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- Target Population Selection: Biomarker profile – applied prior to treatment of locally advanced disease with no lymph node involvement (N0) and low/zero tumor PD-L1 expression (TPS < 10%) or zero identifies patients deriving maximum survival benefit from pre-surgical Multikine® treatment.
- Nearly 3x Higher Pre-Surgical Response: New analysis demonstrated a statistically significant increase in pre-surgical response/disease downstaging (38% for Multikine + SOC vs. 13% for SOC alone; p = 0.003).
- Tumor responses: Significant number of complete and partial tumor responses before surgery.
- Substantial Overall Survival Advantage: Biomarker-selected target population demonstrated a 73.4% 5-year survival rate for Multikine-treatment regimen + SOC versus 45.0% for SOC alone, HR = 0.34, p = 0.0012, representing a 66% reduction in the risk of death.
- Favorable Safety Profile: In a broader new safety analysis, Multikine regimen demonstrated excellent safety without increasing Grade 3/4 adverse events or systemic toxicity compared to standard of care alone, with no Multikine-related deaths.
- Foundation for U.S. Registration Study: Selection criteria form the core of CEL-SCI's upcoming 212-patient U.S. Registration Study, which is designed with about 97% statistical power to prospectively confirm these findings.
- Keynote Presentation: Data presented in a Keynote Address by CSO Dr. Eyal Talor at the 6th World Congress on Otology, Rhinology and Laryngology in Rome, Italy.
Cel-Sci Corporation (NYSE American: CVM) today announced positive new pre-surgical tumor response and safety analyses from its completed Phase 3 study of Multikine® (Leukocyte Interleukin, Injection)*. The findings were presented by the Company's Chief Scientific Officer and developer of Multikine, Dr. Eyal Talor, during a keynote address at the 6th World Congress on Otology, Rhinology and Laryngology in Rome, Italy.
The presentation highlights how pre-surgical patient stratification addresses a long-standing limitation in head and neck cancer management, where patient risk assessment traditionally relies on post-operative pathological evaluation.
This precision oncology approach directs immunotherapy to patients most likely to derive clinical benefit prior to surgery, radiation, or chemotherapy and impacts their survival outcome. By evaluating disease characteristics and tumor biomarkers specific to Multikine's mechanism of action, CEL-SCI identified a patient population achieving a 73.4% 5-year overall survival rate with Multikine plus standard of care (SOC) versus 45.0% for SOC alone. These findings serve as the scientific foundation for CEL-SCI's upcoming Phase 3 U.S. Registration Study.
"This combination of disease stage and pre-treatment tumor biomarkers allows us to identify patients who experience a major survival advantage when treated with Multikine first—before surgery," stated CEL-SCI CEO Geert Kersten. "The new analysis shows that nearly three times as many Multikine-treated patients achieved disease downstaging compared to controls as well as pre-surgical tumor shrinkage. Crucially, Multikine achieved these clinical gains without adding toxicity to standard of care treatments. These data provide the basis for our focused Registration Study toward the potential commercialization of Multikine."
Biomarker-Based Patient Selection
CEL-SCI's selection criteria evaluate two pre-treatment parameters accessible at initial diagnosis:
- Clinical N0 status: Locally advanced primary tumor with no detectable lymph node involvement.
- Low/Zero PD-L1 expression: Tumor Proportion Score (TPS) <10% or 0%.
These criteria define the target population in which neoadjuvant Multikine (administered over 3 weeks prior to surgery) demonstrated its strongest survival effect. These selection criteria will be applied prospectively in the upcoming U.S. Registration Study.
Pre-Surgical Tumor Responses and Improved Survival
In the biomarker-selected population, 38.0% of Multikine-treated patients experienced disease downstaging and/or tumor shrinkage prior to surgery, compared to 13.0% of patients receiving SOC alone—representing a nearly threefold response rate (2-sided Fisher's Exact p = 0.003). Objective tumor responses (shrinkage) were observed in 13% of patients versus 0% receiving SOC alone.
In this target subgroup ( n = 114) from the completed Phase 3 study:
- 5-Year Overall Survival: 73.4% for Multikine + SOC vs. 45.0% for SOC alone, representing a 28.4% absolute overall survival advantage.
- Median Time to Death (Months): Not Reached (NR) [95% CI: 54.8 – NR] for Multikine vs. 50.6 months [95% CI: 43.4 – 72.2] for SOC alone.
- Hazard Ratio (HR): 0.34 [95% CI: 0.18 – 0.66] (Wald p = 0.0012; unstratified log-rank p = 0.0015), representing a 66% reduction in the risk of death.
Favorable Safety Profile
In an analysis across the broader Phase 3 trial, Multikine demonstrated a favorable safety profile:
- No deaths attributed to Multikine.
- No evidence of systemic hyper-immune responses or treatment-induced autoimmune conditions.
- Adverse events associated with pre-surgical Multikine administration were primarily local injection-site reactions that resolved following surgery.
- Multikine did not increase the incidence or severity of Grade 3 or Grade 4 adverse events relative to standard of care alone.
CEL-SCI believes Multikine's safety profile compares favorably to immune checkpoint inhibitors, which frequently carry boxed warnings for severe or fatal organ-specific immune-related adverse events.
About the U.S. Registration Study
CEL-SCI's upcoming 212-patient U.S. Registration Study will prospectively apply these biomarker selection criteria with Overall Survival (OS) as the primary endpoint. The study design provides for approximately 97% statistical power to detect the survival effect observed in the target population of the completed Phase 3 study.
Pre-surgical tumor responses will be evaluated within weeks following full enrollment and treatment, providing an early readout of anti-tumor activity. CEL-SCI intends to explore potential accelerated regulatory pathways using these early response data while continuing to follow patients for overall survival.
About Multikine
Multikine is a novel cancer immunotherapy administered before surgery as a treatment for newly diagnosed previously untreated locally advanced head and neck cancer. It is manufactured at scale, stored in a vial, and injected directly around the tumor. Its goal is to activate a person's immune system to fight cancer before the ravages of surgery, radiation and chemotherapy have weakened the immune system. In the world's largest locally advanced head and neck cancer Phase 3 study, Multikine increased the 5-year survival rate of the target patient population to 73% vs 45% in patients treated with standard of care alone and halved the risk of death from 55% to 27%.
About Cel-Sci Corporation
CEL-SCI believes that boosting a patient's immune system before surgery, radiotherapy and chemotherapy have damaged it, should provide the greatest possible impact on survival. 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 administered to over 740 patients and has an excellent safety profile compared to other cancer drugs. It has 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. 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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Gavin de Windt
Cel-Sci Corporation
(703) 506-9460
