CEL-SCI to Initiate Multikine Confirmatory Phase 3 Cancer Trial
CEL-SCI (NYSE American: CVM) announced the launch of a 212-patient Confirmatory Registration Study for Multikine in head and neck cancer patients. The study aims to confirm results from a previous Phase 3 trial where 5-year survival rates increased to 73% in treated patients versus 45% in control groups.
The study targets previously untreated resectable stage 3 and 4 head and neck cancer patients with no lymph node involvement and low PD-L1 tumor expression. Key highlights include: full enrollment expected by Q2 2026, plans for early approval based on tumor responses, and FDA concurrence with the study design. The final clinical protocol was submitted to FDA in December 2024.
The company reports over 95% chance of success for this Registration Study, as it focuses on the target population that showed the most benefit in the prior Phase 3 study, with Multikine extending median overall survival by nearly 4 years.
CEL-SCI (NYSE American: CVM) ha annunciato il lancio di uno studio di registrazione confermativo su 212 pazienti per Multikine nei pazienti con cancro testa-collo. Lo studio mira a confermare i risultati di un precedente trial di Fase 3 in cui le percentuali di sopravvivenza a 5 anni sono aumentate al 73% nei pazienti trattati rispetto al 45% nei gruppi di controllo.
Lo studio si rivolge a pazienti con cancro testa-collo di stadio 3 e 4, riscontrabili e precedentemente non trattati, senza coinvolgimento dei linfonodi e con bassa espressione del tumore PD-L1. I punti salienti includono: completamento dell’arruolamento previsto entro il secondo trimestre del 2026, piani per un’approvazione anticipata basata sulle risposte tumorali e consenso della FDA sul disegno dello studio. Il protocollo clinico finale è stato inviato alla FDA nel dicembre 2024.
L'azienda riporta oltre il 95% di probabilità di successo per questo studio di registrazione, poiché si concentra sulla popolazione target che ha mostrato il maggior beneficio nello studio di Fase 3 precedente, con Multikine che ha esteso la sopravvivenza globale mediana di quasi 4 anni.
CEL-SCI (NYSE American: CVM) anunció el lanzamiento de un estudio de registro confirmatorio de 212 pacientes para Multikine en pacientes con cáncer de cabeza y cuello. El estudio tiene como objetivo confirmar los resultados de un ensayo de Fase 3 anterior donde las tasas de supervivencia a 5 años aumentaron al 73% en los pacientes tratados en comparación con el 45% en los grupos de control.
El estudio se dirige a pacientes con cáncer de cabeza y cuello en estadio 3 y 4, resecables y previamente no tratados, sin afectación de ganglios linfáticos y con baja expresión tumoral de PD-L1. Los aspectos destacados incluyen: se espera que el reclutamiento completo se complete para el segundo trimestre de 2026, planes para una aprobación anticipada basada en las respuestas tumorales y la conformidad de la FDA con el diseño del estudio. El protocolo clínico final fue presentado a la FDA en diciembre de 2024.
La empresa informa sobre más del 95% de probabilidad de éxito para este estudio de registro, ya que se centra en la población objetivo que mostró el mayor beneficio en el estudio de Fase 3 anterior, con Multikine extendiendo la supervivencia global media en casi 4 años.
CEL-SCI (NYSE American: CVM)는 두경부암 환자를 위한 Multikine의 212명 환자를 대상으로 한 확인 등록 연구를 시작한다고 발표했습니다. 이 연구는 치료받은 환자의 5년 생존율이 45%인 대조군에 비해 73%로 증가한 이전 3상 시험의 결과를 확인하는 것을 목표로 합니다.
이 연구는 림프절 침범이 없고 PD-L1 종양 발현이 낮은 이전에 치료받지 않은 절제 가능한 3기 및 4기 두경부암 환자를 대상으로 하고 있습니다. 주요 내용으로는: 2026년 2분기까지의 완전한 등록이 예상되며, 종양 반응에 기반한 조기 승인을 계획하고 있으며, FDA와의 연구 설계에 대한 합의가 포함됩니다. 최종 임상 프로토콜은 2024년 12월에 FDA에 제출되었습니다.
