Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company
developing transformative cell and exosome-based therapeutics for
the treatment of rare diseases, today announced its financial
results for the first quarter ended March 31, 2024 and provided a
corporate update.
“Capricor continues to make tremendous progress
across our pipeline marked by significant advancements 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. “With enrollment completed in Cohort
A of our Phase 3 pivotal trial, we further aligned with the U.S.
Food and Drug Administration (FDA) on key CMC (chemistry
manufacturing and controls) deliverables necessary for the filing
of a Biologics License Application (BLA) including the
establishment of non-clinical comparability, a major milestone on
the path to clearance for a BLA submission. We are appreciative of
the FDA’s continued guidance under our RMAT designation and we will
provide further updates on our plans as they become available.
Furthermore, we continue to work diligently with our partner,
Nippon Shinyaku (U.S. subsidiary: NS Pharma, Inc.) as we prepare
for the potential launch of CAP-1002. Looking ahead, later this
quarter, we plan to meet with FDA to continue to discuss options
for potential expedited approval pathways as well as announce the
3-year HOPE-2 OLE results.”
Dr. Marbán, continued, “We continue our efforts
to advance our proprietary StealthX™ exosome platform to leverage
exosomes for our vaccine program as well as for therapeutic
development. This quarter, we 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. We continue to remain
focused on advancing this program through partnerships and other
non-dilutive sources of funding.”
First Quarter 2024 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 Regenerative Medicine Advanced Therapy
Designation (RMAT). 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
our HOPE-3 Phase 3 (Cohort A) clinical 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 received in the first
quarter of 2024 under our U.S. Distribution and Commercialization
Agreement with Nippon Shinyaku.
- Announced a positive outcome from a
Type-B CMC FDA meeting held in the first quarter of 2024. In the
meeting, the FDA affirmed alignment on the following topics:
- Comparability between drug product
manufactured at our two different facilities (Los Angeles and San
Diego) has been demonstrated using the provided analytical
comparability data.
- This will now allow for the use of
CAP-1002 drug product manufactured at our San Diego manufacturing
facility upon potential product approval (subject to approval of
the facility).
- Granted a Type-B clinical FDA
meeting scheduled to be held in May 2024 to continue discussing our
pathway to BLA.
- The FDA advised us to include
discussion and a request for a pre-BLA meeting and rolling BLA
schedule at this meeting.
- We also plan to share with FDA our
HOPE-2 OLE 3-year safety and efficacy data.
- Announced further specifics under
our U.S. Distribution and Commercialization Agreement with Nippon
Shinyaku.
- Capricor will receive a meaningful
mid-range double-digit share of product revenue. For clarity,
mid-range falls in between 30-50%, which sum will be offset by the
amounts paid to us as the transfer price for the purchase of the
product.
- As previously disclosed, 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.
- Announced we are on track to enroll
44 U.S. based patients by the end of second quarter in HOPE-3
(Cohort B) clinical trial, however at this time, we are evaluating
various options, with one of such options being the opportunity for
an expansion of Cohort B to include European patients.
- Announced the scale-up of
manufacturing capacity of CAP-1002 in 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.
- Currently, our San Diego facility
is fully operational, staffed and producing doses for clinical use.
Along with BLA readiness activities, we are also actively preparing
for commercial runs.
- Presented at H.C. Wainwright 2nd
Annual Cell Therapy Conference.
- Presented at the Cantor
Fitzgerald’s Muscular Dystrophy Symposium.
- Presented at the 2024 Muscular
Dystrophy Association (MDA) Clinical & Scientific Conference
sharing the previously presented positive 24-month results from our
HOPE-2 OLE study.
- Key results included skeletal
muscle function as measured by the Performance of the Upper Limb
(PUL v2.0) showed a mean PUL v2.0 decline after 24-months of
treatment with CAP-1002 was 2.8 points versus a 7.7 point decline
on average observed over 24-months in the placebo patient group.
Additionally, CAP-1002 revealed clinically meaningful improvements
in ameliorating cardiac function
- Presented at the Parent Project
Muscular Dystrophy (PPMD) Cardiac Workshop III. Capricor was
featured in a panel on industry perspectives on cardiac monitoring
in DMD clinical trials.
StealthX™ Exosome
Platform: 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. They 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 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.
- 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.
- Under the terms of the
collaboration, Capricor will supply the investigational product and
NIAID's Division of Microbiology and Infectious Diseases (DMID)
will conduct the trial.
- 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 data in an oral
presentation at the American Society of Gene and Cell Therapy
(ASGCT) 27th Annual Meeting. The findings highlight a potential
exosome-based approach for the treatment of arginase-1 deficiency
(ARG1-D), a rare genetic metabolic disease characterized by
complete or partial lack of the enzyme arginase in the liver and
red blood cells. The data from this preclinical study further
characterize our StealthX™ exosome platform.
- Presented data at the International
Society of Extracellular Vesicles (ISEV) Annual Meeting 2024.
Highlights from the abstract included data showing targeted cargo
delivery to mouse lower limbs by exosomes carrying a muscle
targeting moiety by intravenous injection.
Anticipated Upcoming Milestones
CAP-1002 DMD Program
- Plan to have a Type-B clinical FDA
meeting in May 2024 to discuss requests for a pre-BLA meeting and
rolling BLA schedule. Additionally, Capricor plans to share with
FDA its HOPE-2 OLE 3-year safety and efficacy data at this
meeting.
