LOS ANGELES, Nov. 10, 2016 /PRNewswire/ -- Capricor
Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology
company developing first-in-class biological therapies for cardiac
and other serious medical conditions, today provided a corporate
update and announced financial results for the third quarter ended
September 30, 2016.
Linda Marbán, Ph.D., president and chief executive officer,
said, "The third quarter was marked by meaningful progress in the
development of our lead candidate, CAP-1002 (allogeneic
cardiosphere-derived cells), as we completed treatment in both our
randomized, controlled Phase I/II HOPE-Duchenne trial in boys and
young men with Duchenne muscular dystrophy (DMD)-associated
cardiomyopathy as well as in the randomized, double-blind,
placebo-controlled Phase II ALLSTAR trial in patients with large
myocardial scar following a heart attack. We believe the rapid
enrollment in HOPE reflects the need to address what is currently
recognized as the number one cause of death in the DMD
population—the progressive deterioration of the heart muscle due to
the lack of the dystrophin protein. We look forward to reporting
top-line six-month data from HOPE early in the second quarter of
next year."
Capricor successfully completed two concurrent common stock
offerings in September, adding approximately $9.9 million in combined net proceeds to the
company's cash resources. Capricor is sufficiently capitalized to
advance CAP-1002 through several important clinical milestones and
activities, including the report of top-line six-month results from
the HOPE trial as well as the initiation of a second clinical trial
in DMD to evaluate CAP-1002's potential to improve peripheral and
respiratory muscle. The company remains on track to submit an
Investigational New Drug application (IND) to develop CAP-2003
(cardiosphere-derived cell exosomes) for the treatment of ocular
graft-versus-host disease, a debilitating eye condition, in the
first half of 2017. Capricor is also exploring its exosome
technology in other therapeutic areas.
"Today we also announced that we have expanded our clinical
development program in DMD to evaluate CAP-1002's ability to
improve skeletal muscle through systemic intra-vascular delivery.
We are committed to developing products that can offer the broadest
therapeutic potential to boys and young men with this progressively
debilitating genetic disorder," added Dr. Marbán.
Third Quarter and Recent Operational Highlights
- Completed treatment in the randomized Phase I/II HOPE-Duchenne
clinical trial (N=25, of which 13 received CAP-1002). The primary
outcome measures of the trial will consist of a broad assessment of
safety and tolerability of CAP-1002. Efficacy will be evaluated
according to pre-specified secondary outcome measures, including
absolute and relative changes in cardiac scar tissue and cardiac
function as measured by magnetic resonance imaging (MRI), as well
as performance on the Six-Minute Walk Test (6MWT), scoring on the
Performance of the Upper Limb (PUL) test, and scoring on the
Pediatric Quality of Life Inventory (PedsQL).
- Completed treatment in the randomized, double-blind,
placebo-controlled Phase II ALLSTAR clinical trial (N=134). For the
pre-specified primary efficacy analysis, ALLSTAR is powered to
detect a reduction in scar size in the CAP-1002 group, relative to
the placebo group, at 12 months post-infusion. Scar size will be
assessed by magnetic resonance imaging (MRI).
- Presented positive 12-month results from the open-label DYNAMIC
clinical trial of CAP-1002 in 14 patients with advanced heart
failure at the 28th Transcatheter Cardiovascular
Therapeutics (TCT), the annual scientific symposium of the
Cardiovascular Research Foundation. The treated patients
demonstrated concordant and durable trends of improvement in
functional status and capacity, cardiac function and dimension, and
quality-of-life measures out to one-year post-treatment. As had
been shown at six months, left ventricular ejection fraction had
significantly improved as compared to baseline (p=0.02).
- Announced ocular graft-versus-host disease (oGVHD) as the first
clinical development opportunity for CAP-2003. This selection was
supported by pre-clinical data which demonstrate the ability of
CAP-2003 to significantly improve clinically-meaningful measures of
ocular injury and inflammation in a relevant model.
- Completed an underwritten registered public offering and
concurrent registered direct offering of common stock, bringing
combined net proceeds of approximately $9.9
million.
