CEL-SCI to Launch FDA Registration-Enabling Phase 3 Confirmatory Study to Bring Multikine® to Market for Newly Diagnosed Head and Neck Cancer
- Patient enrollment expected to commence at clinical centers in the U.S., Europe, Asia and later on in South America
- The 212-patient confirmatory study is powered at approximately 97% to confirm a previously observed 0.34 hazard ratio, representing a 66% reduction in the risk of death
- In the target population, Multikine followed by standard of care increased the 5-year survival rate to 73% versus 45% with standard of care alone
- Pre-surgical tumor responses will be assessed and may serve as the basis for an early conditional or accelerated approval application with the FDA
VIENNA, Va.–(BUSINESS WIRE)–
CEL-SCI Corporation (NYSE American: CVM) today announced it is launching its global Confirmatory Registration Study of Multikine® (Leukocyte Interleukin, Injection)* as a neoadjuvant treatment for newly diagnosed, previously untreated, resectable, locally advanced primary head and neck cancer.
The global, multi-center study will enroll patients in the United States, Europe, Asia, and South America. Orient EuroPharma, CEL-SCI’s strategic partner in Taiwan, will oversee and fund patient enrollment within its territory. The study is highly optimized and designed with approximately 97% statistical power to detect a previously observed 0.34 hazard ratio based on overall survival (OS).
The Confirmatory Study will enroll approximately 212 patients presenting with newly diagnosed, previously untreated, resectable, locally advanced squamous cell carcinoma (head and neck cancer) whose tumors exhibit low or zero PD-L1 expression and who present with no clinical lymph node involvement at entry. This precise cohort represents the patient population that demonstrated the most profound clinical benefit in CEL-SCI’s completed 928-patient randomized controlled Phase 3 study. The patients in the confirmatory study will be required to possess the same disease characteristics as the patients who showed the excellent survival benefit in the completed Phase 3 study. By isolating this specific high-responder group of patients identified in the prior randomized controlled trial, the confirmatory study maintains robust statistical power while requiring a substantially smaller, more efficient patient enrollment target.
“Our Confirmatory Registration Study marks a definitive milestone toward establishing Multikine as the new immunotherapy standard of care for newly diagnosed head and neck cancer with low or zero levels of PD-L1,” said Geert Kersten, CEO of CEL-SCI. “Enrollment will span premier clinical centers across the U.S., Europe, Asia, and South America, targeting the exact population where our previous Phase 3 trial demonstrated compelling, statistically significant improvements in overall survival, quality of life, and safety. Backed by approximately 97% statistical power to confirm this survival advantage, we believe this registration-enabling study provides an exceptional opportunity to validate our earlier Phase 3 results and bring this much-needed therapy to patients.”
Clinical Rationale & Phase 3 Data Foundation
In the target cohort of CEL-SCI’s completed Phase 3 study, 114 patients who received a 3-week regimen of Multikine prior to receiving standard of care (SOC) experienced a significant reduction in the risk of death, achieving a 73% five-year overall survival rate compared to 45% for patients treated with SOC alone.
Furthermore, neoadjuvant administration of Multikine induced rapid, statistically significant pre-surgical tumor responses, including complete tumor responses confirmed by pathology, within 3 weeks of Multikine treatment. In the target population, the pre-surgical objective response rate was 13% in the Multikine arm versus 0% in the control arm. Pre-surgical downstaging was observed in 35% of Multikine-treated patients compared to 13% of controls.
Because these early, pre-surgical objective responses were demonstrated to be highly prognostic and appear to be predictive of long-term overall survival, CEL-SCI intends to utilize these early endpoints to support potential accelerated or conditional approval applications globally. Concurrently, overall survival follow-up will continue to be assessed in a blinded manner until the required event threshold is met.
About Head and Neck Cancer
Head and neck cancer remains one of the world’s most challenging oncology indications, with approximately 900,000 new diagnoses annually. Despite modern iterations of surgery and chemoradiation, long-term survival outcomes have stalled, highlighting a critical global need for non-toxic neoadjuvant immunotherapies that directly improve survival.
About Multikine
Multikine (Leukocyte Interleukin, Injection) is a novel, patented investigational cancer immunotherapy designed to be administered immediately following diagnosis, prior to surgery, radiation, or chemotherapy. By treating patients before any other treatment, while their immune systems are still intact, Multikine aims to stimulate a robust anti-tumor immune response before the immune system is compromised by conventional treatments. Multikine has been administered to over 740 patients across its clinical development program and has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the neoadjuvant therapy of squamous cell carcinoma of the head and neck.
About CEL-SCI Corporation
CEL-SCI is a clinical-stage biotechnology company focused on activating the immune system to fight cancer at the earliest stage of clinical intervention. The Company maintains primary operations in Vienna, Virginia, and manufacturing/operations near 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, 2025. 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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COMPANY CONTACT:
Gavin de Windt
CEL-SCI Corporation
(703) 506-9460
KEYWORDS: Virginia United States North America
INDUSTRY KEYWORDS: Science Radiology Biotechnology Research Oncology Health FDA Clinical Trials
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