Y-mAbs Announces New Interim Analysis of Phase 2 Data for Naxitamab at 2024 ASCO Annual Meeting
Y-mAbs Therapeutics announced interim Phase 2 data for naxitamab at the 2024 ASCO Annual Meeting. The data from Trial 201 show a 50% overall response rate (ORR) in patients with relapsed/refractory high-risk neuroblastoma. Continued naxitamab treatment led to further disease reduction, with 25% achieving complete or partial response after three or more cycles.
Adverse events (AEs) were mostly mild to moderate, with 81% being Grade 1 or 2. Grade ≥3 AEs included hypotension (60%), pain (58%), and others. Notably, the frequency of severe pain and hypotension decreased over treatment cycles.
An independent study from Sant Joan de Déu Barcelona presented data on naxitamab combined with alternative chemotherapies, showing manageable safety profiles and better responses with prior chemo-immunotherapy sensitivity.
- Naxitamab achieved a 50% overall response rate (ORR) in Trial 201.
- 25% of patients reached complete or partial response after three or more cycles of naxitamab.
- Continued naxitamab treatment showed improved patient outcomes.
- Frequency of severe adverse events decreased over time.
- Independent study showed manageable safety profiles for naxitamab combined with alternative chemotherapies.
- Better responses were observed in patients with prior sensitivity to chemo-immunotherapy.
- 60% of patients experienced Grade 3 or higher hypotension.
- 58% of patients reported Grade 3 or higher pain.
- 19% of patients experienced Grade 3 or higher urticaria.
- 18% of patients had Grade 3 or higher bronchospasm.
- 16% of patients reported Grade 3 or higher abdominal pain.
Insights
The interim results of the Phase 2 trial for naxitamab in combination with GM-CSF show promising results for patients with relapsed or refractory high-risk neuroblastoma. A 50% overall response rate (ORR) in a cohort of 52 patients, where nearly a quarter achieved their first complete or partial response after extended cycles, is noteworthy. This suggests the potential for sustained treatment protocols to benefit patients who do not respond initially. While adverse events (AEs) were common, the majority were Grade 1 or 2, pointing to an acceptable safety profile. The gradual reduction of severe pain and hypotension across treatment cycles indicates an improving tolerance over time.
From a clinical perspective, these findings could support extended naxitamab therapy in this patient population. The additional data from independent studies further corroborate the potential flexibility and efficacy of naxitamab in varied chemo-immunotherapy settings. This is especially relevant for patients who are refractory to initial treatments.
Overall, these results provide a compelling argument for the continued development and application of naxitamab-based therapies in high-risk neuroblastoma cases.
The data presented at the ASCO meeting highlights significant advancements in the treatment of high-risk neuroblastoma. Naxitamab's 50% ORR and its ability to induce responses even after multiple treatment cycles indicate a robust therapeutic potential. The study's focus on adverse event patterns provides valuable insights into the drug's safety profile, essential for long-term treatment considerations. Importantly, the reduction in the frequency of severe adverse events like pain and hypotension over time suggests that patients may better tolerate the drug with continued use.
The independent retrospective analysis adds another layer of credibility, showing that naxitamab can be effectively combined with different chemotherapeutic agents, not just the standard irinotecan/temozolomide regimen. This flexibility in treatment combinations could make naxitamab a versatile option in the oncological arsenal, potentially improving outcomes for a broader patient population.
These findings are particularly significant as they not only confirm naxitamab's efficacy but also outline its practical management in clinical settings, paving the way for its broader acceptance and use.
For investors, the interim Phase 2 trial results for naxitamab present a strong indication of Y-mAbs Therapeutics' potential market impact. A 50% ORR in a challenging patient population underscores the drug's efficacy, enhancing its commercial viability. The data on adverse event management is equally critical, as it addresses potential concerns about long-term use, making the therapy more attractive for both clinicians and patients.
Given the drug’s performance, Y-mAbs could see increased investor confidence and potentially higher stock valuations. The positive clinical data may also facilitate smoother regulatory processes, expediting market approval and reducing time to market. Additionally, the independent retrospective analysis supports broader application possibilities, which can significantly expand the drug's market reach and revenue streams.
However, investors should remain cautious about factors such as market competition and potential delays in regulatory approval. While the results are promising, bringing a new therapy to market involves navigating complex regulatory and commercial landscapes.
