New Cancer Cell Study Backs CEL-SCI’s Early Approval Bid for Multikine in Head & Neck Cancer
CEL-SCI Corporation (NYSE American: CVM), a clinical-stage immunotherapy company developing innovative treatments for cancer and infectious diseases, today announced findings that reinforce the scientific rationale behind its lead investigational immunotherapy, Multikine® (Leukocyte Interleukin, Injection). A recently published study in Cancer Cell titled “Distinct CD8+ T cell dynamics associate with response to neoadjuvant cancer immunotherapies” by Li Housaiyin et al. (2025) provides key evidence supporting the predictive value of early tumor response in neoadjuvant cancer treatment, specifically in head and neck squamous cell carcinoma (HNSCC). This development bolsters CEL-SCI’s ongoing efforts to seek regulatory approval for Multikine as a neoadjuvant immunotherapy based on early tumor response outcomes.
The concept that early tumor shrinkage following treatment predicts long-term survival is not new. It has been widely accepted and employed as a surrogate endpoint in regulatory decision-making for numerous oncology indications. The U.S. Food and Drug Administration (FDA) and other global regulators have previously granted accelerated approvals to therapies in breast cancer, non-small cell lung cancer, and other malignancies using pathologic complete response (pCR) or major pathologic response (MPR) as predictors of clinical benefit.
However, this surrogate endpoint had not been extensively validated in head and neck cancer until now. The Cancer Cell study, which involved 41 evaluable patients undergoing neoadjuvant immunotherapy prior to surgery for head and neck cancer, provides direct evidence linking early CD8+ T-cell dynamics and tumor responses with subsequent overall survival outcomes. These findings align closely with CEL-SCI’s clinical experience and data from its pivotal Phase 3 study of Multikine.
Multikine’s Clinical Program: A Focus on Early Intervention
CEL-SCI’s flagship program centers around the use of Multikine as a neoadjuvant immunotherapy administered to patients shortly after diagnosis but prior to undergoing surgery and standard chemoradiation. Multikine is designed to stimulate the immune system within the tumor microenvironment, aiming to generate an anti-tumor immune response that begins before the primary tumor is surgically excised.
The company has already completed a comprehensive Phase 3 randomized controlled trial (RCT) evaluating Multikine in over 900 patients. From this large trial, CEL-SCI identified a prospectively defined subgroup of 212 patients who met specific inclusion criteria and demonstrated statistically significant improvement in survival when treated with Multikine prior to surgery compared to those receiving standard-of-care (SOC) therapies alone.
To confirm these compelling findings, CEL-SCI has received regulatory clearance from the FDA to proceed with a 212-patient Confirmatory Registration Study. This study is intended to validate the survival benefit observed in the original trial and provide additional data necessary for a Biologics License Application (BLA) submission.
Remarkable Efficacy in Target Subgroup
The survival benefit in the selected patient population from CEL-SCI’s original Phase 3 trial was striking. Patients who received Multikine prior to surgery exhibited a 5-year overall survival rate of 73%, compared to only 45% for those in the control arm receiving standard treatment modalities (p=0.0015; hazard ratio [HR] 0.35; 95% CI: 0.18–0.65; Wald p=0.0012). These data suggest a profound reduction in mortality risk when Multikine is integrated into the early stages of therapy.
Further substantiating the impact of Multikine as a neoadjuvant therapy, the trial demonstrated that objective early tumor responses could be detected pathologically at the time of surgery, following just three weeks of Multikine administration. Among the Multikine-treated cohort, 45 patients exhibited objective tumor responses prior to surgical resection, including five complete pathological responses—defined as the complete absence of viable tumor cells.
Notably, none of the patients in the control group showed any tumor response during the pre-surgical window, emphasizing the unique activity of Multikine in the neoadjuvant setting.
Early Tumor Response as a Prognostic Marker
Perhaps most compelling are the data correlating early tumor response with long-term survival outcomes. Patients who responded to Multikine treatment before surgery experienced a 306% increase in overall survival duration compared to non-responders. Moreover, the mortality rate among early responders was dramatically lower—22.2% versus 54.1% for non-responders (Fisher Exact Test, two-sided p<0.0001). These results strongly indicate that pre-surgical tumor response is a valid and powerful prognostic marker in this patient population.
Geert Kersten, CEO of CEL-SCI, commented on the implications of the recent publication and its alignment with Multikine’s clinical data:
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 more likely to experience better overall survival outcomes. This is quite logical—when a tumor shrinks in response to immunotherapy before surgical resection, we expect that patient’s long-term prognosis to improve. We believe the growing body of peer-reviewed data specific to neoadjuvant immunotherapy in head and neck cancer reinforces our regulatory and development pathway.”
Regulatory Pathway and Future Outlook
CEL-SCI’s regulatory strategy hinges on leveraging the robust survival benefit demonstrated in the target subgroup, supported by early tumor response data as a surrogate for clinical benefit. The precedent for using such surrogate endpoints exists across multiple cancer indications and may facilitate earlier approval decisions by the FDA or other regulatory bodies.
The Company has also expressed optimism that the increasing availability of published data supporting neoadjuvant immune-based strategies in head and neck cancer will catalyze a paradigm shift in how these patients are treated. Historically, head and neck squamous cell carcinoma has been managed primarily through surgery and chemoradiotherapy, with immunotherapy reserved for recurrent or metastatic disease. CEL-SCI’s work with Multikine aims to change that narrative—bringing immune modulation to the forefront of treatment strategy right from diagnosis.
Broader Implications for Cancer Immunotherapy
The insights gleaned from CEL-SCI’s work and the recent Cancer Cell publication have implications that extend beyond head and neck cancer. They highlight the growing importance of neoadjuvant immunotherapy as a frontier in cancer treatment, where the immune system is activated while the tumor is still present, potentially yielding stronger responses and long-lasting systemic immunity.
Multikine’s development trajectory and clinical data also underscore the relevance of personalized and timed immune intervention, suggesting that when and how immunotherapy is delivered can be as critical as the therapy itself.
As the Confirmatory Registration Study progresses and more peer-reviewed studies emerge, CEL-SCI is well-positioned to be at the forefront of this evolution in oncology care.