- Plan to report 3-year HOPE-2 OLE
data in the second quarter of 2024.
- Plan to announce further updates
with respect to next steps for HOPE-3 (Cohort B), when
available.
- 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.
StealthX™Exosome Platform
- Plan to provide updates on our
NIAID collaboration for our StealthX™ vaccine as they become
available. NIAID plans to initiate the Phase 1 clinical trial in
late 2024, subject to regulatory approval.
- Continue to explore opportunities
for partnerships and non-dilutive sources of funding to support
advancement of our StealthX™ exosome platform technology.
First Quarter 2024 Financial
Results
Cash position: Cash, cash equivalents and
marketable securities totaled approximately $39.9 million as of
March 31, 2024 compared to approximately $39.5 million as of
December 31, 2023. In the first quarter of 2024, the Company
received $10.0 million from the first milestone payment under our
U.S. Distribution and Commercialization Agreement with Nippon
Shinyaku and raised approximately $2.3 million in net proceeds
through issuances of common stock at an average price of
approximately $5.33 per share under its at-the-market offering
program.
Revenues: Revenues for the first quarter of
2024 were approximately $4.9 million compared with approximately
$3.0 million for the first quarter of 2023. 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
Expenses: Operating expenses for the first
quarter of 2024 were approximately $15.2 million compared with
approximately $11.2 million for the first quarter of 2023.
Net loss: The Company reported a net loss
of approximately $9.8 million, or $0.31 per share, for the first
quarter of 2024, compared to a net loss of approximately $7.8
million, or $0.31 per share, for the first quarter of 2023.
Financial Outlook: We believe that based on the
current operating plan and financial resources, Capricor’s
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 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.
Upcoming Events
The Company plans to participate in the following upcoming
events:
- CureDuchenne 2024 FUTURES National
Conference, May 23-26, 2024, Orlando
- International Society for Cell
& Gene Therapy (ISCT) 2024 Meeting, May 29-June 1, 2024,
Vancouver
- BIO International Convention 2024,
June 3-6, 2024, San Diego
- Parent Project Muscular Dystrophy
(PPMD) 30th Annual Conference, June 27-29, 2024, Orlando
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: 01529679.
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 in
preclinical development 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 revenue streams and
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, 2023, as filed with the Securities and
Exchange Commission on March 11, 2024. 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 March 31, |
|
|
|
2024 |
|
|
|
2023 |
|
|
|
|
|
|
REVENUE |
|
|
|
|
Revenue |
|
$ |
4,906,877 |
|
|
$ |
2,986,696 |
|
|
|
|
|
|
TOTAL REVENUE |
|
|
4,906,877 |
|
|
|
2,986,696 |
|
|
|
|
|
|
OPERATING EXPENSES |
|
|
|
|
Research and development |
|
|
11,101,013 |
|
|
|
7,661,519 |
|
General and administrative |
|
|
4,071,766 |
|
|
|
3,509,885 |
|
|
|
|
|
|
TOTAL OPERATING EXPENSES |
|
|
15,172,779 |
|
|
|
11,171,404 |
|
|
|
|
|
|
LOSS FROM OPERATIONS |
|
|
(10,265,902 |
) |
|
|
(8,184,708 |
) |
|
|
|
|
|
OTHER INCOME (EXPENSE) |
|
|
|
|
Investment income |
|
|
471,829 |
|
|
|
416,442 |
|
|
|
|
|
|
TOTAL OTHER INCOME (EXPENSE) |
|
|
471,829 |
|
|
|
416,442 |
|
|
|
|
|
|
NET LOSS |
|
|
(9,794,073 |
) |
|
|
(7,768,266 |
) |
|
|
|
|
|
OTHER COMPREHENSIVE INCOME (LOSS) |
|
|
|
|
Net unrealized gain (loss) on marketable securities |
|
|
71,888 |
|
|
|
(10,258 |
) |
|
|
|
|
|
COMPREHENSIVE LOSS |
|
$ |
(9,722,185 |
) |
|
$ |
(7,778,524 |
) |
|
|
|
|
|
Net loss per share, basic and diluted |
|
$ |
(0.31 |
) |
|
$ |
(0.31 |
) |
Weighted average number of shares, basic and diluted |
|
|
31,354,629 |
|
|
|
25,247,354 |
|
CAPRICOR THERAPEUTICS, INC. |
SUMMARY BALANCE SHEETS |
|
|
|
March 31, 2024 (unaudited) |
|
December 31, 2023 |
Cash, cash equivalents and marketable securities |
|
$ |
39,917,078 |
|
|
$ |
39,487,703 |
|
Total assets |
|
$ |
49,444,442 |
|
|
$ |
58,734,327 |
|
|
|
|
|
|
Total liabilities |
|
$ |
31,009,504 |
|
|
$ |
36,132,860 |
|
|
|
|
|
|
Total stockholders'
equity – 31,600,183 and 31,148,320 common shares issued and
outstanding at March 31, 2024 and December 31, 2023,
respectively |
|
|
18,434,938 |
|
|
|
22,601,467 |
|
Total liabilities and
stockholders' equity |
|
$ |
49,444,442 |
|
|
$ |
58,734,327 |
|
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