CEL-SCI Corporation (NYSE American: CVM) reported financial results for the fiscal year ended September 30, 2023, as well as key clinical and corporate developments.

Clinical and Corporate Developments:

• CEL-SCI identified the target head and neck cancer patient population for Multikine that will be the basis for the Company’s regulatory filings for marketing clearance. The new data were presented at the European Society for Medical Oncology (ESMO) Congress. The target population, which saw its 5-year risk of death cut in half, can be identified prior to surgery upon diagnosis with tests that physicians routinely use in cancer screenings, a key finding for Multikine, which is a neoadjuvant therapy. A summary of Multikine’s results in the target population include the following:
  • 73% survival for Multikine vs 45% in the control at 5 years
  • 28% absolute survival benefit
  • Statistically significant p = 0.0015 and hazard ratio = 0.35
  • Tumor reduction rate >13% and tumor downstaging >35%
  • No safety signals or toxicities vs standard of care
  • Target population of an estimated 145,000 patients (global, annual) who present with:
    • No nodal involvement and no extracapsular spread
    • Low PD-L1 tumor expression (different from high PD-L1 targeted by checkpoint inhibitors)
  • Physicians routinely assess these features at baseline; no extra tests needed
  • These features make it easy to write a label for Multikine, which is essential for drug approval

• Marking a major milestone toward regulatory approval, the UK’s National Institute for Health and Care Excellence (NICE) selected Multikine to be evaluated as the potential new standard of care for squamous cell carcinoma of the head and neck (SCCHN). NICE posted a detailed report from the UK’s National Institute for Health and Care Research (NIHR) regarding Multikine, its clinical data, and its potential to become a better standard of care in treating newly diagnosed head and neck cancer in the UK. This published report informs UK doctors, patients, and other interested parties that NICE has started the review of Multikine and is soliciting public comment. CEL-SCI has submitted its SCCHN target population data to the UK’s health regulator, the Medicines and Healthcare Products Regulatory Agency, and is anticipating a Scientific Advise meeting in H1 2024.

• CEL-SCI expects to submit the target population data to the U.S. Food and Drug Administration (FDA) and Health Canada in Q1 2024. Health Canada advised CEL-SCI to request advance consideration for approval under a Notice of Compliance with Conditions policy which could lead to commercialization as early as 2024 if approved. The target population data have also been submitted to the European Medicines Agency (EMA) with a Scientific Advise Meeting expected H1 2024.

• Additional results from the Phase 3 clinical trial of Multikine in advanced primary head and neck cancer were presented at the following conferences:
  • 10th European Congress on Head & Neck Oncology (ECHNO) 2023
    • “Leukocyte Interleukin Injection (LI) immunotherapy followed by radiotherapy extends overall survival (OS) in treatment naïve locally advanced primary squamous cell carcinoma of the head & neck: the IT-MATTERS Study” (Link to data)
  • European Society for Radiotherapy and Oncology (ESTRO) 2023
    • “Histopathology population (HPP) confirms Multikine* [Leukocyte Interleukin Injection (LI)] treatment (Tx) outcome in naïve locally advanced primary head & neck squamous cell carcinoma SCCHN)” (Link to data)
  • American Head and Neck Cancer Society’s (AHNS) 11th Annual International Conference on Head and Neck Cancer 2023
    • “Tumor cell PD-L1 biomarker confirms Leukocyte Interleukin Injection (LI) treatment (Tx) survival outcome advantage in naive locally advanced primary head & neck squamous cell carcinoma (SCCHN), the IT-MATTERS Study” (Link to data)
  • European Society for Medical Oncology (ESMO) Annual Congress 2023
    • “Early response to Neoadjuvant Leukocyte Interleukin Injection (LI) immunotherapy extends overall survival (OS) in locally advanced primary squamous cell carcinoma (SCC) of the head & neck (HN): the IT-MATTERS Study” (Link to data)

• New PD-L1 biomarker findings from the Phase 3 study, which have been integrated into the new target population, were presented at AHNS. The new data demonstrated that Multikine significantly increases overall survival in patients with low levels of tumor cell PD-L1 expression in contrast to approved checkpoint inhibitors, such as Keytruda and Opdivo, which most often show longer survival in a proportion of patients with a higher level of tumor cell PD-L1 expression, suggesting a combination therapy could boost patient outcomes. CEL-SCI filed a patent for the use of Multikine in tumors expressing low levels of PD-L1.

• CEL-SCI’s cGMP state-of-the-art dedicated manufacturing facility commissioning is substantially complete, a significant milestone toward a planned Biologics License Application (BLA) with several regulatory agencies for approval of Multikine.

“The identification of our target patient population is a tremendous achievement that we accomplished in conjunction with regulators and the top key opinion leaders in head and neck cancer, backed by our Phase 3 data from the largest study of its kind in the world. Based on these findings, Multikine’s approval pathway focuses on the 70% of patients not well served by the two leading approved head and neck cancer drugs, Keytruda and Opdivo, which are not approved as pre-surgical treatments, CEL-SCI’s intended market,” stated CEL-SCI CEO, Geert Kersten. “We are working diligently to move forward on the regulatory front, with several meetings and filing upcoming next year.”

Financial Results

During the year ended September 30, 2023, research and development expenses were $22.5 million, a decrease of approximately $2.9 million, or 11%, from $25.4 million in the prior fiscal year. General and administrative expenses in fiscal 2023 were $9 million, a decrease of approximately $1.7 million, or 16%, compared to the year ended September 30, 2022. Net loss narrowed by $4.5 million, or 12%, to approximately $32.2 million for the twelve months ended September 30, 2023 from $36.7 million in fiscal 2022. Almost 40% of the loss is non-cash expenditures. CEL-SCI’s audited financial statements contained an audit opinion from its independent registered public accounting firm that included an explanatory paragraph related to CEL-SCI’s ability to continue as a going concern.

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. Therefore, in the 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 first, BEFORE they received surgery and radiotherapy or surgery plus concurrent radiotherapy and chemotherapy (the current standard of care for these patients). We believe this approach is unique. Most other cancer immunotherapies are administered only after conventional therapies have been tried and/or failed.

Multikine 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. The Phase 3 study enrolled 928 patients.

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.

The Company has operations in Vienna, Virginia, and near/in Baltimore, Maryland.

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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