HOPE-2 Clinical Trial is Currently
Enrolling Patients
Capricor Therapeutics (NASDAQ:CAPR), a clinical-stage biotechnology
company focused on the development of first-in-class biological
therapeutics for the treatment of Duchenne muscular dystrophy and
other rare disorders, today announced its financial results for the
first quarter, which ended March 31, 2018, and provided a corporate
update.
“We are excited by the recent initiation of the
HOPE-2 clinical trial of CAP-1002, our lead cell therapy product,
to treat Duchenne muscular dystrophy,” said Linda Marbán, Ph.D.,
Capricor president and chief executive officer. “CAP-1002 is one of
the very few clinical initiatives to focus on helping boys and
young men who are in later stages of the disease process and whose
ability to walk has been seriously impaired by the loss of muscle
function as a result of the disease. We have seen the potential for
improvements in muscle function in both pre-clinical studies and in
our earlier HOPE-Duchenne trial, and we have been granted the RMAT
and orphan disease designations by the U.S. Food and Drug
Administration (FDA) for CAP-1002 for Duchenne muscular
dystrophy.”
These designations enable Capricor to work
closely with the FDA in finalizing the regulatory approval pathway
for CAP-1002 and to receive expedited FDA reviews.
“We are hopeful that HOPE-2 may potentially be a
registration trial,” said Dr. Marbán. “We see CAP-1002 as an
important tool in the toolbox to treat Duchenne muscular dystrophy
because it may work synergistically with the emerging
disease-modifying therapies. While gene and other therapies have
the potential to restore dystrophin expression and sustain muscle
function, there will still be significant inflammation and
fibrosis, which can offset the restorative effects. CAP-1002’s
primary mechanism of action is immunomodulatory, meaning it can
help balance inflammation in this chronic inflammatory
disease.”
First Quarter 2018 Highlights and Recent
Clinical and Operational Developments
- In April, Capricor initiated the HOPE-2 clinical trial at UC
Davis Medical Center. Up to 84 boys and young men with Duchenne
muscular dystrophy will be enrolled in HOPE-2, a Phase II,
randomized, double-blind, placebo-controlled trial that will test
CAP-1002 in participants with advanced stages of Duchenne muscular
dystrophy. Approximately 12-15 investigative sites are expected to
participate in the trial. Craig McDonald, M.D., UC Davis professor
and chair of its Department of Physical Medicine and
Rehabilitation, is the national principal investigator for HOPE-2.
More information is available at www.HOPE2trial.com
- In an abstract presentation at the 11th Annual Neuromuscular
Translational Research Conference in Cambridge, England in April,
Capricor announced results of a new study that found that repeat
dosing of CAP-1002 yields an increase in exercise performance in a
mouse model of Duchenne muscular dystrophy.
- A newly published study in “Stem Cell Reports” from
Cedars-Sinai Medical Center reported that cardiosphere-derived
cells (CDCs) improved skeletal, diaphragm and cardiac muscle
function in a mouse model of Duchenne muscular dystrophy.
- Capricor secured the Regenerative Medicine Advanced Therapy
(RMAT) designation for CAP-1002 for Duchenne muscular dystrophy,
which makes therapies eligible for expedited review.
- Capricor added seven new patent applications to its existing
Exclusive License Agreements with Cedars-Sinai Medical Center,
giving Capricor worldwide, exclusive rights to inventions related
to cardiosphere-derived cells (CDCs and CAP-1002) and CDC-derived
extracellular vesicles, including exosomes.
- Capricor hosted a Key Opinion Leaders Lunch in New York City on
March 9, which included four distinguished speakers discussing the
emerging paradigms in gene and cellular therapies to treat Duchenne
muscular dystrophy. The speakers included Craig McDonald, M.D.,
professor and chair of the Department of Physical Medicine and
Rehabilitation and director of the Neuromuscular Disease Clinics at
the University of California, Davis. Other speakers were Jeffrey
Chamberlain, Ph.D., professor in the departments of Neurology,
Medicine and Biochemistry and director of the Seattle Wellstone
Muscular Dystrophy Center, and Michelle Eagle, Ph.D., the managing
director of ATOM International LTD and one of the creators of, and
who has published extensively on, the Performance of the Upper Limb
(PUL) test, a validated test for skeletal muscle function in
Duchenne muscular dystrophy. The fourth speaker was Pat Furlong,
the founding president and CEO of the Parent Project Muscular
Dystrophy (PPMD), the largest non-profit organization in the U.S.
focused solely on Duchenne.
Anticipated Milestones in
2018
- Continue to add additional sites and enroll patients in the
HOPE-2 clinical trial.
- Continue to conduct pre-clinical research for Capricor’s
investigational exosome-based therapy, CAP-2003, to treat various
diseases of inflammation and fibrosis.
- Continue preparations for manufacturing scale-up and technology
transfer of CAP-1002.
