Y-mAbs Announces Publication of Phase 2 Interim Results in Nature Communications
Y-mAbs Therapeutics (YMAB) has published interim results from a Phase 2 clinical trial of naxitamab with GM-CSF in Nature Communications. The trial evaluated patients with relapsed/refractory high-risk neuroblastoma and residual disease in bone/bone marrow.
Key findings include:
- Overall response rate (ORR) of 50% (95% CI: 36-64%, N=52)
- Complete response in 38% and partial response in 12% of patients
- Bone compartment response of 58% (29/50)
- Bone marrow compartment response of 74% (17/23)
- One-year overall survival of 93% and progression-free survival of 35%
The treatment showed manageable safety with primarily infusion-related adverse events (90%), including hypotension and bronchospasm. The study met its primary endpoint with the ORR's lower confidence interval exceeding 20%.
Y-mAbs Therapeutics (YMAB) ha pubblicato risultati intermedi di uno studio clinico di Fase 2 su naxitamab con GM-CSF in Nature Communications. Lo studio ha valutato pazienti con neuroblastoma ad alto rischio in recidiva/resistente e malattia residua nelle ossa/nel midollo osseo.
I risultati chiave includono:
- Un tasso di risposta globale (ORR) del 50% (95% CI: 36-64%, N=52)
- Risposta completa nel 38% e risposta parziale nel 12% dei pazienti
- Risposta nel compartimento osseo del 58% (29/50)
- Risposta nel compartimento del midollo osseo del 74% (17/23)
- Sopravvivenza globale a un anno del 93% e sopravvivenza libera da progressione del 35%
Il trattamento ha mostrato una sicurezza gestibile con eventi avversi principalmente correlati all'infusione (90%), inclusi ipotensione e broncospasmo. Lo studio ha raggiunto il suo obiettivo primario con l'intervallo di confidenza inferiore dell'ORR che supera il 20%.
Y-mAbs Therapeutics (YMAB) ha publicado resultados intermedios de un ensayo clínico de Fase 2 de naxitamab con GM-CSF en Nature Communications. El ensayo evaluó a pacientes con neuroblastoma de alto riesgo en recaída/refractario y enfermedad residual en huesos/médula ósea.
Los hallazgos clave incluyen:
- Tasa de respuesta global (ORR) del 50% (95% CI: 36-64%, N=52)
- Respuesta completa en el 38% y respuesta parcial en el 12% de los pacientes
- Respuesta en el compartimento óseo del 58% (29/50)
- Respuesta en el compartimento de médula ósea del 74% (17/23)
- Sobrevivencia global a un año del 93% y supervivencia libre de progresión del 35%
El tratamiento mostró una seguridad manejable con eventos adversos principalmente relacionados con la infusión (90%), incluidos hipotensión y broncoespasmo. El estudio cumplió su objetivo primario con el intervalo de confianza inferior del ORR que supera el 20%.
Y-mAbs Therapeutics (YMAB)는 Nature Communications에 GM-CSF와 함께한 naxitamab의 2상 임상 시험 중간 결과를 발표했습니다. 이 시험은 재발/내성 고위험 신경모세포종과 뼈/골수에 잔여 질환이 있는 환자를 평가했습니다.
주요 발견 사항은 다음과 같습니다:
- 전체 반응률 (ORR) 50% (95% CI: 36-64%, N=52)
- 환자의 38%에서 완전 반응, 12%에서 부분 반응
- 뼈 부위 반응률 58% (29/50)
- 골수 부위 반응률 74% (17/23)
- 1년 전체 생존율 93% 및 무진행 생존율 35%
치료는 주로 주입 관련 부작용(90%)이 포함된 관리 가능한 안전성을 보여주었으며, 저혈압 및 기관지 경련이 포함됩니다. 연구는 ORR의 하한 신뢰 구간이 20%를 초과하여 주요 목표를 달성했습니다.
Y-mAbs Therapeutics (YMAB) a publié des résultats intermédiaires d'un essai clinique de Phase 2 sur le naxitamab avec GM-CSF dans Nature Communications. L'essai a évalué des patients atteints de neuroblastome à haut risque en rechute/résistant et de maladie résiduelle dans les os/la moelle osseuse.
