Full Press Release Details
Therapeutics Announces Positive Preclinical Data for Multivalent Exosome-mRNA Vaccine For COVID-19
Vaccine Candidate Employs Novel Exosome-Based mRNA Delivery Platform that Induces Long-Lasting Immunity to Multiple SARS-CoV-2
Scheduling Pre-IND Meeting with FDA to Discuss Clinical Strategy
CALIF., Nov. 9, 2020 - Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company
focused on the discovery, development and commercialization of first-in-class cell- and exosome-based therapeutics for the treatment
and prevention of a variety of diseases and disorders, in collaboration with researchers at Johns Hopkins University, announced
today that positive data from a preclinical study for a multivalent exosome-based mRNA vaccine for COVID-19 has been posted on
the bioRxiv preprint server and will be submitted for publication.
"Capricor's unique exosome-based
mRNA delivery platform is a novel type of SARS-CoV-2 potential vaccine being developed to aid in the worldwide fight against this
virus, which continues to plague the world," said Dr. Linda Marb n, Ph.D., CEO of Capricor. "Exosomes are the
body's own drug delivery vehicle, produced by all cells, abundant in all biofluids, and demonstrated to be safe by decades
of transfusion and transplantation medicine. This study represents a major step forward for our joint effort to develop exosome-based
therapeutics. Furthermore, it highlights the ability of our exosome-based RNA delivery platform to deliver multiple mRNAs, induce
long-lasting immune responses to multiple SARS-CoV-2 proteins, and potentially elicit a broad-based, cellular immunity that extends
beyond the Spike protein alone, which is the sole target of leading vaccine candidates."
Key findings of the pre-clinical study
Exosomes represent a natural drug delivery
vehicle. Their small size, biological origin, minimal immunogenicity and normal role in delivering signals and RNAs to human cells
indicates that they have the potential to expand the range of therapeutics that can be deployed in the fight against human disease.
As a cell-free substance, exosomes can be stored, handled, reconstituted and administered in a similar fashion to common biopharmaceutical
products, such as antibodies and other recombinant protein drugs.
Dr. Marb n continued, "Over
the last few years, Capricor has worked diligently to build our exosome platform to deliver biologics, primarily nucleic acids.
The work reported reflects our commitment to translating our know-how in the area of exosomes to deliver biologic payloads. We
are excited to continue the expansion of this platform of RNA-delivery using exosomes into other therapeutic indications where
delivery has proved challenging. We are planning to meet with the FDA to discuss next steps for a clinical development strategy
and look forward to sharing additional updates as they become available."
The pre-clinical study was designed to
interrogate the utility of Capricor's exosome-based mRNA delivery system, to test this system's ability to express
multiple combinations of the SARS-CoV-2 virus spike (S), nucleocapsid (N), membrane (M) and envelope (E) proteins, and to assess
immune responses to this vaccine in a standard mouse model. The value of this multiplexed, multivalent vaccination approach is
highlighted by the fact that immunodominant epitopes in the T-cell response to SARS-CoV-2 infection extend well beyond the Spike
protein and are particularly focused on the nucleocapsid (N) protein. Following a low-dose immunization protocol, we show that
vaccinated animals generate persistent, long-lasting cellular and humoral immune responses, with no evidence of vaccine-induced
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 cell and
exosome-based therapeutics for the treatment and prevention of diseases. Capricor's lead candidate, CAP-1002, is an allogeneic
cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy and the cytokine storm
associated with COVID-19. Capricor is also investigating the field of extracellular vesicles and exploring the potential of exosome-based
candidates to treat or prevent a variety of disorders. We are now developing two potential vaccines for COVID-19 as part of our
exosome platform. For more information, visit www.capricor.com and follow the Company on Facebook, Instagram and Twitter.
Financial Update for the Third Quarter
The Company reported a net loss of approximately
$3.9 million, or $0.20 per share, for the third quarter of 2020, compared to a net loss of approximately $1.6 million, or $0.43
per share, for the third quarter of 2019. As of September 30, 2020, the Company's cash, cash equivalents and marketable
securities totaled approximately $35.3 million, compared to approximately $9.9 million on December 31, 2019. As of November 6,
2020, the Company has 20,251,602 shares issued and outstanding. The Company will report more detailed results later this week
in its quarterly earnings report.
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, 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, 2019 as filed with the Securities and Exchange Commission on March 27, 2020
and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 as filed with the Securities and Exchange Commission
on August 10, 2020. 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.
None of Capricor's exosome-based
candidates have been approved for clinical investigation.
For more information, please contact:
Caitlin Kasunich / Raquel Cona
KCSA Strategic Communications
212.896.1241 / 212.896.1204
LifeSci Advisors, LLC
AJ Bergmann, Chief Financial Officer