Full Press Release Details
Therapeutics Presents Positive Six-Month Results in
Muscular Dystrophy at World Muscle Society
Data Set the Stage for the Upcoming HOPE-2
Clinical Trial of CAP-1002
SAINT MALO, FRANCE - (Oct. 4, 2017)
- Capricor Therapeutics, Inc. (NASDAQ: CAPR), in its presentation today at the 22nd
Annual International Congress of the World Muscle Society, reported that teens and young men in the advanced stages of Duchenne
muscular dystrophy (DMD) experienced meaningful improvements in cardiac and upper limb function after a single dose of Capricor's
lead investigational product, CAP-1002. DMD is a rare, life-threatening genetic disorder for which treatment options are limited.
The late breaking abstract and results
presented at the late breaking poster session describe the first six months of follow-up data from the randomized 12-month Phase
I/II HOPE clinical trial of CAP-1002. CAP-1002 is a cell-based therapeutic candidate and consists of allogeneic cardiosphere-derived
cells, whose mechanism of action is immunomodulatory and anti-fibrotic, and which have been shown to generate new muscle cells
in preclinical models.
"These findings are especially significant
because the patients in the HOPE trial were preteens or young men who were in advanced stages of Duchenne muscle disease,"
said Ron Victor, M.D., professor of medicine and Burns and Allen Chair in Cardiology Research at Cedars-Sinai Heart Institute and
a principal investigator for the HOPE Trial. "Most other studies in DMD have focused on pediatric patients in earlier stages
of the disease. To see such positive results in a clinical trial with just one dose of CAP-1002 sets the stage for the next step
of evaluating multiple doses of this innovative cellular therapy in a larger trial."
The HOPE Trial was a randomized, open label
trial of 25 males with DMD, of ages 12 to 25 years (mean 17.8). For 17 of them, the disease had progressed to the point of wheelchair
dependence for mobility. Cardiomyopathy, or heart disease, secondary to DMD was an eligibility criterion and was evidenced by scar
in four or more left ventricular segments. All participants had been receiving chronic corticosteroid therapy at entry. Thirteen
received a single dose of CAP-1002, while the others received the standard of care, and all participants were to be followed for
12 months. CAP-1002 was administered by infusion into each of the three main coronary arteries for a total dose of 75 million cells.
In the trial, cardiac muscle was assessed
by magnetic resonance imaging (MRI) studies performed at baseline, six months, and 12 months. All MRI interpretations were conducted
in a manner blinded to treatment assignment and clinical outcomes.
Regional left ventricular (LV) function
significantly differed between treatment groups following a single dose of CAP-1002, as determined by assessments of systolic thickening
of LV wall segments. At six months, a statistically-significant increase in mean (standard deviation, or SD) change from baseline
in inferior wall segments was observed with CAP-1002 (+31.2% (46.9)) compared to the usual care group (-8.8% (27.7)) (p=0.02).
Six-month mean changes in anterior (+16.3 (46.5)) and lateral (+24.5 (51.2)) wall segments numerically favored CAP-1002 as compared
to usual care ((-14.1 (24.9)) (p=0.11) and (-4.5 (35.0)) (p=0.24), respectively).
Differences observed in the change from
baseline in cardiac scar size are consistent with a treatment effect on the heart. At six months, the mean (SD) percent change
from baseline in observed scar size was -5.1 (8.5) in the CAP-1002 group (p=0.04) and -0.2 (11.5) in the usual care group (p=0.71)
(p=0.09 for treatment group difference).
Skeletal muscle was assessed by the Performance
of the Upper Limb test (PUL), a validated instrument for the assessment of upper limb motor function in individuals with DMD and
consists of manual tasks that relate to activities of daily living. Scoring on the PUL was evaluated at baseline and at six weeks,
and then at three, six, and 12 months.
Following a single dose of CAP-1002, the
mean (SD) percent changes from baseline to six weeks and three months, respectively, in combined middle-plus-distal PUL dimension
were +8.8 (15.0) and +8.9 (15.4) in the CAP-1002 group and -1.7 (3.7) and +0.8 (3.7) in the usual care group. By a post hoc responder
analysis, mid-distal PUL score increased at six weeks by 10% (or maximum possible) in 42% of CAP-1002 participants compared
to none of the usual-care participants (p=0.045). At three months, the group difference in response was 33% CAP-1002 vs. 10% usual
care (p=0.32). Given the ages of the trial participants, shoulder function (upper PUL scoring) had been essentially lost prior
Treatment with CAP-1002 was generally safe
and well-tolerated over the initial six-month follow-up period of the HOPE Trial. There was no significant difference in the incidence
of treatment-emergent adverse events of either group.
"These exciting findings propel us
into our next phase of development of CAP-1002 for the treatment of DMD," said Linda Marb n, Ph.D., Capricor's
president and chief executive officer. "Subject to regulatory approvals, we expect to initiate the randomized, double-blind,
placebo-controlled HOPE-2 clinical trial of intravenous (IV), repeat-dose CAP-1002 in the first quarter of 2018. The primary efficacy
endpoint will be based on the PUL, and the HOPE-2 Trial may potentially serve as a registration study. We also look forward to
presenting 12-month follow-up results from the HOPE Trial at a major medical conference later this quarter."
The poster is available at the Events &
Presentations section of Capricor's website.
DMD is a devastating genetic disorder that
causes muscle degeneration and leads to death generally before the age of 30, most commonly from heart failure. DMD occurs in one
in every 3,600 live male births across all races, cultures and countries. DMD afflicts approximately 15,000 to 20,000 boys and
young men in the U.S. Treatment options are limited and there is no cure.
The HOPE trial was funded in part by the
California Institute for Regenerative Medicine.
CAP-1002 consists of allogeneic cardiosphere-derived
cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory
activity and alters 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 140 human subjects across several clinical trials.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company developing biological therapies for Duchenne
muscular dystrophy (DMD) and other rare diseases. Capricor's lead candidate, CAP-1002, is a cell-based candidate currently in
clinical development for the treatment of DMD. Capricor is also exploring the potential of CAP-2003, a cell-free, extracellular
vesicle-based candidate, to treat a variety of disorders. For more information, visit www.capricor.com.
Cautionary Note Regarding Forward-Looking
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; the timing of regulatory approvals; 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, 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, 2016, as filed with the Securities and Exchange Commission on March 16, 2017,
in its Registration Statement on Form S-3, as filed with the Securities and Exchange Commission on September 28, 2015, together
with prospectus supplements thereto, and in its Quarterly Report on Form 10-Q for the quarter ended June 30, 2017, as filed with
the Securities and Exchange Commission on August 14, 2017. 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. Capricor's exosomes technology, including CAP-2003, has not yet been approved for clinical
For more information, please contact:
AJ Bergmann, Vice President of Finance