- Announced a grant award of approximately $2.4 million from the Department of Defense (DoD)
to be used toward establishing a scalable, commercially-ready
manufacturing process for CAP-2003.
- Announced a grant award of up to approximately $4.2 million from the National Institutes of
Health (NIH) to support the investigation of CAP-2003 in
hypoplastic left heart syndrome (HLHS).
Anticipated Events and Milestones
- Report top-line six-month data from the HOPE-Duchenne clinical
trial of CAP-1002 early in the second quarter of 2017.
- Plan to evaluate re-dosing of CAP-1002 in patients who complete
12-month follow-up in the HOPE-Duchenne trial.
- Initiate a second clinical trial of CAP-1002 in DMD,
administered by systemic intra-vascular delivery, in 2017.
- Janssen decision on its license option for CAP-1002 by
mid-2017.
- Submit an IND for CAP-2003 for the treatment of ocular
graft-versus-host disease in the first half of 2017.
Third Quarter Results
The Company reported a net loss of approximately $5.3 million, or $0.29 per share, for the third quarter of 2016,
compared to a net loss of approximately $2.9
million, or $0.18 per share,
for the third quarter of 2015. At September
30, 2016, the Company's cash, cash equivalents and
marketable securities totaled approximately $20.5 million compared to approximately
$13.6 million at December 31, 2015.
Financial Outlook
During the third quarter, Capricor sold approximately 3.4
million shares of common stock at a price of $3.20 per share in an underwritten registered
public offering and concurrent registered direct offering that
resulted in combined net proceeds of approximately $9.9 million. Capricor currently expects that its
cash, cash equivalents and marketable securities will fund its
research and development programs and other operations into the
fourth quarter of 2017.
Conference Call and Webcast
Capricor management will hold a conference call at 5:00 p.m. ET today. The live call may be accessed
by dialing (866)-320-0174 for domestic callers and (785) 424-1631
for international callers, and by using "CAPRICOR" as the
conference ID. Access to the live webcast as well as the link to
the replay of the call can be found at
http://capricor.com/news/events/. The webcast will be archived for
approximately 30 days.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage
biotechnology company focused on the discovery, development and
commercialization of first-in-class biological therapies for the
treatment of cardiac and other serious medical conditions.
Capricor's lead candidate, CAP-1002, is a cardiac cell therapy that
is currently being evaluated for the treatment of heart disease
associated with Duchenne muscular dystrophy and myocardial
infarction (heart attack). Capricor is advancing its proprietary
exosome product candidate, CAP-2003, for the treatment of
ophthalmic disorders and is exploring other therapeutic areas.
Capricor's portfolio also features Cenderitide, a dual natriuretic
peptide receptor agonist, which may have application for the
outpatient treatment of advanced heart failure and other potential
indications. For additional information, visit
www.capricor.com.
ALLSTAR and HOPE-Duchenne are funded in part by the California
Institute for Regenerative Medicine. DYNAMIC was funded in part by
the National Institutes of Health.
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; 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 offering 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 are set forth in Capricor's Annual Report on Form 10-K for
the year ended December 31, 2015, as
filed with the Securities and Exchange Commission on March 30, 2016, in its Registration Statement on
Form S-3, as filed with the Securities and Exchange Commission on
September 28, 2015, and in its
Quarterly Report on Form 10-Q for the quarter ended June 30, 2016, as filed with the Securities and
Exchange Commission on August 15,
2016. 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 and Cenderitide are Investigational New Drugs and
are not approved for any indications. Capricor's exosomes
technology, including CAP-2003, has not yet been approved for
clinical investigation.
CAPRICOR
THERAPEUTICS, INC.
