Potential Vaccine Candidate Employs Novel Exosome-Based mRNA Delivery Platform that Induces Long-Lasting Immunity to Multiple SARS-CoV-2 Proteins

Capricor Scheduling Pre-IND Meeting with FDA to Discuss Clinical Strategy

LOS ANGELES, CA, USA I November 09, 2020 I 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 Marbán, 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 in mice include:

  • Development of safe, non-toxic exosome formulation capable of delivering functional mRNA in vitro and in vivo.
  • Creation of a multiplexed exosome-RNA vaccine that expresses viral antigens engineered to induce cellular immunity and antibody responses to multiple proteins of SARS-CoV-2.
  • Validation that an exosome mRNA vaccine can induce:
    ° Persistent cellular immune responses to the SARS-CoV-2 N and S proteins.
    ° Moderate but sustained antibody responses to the SARS-CoV-2 N and S proteins.

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. Marbán 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.”

Study Design

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 adverse 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 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 FacebookInstagram and Twitter.

Financial Update for the Third Quarter of 2020

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.

SOURCE: Capricor Therapeutics