회사는 이 등록 연구의 성공 확률이 95% 이상이라고 보고하며, 이는 이전 3상 연구에서 가장 큰 혜택을 보인 대상 집단에 초점을 맞추고 있으며, Multikine이 중앙값 전반적인 생존 기간을 거의 4년 연장했음을 나타냅니다.
CEL-SCI (NYSE American: CVM) a annoncé le lancement d'une étude de registration confirmatoire de 212 patients pour Multikine chez des patients atteints de cancer de la tête et du cou. L'étude vise à confirmer les résultats d'un précédent essai de Phase 3 où les taux de survie à 5 ans ont augmenté à 73 % chez les patients traités contre 45 % dans les groupes de contrôle.
L'étude cible des patients atteints de cancer de la tête et du cou, classés en stade 3 et 4, résécables et précédemment non traités, sans atteinte des ganglions lymphatiques et avec une faible expression tumorale de PD-L1. Les points clés incluent : un recrutement complet attendu d'ici le deuxième trimestre 2026, des plans pour une approbation anticipée basée sur les réponses tumorales et un accord de la FDA sur le design de l'étude. Le protocole clinique final a été soumis à la FDA en décembre 2024.
L'entreprise rapporte plus de 95 % de chances de succès pour cette étude de registration, car elle se concentre sur la population cible qui a montré le plus de bénéfice dans l'étude de Phase 3 précédente, avec Multikine prolongeant la survie globale médiane de près de 4 ans.
CEL-SCI (NYSE American: CVM) hat den Start einer Bestätigungsregistrierungsstudie mit 212 Patienten für Multikine bei Patienten mit Kopf- und Halskrebs bekannt gegeben. Die Studie zielt darauf ab, die Ergebnisse einer vorherigen Phase-3-Studie zu bestätigen, bei der die 5-Jahres-Überlebensraten bei behandelten Patienten auf 73% im Vergleich zu 45% in den Kontrollgruppen anstiegen.
Die Studie richtet sich an zuvor unbehandelte, resektable Patienten im Stadium 3 und 4 mit Kopf- und Halskrebs, ohne Lymphknotenbeteiligung und mit niedriger PD-L1-Tumorexpression. Zu den wichtigsten Punkten gehören: die vollständige Rekrutierung wird bis zum 2. Quartal 2026 erwartet, Pläne für eine vorzeitige Genehmigung basierend auf Tumorreaktionen und die Zustimmung der FDA zum Studiendesign. Das endgültige klinische Protokoll wurde im Dezember 2024 bei der FDA eingereicht.
Das Unternehmen berichtet von einer über 95%igen Erfolgswahrscheinlichkeit für diese Registrierungsstudie, da der Fokus auf der Zielpopulation liegt, die im vorherigen Phase-3-Studie den größten Nutzen zeigte, wobei Multikine die mediane Gesamtüberlebenszeit um fast 4 Jahre verlängerte.
- Previous Phase 3 trial showed significant survival improvement (73% vs 45% in control group)
- FDA has concurred with the study design
- Median overall survival extended by nearly 4 years in prior Phase 3 study
- 95% chance of success predicted for the Registration Study
- No safety signals identified in previous trials
- Full enrollment not expected until Q2 2026
- to specific patient subset (no lymph node involvement and low PD-L1 expression)
- Requires 3-week treatment period before surgery
Insights
The announcement of CEL-SCI's confirmatory Phase 3 trial for Multikine represents a strategically designed path to market with several notable advantages. The trial's focused approach on a biomarker-selected population - specifically patients with low PD-L1 expression and no nodal involvement - demonstrates sophisticated trial design that could significantly increase the likelihood of success.
The previously observed hazard ratio of 0.35 (with upper 95% CI of 0.66) in the target population is particularly compelling. For context, this level of survival benefit is rarely seen in oncology trials, where hazard ratios of 0.7-0.8 are often considered clinically meaningful. The nearly 4-year median survival improvement in the previous Phase 3 study represents a potential paradigm shift in head and neck cancer treatment.
The 212-patient trial size is notably efficient, reflecting confidence in the treatment effect and potentially reducing time and costs to market. The company's strategy to seek early approval based on tumor responses is particularly astute, as it could accelerate the path to commercialization while the longer-term survival data matures.