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE
LOSS
|
(unaudited)
|
|
|
Three months ended
September 30,
|
|
Nine months ended
September 30,
|
|
2016
|
|
2015
|
|
2016
|
|
2015
|
|
|
|
|
|
|
|
|
INCOME
|
|
|
|
|
|
|
|
Collaboration
income
|
$
683,595
|
|
$
911,458
|
|
$
2,506,511
|
|
$
2,864,583
|
Grant
income
|
63,186
|
|
403,426
|
|
585,177
|
|
1,529,669
|
|
|
|
|
|
|
|
|
TOTAL
INCOME
|
746,781
|
|
1,314,884
|
|
3,091,688
|
|
4,394,252
|
|
|
|
|
|
|
|
|
OPERATING
EXPENSES
|
|
|
|
|
|
|
|
Research and
development
|
4,727,111
|
|
3,192,657
|
|
13,376,178
|
|
10,426,548
|
General and
administrative
|
1,259,744
|
|
972,782
|
|
3,778,699
|
|
3,294,601
|
|
|
|
|
|
|
|
|
TOTAL OPERATING
EXPENSES
|
5,986,855
|
|
4,165,439
|
|
17,154,877
|
|
13,721,149
|
|
|
|
|
|
|
|
|
LOSS FROM
OPERATIONS
|
(5,240,074)
|
|
(2,850,555)
|
|
(14,063,189)
|
|
(9,326,897)
|
|
|
|
|
|
|
|
|
OTHER INCOME
(EXPENSE)
|
|
|
|
|
|
|
|
Investment
income
|
5,410
|
|
292
|
|
16,347
|
|
723
|
Interest
expense
|
(98,749)
|
|
(61,681)
|
|
(241,760)
|
|
(185,043)
|
|
|
|
|
|
|
|
|
TOTAL OTHER INCOME
(EXPENSE)
|
(93,339)
|
|
(61,389)
|
|
(225,413)
|
|
(184,320)
|
|
|
|
|
|
|
|
|
NET LOSS
|
(5,333,413)
|
|
(2,911,944)
|
|
(14,288,602)
|
|
(9,511,217)
|
|
|
|
|
|
|
|
|
OTHER COMPREHENSIVE
GAIN (LOSS)
|
|
|
|
|
|
|
|
Net unrealized gain
(loss) on marketable securities
|
(3,855)
|
|
5,414
|
|
(8,462)
|
|
11,949
|
|
|
|
|
|
|
|
|
COMPREHENSIVE
LOSS
|
$
(5,337,268)
|
|
$
(2,906,530)
|
|
$
(14,297,064)
|
|
$
(9,499,268)
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
(0.29)
|
|
$
(0.18)
|
|
$
(0.81)
|
|
$
(0.60)
|
|
|
|
|
|
|
|
|
Weighted average
number of shares, basic and
diluted
|
|
|
|
|
|
|
|
18,286,816
|
|
16,242,090
|
|
17,594,749
|
|
15,783,224
|
CAPRICOR
THERAPEUTICS, INC.
SUMMARY BALANCE SHEETS
|
|
|
|
September 30, 2016
(unaudited)
|
|
December 31,
2015
|
Cash, cash
equivalents and marketable securities
|
|
$
20,520,850
|
|
$
13,567,316
|
Total
assets
|
|
$
24,471,820
|
|
$
16,069,572
|
|
|
|
|
|
Total deferred
revenue
|
|
2,050,781
|
|
4,557,292
|
Total
liabilities
|
|
$
24,422,689
|
|
$
17,101,346
|
|
|
|
|
|
Total stockholders'
equity (deficit) - 21,399,019 and 16,254,985 common shares issued
and
outstanding at
September 30, 2016 and December 31, 2015, respectively
|
|
|
|
|
|
49,131
|
|
(1,031,774)
|
Total liabilities and
stockholders' equity
|
|
$
24,471,820
|
|
$
16,069,572
|
For more information, please contact:
Corporate
Capricor Therapeutics, Inc.
AJ Bergmann, Vice President of Finance
+1-310-358-3200
abergmann@capricor.com
Investor Relations
Argot Partners
Kimberly Minarovich
+1-212-600-1902
kimberly@argotpartners.com
Media
Argot Partners
Eliza Schleifstein
+1-917-763-8106
eliza@argotpartners.com
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/capricor-therapeutics-reports-third-quarter-2016-financial-results-and-provides-corporate-update-300360950.html
SOURCE Capricor Therapeutics, Inc.