Journal of the American Medical Association (JAMA) Study Provides Further Evidence that CEL-SCI’s Multikine Can Treat Unmet Need in ~70% of Head and Neck Patients Based on PD-L1 Expression
CEL-SCI (NYSE: CVM) reports that a JAMA Oncology study published on March 6, 2025, supports Multikine's potential as a neoadjuvant treatment for head and neck cancer patients with low PD-L1 expression. The DEPEND study demonstrates that while checkpoint inhibitor nivolumab works best with high PD-L1 levels, it's less effective with low levels.
Multikine stands out as the only neoadjuvant oncology drug showing overall survival benefit in head and neck cancer patients with low PD-L1 expression, which represents approximately 70% of patients. In CEL-SCI's Phase 3 trial, the 5-year survival rate for the target patient population increased to 73% with Multikine treatment compared to 45% in the control group.
The company has received FDA approval to proceed with a confirmatory Registration Trial, focusing on previously untreated resectable stage 3 and 4 head and neck cancer patients with low PD-L1 tumor expression and no lymph node involvement.
CEL-SCI (NYSE: CVM) riporta che uno studio pubblicato su JAMA Oncology il 6 marzo 2025 supporta il potenziale di Multikine come trattamento neoadiuvante per i pazienti con cancro testa-collo e bassa espressione di PD-L1. Lo studio DEPEND dimostra che, mentre l'inibitore del checkpoint nivolumab è più efficace con alti livelli di PD-L1, risulta meno efficace con livelli bassi.
Multikine si distingue come l'unico farmaco oncologico neoadiuvante che mostra un beneficio in termini di sopravvivenza globale nei pazienti con cancro testa-collo e bassa espressione di PD-L1, che rappresenta circa il 70% dei pazienti. Nella fase 3 della sperimentazione di CEL-SCI, il tasso di sopravvivenza a 5 anni per la popolazione target è aumentato al 73% con il trattamento con Multikine rispetto al 45% nel gruppo di controllo.
L'azienda ha ricevuto l'approvazione dalla FDA per procedere con uno studio di registrazione confermativo, focalizzandosi su pazienti con cancro testa-collo resecabile di stadio 3 e 4, precedentemente non trattati, con bassa espressione di PD-L1 e senza coinvolgimento dei linfonodi.
CEL-SCI (NYSE: CVM) informa que un estudio publicado en JAMA Oncology el 6 de marzo de 2025 respalda el potencial de Multikine como tratamiento neoadyuvante para pacientes con cáncer de cabeza y cuello con baja expresión de PD-L1. El estudio DEPEND demuestra que, aunque el inhibidor de checkpoint nivolumab es más efectivo con altos niveles de PD-L1, es menos efectivo con niveles bajos.
Multikine se destaca como el único medicamento oncológico neoadyuvante que muestra un beneficio en la supervivencia global en pacientes con cáncer de cabeza y cuello y baja expresión de PD-L1, que representa aproximadamente el 70% de los pacientes. En el ensayo de fase 3 de CEL-SCI, la tasa de supervivencia a 5 años para la población objetivo aumentó al 73% con el tratamiento de Multikine en comparación con el 45% en el grupo de control.
La empresa ha recibido la aprobación de la FDA para proceder con un ensayo de registro confirmatorio, centrándose en pacientes con cáncer de cabeza y cuello resecable en estadio 3 y 4, previamente no tratados, con baja expresión de PD-L1 y sin involucramiento de ganglios linfáticos.
CEL-SCI (NYSE: CVM)는 2025년 3월 6일 JAMA Oncology에 발표된 연구가 낮은 PD-L1 발현을 가진 두경부암 환자에 대한 Multikine의 잠재력을 지지한다고 보고했습니다. DEPEND 연구는 체크포인트 억제제인 nivolumab이 높은 PD-L1 수치에서 가장 효과적이지만 낮은 수치에서는 덜 효과적이라는 것을 보여줍니다.
Multikine은 낮은 PD-L1 발현을 가진 두경부암 환자에서 전체 생존 이점을 보여주는 유일한 신 보조 항암제로, 이는 약 70%의 환자를 차지합니다. CEL-SCI의 3상 시험에서 목표 환자 집단의 5년 생존율은 Multikine 치료를 통해 73%로 증가했으며, 대조군의 45%와 비교됩니다.
