Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company
developing transformative cell and exosome-based therapeutics for
the treatment and prevention of rare diseases, announced today its
financial results for the fourth quarter and full year ended
December 31, 2023 and provided a corporate update.
“2023 was a pivotal year for Capricor marked by
major advances in our CAP-1002 cell therapy program for the
treatment of Duchenne muscular dystrophy (DMD),” said Linda Marbán,
Ph.D., Capricor’s chief executive officer. “Over the course of
the year, we completed enrollment in Cohort A of our Phase 3,
HOPE-3 pivotal trial, reported the positive outcome of our interim
futility analysis, aligned with the U.S. Food and Drug
Administration (FDA) on key deliverables necessary for the filing
of a Biologics License Application (BLA), completed construction on
our new San Diego manufacturing facility and began clinical dosing
of Cohort B of the HOPE-3 trial from our San Diego facility. Each
of these achievements are major milestones on our path towards
potential approval of CAP-1002 for the treatment of DMD. While
there are limited treatment options currently available for
patients with DMD, we believe in the potential of CAP-1002’s
ability to significantly slow down disease progression.
Observed robust and consistent efficacy combined with a favorable
safety/tolerability profile, positions CAP-1002 as a potential
anchor therapy for patients. In 2024, we plan to meet with FDA to
continue to discuss options for potential expedited approval
pathways and Capricor is well-positioned to execute on several
important milestones including reporting top-line data from our
pivotal trial in the fourth quarter of 2024.”
Dr. Marbán, continued, “We continue to progress
our proprietary StealthX™ exosome platform technology as part of
our strategy to leverage exosomes for therapeutic development. We
recently announced a collaboration for our exosome-based
multivalent vaccine for the prevention of SARS-CoV-2 with the
National Institute of Allergy and Infectious Diseases (NIAID) where
they will conduct and fully fund a Phase 1 clinical trial, subject
to regulatory approval. In addition, we are engaged in several
discussions with potential partners focused on the development of
an engineered exosome therapeutic, which would support our goal of
building a pipeline of targeted exosome-based therapeutics.”
Fourth Quarter 2023 and Recent Operational
Highlights
CAP-1002 Duchenne Muscular Dystrophy
Program: CAP-1002 is an investigational cell therapy in
Phase 3 development for the treatment of DMD. CAP-1002 aims to slow
disease progression through immunomodulatory, anti-inflammatory,
and anti-fibrotic actions, with the goal of potentially improving
skeletal and cardiac muscle function in patients with DMD. HOPE-3,
our Phase 3 study, is a multi-center, randomized, double-blind,
placebo-controlled clinical trial comprised of two cohorts
evaluating the safety and efficacy of CAP-1002 in participants with
DMD and impaired skeletal muscle function. The trial is being
conducted in the United States. Approximately 102 eligible study
subjects will participate in this dual-cohort study (Cohort A and
B). CAP-1002 for the treatment of DMD has received Orphan Drug
Designation and RMAT (Regenerative Medicine Advanced Therapy
Designation). In addition, if Capricor receives FDA marketing
approval for CAP-1002 for the treatment of DMD, Capricor would be
eligible to receive a Priority Review Voucher (PRV) based on our
previous receipt of a rare pediatric disease designation.
- Enrollment has
been completed for Cohort A in our Phase 3 trial which enrolled 61
subjects randomized to either CAP-1002 or placebo in a 1:1
ratio.
- Reported a
positive outcome from the interim futility analysis for Cohort A
which triggered the first milestone payment of $10.0 million under
our U.S. Distribution and Commercialization Agreement with Nippon
Shinyaku. There is an additional $90.0 million in potential
milestone payments up to the time of approval which are triggered
upon certain regulatory-based achievements. Following potential
approval, there is an additional $605.0 million in potential
milestones payments which may be payable to Capricor based on
various sales-based targets being met. Further, Capricor will
receive a meaningful mid-range double-digit revenue share of
product revenue under the terms of this Agreement.
- Next steps for
Cohort A: plan to report top-line data in the fourth quarter of
2024.
- Enrollment is
underway for Cohort B designed to enroll approximately 44 subjects
randomized to either CAP-1002 or placebo in a 1:1 ratio.
- Next steps for
Cohort B: expect to complete enrollment in the second quarter of
2024.
- Announced the
scale-up to expand manufacturing capacity of CAP-1002 to our new
San Diego facility, intended for commercial use, subject to
regulatory approval. This facility was designed to be a versatile
and cost-effective way to bring CAP-1002 to market efficiently and
it is expected that our enhanced manufacturing capacity will
increase our supply capabilities and improve our margins on
ultimate product sales, if any. We are currently producing CAP-1002
doses at our San Diego facility for use in Cohort B.
