A 212-patient Confirmatory Registration Study for Multikine in PD-L1 low newly diagnosed head and neck cancer patients is currently in final stage of start-up preparations - full enrollment expected by Q2 2026
Plans to seek early approval based on pre-surgical response rates
A positive study outcome will position Multikine as the first treatment in over 50 years for newly diagnosed locally advanced head and neck cancer patients – a population with a severe unmet medical need
Development of Multikine in other cancers is planned
VIENNA, Va., January 14, 2025--(BUSINESS WIRE)--CEL-SCI Corporation (NYSE American: CVM) reported financial results for the fiscal year ended September 30, 2024, as well as key clinical and corporate developments.
"We are very optimistic about the prospects of our investigational drug Multikine in 2025 and 2026. We look ahead to Multikine being able to deliver much needed relief to a patient population with a severe unmet medical need. We see several very important milestones and value drivers upcoming. Positive data on presurgical response rates, previously shown to be indicative of overall survival, could enable us to apply for early approval," stated CEL-SCI CEO, Geert Kersten. "This confirmatory Registration Study is designed to do two things: create a new standard of care for newly diagnosed treatment naïve locally advanced head and neck cancer patients; and provide survival benefit for the population of patients with low/negative tumor expression of PD-L1, those patients who have historically not responded well to widely used checkpoint inhibitors such as Keytruda and Opdivo."
Clinical and Corporate Developments:
The U.S. Food and Drug Administration (FDA) gave CEL-SCI the ‘go-ahead’ to conduct a confirmatory 212-patient Registration Study of Multikine in the treatment of locally advanced head and neck cancer based on strong safety and efficacy data from the completed Phase 3 study which enrolled 928 patients. The FDA agreed with the pre-surgical selection of patients most likely to benefit from Multikine—those with newly diagnosed locally advanced primary head and neck cancer with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy). In this target population in the completed Phase 3 study, Multikine demonstrated a 5-year survival of 73% vs a 45% survival in the control patients and a hazard ratio of 0.35. A report on the FDA’s agreement and Multikine’s path forward is available on CEL-SCI’s website and at the following LINK.
CEL-SCI selected Ergomed, a clinical research organization (CRO) with a strong track record of fast enrollment and high-quality study delivery, as the CRO for its relatively small registration study. Ergomed has been a strategic partner and collaborator for over 10 years and was instrumental in successfully completing the Phase 3 study.
Data from an analysis of CEL-SCI’s Phase 3 study were shared at the International Drug Discovery Science & Technology (IDDST) 20th Annual Congress in a presentation titled "Neoadjuvant Immunotherapy for Head and Neck Cancer: Low Tumor PD-L1 Expression - IT-MATTERS – RCT". Highlights of the presentation include:
Multikine significantly increases overall survival in patients with low levels of tumor cell PD-L1 expression, a population in which approved checkpoint inhibitors, which inhibit PD-L1, are generally not successful
73% survival for Multikine vs 45% in the control arm at 5 years
Statistically significant log rank p = 0.0015
5-year risk of death reduced in half from 55% to 27%
Hazard ratio = 0.35 (95% CIs [0.19, 0.66], Wald p=0.0015)
The presentation may be accessed on CEL-SCI’s website at the following LINK
New data from the Phase 3 study were also presented at the European Society for Medical Oncology (ESMO) 2024 Congress in a poster titled "Prognostic significance of diagnostic staging in treatment naïve, resectable locally advanced primary oral cavity squamous cell carcinoma for neoadjuvant Leukocyte Interleukin Injection immunotherapy".
Positive regulatory decision letters were received from the European Medicines Agency (EMA) Paediatric Committee and the United Kingdom’s Healthcare Products Regulatory Agency (MHRA) granting Multikine a product-specific waiver for the treatment of head and neck cancer in a pediatric population up to 18 years of age. As a result, CEL-SCI is not required to evaluate Multikine in a pediatric population as part of its license and marketing clearance review in the UK and in the European Union (EU).
A patient population analysis of CEL-SCI’s Phase 3 study demonstrated well-balanced populations for both overall population and the selected population for the upcoming Registration Study (N0, PD-L1 low), providing greater confidence in the clinical results of the confirmatory Registration Study. This bias analysis, a standard process to ensure a trial’s findings are reliable, analyzed detailed data on parameters including patient age, sex, race, tumor locations, and staging, demonstrating a balance between the treatment and control arms. No bias favoring Multikine treated patients was found, lending confidence that Multikine is the reason for the large increase in patient survival.
CEL-SCI’s cGMP state-of-the-art dedicated manufacturing facility commissioning was completed, a significant milestone toward regulatory approval of Multikine.
Financial Results
During the fiscal year ended September 30, 2024, research and development expenses were $18.2 million, which decreased by approximately $4.3 million, or 19%, compared to the year ended September 30, 2023. General and administrative expenses in fiscal 2024 were $8.2 million, which decreased by approximately $0.8 million, or 9%, compared to the year ended September 30, 2023. Net loss decreased by $4.6 million to approximately $26.9 million for the twelve months ended September 30, 2024 from $32.2 million in fiscal 2023. The operating cash expenditures for the year were approximately $18.8 million. 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. 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), a true first-line cancer therapy, 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 which will enroll 212 patients. CEL-SCI will enroll 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 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.
