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as 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 assumes no obligation to update these forward-looking statements.
Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.
For more information, please contact:
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
FAQ
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