CEL-SCI Reports Fiscal 2024 Results: Set to Commence Confirmatory Study That Could Bring New Standard of Care to Head & Neck Cancer
CEL-SCI (CVM) reported fiscal year 2024 results and announced preparations for a 212-patient Confirmatory Registration Study for Multikine in head and neck cancer patients. The study, expected to complete enrollment by Q2 2026, focuses on PD-L1 low newly diagnosed patients.
Key financial results include a reduced net loss of $26.9 million compared to $32.2 million in 2023. Research and development expenses decreased 19% to $18.2 million, while general and administrative expenses fell 9% to $8.2 million.
The FDA has approved the confirmatory study based on previous Phase 3 results, where Multikine demonstrated 73% survival rate versus 45% in control patients at 5 years. The company plans to seek early approval based on pre-surgical response rates and aims to establish Multikine as the first new treatment in over 50 years for newly diagnosed locally advanced head and neck cancer.
CEL-SCI (CVM) ha riportato i risultati dell'anno fiscale 2024 e ha annunciato i preparativi per uno studio di registrazione confermativo su 212 pazienti per Multikine in pazienti affetti da cancro testa-collo. Lo studio, il cui completamento dell'arruolamento è previsto entro il secondo trimestre del 2026, si concentra su pazienti recentemente diagnosticati con basse espressioni di PD-L1.
I risultati finanziari chiave includono una perdita netta ridotta di 26,9 milioni di dollari rispetto ai 32,2 milioni di dollari del 2023. Le spese per ricerca e sviluppo sono diminuite del 19% a 18,2 milioni di dollari, mentre le spese generali e amministrative sono scese del 9% a 8,2 milioni di dollari.
La FDA ha approvato lo studio di conferma sulla base dei precedenti risultati della Fase 3, in cui Multikine ha dimostrato un tasso di sopravvivenza del 73% rispetto al 45% nei pazienti di controllo a 5 anni. L'azienda prevede di cercare un'approvazione anticipata basata sui tassi di risposta pre-operatoria e punta a stabilire Multikine come il primo nuovo trattamento dopo oltre 50 anni per il cancro testa-collo localmente avanzato recentemente diagnosticato.
CEL-SCI (CVM) informó sobre los resultados del año fiscal 2024 y anunció preparativos para un estudio de registro confirmatorio de 212 pacientes para Multikine en pacientes con cáncer de cabeza y cuello. Se espera que el estudio complete la inscripción para el segundo trimestre de 2026 y se centra en pacientes recién diagnosticados con bajo nivel de PD-L1.
Los resultados financieros clave incluyen una pérdida neta reducida de 26.9 millones de dólares en comparación con 32.2 millones de dólares en 2023. Los gastos de investigación y desarrollo disminuyeron un 19% a 18.2 millones de dólares, mientras que los gastos generales y administrativos cayeron un 9% a 8.2 millones de dólares.
La FDA ha aprobado el estudio confirmatorio basado en los resultados anteriores de la Fase 3, donde Multikine mostró una tasa de supervivencia del 73% frente al 45% en los pacientes de control a 5 años. La empresa planea buscar una aprobación anticipada basada en las tasas de respuesta prequirúrgicas y tiene como objetivo establecer a Multikine como el primer nuevo tratamiento en más de 50 años para el cáncer de cabeza y cuello localmente avanzado recientemente diagnosticado.
CEL-SCI (CVM)는 2024 회계연도 결과를 보고하고 목과 두경부 암 환자를 위한 Multikine의 212명 환자를 대상으로 하는 확인 등록 연구를 준비 중이라고 발표했습니다. 이 연구는 2026년 2분기까지 등록을 완료할 것으로 예상되며, PD-L1 수치가 낮은 신규 진단 환자에 초점을 맞추고 있습니다.
주요 재무 결과에는 2690만 달러의 감소된 순손실이 포함되며, 이는 2023년의 3220만 달러에 비해 감소한 수치입니다. 연구 및 개발 비용은 19% 감소하여 1820만 달러가었고, 일반 관리 비용은 9% 감소하여 820만 달러로 줄어들었습니다.
FDA는 이전 3상 결과를 바탕으로 확인 연구를 승인하였으며, 해당 연구에서 Multikine은 5년 동안 대조군 환자 45%에 비해 73%의 생존율을 입증했습니다. 회사는 수술 전 반응률을 바탕으로 조기 승인을 요청할 계획이며, 50년 이상 간 신규 진단된 국소 진행성 두경부 암에 대한 첫 번째 신규 치료제로서 Multikine을 확립하는 것을 목표로 하고 있습니다.
