VIENNA, Va.--(BUSINESS WIRE)--CEL-SCI Corporation (NYSE American: CVM) announced today its LEAPS COV-19 peptides, delivered as a therapeutic treatment following SARS-CoV-2 virus challenge, achieved a 40% survival rate in transgenic mouse models as compared to 0% survival in the two control groups in studies conducted at the University of Georgia Center for Vaccines and Immunology.
Scientists at the University of Georgia working in conjunction with CEL-SCI’s scientific team conducted a challenge study in human(h) ACE2 receptor transgenic mice infected with a dose of SARS-CoV-2 (the causative agent of COVID-19 disease) virus sufficient to cause death in all animals within 8 days. This transgenic animal model is useful to study COVID-19 disease because the mice express the molecule that provides entry for the SARS-CoV-2 virus into human cells. Virus infection is optimized in this animal model providing an ideal system to determine vaccine induced immune protection or therapy against a SARS-CoV-2 infection of humans.
The animals were therapeutically treated with CEL-SCI’s LEAPS COV-19 peptides one day after infection with a lethal dose of SARS-CoV-2. Of the LEAPS treated mice, forty percent (40%) were alive, recovering and regained lost weight, attaining > 90% of their starting weight, by the study’s end. In contrast, mice in the two control groups lost 20% or more of their body weight by day 8 and all of them died between day 5 and day 8 post challenge. The success of this therapy was statistically significant at a 95% level.
An additional study conducted using LEAPS as a vaccine to prevent disease resulted in similar findings to the above described study, but with a slightly lower level of statistical significance. In this study, the Human(h) ACE2 transgenic mice were dosed twice with the LEAPS conjugate 28 and 14 days prior to being challenged with a lethal dose of SARS-CoV-2 virus.
“We are highly encouraged by these results, which clearly show our LEAPS COV-19 peptides produced a survival benefit in a very credible animal model, which mimics the virus’ effects in humans. A critical benefit of LEAPS’s mechanism of action is its focus on highly conserved portions within the nucleoprotein of the virus. This may become very important as mutations in SARS-CoV-2 that could limit a vaccine or treatment induced protection have already been reported. While LEAPS can potentially work as a vaccine, the greater need though is to provide better treatment for the acute disease stage and also for the longer-term complications of the virus. Helping those patients is our goal,” stated CEL-SCI CEO Geert Kersten.
Daniel Zimmerman, Ph.D., Senior VP of Research Cellular Immunology at CEL-SCI and discoverer of the LEAPS technology added, “Based on prior animal studies we also know that LEAPS peptides can reduce inflammation and cytokine storm. We think it is likely to have a similar effect on the cytokine storm that causes severe systemic COVID-19 disease. Our next step is to leverage the findings from these two animal studies into future studies that will optimize treatment dosing and test additional LEAPS peptides as a therapy.”
Background on LEAPS and its Relevance to COVID-19
CEL-SCI’s goal is to develop an immunotherapy with the potential to treat the SARS-CoV-2 virus using its patented LEAPS peptide technology. The LEAPS peptides utilize conserved regions of SARS-CoV-2 proteins to stimulate protective antibody and cell mediated T cell responses to reduce viral load and systemic inflammatory immune responses. The LEAPS peptide technology can be used to construct immunotherapeutic peptides that exhibit both antiviral and anti-inflammatory properties at the same time. Consequently, these products not only target the virus infection against which they are directed, but also elicit the appropriate protective response(s) against it, without exacerbating an overly active inflammation.
CEL-SCI’s studies are utilizing the LEAPS peptide approach, which is unique in its proven ability in animals to elicit both a cell mediated antiviral response and an anti-inflammatory immunomodulating response by activating CD8 T lymphocytes. Previous studies showed that LEAPS immunogens can prevent lethal infection by herpes simplex virus (HSV) and stop the inflammatory disease progression of rheumatoid arthritis in animal models. LEAPS peptides against HSV demonstrated that the T cell response was sufficient to prevent viral disease, and if there was residual virus production, anti-viral antibody was generated to further control the spread of the virus.
