Full Press Release Details
Capricor Therapeutics
Announces Positive Six-Month Results from the Randomized Phase I/II HOPE Clinical Trial in Duchenne Muscular Dystrophy
Statistically-Significant Improvements
in Measures of Cardiac and Upper Limb Function in Patients Treated with CAP-1002
to Seek Breakthrough Therapy or Regenerative Medicine Advanced Therapy Designation
Conference Call and Webcast to be Held
Today at 8:00 a.m. Eastern Time
LOS ANGELES, April 25, 2017 -
Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company
developing first-in-class biological therapies for cardiac and other medical conditions, today announced positive top-line
results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical
Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne
muscular dystrophy, or DMD. Duchenne muscular dystrophy is a rare, life-threatening genetic disorder for which treatment options
Highlights from the Phase I/II HOPE Clinical
Trial six-month results:
John L. Jefferies, M.D., Professor of Pediatric
Cardiology and Adult Cardiovascular Diseases at the University of Cincinnati and Director, Advanced Heart Failure and Cardiomyopathy,
and Principal Investigator of the HOPE Trial, said, "In HOPE, we saw potential effects in both the heart and skeletal muscle
that appear quite compelling in an exploratory trial. These results clearly support the conduct of a confirmatory clinical trial
in DMD to further evaluate the potential of CAP-1002. We look forward to an effective medication becoming available for people
with this progressive and fatal disease, one that is poorly met by current options."
Joao A. C. Lima, M.D., Professor of Medicine
and Director of the Magnetic Resonance Imaging, or MRI, Core Lab at The Johns Hopkins University School of Medicine, said, "The
observed signal in global cardiac scar reduction and the increase in the thickening of the left ventricle during contraction are
very encouraging. The population treated in HOPE had very advanced cardiac involvement, and to see such positive results following
just a single dose of CAP-1002 is remarkable. Cardiac disease is the most common cause of mortality among those with DMD."
Pat Furlong, Founding President and CEO
of Parent Project Muscular Dystrophy, the largest nonprofit organization in the U.S. solely focused on DMD, said, "I'm
excited to see these data, especially given the advanced nature of the patients in the HOPE trial. It is also gratifying to see
the field of cell therapy making progress after more than two decades in development. It is our hope that CAP-1002 will have broad
potential to improve the lives of patients with Duchenne muscular dystrophy."
The randomized HOPE (Halt cardiomyOPathy
progrEssion in Duchenne) Clinical Trial was designed to evaluate the safety and exploratory efficacy of CAP-1002 in patients 12
years and older with DMD who had cardiomyopathy, or heart disease, secondary to DMD as evidenced by scar in four or more left ventricular
segments as detected by late gadolinium-enhancement MRI. Twenty-five patients were randomized to receive either a single dose of
CAP-1002 (13 patients) or usual care (12 patients). CAP-1002 was infused into each of the three main coronary arteries at a total
dose of 75 million cells.
All cardiac assessments were performed
by MRI. A validated test of upper limb function, the Performance of the Upper Limb test, or PUL, was used to assess skeletal muscle
performance. The PUL was designed specifically for use in the DMD setting, and evaluates a variety of manual tasks, such as lifting
cans, tearing paper, and removing a container lid, which simulate activities of daily living. Boys and young men with advanced
DMD have difficulty with such common activities as feeding themselves and brushing their teeth.
CAP-1002 has been well-tolerated in the
HOPE trial. During and immediately following CAP-1002 infusion, no treated subjects experienced a pre-specified composite safety
endpoint, which included the major adverse cardiac events of death, myocardial infarction, or hospitalization for a cardiovascular
event. There were no early study discontinuations due to adverse events.
In exploratory efficacy analyses, statistically-significant
improvements in systolic thickening of the inferior wall of the heart (p=0.030), and in the function of the middle and distal upper
limb according to a PUL responder analysis (p=0.045), were observed in patients treated with CAP-1002 as compared to usual care
control patients. In addition, differences observed in several other cardiac and skeletal muscle measures, including cardiac scar
(p=0.09), are consistent with a treatment effect. At three months, a statistically-significant difference in quality-of-life (p=0.03),
according to the PODCI Adolescent Questionnaire, that favored the CAP-1002 arm was also observed. A more comprehensive summary
of the top-line data will be provided in slides to be presented during today's webcast.
"These initial positive clinical
results build upon a large body of preclinical data which illustrate CAP-1002's potential to broadly improve the condition
of those afflicted by DMD, as they show that cardiosphere-derived cells exert salutary effects on cardiac and skeletal muscle,"
said Linda Marb n, Ph.D., Capricor's president and chief executive officer.
"We have submitted an FDA meeting
request to discuss these results as well as next steps in our development of CAP-1002 for Duchenne muscular dystrophy, which includes
our plan to begin a clinical trial of intravenously-administered CAP-1002 in the latter half of this year. We believe the interim
HOPE results may enable us to pursue one of the FDA's Expedited Programs for Serious Conditions, and we will apply for either
or both of the Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations for CAP-1002," added Dr.
Capricor expects to report top-line 12-month
results from the HOPE-Duchenne Trial in the fourth quarter of 2017.
A pre-publication manuscript reporting
on academic studies of cardiosphere-derived cells in animal models of DMD may be accessed at http://biorxiv.org/content/early/2017/04/20/128900.
The HOPE-Duchenne trial is funded in part
by the California Institute for Regenerative Medicine.
Conference Call and Webcast Information
Capricor will host a conference call and
webcast with slides today, April 25, 2017, at 8:00 a.m. Eastern Time to discuss the top-line six-month HOPE-Duchenne clinical
trial results. Capricor's executive management team will be joined on the call by Dr. Lima. The conference call can be accessed
by dialing (866) 901-2585 for participants in the U.S. and Canada and (404) 835-7099 for international callers (reference passcode
44797929). The conference call will be webcast live and can be accessed at Capricor's website or by clicking this link (www.wsw.com/webcast/cc/capr).
The webcast will be archived on the Capricor website for approximately 90 days.
About Duchenne Muscular Dystrophy
DMD is a genetic disorder characterized
by progressive muscle degeneration and weakness. It is caused by an abnormality in the dystrophin complex, a structural element
that plays a critical role in muscle fiber integrity, which leads to chronic skeletal and cardiac muscle damage. Patients with
DMD typically die in their twenties, most commonly due to heart disease. The incidence of DMD is estimated to be one in every 3,600
live male births, and DMD is believed to afflict approximately 15,000 to 20,000 boys and young men in the U.S.
CAP-1002 consists of allogeneic cardiosphere-derived
cells, or CDCs, a type of cardiac progenitor cell. 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. CAP-1002 is currently being evaluated
in the randomized, double-blind, placebo-controlled Phase II ALLSTAR Clinical Trial in adults who have suffered a large heart attack
and in the Phase I/II HOPE Clinical Trial in boys and young men with DMD.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR)
is a clinical-stage biotechnology company developing first-in-class biological therapies
for cardiac and other medical conditions. Capricor's lead candidate, CAP-1002, is a cell-based candidate currently in clinical
development for the treatment of Duchenne muscular dystrophy, myocardial infarction (heart attack), and heart failure. Capricor
is exploring the potential of CAP-2003, a cell-free, exosome-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; 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