Three key aspects warrant investor attention:
- The pre-surgical immunotherapy approach represents a novel treatment paradigm that could expand beyond head and neck cancer if successful
- The biomarker-driven patient selection strategy aligns with current trends in precision oncology and could enhance reimbursement prospects
- The rapid enrollment timeline (by Q2 2026) appears achievable given the high unmet need and positive previous results
However, investors should note that while the 95% success probability cited by the biostatistician is encouraging, such high confidence levels should be viewed cautiously in the context of historical oncology trial success rates. The key risk mitigation factor here is the precise patient selection based on previous positive data.
- Confirmatory Phase 3 Trial Designed to Support Commercialization of Multikine for Head and Neck Cancer Treatment
- Full enrollment expected by Q2 2026 with plans to seek early approval at that time based on early tumor responses—Potential to set a new standard of care
Multikine is given via injection for 3 weeks after diagnosis and before surgery. The pre-surgical tumor responses shown in the chart demonstrate that patients whose tumors shrink in response to Multikine are more likely to have longer survival. (Graphic: Business Wire)
This new Registration Study targets the population of previously untreated resectable stage 3 and 4 head and neck cancer patients who had no lymph node involvement and low PD-L1 tumor expression. During the completed Phase 3 clinical trial, the 5-year survival rate of the target patient population increased to
Key aspects of the new Confirmatory Registration Study are as follows:
- Enlisting clinical sites and investigators in numerous countries across 3 continents
- Full enrollment expected by Q2 2026 with plans to seek early approval at that time based on early tumor responses—Potential to set a new standard of care
- FDA concurred with the overall design of the Registration Study in meetings last year
- Final clinical protocol submitted to FDA in December 2024
- Multikine extended median overall survival by nearly 4 years in prior Phase 3 study
- A new model for healthcare—Immunotherapy given before surgery to boost the immune system and make the first cancer treatment more successful - may set the first new standard of care in more than half a century
“Now that the clinical protocol for our final marketing Registration Study has been submitted, we are proceeding to sign up investigators and open clinical sites. We are receiving a very positive response and high levels of interest from head and neck cancer physicians who see the abundance of data on Multikine and want to deliver this option to their patients,” stated CEL-SCI CEO Geert Kersten. “We believe that this small 212-patient trial will enroll relatively quickly precisely because Multikine has proven what it can do in terms of safety and efficacy in this specific target patient population which has not had a new option for treatment in decades.”
“We are very confident in this Registration Study’s chance of success since we already know that this type of patient showed an almost 4 year increase in median survival in our last study. The many studies we have done have helped us figure out who benefits from the drug and who does not. Early tumor responses measured after treatment with Multikine and prior to surgery have been shown to be correlated with 5-year overall survival outcomes. We plan to seek accelerated and/or conditional marketing approval based on this finding. Data regarding early tumor responses following Multikine treatment should become available shortly after the last patient has been treated, expected by the second quarter of 2026,” Kersten concluded.
CEL-SCI has been advised by a biostatistician with expertise in the design and analysis of oncology studies that this final Confirmatory Registration Study has an over
There were no safety signals identified, Multikine administration did not interfere with disease directed treatment/therapy, and Multikine did not add to the treatment burden imparted by the standard of care.
Following the
“A Phase III, Open Label, Randomized, Controlled, Multi-Center Study of the Effects of Neoadjuvant Leukocyte Interleukin, Injection (LI) Plus Standard of Care Versus Standard of Care Only in Treatment Naïve Adults With Resectable Locally Advanced Primary Squamous Cell Carcinoma of the Head and Neck (Oral Cavity) Who Present With No Nodal Involvement and Low Tumor Programmed Death Ligand 1 Expression (Defined as Tumor Proportion Score <10)”
Ergomed, the clinical research organization (CRO) for the Registration Study, is currently identifying clinical sites and investigators. Sites are initially expected to open in four countries across three continents, with the first clinical site expected to initiate in
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 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. The study will enroll 212 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/20250220081468/en/
Gavin de Windt
(703) 506-9460
CEL-SCI Corporation
Source: CEL-SCI Corporation
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