Les résultats clés comprennent:
- Taux de réponse global (ORR) de 50% (95% CI: 36-64%, N=52)
- Réponse complète chez 38% et réponse partielle chez 12% des patients
- Taux de réponse dans le compartiment osseux de 58% (29/50)
- Taux de réponse dans le compartiment de la moelle osseuse de 74% (17/23)
- Sursistance globale à un an de 93% et survie sans progression de 35%
Le traitement a montré une sécurité gérable avec principalement des événements indésirables liés à l'infusion (90%), y compris l'hypotension et le bronchospasme. L'étude a atteint son objectif principal avec l'intervalle de confiance inférieur de l'ORR dépassant 20%.
Y-mAbs Therapeutics (YMAB) hat Zwischenberichte aus einer Phase-2-Studie zu naxitamab mit GM-CSF in Nature Communications veröffentlicht. Die Studie bewertete Patienten mit rezidivierendem/refraktärem hochriskantem Neuroblastom und Restkrankheit in Knochen/Knochenmark.
Wichtige Ergebnisse umfassen:
- Gesamtansprechrate (ORR) von 50% (95% CI: 36-64%, N=52)
- Vollständige Ansprechrate bei 38% und partielle Ansprechrate bei 12% der Patienten
- Knochenkompartimentsansprechrate von 58% (29/50)
- Knochenmarkkompartimentsansprechrate von 74% (17/23)
- Eins-Jahres-Gesamtüberlebensrate von 93% und progressionsfreies Überleben von 35%
Die Behandlung zeigte eine handhabbare Sicherheit mit hauptsächlich infusionsbedingten unerwünschten Ereignissen (90%), einschließlich Hypotonie und Bronchospasmus. Die Studie erreichte ihr primäres Ziel, da das untere Konfidenzintervall der ORR 20% überschritt.
- 50% overall response rate in Phase 2 trial
- 93% one-year overall survival rate
- 74% bone marrow compartment response rate
- 31% response rate in patients previously treated with anti-GD2 antibodies
- Met primary endpoint threshold
- 35% one-year progression-free survival rate
- High rate of infusion-related adverse events (90%)
- Severe hypotension observed (58% Grade 3, 3 Grade 4 cases)
Insights
Y-mAbs' publication in Nature Communications delivers compelling Phase 2 data for naxitamab in treating relapsed/refractory high-risk neuroblastoma, representing a significant clinical milestone. The
The efficacy signals are particularly meaningful given this challenging patient population. The
The bone compartment response rate of
Safety data reveals manageable adverse events primarily consisting of infusion-related reactions like hypotension and bronchospasm, along with expected anti-GD2 class effect pain that generally resolved promptly after infusion. This safety profile appears consistent with mechanism of action without unexpected signals that would limit clinical utility.
The Nature Communications publication significantly strengthens Y-mAbs' scientific positioning for DANYELZA® (naxitamab-gqgk) in the competitive oncology landscape. High-tier journal validation provides important third-party validation that could accelerate clinical adoption and potentially strengthen reimbursement negotiations.
The study demonstrates meaningful efficacy in patients with treatment options, addressing an unmet medical need in pediatric oncology. The substantial
Most notably, the
The single-arm trial design focusing specifically on bone/bone marrow disease represents a strategically targeted approach to product differentiation. By demonstrating efficacy in these specific disease compartments, Y-mAbs establishes a focused clinical niche for DANYELZA® rather than competing broadly against all neuroblastoma treatments. This targeted approach, combined with the robust safety profile described, should support continued commercial momentum for this key product in Y-mAbs' portfolio.
This Nature Communications publication represents a meaningful clinical and commercial catalyst for Y-mAbs' lead product DANYELZA® (naxitamab-gqgk). The robust
Particularly noteworthy is the
The publication strategically focuses on DANYELZA's efficacy in bone and bone marrow compartments—highlighting response rates of
The single-arm trial design with a predefined efficacy threshold (
This data publication in a high-impact journal enhances DANYELZA's scientific credibility and may accelerate its integration into treatment protocols, supporting ongoing commercialization efforts for this key revenue driver in Y-mAbs' portfolio.