The LEAPS-COV-19 peptide conjugates are directed towards antigens within the NP protein of SARS-Cov-2, the causative agent of COVID-19. These conjugates elicit cytolytic T cell responses to virus infected cells and immunomodulating responses. Unlike glycoprotein spike antigens, which are utilized in antibody-based vaccines, the T cell antigens are less variable between viral strains and less likely to change in response to antibodies elicited by prior infection or other vaccines. Cytolytic T cell responses attack the virus infected cellular “factories” within the infected host in order to eliminate the source of virus and help subdue the infection.
Ligand Epitope Antigen Presentation System (LEAPS) platform technology has demonstrated in several animal models the ability to design antigen-specific immunotherapeutic peptides that preferentially direct the immune response to a cellular (e.g., T cell), humoral (antibody) or mixed response and are also capable of enhancing important T-regulatory (Treg) responses. Therefore, the LEAPS technology provides the opportunity to develop immunotherapeutic products for diseases for which disease associated antigenic peptide(s) sequences have already been identified, such as: a number of infectious diseases, some cancers, autoimmune diseases (e.g., RA), allergic asthma and allergy, select CNS diseases (e.g., Alzheimer's) and the SARS-Cov-2 virus.
The Company's LEAPS technology is currently also being developed as a therapeutic vaccine for rheumatoid arthritis and is supported by $1.5 million grant for IND enabling studies from the National Institute of Arthritis and Musculoskeletal and Skin Diseases.
About CEL-SCI Corporation
The Company’s LEAPS technology is currently being developed for rheumatoid arthritis and as a potential treatment for COVID-19 infection/disease.
CEL-SCI is also close to the readout for a very large head and neck cancer study using another immunotherapy product called Multikine*. CEL-SCI believes that boosting a patient’s immune system while it is still intact should provide the greatest possible impact on survival. Therefore, in the Phase 3 study CEL-SCI treated patients who are newly diagnosed with advanced primary squamous cell carcinoma of the head and neck with the investigational product Multikine first, BEFORE they received surgery, radiation and/or chemotherapy. This approach is unique. Most other cancer immunotherapies are administered only after conventional therapies have been tried and/or failed. Multikine (Leukocyte Interleukin, Injection), has received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck.
CEL-SCI believes that this Phase 3 study is the largest Phase 3 study in the world for the treatment of head and neck cancer. Per the study’s protocol, newly diagnosed patients with advanced primary squamous cell carcinoma of the head and neck were treated with the Multikine treatment regimen first - for 3 weeks prior to receiving the Standard of Care (SOC), which involves surgery, radiation or concurrent radiochemotherapy. Multikine is designed to help the immune system “see” the tumor at a time when the immune system is still relatively intact and thereby thought to better be able to mount an attack on the tumor. The aim of treatment with Multikine is to boost the body’s immune system prior to SOC to attack the cancer. The Phase 3 study is fully enrolled with 928 patients and the last patient was treated in September 2016. To prove an overall survival benefit, the study requires CEL-SCI to wait until 298 events have occurred among the two main comparator groups. This study milestone occurred in late April 2020.
The Company has operations in Vienna, Virginia, and near/in Baltimore, Maryland.
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, including statements with respect to Multikine and the Phase 3 clinical trial of Multikine in patients with advanced primary squamous cell carcinoma of the head and neck. When used in this press release, the words "intends," "believes," "anticipated," "plans" and "expects," and similar expressions, are intended to identify forward-looking statements. Such statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Factors that could cause or contribute to such differences include, an inability to duplicate the clinical trials or nonclinical results demonstrated in clinical studies, timely development of any potential products that can be shown to be safe and effective, receiving necessary regulatory approvals, difficulties in manufacturing any of the Company's potential products, inability to raise the necessary capital and the risk factors set forth from time to time in CEL-SCI's filings with the Securities and Exchange Commission, including but not limited to its report on Form 10-K/A for the year ended September 30, 2019. The Company undertakes no obligation to publicly release the result of any revision to these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.
* Multikine (Leukocyte Interleukin, Injection) is the trademark that CEL-SCI has registered for this investigational therapy, and this proprietary name is subject to FDA review in connection with the Company's future anticipated regulatory submission for approval. Multikine has not been licensed or approved for sale, barter or exchange by the FDA or any other regulatory agency. Similarly, its safety or efficacy has not been established for any use. Moreover, no definitive conclusions can be drawn from the early-phase, clinical-trials data involving the investigational therapy Multikine. Further research is required, and early-phase clinical trial results must be confirmed in the Phase 3 clinical trial of this investigational therapy that is in progress.