Full Press Release Details
Capricor Therapeutics
Announces Positive Results from its Interim Analysis in the HOPE-2 Trial to Treat Patients with Duchenne Muscular Dystrophy
--Interim Analysis Showed Statistically
Significant Improvements in the Performance of the Upper Limb, Grip Strength and Inspiratory Flow Reserve in the Randomized Double-Blind
Placebo Controlled Study --
--Company to Hold Conference Call Today
at 5:00 AM PT / 8:00 AM ET--
LOS ANGELES, July 15, 2019 - Capricor
Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company, today announced that a pre-specified interim analysis
performed on 6-month data from the HOPE-2 trial showed statistically significant results across several independent clinical measures.
"I am incredibly pleased with the
outcome of the interim analysis as it has demonstrated the biologic activity of CAP-1002 that has resulted in changes of clinically
relevant outcomes including the upper limb, the hand and diaphragmatic function" said Craig McDonald, M.D., the national
principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of its Department of Physical Medicine and
Rehabilitation. "For these older boys who have no further therapeutic options, these data support the hope that CAP-1002
may one day become an important therapeutic option and possibly slow the advancement of the disease.
HOPE-2 is a randomized, double-blind, placebo-controlled,
Phase II clinical trial of the company's lead investigational therapy, CAP-1002 in steroid-treated boys and young men who
are in advanced stages of Duchenne muscular dystrophy (DMD), a debilitating genetic disorder. DMD is characterized by progressive
weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Study patients were treated via intravenous
delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months.
In the interim analysis, top-line data
from a total of 17 patients was analyzed in the per protocol population (10 placebo and 7 treated) at the 3 month time-point and
12 patients (6 placebo and 6 treated) were analyzed at the 6 month time-point. Approximately 80% of the patients were non-ambulant.
Demographic and baseline characteristics were similar between the two treatment groups.
Skeletal Assessments
To assess skeletal muscle function, investigators
used the mid-level dimension of the Performance of the Upper Limb (PUL) 1.2 and 2.0 tools. The PUL evaluates manual tasks that
relate to activities of daily living that are very important for quality of life. The U.S. Food and Drug Administration (FDA)
has suggested the use of the updated PUL 2.0 version as the primary efficacy endpoint in support of a Biologics License Application
(BLA). Positive results were seen in the PUL 1.2 version which is consistent with the positive results seen in Capricor's
HOPE-Duchenne Phase I/II clinical trial published in Neurology, the medical journal of the American Academy of Neurology.
Additional independent tests assessing grip strength showed statistically significant results at 6 months and tests assessing
tip to tip pinch strength showed positive results.
| Time-point | 3 months | 6 months | ||||
| Treatment | CAP-1002 | Placebo | p-value | CAP-1002 | Placebo | p-value |
| Mid-level Performance of the Upper Limb PUL 2.0 | 0.1 (1.07) | -0.4 (0.52) | 0.0591 | -0.2 (1.17) | -0.8 (0.75) | 0.0389 |
| Tip to Tip Pinch Strength | 0.9 (3.44) | 1.9 (4.12) | 0.9057 | 3.3 (2.88) | -0.3 (1.51) | 0.0674 |
| Grip Strength | -0.6 (3.15) | -0.8 (2.30) | 0.5897 | 0.8 (4.54) | -2.2 (1.83) | 0.0389 |
Table: Skeletal Assessments at 3- and
6-month time-points.
Mean Change from baseline (standard
Pulmonary Assessments
To assess pulmonary function, investigators
measured several clinically relevant parameters. At 3 months, inspiratory flow reserve (absolute), a reflection of diaphragmatic
strength, showed a statistically significant improvement (p=0.0473). Additionally, positive trends were seen at 3 months in peak
expiratory flow (% predicted), another measure of diaphragmatic strength.
Magnetic resonance imaging (MRI) was used
to assess cardiac structure and function at 6 months. Positive trends were found in cardiac muscle function including systolic
wall thickening and cardiac mass among those treated with CAP-1002 compared to placebo. Duchenne hearts atrophy progressively
and have impaired systolic function. Improved mass and wall thickening suggest possible cardiac regeneration and functional improvement.
Although these trends did not reach statistical significance, they were consistent with the cardiac findings seen in the previously
published HOPE-Duchenne study.
In late December 2018, Capricor put a voluntary
hold on dosing after two patients in the HOPE trials had a serious adverse event in the form of an immediate immune reaction.
The investigation suggested the patient may have developed hypersensitivity to something contained in the investigational product,
including an excipient or inactive ingredient in the formulation. To reduce the risk of future adverse events, Capricor initiated
a commonly used pre-medication strategy including intravenous steroids and antihistamines to prevent or mitigate potential immune
reactions during the administration. Since the initiation of the pre-treatment regimen, 30 infusions of investigational drug (CAP-1002
or placebo) have been administered to HOPE-2 patients with only one serious adverse event reported that required an overnight observation
In summary, a statistically significant
outcome relative to placebo controls was shown in PUL 2.0 at 6 months, with supportive, positive treatment effects also seen in
some independent skeletal and pulmonary assessments. Positive trends, although not statistically significant, were observed in
other skeletal, pulmonary and cardiac measures.
Although we have collected data from 2
treated patients at the 9-month timepoint, 1 of which at the 12-month time-point, Capricor is not able to draw any conclusions
at this time with respect to this data.
"We are extremely pleased and it
is truly extraordinary that even in such as small sample size, we achieved statistically significant improvements in several clinically
relevant parameters. In these older patients, functional improvement in the upper limb is highly meaningful for their quality of
life. To our knowledge, this is the first randomized double-blind, placebo-controlled study in DMD that has shown statistically
significant functional improvement in steroid treated boys." said Linda Marb n, president and CEO of Capricor.
Pat Furlong, Founding President and CEO
of Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne said, "I am encouraged by
the prospects that this data has for people with Duchenne, especially our non-ambulatory community who have limited therapeutic
options. Additionally, CAP-1002 potentially provides cardiac benefits in this patient population where heart failure continues
to be the leading cause of mortality."
The FDA has granted Capricor RMAT
and Orphan Drug Designation. Capricor met with the FDA in December 2018 as part of the expedited review afforded under
the RMAT designation which the FDA granted to CAP-1002 in February 2018. Additionally, the FDA has granted a Rare Pediatric Disease
Designation to CAP-1002. The Rare Pediatric Disease Designation, as well as the Orphan Drug Designation previously granted, covers
the broad treatment of DMD. Upon receiving market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority
Capricor will continue its ongoing discussions
with the FDA about the DMD program and future plans.
Conference Call and Webcast
Capricor will host a conference call and
webcast with slides today, July 15, 2019, at 8:00 a.m. ET to discuss the top-line interim results of the HOPE-2 clinical trial.
To participate in the conference call,
please dial 866-717-4562 (domestic) or 210-874-7812 (international) and reference the access code: 1081257
To participate via a webcast, please visit:
https://edge.media-server.com/mmc/p/y9iygowj to view the slides. The webcast will be archived for approximately 30
days and will be available at http://capricor.com/news/events/.
About Duchenne Muscular
Duchenne muscular dystrophy
is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly
from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular
dystrophy afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.
CAP-1002 consists of allogeneic
cardiosphere-derived cells, or CDCs, a type of progenitor cell that has been shown in pre-clinical and clinical studies to exert
potent immuno-modulatory activity, and is being investigated for its potential to modify 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 150 human subjects across several clinical trials.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR)