Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy
04 Juin 2024 - 3:00PM
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company
developing transformative cell and exosome-based therapeutics for
the treatment of rare diseases, today announced positive 3-year
results from the ongoing HOPE-2 open-label extension (OLE) study
with CAP-1002 for the treatment of Duchenne muscular dystrophy
(DMD). Patients treated with CAP-1002 continue to show positive
benefits after 3 years of treatment in both the PUL 2.0 and LVEF
measures when compared to an external comparator dataset of similar
DMD patients (dataset provided by Cincinnati Children’s Hospital
Medical Center (CCHMC)). Further, CAP-1002 treatment during the
HOPE-2-OLE study continues to have a consistently well-tolerated
safety profile throughout the study. The Company plans to present
these results in more detail at the upcoming Parent Project
Muscular Dystrophy (PPMD) 30th Annual Conference being held June
27-29, 2024.
3-Year HOPE-2 OLE Topline Study
Results
Primary Endpoint |
3-Year Timepoint Change from
Baseline* |
Delta Changep-value |
Skeletal-Muscle (Upper Limb Function)Full
Performance of Upper Limb (PUL 2.0) |
CAP-1002 (n=12) |
External Comparator (n=32) |
-4.1 points |
-7.8 points |
+3.7 pointsp< 0.001 |
*Baseline is referred to as start of HOPE-2 OLE
study, changes in means are shown
“I am extremely encouraged by these positive
results from our HOPE-2 OLE study which continue to support the
long-term impact of CAP-1002 for the treatment of DMD,” said Linda
Marbán, Ph.D., Capricor’s chief executive officer. “Importantly,
the external comparator of the disease shows a steady state of
decline in both skeletal and cardiac function; however over the
course of 3-years, we observed a statistically significant
reduction in PUL decline as well as stabilization in LVEF in
CAP-1002 treated patients suggesting sustained disease attenuation.
Furthermore, at our recent Type-B meeting with the U.S. Food and
Drug Administration (FDA), we shared these results with the FDA to
support what we expect to see in the HOPE-3 pivotal study as we
continue to work on strategies for the most expeditious pathway to
potential BLA approval and commercialization. The details of our
meeting will be shared later this month once the meeting minutes
are final. Looking ahead, we remain on track to announce topline
results from our Phase 3 HOPE-3 pivotal trial in the fourth quarter
of 2024. We continue to thank the patients, families and the
broader Duchenne community for continuing to work with us on this
promising therapy.”
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, CAP-1002, in boys and young men who have
DMD. Study patients were treated via intravenous delivery with
either CAP-1002 (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 CAP-1002 (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 2.0
(p=0.02). The HOPE-2-OLE study remains ongoing and into its fourth
year and participants continue to be monitored for safety 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, CAP-1002, an allogeneic cardiac-derived cell
therapy. Extensive preclinical and clinical studies have shown
CAP-1002 to demonstrate immunomodulatory, antifibrotic, and
regenerative actions specifically tailored for dystrophinopathies
and heart disease. CAP-1002 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
a partnership for the exclusive commercialization and
distribution of 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. CAP-1002 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 Officerabergmann@capricor.com
858.727.1755
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