CEL-SCI (CVM) a annoncé les résultats de l'exercice fiscal 2024 et a annoncé les préparatifs d'une étude d'enregistrement confirmatoire impliquant 212 patients pour Multikine chez des patients atteints de cancer de la tête et du cou. L'étude, dont l'inscription devrait être finalisée d'ici le deuxième trimestre 2026, se concentre sur les patients nouvellement diagnostiqués avec un faible niveau de PD-L1.
Les résultats financiers clés incluent une perte nette réduite de 26,9 millions de dollars par rapport à 32,2 millions de dollars en 2023. Les dépenses de recherche et développement ont diminué de 19 % pour atteindre 18,2 millions de dollars, tandis que les dépenses générales et administratives ont baissé de 9 % pour atteindre 8,2 millions de dollars.
La FDA a approuvé l'étude de confirmation sur la base de résultats précédents de la phase 3, où Multikine a démontré un taux de survie de 73 % contre 45 % chez les patients témoins après 5 ans. L'entreprise prévoit de demander une approbation anticipée basée sur les taux de réponse préopératoire et vise à établir Multikine comme le premier nouveau traitement en plus de 50 ans pour les cancers de la tête et du cou localement avancés récemment diagnostiqués.
CEL-SCI (CVM) hat die Ergebnisse des Geschäftsjahres 2024 veröffentlicht und Vorbereitungen für eine Bestätigungsregistrierungsstudie mit 212 Patienten für Multikine bei Patienten mit Kopf-Hals-Krebs angekündigt. Die Studie soll im zweiten Quartal 2026 mit der Einschreibung fertig sein und konzentriert sich auf neu diagnostizierte Patienten mit niedrigem PD-L1.
Die wichtigsten finanziellen Ergebnisse umfassen einen reduzierten Nettoverlust von 26,9 Millionen Dollar im Vergleich zu 32,2 Millionen Dollar im Jahr 2023. Die Forschung- und Entwicklungsausgaben sanken um 19 % auf 18,2 Millionen Dollar, während die allgemeinen und verwaltenden Ausgaben um 9 % auf 8,2 Millionen Dollar fielen.
Die FDA hat die Bestätigungsstudie basierend auf früheren Ergebnissen der Phase 3 genehmigt, in denen Multikine eine Überlebensrate von 73 % gegenüber 45 % bei Kontrollpatienten nach 5 Jahren zeigte. Das Unternehmen plant, eine vorzeitige Genehmigung basierend auf präoperativen Ansprechraten zu beantragen und möchte Multikine als die erste neue Behandlung in über 50 Jahren für neu diagnostizierten lokal fortgeschrittenen Kopf-Hals-Krebs etablieren.
- FDA approval to conduct confirmatory Registration Study based on strong Phase 3 data
- Phase 3 showed 73% vs 45% 5-year survival rate in target population
- 19% decrease in R&D expenses to $18.2 million
- Net loss reduced by $4.6 million to $26.9 million
- Completion of cGMP manufacturing facility commissioning
- Going concern warning in auditor's opinion
- Operating cash expenditures of $18.8 million for the year
- Full enrollment not expected until Q2 2026
Insights
The fiscal 2024 results reveal both challenges and opportunities for CEL-SCI. The company's net loss of
However, the upcoming 212-patient confirmatory study represents a critical catalyst. With full enrollment expected by Q2 2026 and potential early approval pathway based on pre-surgical response rates, this could dramatically shift the company's trajectory. The previous Phase 3 data showing
The clinical data presents compelling evidence for Multikine's efficacy in a specific patient subset. The hazard ratio of 0.35 in PD-L1 low expressing tumors is remarkably strong, suggesting a
The strategic focus on patients with no lymph node involvement and low PD-L1 expression demonstrates sophisticated patient selection, potentially increasing the probability of regulatory success. The completion of manufacturing facility commissioning also removes a critical regulatory hurdle, though cash constraints could impact commercialization timeline.
The head and neck cancer market represents a substantial opportunity, with no new standard of care established in over 50 years. The patient selection strategy targeting PD-L1 low expressors is particularly shrewd, as it addresses a clear market gap where current immunotherapies underperform. The partnership with Ergomed for the registration study adds credibility and execution capability.
However, the current market cap of
- 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
“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:
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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 of73% vs a45% 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
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73% survival for Multikine vs45% in the control arm at 5 years - Statistically significant log rank p = 0.0015
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5-year risk of death reduced in half from
55% to27% -
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”.
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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
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
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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20250114061710/en/
Gavin de Windt
CEL-SCI Corporation
(703) 506-9460
Source: CEL-SCI Corporation
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