- Announced a
positive outcome from a Type-B meeting held with FDA in the third
quarter of 2023. In the meeting, the FDA affirmed alignment on the
current HOPE-3 clinical trial design comprised of two cohorts and
our plan to submit a BLA supported by results from Cohort A which
uses product manufactured from our Los Angeles manufacturing
facility.
- We plan to meet
with FDA in the first quarter of 2024 to continue discussing our
pathway to BLA. In the upcoming Type-B meeting, we intend to
discuss our further CMC plans for commercial launch, if approved,
with an aim to expedite the approval pathway to our BLA filing. Our
ultimate goal is to transition to our San Diego manufacturing
facility for commercial manufacturing as quickly as possible.
- Hosted
a webinar in conjunction with Parent Project Muscular
Dystrophy (PPMD) where key updates on our DMD program were
outlined.
- Presented a
late-breaking poster at the 28th International Annual Congress of
the World Muscle Society (WMS). Highlights from the poster included
data from the HOPE-2 OLE trial measured by the Performance of the
Upper Limb (PUL 2.0) showing a delta change=4.9 points, p=0.021
after 24-months of treatment, compared with the placebo patient
group.
Exosome Program: Exosomes are
membrane-bound extracellular vesicles which are secreted by most
cells and contain characteristic lipids, proteins and nucleic acids
such as mRNA and microRNAs. Exosomes act as messengers to regulate
the functions of neighboring or distant cells and have been shown
to regulate functions such as cell survival, proliferation,
inflammation and tissue regeneration. We are developing our exosome
technology, using our proprietary StealthX™ platform which is
focused on the areas of vaccinology, targeted delivery of
oligonucleotides, proteins and small molecule therapeutics to
potentially treat and prevent a diverse array of diseases.
- Announced that our proprietary
StealthX™ exosome-based multivalent vaccine (StealthX™ vaccine) for
the prevention of SARS-CoV-2 was selected to be part
of Project NextGen, an initiative by the U.S. Department
of Health and Human Services to advance a pipeline of new,
innovative vaccines providing broader and more durable protection
for COVID-19. As part of Project NextGen, NIAID, part of the
National Institutes of Health, will conduct and fund a Phase 1
clinical trial with our StealthX™ vaccine, subject to regulatory
approval. Under the terms of the collaboration, Capricor will
supply the investigational product and NIAID's Division of
Microbiology and Infectious Diseases (DMID) will oversee the
trial.
- Next steps for this project: NIAID
plans to initiate the Phase 1 clinical trial in late 2024, subject
to regulatory approval. If NIAID finds that our StealthX™ vaccine
meets its criteria for safety and efficacy, they may consider our
program for a funded Phase 2.
- Currently, in collaboration with an
undisclosed pharmaceutical company, we are also investigating the
therapeutic application of our StealthX™ exosome platform.
- Presented a late-breaking poster at
the WMS on the application of our StealthX™ exosome platform for
the delivery of antisense oligonucleotides (ASO). Highlights from
the poster included data showing the presence of exosomes loaded
with a labeled ASO in the lower limbs of mice 24 hours
post-intravenous (IV) injection. Notably, the exosomes carrying the
muscle-targeting moiety were not detected in any other tissues
except for the expected clearance pathways (kidney and liver) with
a single dose.
Corporate Updates
- Announced receipt of our first
milestone payment of $10.0 million under our U.S. Distribution and
Commercialization Agreement with Nippon Shinyaku.
- Announced completion of a
registered direct offering with participation from Nippon Shinyaku
for gross proceeds of approximately $23.0 million.
Anticipated Upcoming Milestones
CAP-1002 Duchenne Muscular Dystrophy
Program
- Plan to have a Type-B meeting with FDA in the first quarter of
2024 to discuss commercial manufacturing planning with an aim to
expedite the BLA pathway.
- Plan to report 3-year HOPE-2 OLE data in the second quarter of
2024.
- Plan to complete HOPE-3 (Cohort B) enrollment in the second
quarter of 2024.
- Plan to report topline data from HOPE-3 (Cohort A) in the
fourth quarter of 2024.
- Continue to explore opportunities for additional partnerships
outside of the U.S. and Japan to support the potential
commercialization of CAP-1002 in DMD.
Exosome Program
- Subject to regulatory approval, plan to announce IND clearance
for our StealthX™ vaccine for the prevention of SARS-CoV-2.
- Plan to provide updates on our NIAID collaboration for our
StealthX™ vaccine as they become available.
