Benzinga | Dec 13, 2021 09:19AM EST

Capricor Therapeutics Announces Updates On Its Vaccine And Therapeutic Programs For COVID-19

Capricor's Exosome-based mRNA Vaccine for SARS-CoV-2

-In-Vivo Preclinical Data Published in the Journal of Biological Chemistry-

-Preclinical Data Demonstrates Strong T-Cell Response for High Conserved N Protein which may Confer Longer Lasting Immunity-

-Completing Non-Clinical Studies for IND Submission-

CAP-1002 -- Capricor's Cell Therapy Program for Severe COVID-19 Patients

-Phase 2 Trial Enrollment Complete with 63 Patients Randomized-

-Data Expected in First Quarter 2022-

SAN DIEGO, Dec. 13, 2021 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, today announced new updates on its vaccine and therapeutic development programs focused on COVID-19. The preclinical data describes, for the first time, the potential for Capricor's multivalent exosome mRNA vaccine candidate for SARS-CoV-2 to generate a strong T-cell response against viral infection in addition to eliciting an antibody response. In addition, Capricor will announce topline data from its Phase 2 clinical trial, INSPIRE, in the first quarter of 2022. This study is designed to assess the ability of CAP-1002 to modulate the cytokine storm and attenuate the sequelae caused by severe COVID-19 disease.

Capricor's multivalent mRNA vaccine utilizes exosomes, which 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 proprietary SARS-CoV-2 vaccine is multivalent, delivering both the highly mutagenic S protein (Spike) and the more conserved N protein (Nucleocapsid) which may offer broader immunity against SARS-CoV-2. Utilization of a multivalent vaccine may potentially offer enhanced protection against future coronavirus variants and may be critical in ensuring long-term management of the COVID-19 pandemic.

"This published study represents a significant milestone in Capricor's efforts to develop mRNA-loaded exosomes for the prevention and treatment of human disease," said Dr. Linda Marb?n, Ph.D., CEO of Capricor. "Exosomes have been demonstrated to be safe and effective for mRNA delivery by this new study. This new report, which arises from a close collaboration with Dr. Stephen J. Gould and colleagues of Johns Hopkins University, lays the foundation for our platform by demonstrating how to make clinical-grade exosomes, how to load them at high efficiency with synthetic mRNAs, and, once formulated, how they are able to drive functional expression of the encoded proteins over sustained cycles of weekly and semi-weekly injections for more than two months. Further, it demonstrates that this approach can be used to simultaneously deliver multiple mRNAs encoding both Spike and Nucleocapsid, a dual immunization approach that has recently emerged as key to protecting against both proximal and distal COVID-19 disease. At this time, we plan to file an IND and position this vaccine as a potential booster to currently available vaccines."

Capricor's therapeutic approach to COVID-19 is based on multiple published peer-reviewed studies of CAP-1002 demonstrating favorable modulation of various inflammatory cytokines and regulation of the immune response. COVID-19's more severe presentation is thought to be due to overstimulation of the immune system, which triggers a cytokine storm in which the body is overwhelmed with pro-inflammatory molecules. This immune response may become excessive and pathologic, inducing ARDS, multi-system organ failure and death.

Dr. Marb?n continued, "Our ability to leverage a therapeutic CAP-1002 program to intervene in the progression of the cytokine storm in COVID-19 patients may reduce the need for ventilatory support and may potentially not only save lives but also reduce long-term morbidity that can occur after severe respiratory compromise. We look forward to sharing the results from this double-blind, placebo-controlled Phase 2 study in the first quarter of 2022 and then moving rapidly to discuss this program with FDA if a pathway towards further development becomes clear."