• Head and neck cancer patients treated with Multikine who had complete or partial tumor responses before surgery had improvements in overall survival, suggesting pre-surgical tumor response is a strong indicator of efficacy and leads to improved overall survival
• A third-party head and neck cancer study published in “Cancer Cell” confirms the observation that pre-surgical tumor responses in head and neck cancer leads to improved survival

VIENNA, Va. / Apr 08, 2025 / Business Wire / CEL-SCI Corporation (NYSE American: CVM) today announced that a study titled “Distinct CD8+ T cell dynamics associate with response to neoadjuvant cancer immunotherapies” by Li Housaiyin et. al., Cancer Cell (2025) provides support for CEL-SCI’s approach aimed at seeking early regulatory approval for Multikine* (Leukocyte Interleukin, Injection) as a neoadjuvant in the treatment of newly diagnosed previously untreated locally advanced head and neck cancer based on early tumor responses.

The concept that tumor responses predict survival has been acknowledged for many cancer types and has led to accelerated approval of many cancer drugs. The study in Cancer Cell, which had 41 evaluable patients, gives further support that this is also true in the neoadjuvant pre-surgical immunotherapy treatment of head and neck cancer.

“The data from both our completed Multikine neoadjuvant Phase 3 study and the study published in Cancer Cell show that newly diagnosed locally advanced head and neck cancer patients who were treated with immune therapies before surgery and had tumor responses, were also likely to have better overall survival. This is quite logical—when a person’s tumor shrinks in response to immunotherapy treatment before the tumor is removed by surgery—their survival outcome is expected to be better,” stated CEL-SCI CEO Geert Kersten. “We believe that the recent greater availability of peer-reviewed published data specific to neoadjuvant therapy and early tumor response in head and neck cancer supports our development and regulatory efforts.”

CEL-SCI’s 212-patient Confirmatory Registration Study, which has received the FDA’s go-ahead, is designed to verify the statistically significant efficacy and safety results from the Company’s previously completed Phase 3 randomized controlled Multikine trial.

During the Multikine Phase 3 clinical trial, the 5-year survival rate of the target patient population for the confirmatory study increased to 73% when patients were treated with Multikine before surgery vs 45% for control patients who received only the standard of care treatments [Log rank p=0.0015 and a hazard ratio of 0.35 (0.18, 0.65; Wald p=0.0012)].

As a neoadjuvant therapy, Multikine is given to patients right after diagnosis, prior to surgery. In the Phase 3 study, pre-surgery objective early tumor response to treatment with Multikine was confirmed by pathology at surgery. There were 45 objective early responders, of which 5 were complete pathological responders, whose tumors completely disappeared in the Multikine treated group, within the 3-weeks of Multikine treatment. There were zero responders reported in the control group pre-surgery. Pre-surgical responders exhibited a 306% overall survival prolongation and had a significantly lowered death rate -vs- non-responders 22.2% vs 54.1% (2-sided Fisher Exact Test, p <0.0001) in support of pre-surgical response being prognostic.

About CEL-SCI Corporation

CEL-SCI has been a pioneer in advocating the use of neoadjuvant cancer immunotherapy. We believe that the greatest benefit for patients can only be achieved if you boost a patient’s immune system before surgery, radiotherapy and chemotherapy have damaged it. 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.

Multikine (Leukocyte Interleukin, Injection), given right after diagnosis and before surgery, has been dosed in over 740 patients and received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck. Based on the data from the completed randomized controlled Phase 3 study, the FDA concurred with CEL-SCI’s target patient selection criteria and gave the go-ahead to conduct a confirmatory Registration Study. The study will enroll 212 newly diagnosed locally advanced primary treatment naïve resectable head and neck cancer patients with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy), representing about 100,000 patients annually.

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. 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, inability to finalize a partnering agreement 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, 2024. 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.