- Continue to explore opportunities for partnerships and
non-dilutive sources of funding to support advancement of our
StealthX™ exosome platform technology.
Fourth Quarter and Full Year 2023
Financial Results
Cash position: The Company’s cash, cash
equivalents and marketable securities totaled approximately $39.5
million as of December 31, 2023 compared to approximately $41.4
million on December 31, 2022. In the fourth quarter of 2023,
Capricor raised approximately $1.5 million in net proceeds through
issuances of common stock at an average price of approximately
$4.47 per share under its at-the-market offering program.
Additionally in January 2024, the Company received $10.0 million
from the first milestone payment under our U.S. Distribution and
Commercialization Agreement with Nippon Shinyaku.
Revenues: Capricor’s primary source of
revenue was from the ratable recognition of the $40.0 million
(upfront and milestone payments) in accordance with its U.S.
Commercialization and Distribution Agreement with Nippon Shinyaku.
Revenues for the fourth quarter of 2023 were approximately $12.1
million compared with approximately $1.0 million for the fourth
quarter of 2022.
Operating expenses: Total operating
expenses for the fourth quarter of 2023 were approximately $13.2
million compared with approximately $9.0 million for the fourth
quarter of 2022.
Net loss: The Company reported a net loss
of approximately $0.8 million, or $0.02 per share, for the fourth
quarter of 2023, compared to a net loss of approximately $7.7
million, or $0.31 per share, for the fourth quarter of 2022. For
the year ended December 31, 2023, the Company reported a net loss
of approximately $22.3 million, or $0.83 per share, compared to a
net loss of approximately $29.0 million, or $1.18 per share, for
the year ended December 31, 2022.
Financial Outlook
Capricor believes that based on the current
operating plan and financial resources, its available cash, cash
equivalents and marketable securities will be sufficient to cover
anticipated expenses and capital requirements into the first
quarter of 2025. This expectation excludes any additional potential
milestone payments under its exclusive commercialization and
distribution agreements with Nippon Shinyaku, as well as any
strategic use of capital not currently in the Company’s base-case
planning assumptions.
Conference Call and Webcast
To participate in the conference call, please
dial 1-888-886-7786 (Domestic/Toll-Free) or 1-416-764-8658
(International) and reference the conference ID: 83986877.
Participants can use guest dial-in numbers above and be answered by
an operator or click the Call me™ link for instant telephone
access. To participate via a webcast, please click here. A replay
of the webcast will be available following the conclusion of the
live broadcast and will be accessible on the Company’s website.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a
biotechnology company dedicated to advancing transformative cell
and exosome-based therapeutics to redefine the treatment landscape
for rare diseases. At the forefront of our innovation is our lead
product candidate, CAP-1002 — an allogeneic cardiac-derived cell
therapy. Extensive preclinical and clinical studies have shown
CAP-1002 to demonstrate immunomodulatory, antifibrotic, and
regenerative actions specifically tailored for dystrophinopathies
and heart disease. CAP-1002 is currently advancing through Phase 3
clinical development for the treatment of Duchenne muscular
dystrophy (DMD). Capricor is also harnessing the power of our
exosome technology, using our proprietary StealthX™ platform which
is focused on the areas of vaccinology, targeted delivery of
oligonucleotides, proteins and small molecule therapeutics to
potentially treat and prevent a diverse array of diseases. At
Capricor, we stand committed to pushing the boundaries of
possibility and forging a path toward transformative treatments for
those in need. For more information, visit capricor.com, and
follow Capricor
on Facebook, Instagram and Twitter.
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; manufacturing capabilities;
dates for regulatory meetings; statements about our financial
outlook; the ability to achieve product milestones and to receive
milestone payments from commercial partners; 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 and revenue
projections; 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, 2022, as filed with the Securities and
Exchange Commission on March 17, 2023 and in our Quarterly Report
on Form 10-Q for the quarter ended September 30, 2023, as filed
with the Securities and Exchange Commission on November 14, 2023.
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.
Capricor has entered into
a partnership for the exclusive commercialization and
distribution of CAP-1002 for DMD in the United States and Japan
with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma,
Inc.), subject to regulatory approval. CAP-1002 is an
Investigational New Drug and is not approved for any indications.
None of Capricor’s exosome-based candidates have been approved for
clinical investigation.