회사는 낮은 PD-L1 종양 발현과 림프절 침범이 없는 이전에 치료받지 않은 절제 가능한 3기 및 4기 두경부암 환자에 대한 확인 등록 시험을 진행할 수 있도록 FDA 승인을 받았습니다.
CEL-SCI (NYSE: CVM) rapporte qu'une étude publiée dans JAMA Oncology le 6 mars 2025 soutient le potentiel de Multikine en tant que traitement néoadjuvant pour les patients atteints de cancer de la tête et du cou avec une faible expression de PD-L1. L'étude DEPEND démontre que, bien que l'inhibiteur de point de contrôle nivolumab soit plus efficace avec des niveaux élevés de PD-L1, il est moins efficace avec des niveaux bas.
Multikine se distingue comme le seul médicament oncologique néoadjuvant montrant un bénéfice en termes de survie globale chez les patients atteints de cancer de la tête et du cou avec une faible expression de PD-L1, représentant environ 70% des patients. Dans l'essai de phase 3 de CEL-SCI, le taux de survie à 5 ans pour la population cible a augmenté à 73% avec le traitement par Multikine par rapport à 45% dans le groupe témoin.
L'entreprise a reçu l'approbation de la FDA pour procéder à un essai de confirmation d'enregistrement, axé sur des patients atteints de cancer de la tête et du cou resectables de stade 3 et 4, précédemment non traités, avec une faible expression de PD-L1 et sans atteinte des ganglions lymphatiques.
CEL-SCI (NYSE: CVM) berichtet, dass eine am 6. März 2025 veröffentlichte Studie in JAMA Oncology das Potenzial von Multikine als neoadjuvante Behandlung für Patienten mit Kopf- und Halskrebs mit niedriger PD-L1-Expression unterstützt. Die DEPEND-Studie zeigt, dass der Checkpoint-Hemmer Nivolumab bei hohen PD-L1-Werten am besten wirkt, bei niedrigen Werten jedoch weniger effektiv ist.
Multikine hebt sich als das einzige neoadjuvante onkologische Medikament hervor, das einen Nutzen für das Überleben insgesamt bei Kopf- und Halskrebspatienten mit niedriger PD-L1-Expression zeigt, was etwa 70% der Patienten ausmacht. In der Phase-3-Studie von CEL-SCI stieg die 5-Jahres-Überlebensrate für die Zielpatientenpopulation auf 73% mit Multikine-Behandlung im Vergleich zu 45% in der Kontrollgruppe.
Das Unternehmen hat von der FDA die Genehmigung erhalten, mit einer bestätigenden Registrierungstudie fortzufahren, die sich auf zuvor unbehandelte resezierbare Patienten mit Stadium 3 und 4 von Kopf- und Halskrebs mit niedriger PD-L1-Tumorexpression und ohne Lymphknotenbefall konzentriert.
- Significant survival benefit: 73% 5-year survival rate vs 45% in control group
- FDA approval received for confirmatory Registration Trial
- Addresses unmet need in 70% of head and neck cancer patients
- Only neoadjuvant oncology drug showing survival benefit in low PD-L1 expression patients
- Independent JAMA study validates treatment approach
- Still requires completion of confirmatory Registration Trial before potential approval
- Treatment to specific subset of head and neck cancer patients
Insights
The JAMA Oncology publication provides crucial validation for CEL-SCI's Multikine in treating head and neck cancer patients with low PD-L1 expression - a population representing approximately
This third-party DEPEND study reinforces a critical biomarker-based treatment paradigm: checkpoint inhibitors like nivolumab work effectively with high PD-L1 expression but underperform with low expression. Multikine's mechanism appears complementary, showing greatest benefit precisely where checkpoint inhibitors struggle.
The survival data is particularly compelling - Multikine-treated patients with low PD-L1 expression and no lymph node involvement achieved
The FDA's approval for a confirmatory Registration Trial signals regulatory acknowledgment of Multikine's potential pathway to approval. This trial design strategically focuses on the precise patient subset showing strongest benefit, optimizing chances for success.