- Plan to meet with the FDA to discuss Capricor’s Duchenne
program through the RMAT process.
First Quarter 2018 Financial
Results
The Company reported a net loss of approximately
$3.7 million, or $0.14 per share, for the first quarter of 2018,
compared to a net loss of approximately $3.7 million, or $0.17 per
share, for the first quarter of 2017.
As of March 31, 2018, the Company's cash, cash
equivalents and marketable securities totaled approximately $13.2
million, compared to approximately $14.1 million on December 31,
2017. Additionally, in the first quarter of 2018, Capricor raised
approximately $2.4 million in net proceeds at an average price of
approximately $1.88 per share under its at-the-market offering
program. Capricor believes that its current financial resources
should be sufficient to fund its operations and meet its financial
obligations into the first quarter of 2019 based on the Company's
current projections.
Conference Call and Webcast
To participate in the conference call, please
dial 866-717-4562 (domestic) or 210-874-7812 (international) and
reference the access code 5795736.
To participate via a webcast, please visit
https://edge.media-server.com/m6/p/qz4u7wox. The webcast will be
archived for approximately 30 days and will be available at
http://capricor.com/news/events/.
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
therapeutics for the treatment of rare disorders. Capricor’s lead
candidate, CAP-1002, is an allogeneic cell therapy that is
currently in clinical development for the treatment of Duchenne
muscular dystrophy. Capricor has also established itself as one of
the leading companies investigating the field of extracellular
vesicles and 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.
Keep up with Capricor on social media:
www.facebook.com/capricortherapeutics,
www.instagram.com/capricortherapeutics/ and
https://twitter.com/capricor
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; 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, 2017 as filed with the Securities and Exchange
Commission on March 22, 2018, and in its Registration Statement on
Form S-3, as filed with the Securities and Exchange Commission on
September 28, 2015, together with the prospectus included therein
and prospectus supplements thereto. 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. 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 March 31, |
|
2018 |
|
2017 |
|
|
|
|
INCOME |
|
|
|
Collaboration income |
$ |
- |
|
|
$ |
683,594 |
|
Grant
income |
|
306,750 |
|
|
|
197,214 |
|
Other
income |
|
93,316 |
|
|
|
- |
|
|
|
|
|
TOTAL
INCOME |
|
400,066 |
|
|
|
880,808 |
|
|
|
|
|
OPERATING EXPENSES |
|
|
|
Research
and development |
|
2,696,517 |
|
|
|
3,257,149 |
|
General
and administrative |
|
1,389,732 |
|
|
|
1,189,238 |
|
|
|
|
|
TOTAL
OPERATING EXPENSES |
|
4,086,249 |
|
|
|
4,446,387 |
|
|
|
|
|
LOSS FROM
OPERATIONS |
|
(3,686,183 |
) |
|
|
(3,565,579 |
) |
|
|
|
|
OTHER
INCOME (EXPENSE) |
|
|
|
Investment income |
|
14,653 |
|
|
|
4,282 |
|
Interest
expense |
|
- |
|
|
|
(105,320 |
) |
|
|
|
|
TOTAL
OTHER INCOME (EXPENSE) |
|
14,653 |
|
|
|
(101,038 |
) |
|
|
|
|
NET
LOSS |
|
(3,671,530 |
) |
|
|
(3,666,617 |
) |
|
|
|
|
OTHER
COMPREHENSIVE INCOME (LOSS) |
|
|
|
Net
unrealized gain on marketable securities |
|
8,709 |
|
|
|
6,187 |
|
|
|
|
|
COMPREHENSIVE LOSS |
$ |
(3,662,821 |
) |
|
$ |
(3,660,430 |
) |
|
|
|
|
Net loss
per share, basic and diluted |
$ |
(0.14 |
) |
|
$ |
(0.17 |
) |
Weighted
average number of shares, basic and diluted |
|
26,905,331 |
|
|
|
21,399,019 |
|
|
|
|
|
|
CAPRICOR THEAPEUTICS, INC. |
SUMMARY BALANCE SHEETS |
|
|
|
March 31, 2018(unaudited) |
|
December 31, 2017 |
Cash, cash equivalents
and marketable securities |
|
$ |
13,164,642 |
|
$ |
14,124,935 |
Total assets |
|
$ |
15,388,422 |
|
$ |
16,273,789 |
|
|
|
|
|
Total liabilities |
|
$ |
4,854,158 |
|
$ |
5,046,934 |
|
|
|
|
|
Total stockholders'
equity - 27,970,879 and 26,270,491 common shares issued and |
|
|
|
|
outstanding at March 31, 2018 and December 31, 2017,
respectively |
|
|
10,534,264 |
|
|
11,226,855 |
Total liabilities and
stockholders' equity |
|
$ |
15,388,422 |
|
$ |
16,273,789 |
|
|
|
|
|
For more information, please contact:
AJ Bergmann, Chief Financial Officer
+1-310-358-3200
abergmann@capricor.com
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