For more information, please
contact:
Capricor Company Contact:AJ
Bergmann, Chief Financial Officerabergmann@capricor.com
858.727.1755
CAPRICOR
THERAPEUTICS, INC. |
|
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS |
|
(UNAUDITED) |
|
|
|
|
|
|
|
|
|
|
|
Three months ended December 31, |
|
Year ended December 31, |
|
|
2023 |
|
2022 |
|
2023 |
|
2022 |
|
|
|
|
|
|
|
|
|
|
REVENUE |
|
|
|
|
|
|
|
|
Revenue |
$ |
12,088,089 |
|
|
$ |
959,903 |
|
|
$ |
25,178,066 |
|
|
$ |
2,551,469 |
|
|
|
|
|
|
|
|
|
|
|
TOTAL REVENUE |
|
12,088,089 |
|
|
|
959,903 |
|
|
|
25,178,066 |
|
|
|
2,551,469 |
|
|
|
|
|
|
|
|
|
|
|
OPERATING
EXPENSES |
|
|
|
|
|
|
|
|
Research and development |
|
9,940,167 |
|
|
|
6,231,806 |
|
|
|
36,448,039 |
|
|
|
21,816,949 |
|
|
General and administrative |
|
3,210,947 |
|
|
|
2,794,442 |
|
|
|
12,589,619 |
|
|
|
10,431,903 |
|
|
|
|
|
|
|
|
|
|
|
TOTAL OPERATING EXPENSES |
|
13,151,114 |
|
|
|
9,026,248 |
|
|
|
49,037,658 |
|
|
|
32,248,852 |
|
|
|
|
|
|
|
|
|
|
|
LOSS FROM OPERATIONS |
|
(1,063,025 |
) |
|
|
(8,066,345 |
) |
|
|
(23,859,592 |
) |
|
|
(29,697,383 |
) |
|
|
|
|
|
|
|
|
|
|
OTHER INCOME
(EXPENSE) |
|
|
|
|
|
|
|
|
Other income |
|
67,657 |
|
|
|
- |
|
|
|
67,657 |
|
|
|
190,582 |
|
|
Investment income |
|
233,932 |
|
|
|
379,699 |
|
|
|
1,510,434 |
|
|
|
521,535 |
|
|
Loss on disposal of fixed assets |
|
(653 |
) |
|
|
(34,266 |
) |
|
|
(6,041 |
) |
|
|
(34,266 |
) |
|
|
|
|
|
|
|
|
|
|
TOTAL OTHER INCOME (EXPENSE) |
|
300,936 |
|
|
|
345,433 |
|
|
|
1,572,050 |
|
|
|
677,851 |
|
|
|
|
|
|
|
|
|
|
|
NET LOSS |
|
(762,089 |
) |
|
|
(7,720,912 |
) |
|
|
(22,287,542 |
) |
|
|
(29,019,532 |
) |
|
|
|
|
|
|
|
|
|
|
OTHER
COMPREHENSIVE INCOME (LOSS) |
|
|
|
|
|
|
|
|
Net unrealized gain (loss) on marketable securities |
|
122,605 |
|
|
|
(17,223 |
) |
|
|
130,569 |
|
|
|
105,244 |
|
|
|
|
|
|
|
|
|
|
|
COMPREHENSIVE LOSS |
$ |
(639,484 |
) |
|
$ |
(7,738,135 |
) |
|
$ |
(22,156,973 |
) |
|
$ |
(28,914,288 |
) |
|
|
|
|
|
|
|
|
|
|
Net loss per
share, basic and diluted |
$ |
(0.02 |
) |
|
$ |
(0.31 |
) |
|
$ |
(0.83 |
) |
|
$ |
(1.18 |
) |
|
Weighted
average number of shares, basic and diluted |
|
30,664,100 |
|
|
|
25,163,711 |
|
|
|
26,778,360 |
|
|
|
24,552,688 |
|
|
|
|
|
|
|
|
|
|
|
CAPRICOR
THERAPEUTICS, INC. |
|
|
SUMMARY
BALANCE SHEETS |
|
|
|
|
|
|
|
December 31, 2023 |
|
December 31, 2022 |
|
|
Cash, cash equivalents and marketable securities |
|
$ |
39,487,703 |
|
$ |
41,421,262 |
|
|
Total assets |
|
$ |
58,734,327 |
|
$ |
50,094,910 |
|
|
|
|
|
|
|
|
|
Total liabilities |
|
$ |
36,132,860 |
|
$ |
38,308,816 |
|
|
|
|
|
|
|
|
|
Total stockholders' equity - 31,148,320 and 25,241,402 common
shares issued and |
|
|
|
|
|
|
outstanding at December 31, 2023 and December 31, 2022,
respectively |
|
|
22,601,467 |
|
|
11,786,094 |
|
|
Total liabilities and stockholders' equity |
|
$ |
58,734,327 |
|
$ |
50,094,910 |
|
|
|
|
|
|
|
|
|
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