What's most significant is Multikine's positioning as a neoadjuvant therapy - administered before standard treatments. This approach, combined with its apparent efficacy in low PD-L1 patients, creates a unique therapeutic niche addressing a substantial unmet need largely overlooked by current immunotherapy giants.
The JAMA Oncology study represents a significant external validation for CEL-SCI's Multikine, establishing its unique position in addressing a major treatment gap for head and neck cancer patients. With checkpoint inhibitors like nivolumab and pembrolizumab showing efficacy in low PD-L1 expression patients, Multikine's demonstrated survival benefit in this
The FDA's approval of the confirmatory Registration Trial design represents a critical regulatory milestone. Importantly, the trial will target the precise patient population where Phase 3 results showed the most dramatic benefit - a strategic decision that enhances probability of success while addressing an unmet medical need.
The survival data is compelling - the
For investors, this third-party validation from a prestigious medical publication coupled with FDA agreement on the registration pathway significantly de-risks the Multikine program. With CEL-SCI's current market cap of just
The DEPEND study demonstrates that checkpoint inhibitor nivolumab (Opdivo®) works best with high levels of PD-L1 biomarker, but does not work well with low levels of PD-L1
Multikine* is uniquely positioned in the field as the only oncology drug used as a neoadjuvant that has demonstrated overall survival benefit in head and neck cancer patients whose tumors express low levels of PD-L1
Multikine aims to address a major treatment gap for head and neck cancer patients where about
“With these latest findings published in JAMA, industry is taking notice. We believe there is growing interest in Multikine as an advanced clinical stage asset that can prolong life for about
The DEPEND study evaluated nivolumab as a neoadjuvant immunotherapy in human papilloma virus (HPV)–negative locoregionally advanced head and neck cancer. Nivolumab is already an FDA approved treatment for recurrent metastatic squamous cell carcinoma of the head and neck. The authors of the JAMA publication stated: “Taken together the DEPEND results further support the importance of PD-L1 expression as a predictive biomarker with immunotherapy trials in curative intent setting and may be an important selection criterion in subsequent trials”.
The findings of the DEPEND study are very important and timely. They are similar to the findings in CEL-SCI’s Phase 3 study, namely that PD-L1 inhibitors such as Opdivo work best in patients who have high levels of PD-L1, but do not work well in patients with low or zero levels of PD-L1. Conversely, Multikine, which has a very different mechanism of action, worked best in patients who have low to zero levels of PD-L1. This underscores the potential of Multikine to address a critical unmet need amongst newly diagnosed head and neck cancer patients whose tumors have low PD-L1 expression, representing about
The DEPEND Phase 2 data also confirm the independent findings reviewed by the FDA’s recent Oncologic Advisory Committee meeting (September 2024) on the use of checkpoint inhibitors including blockbuster drugs nivolumab and pembrolizumab, which appear to not work well in patients with low PD-L1 expression. To CEL-SCI’s knowledge, Multikine is the only oncology drug with solid data showing overall survival benefit when used as a neoadjuvant treatment in newly diagnosed locally advanced head and neck cancer patients whose tumors have low PD-L1 expression.
Multikine is an investigational cancer immunotherapy (treatment) given to newly diagnosed head and neck cancer patients before the primary standard of care treatment. CEL-SCI’s confirmatory Registration Trial, which has received the FDA’s go-ahead, will enroll patients with previously untreated resectable disease, stage 3 and 4 head and neck cancer who have low PD-L1 tumor expression and no lymph node involvement. During CEL-SCI’s completed Phase 3 clinical trial, the 5-year survival rate of this target patient population increased to
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/20250314970798/en/
COMPANY CONTACT:
Gavin de Windt
CEL-SCI Corporation
(703) 506-9460
Source: CEL-SCI Corporation
FAQ
What were the survival rates in CVM's Phase 3 trial for Multikine in head and neck cancer?
What percentage of head and neck cancer patients could potentially benefit from CVM's Multikine treatment?
How does CVM's Multikine differ from nivolumab in treating head and neck cancer?
What is the target patient population for CVM's upcoming Multikine Registration Trial?
When was the JAMA study supporting CVM's Multikine published?