NEW YORK, June 01, 2024 (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 new interim data from the Phase 2 Trial 201 of naxitamab in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”) in patients with relapsed or refractory high-risk neuroblastoma with residual disease limited to bone and/or bone marrow. The results are summarized in poster presentations scheduled to be presented today, June 1, 2024, at the 2024 American Society of Clinical Oncology (“ASCO”) Annual Meeting in Chicago, IL.
Improved patient outcomes with extended naxitamab therapy: A poster titled “Patterns of improvement following initial response in patients treated with naxitamab for relapsed/refractory high-risk neuroblastoma” (poster #10033) demonstrates that continued treatment with naxitamab can further reduce disease following the initial response to naxitamab therapy. The Trial 201 prespecified interim analysis achieved a
Evolution of adverse events over the course of treatment: A poster titled “Naxitamab-related adverse events within and across treatment cycles in patients with relapsed/refractory high-risk neuroblastoma” (poster #10032) demonstrates the frequency and patterns of adverse events (“AEs”) based on the interim analysis of Trial 201. In this analysis,
“We are encouraged to see that some patients who did not demonstrate an objective response after initial assessment went on to achieve a complete or partial response after continued naxitamab treatment. In addition, these data provide a practical understanding of the temporal patterns of treatment-related adverse events,” said Vignesh Rajah, MBBS, DCH, MRCP (UK), Chief Medical Officer.
In addition, Dr. Jaume Mora and researchers from the Sant Joan de Déu Barcelona Children’s Hospital in Spain will present results from an independent retrospective analysis of patients treated with naxitamab in combination with chemotherapeutics in a poster titled “Naxitamab chemo-immunotherapy regimens other than with irinotecan/temozolomide for patients with relapsed/refractory high-risk neuroblastoma” (poster #10037). In this study, 29 patients with mostly chemo-immunotherapy refractory disease received compassionate use of naxitamab plus sargramostim in combination with alternative chemotherapeutics. Importantly, prior sensitivity to chemo-immunotherapy predicted better responses with the new combinations. The safety profiles of the alternative naxitamab chemo-immunotherapy regimens were considered manageable.
Y-mAbs will be available for comment at booth #35151 on the Exhibition Floor of McCormick Place.
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 DANYELZA® (naxitamab-gqgk)
DANYELZA® (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA® includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information for complete Boxed Warning and other important safety information.
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.
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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 2023 and beyond, including estimated operating expenses, cash burn and DANYELZA product revenue and sufficiency of cash resources and related assumptions; implied and express statements regarding the future of the Company’s business, including with respect to expansion and its goals; the Company’s plans and strategies, development, commercialization and product distribution plans, including potential partnerships; expectations with respect to the Company’s products and product candidates, including potential territory and label expansion of DANYELZA and the potential market opportunity related thereto and potential benefits thereof, and the potential of the SADA Technology and potential benefits and applications thereof; statements with respect to DANYELZA as a growing commercial product and SADA as a differentiated radioimmunotherapy platform positioning the Company on a path to potentially transform the treatment paradigm for a variety of cancers and improve patients’ lives; expectations relating to key anticipated development milestones, including potential expansion of international commercialization efforts with respect to DANYELZA development efforts and the SADA Technology, including potential indications and applications, and the timing thereof; expectations with respect to current and future clinical and pre-clinical studies and the Company’s research and development programs, including with respect to timing and results; expectations related to the timing of the initiation and completion of regulatory submissions; additional product candidates and technologies; expectations regarding collaborations or strategic partnerships and the potential benefits thereof; expectations related to the use of cash and cash equivalents, and the need for, timing and amount of any future financing transaction; expectations with respect to the Company’s future financial performance; and other statements that are not historical facts. 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Investor Contact:
Courtney Dugan
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cdu@ymabs.com
FAQ
What is the overall response rate for naxitamab in the Phase 2 Trial 201?
What percentage of patients achieved a complete or partial response after three or more cycles of naxitamab?
What are the common severe adverse events associated with naxitamab?
How did the frequency of severe pain change over the course of naxitamab treatment?
What did the independent study from Sant Joan de Déu Barcelona reveal about naxitamab combined with alternative chemotherapies?