Naxitamab demonstrated clinically meaningful efficacy with manageable safety in patients with relapsed/refractory high-risk neuroblastoma and residual disease in bone/bone marrow
NEW YORK, March 03, 2025 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel radioimmunotherapy and antibody-based therapeutic products for the treatment of cancer, today announced the publication of interim data from a Phase 2 clinical trial evaluating naxitamab with granulocyte-macrophage colony-stimulating factor (GM-CSF) in patients with relapsed/refractory high-risk neuroblastoma in the journal Nature Communications.
The article, titled “The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial,” details the results of a single-arm, global Phase 2 trial (Trial 201, NCT03363373) of patients with relapsed/refractory high-risk neuroblastoma and residual disease in the bone/bone marrow who received naxitamab on days 1, 3, and 5 (3 mg/kg/day) with GM-CSF (days -4 to 5) every 4 weeks, until a complete response (CR) or partial response (PR) was achieved, followed by 5 additional cycles every 4 weeks. Overall, naxitamab demonstrated statistically significant efficacy with a manageable safety profile.
“In this trial, naxitamab showed clinically meaningful outcomes in a well-defined patient population with an urgent need for efficacious treatment options,” said Dr. Jaume Mora, Pediatric Cancer Center Barcelona, Hospital Sant Joan de Déu, Barcelona, Spain and lead author. “The study provides robust evidence for targeting residual disease in the bone and bone marrow, a common niche for chemoresistant cells in patients with primary refractory or relapsed disease.”
The primary endpoint in the prespecified interim analysis was overall response (per 2017 International Neuroblastoma Response Criteria). Overall response rate (ORR) was
In the safety population (N=74), treatment-related adverse events (AEs) were primarily infusion-related (
“We are pleased to have worked with the investigators and are grateful to our patients and caregivers for participating in this global trial, specifically designed to evaluate responses to a single-agent anti-GD2 monoclonal antibody (with GM-CSF) in patients with residual disease in bone and bone marrow,” said Norman LaFrance, MD, Chief Development Officer at Y-mAbs. “Results from the study support naxitamab as an important treatment option for patients with relapsed and refractory high-risk neuroblastoma and their caregivers.”
Researchers at Memorial Sloan Kettering Cancer Center (“MSK”) developed DANYELZA® (naxitamab-gqgk), which is exclusively licensed by MSK to Y-mAbs. MSK has institutional financial interests in the compound and Y-mAbs.
About Y-mAbs
Y-mAbs is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, radioimmunotherapy and antibody-based therapeutic cancer products. The Company’s technologies include its investigational Self-Assembly DisAssembly (“SADA”) Pretargeted Radioimmunotherapy Platform (“PRIT”) and bispecific antibodies generated using the Y-BiClone platform. The Company’s broad and advanced product pipeline includes the anti-GD2 therapy DANYELZA® (naxitamab-gqgk), the first FDA-approved treatment for patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow after a partial response, minor response, or stable disease to prior therapy.
Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements include, but are not limited to, statements about our business model, including financial outlook for 2024 and beyond.Words such as ‘‘anticipate,’’ ‘‘believe,’’ “contemplate,” ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ “hope,” ‘‘intend,’’ ‘‘may,’’ ‘‘might,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘should,’’ ‘‘target,’’ “will,” ‘‘would’,’ “guidance,” “goal,” “objective,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company’s business is subject to risks and uncertainties affecting the Company including those described in the “Risk Factors” section included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, and the Company’s Quarterly Report on Form 10-Q for the quarterly periods ended March 31, 2024, and September 30, 2024, and future filings and reports by the Company. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company undertakes no obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
SADA®, SADA PRIT™, DANYELZA® and Y-mAbs® are registered trademarks of Y-mAbs Therapeutics, Inc.
Investor Contact:
Courtney Dugan
VP, Head of Investor Relations
cdu@ymabs.com
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