CEL-SCI’s LEAPS Vaccine Offers Promising New Paradigm to Treat Rheumatoid Arthritis: Published in Frontiers in Immunology
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Insights
The development of CEL-4000, an antigen-specific immunomodulating vaccine, represents a significant advancement in the field of immunotherapy for autoimmune diseases like rheumatoid arthritis (RA). The traditional approach to treating RA has largely been through immunosuppressive drugs, which, while effective in controlling symptoms, come with notable side effects, such as increased susceptibility to infections and potentially cancer. In contrast, CEL-4000 aims to rebalance the immune response, potentially offering a safer alternative that maintains the body's natural defense mechanisms.
From an immunological perspective, the ability to elicit an antigen-specific T cell response that targets the disease process without compromising overall immune function could be a game-changer. It addresses the underlying pathology of RA rather than merely suppressing symptoms. However, the transition from preclinical models to human clinical trials will be critical. The success of this vaccine in humans will depend on its efficacy and safety profile, which will need to be demonstrated in well-designed adaptive clinical trials using the Bayesian optimal design model, as suggested by the authors.
CEL-SCI's announcement regarding the publication in Frontiers in Immunology is a positive development for the company and could have implications for its stock valuation. Investors often respond favorably to advancements in a company's technology platform, especially when it addresses a significant unmet medical need like safer treatments for RA. The potential of CEL-4000 to offer a novel approach to RA treatment without the side effects associated with current therapies could position CEL-SCI favorably in the biotech market.
However, it is essential to consider the long development timelines and the inherent risks associated with clinical trials in biotechnology. While preclinical data may be promising, the real test will come with human clinical trials and the ability of CEL-4000 to demonstrate a clear benefit over existing therapies. Market analysts will be watching for further developments closely, as successful trials could lead to significant market opportunities for CEL-SCI.
The emphasis on an adaptive study design and the use of Bayesian optimal design models for advancing LEAPS vaccines like CEL-4000 into clinical trials is noteworthy. These methodologies allow for more flexible and efficient clinical trials, which can adapt to data as it is collected, potentially reducing the time and cost associated with traditional fixed-design trials. Moreover, the focus on enrolling patients with early-stage RA and using disease-related biomarkers to measure treatment responsiveness indicates a targeted approach to clinical development, which could improve the chances of demonstrating efficacy and safety.
For stakeholders, including investors and potential partners, the adoption of these advanced clinical trial designs could signal a commitment to rigorous and innovative research practices. However, the path to regulatory approval is complex and success is not guaranteed. It is important for clinical research analysts to monitor the progress of the trials and to evaluate interim data as it becomes available to gauge the likelihood of CEL-4000 becoming a viable therapeutic option for RA.
CEL-4000 may deliver safe and effective therapy for RA by rebalancing the inflammatory response, without weakening important immune defense mechanisms, risking infections or cancer, as current treatments may
In the article, the authors note that the currently available therapeutic arsenal for the treatment of RA consists mainly of immunosuppressive or ablative drugs, which may carry the potential risk of facilitating recurrent or primary infectious diseases or cancer.
They also underscore that a therapeutic vaccine that can rebalance the inflammatory disease process, promoting antigen-specific disease-directed T cell immune responses, is highly desirable. CEL-4000, an antigen specific immunomodulating vaccine, is provided as a new treatment paradigm that should deliver therapy for RA without weakening important immune defense mechanisms against microbial infections or cancer.
The authors also emphasized that the most appropriate way to advance the LEAPS vaccines to the clinic would be an adaptive study design for clinical trials, enrollment of patients with early-stage RA, and using the Bayesian optimal design model, currently used in oncology studies, as well as the use of disease-related biomarkers to ascertain responsiveness to treatment and introduce additional tools to assess outcome at the disease level.
“At CEL-SCI, we focus on utilizing a healthy immune system to overcome disease safely and with minimal side effects. Our LEAPS platform, and CEL-4000 specifically approaches the treatment of auto immune diseases like rheumatoid arthritis in a completely different way. By rebalancing the immune system to its original healthy state, CEL-4000 could protect patients from the risk of cancer and infection,” stated CEL-SCI CEO, Geert Kersten. “The growing body of preclinical data on LEAPS supports the advancement of this asset into human studies.”
About CEL-SCI Corporation
CEL-SCI believes that boosting a patient’s immune system while it is still intact should provide the greatest possible impact on survival. Multikine* (Leukocyte Interleukin, Injection) is designed to help the immune system "target" the tumor at a time when the immune system is still relatively intact and thereby thought to be better able to mount an attack on the tumor. In a Phase 3 study, CEL-SCI studied patients who were newly diagnosed with locally advanced primary squamous cell carcinoma of the head and neck (oral cavity and soft-palate) with the investigational product Multikine administered first, before they received the standard of care, which involved surgery followed by either radiation or chemoradiation. The Phase 3 study enrolled 928 patients. Our approach is unique because most other cancer immunotherapies are administered only after conventional therapies have been tried and/or failed.
After analyzing data from the Phase 3 study, we have better defined the target head and neck cancer population for Multikine, which is advanced primary head and neck cancer patients with no lymph node involvement and with low PD-L1 tumor expression. In the Phase 3 study, we observed statistically significant survival data in the final target population, showing that Multikine cut the risk of death in half at five years vs control. We plan to submit the proposed study protocol to the FDA in Q1 2024, with the goal to get FDA buy-in for a confirmatory clinical trial, and also to discuss potential accelerated approval pathways.
Multikine (Leukocyte Interleukin, Injection) received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck.
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Forward-Looking Statements
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. 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. Such statements include, but are not limited to, statements about the terms, expected proceeds, use of proceeds and closing of the offering. Factors that could cause or contribute to such differences include an inability to duplicate the clinical 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 for the year ended September 30, 2023. 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. 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.
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Gavin de Windt
CEL-SCI Corporation
(703) 506-9460
www.cel-sci.com
Source: CEL-SCI Corporation
