-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
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 (> 45%) at the beginning of the HOPE-2 randomized trial. Published data supports the need for early intervention in order to maintain function and potentially slow the progression of the cardiomyopathy, one of the leading causes of death in patients with DMD. Currently, there is no approved treatment specifically for DMD cardiomyopathy, which underscores the need for additional therapies to treat DMD. Additionally, as previously reported, patients showed a statistically significant benefit (+3.7 points, p< 0.001) in the PUL v2.0 total score when compared to an external comparator dataset of similar DMD patients. The HOPE-2 OLE study continues to show a favorable safety profile for long-term treatment of deramiocel. These data will be shared at this year’s Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference being held in
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
*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 >45% LVEF at end of HOPE-2 | |||
Left ventricular ejection fraction (LVEF %) Positive value indicates improvement | +1.2% | +3.0% | ||
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
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.
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
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
About
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
For more information, please contact:
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
Source:
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