Y-mAbs Presents Neuroblastoma Research on Naxitamab and SADA PRIT at AACR Pediatric Cancer Conference

Y-mAbs Therapeutics, Inc. (Nasdaq: YMAB), a biopharmaceutical company specializing in innovative radioimmunotherapy and antibody-based treatments for cancer, has revealed new clinical and preclinical findings regarding its drugs naxitamab and GD2-SADA. These results will be showcased in poster presentations at the American Academy of Cancer Research’s Special Conference on Advances in Pediatric Cancer Research in Toronto, Canada, held from September 6-7, 2024.

A poster titled “Disease control in patients treated with naxitamab for refractory/relapsed high-risk neuroblastoma” (poster #B055) will be presented on September 7, 2024. This study focused on the effectiveness of naxitamab in maintaining disease control in patients with high-risk neuroblastoma that is refractory or has relapsed. The analysis, derived from an interim evaluation of Trial 201 (NCT03363373), found that patients treated with naxitamab combined with granulocyte-macrophage colony-stimulating factor (GM-CSF) achieved a disease control rate of 63%. Remarkably, this disease control was consistent irrespective of the patients' initial Curie scores.

Dr. Vignesh Rajah, Chief Medical Officer of Y-mAbs, highlighted the significance of these findings, stating that naxitamab’s ability to control disease progression is vital for patients at high risk of deterioration. He emphasized that these results demonstrate Y-mAbs' ongoing dedication to advancing neuroblastoma treatment.

The second study, titled “GD2-SADA, a bispecific fusion protein that forms self-assembling and disassembling GD2-avid tetramers with high affinity for chelated radiolanthanides” (poster #A075), will be presented on September 6, 2024. This research illustrated the high-affinity interactions between GD2-SADA and DOTA-chelated terbium, a lanthanide metal with several useful medical isotopes. The study explored the dynamic assembly and disassembly of GD2-SADA tetramers, a mechanism that allows for high-avidity binding to GD2-expressing tumors during the first step of pre-targeted radiotherapy (PRIT) while enabling the renal clearance of disassembled monomers.

In a preclinical neuroblastoma model, the chelated radioisotope, administered in the second step of PRIT, binds to GD2-SADA on tumor cells, delivering targeted radiation with minimal off-target effects. These findings have been crucial in the pharmacokinetic and pharmacodynamic modeling that informs the initial dosing in Trial 1001 (NCT05130255), a Phase 1 human trial using GD2-SADA PRIT with 177Lu-DOTA in patients with GD2-positive solid tumors. Additionally, Trial 1002 is planned for pediatric patients with high-risk neuroblastoma.

Dr. Rajah commented on the importance of these advancements, stressing that high-risk neuroblastoma is a severe and challenging type of tumor, particularly in children. He reiterated Y-mAbs’ commitment to furthering the development of naxitamab and the novel SADA PRIT technology to improve patient outcomes.

Y-mAbs’ collaboration with Memorial Sloan Kettering Cancer Center (MSK) has been pivotal in these developments. MSK researchers developed DANYELZA® (naxitamab-gqgk), which is exclusively licensed to Y-mAbs and has received FDA approval for treating relapsed or refractory high-risk neuroblastoma in specific patients. The SADA technology for radioimmunotherapy, also developed at MSK, is licensed to Y-mAbs, with Dr. Nai-Kong Cheung holding intellectual property rights related to the technology.

DANYELZA®, used with GM-CSF, is indicated for pediatric patients aged one year and older and adults with relapsed or refractory high-risk neuroblastoma involving the bone or bone marrow, contingent upon their response to prior therapy. This approval was granted based on response rates and duration of response, with continued approval dependent on further clinical validation. DANYELZA® carries warnings for serious infusion-related reactions and neurotoxicity.

GD2-SADA, a bispecific fusion protein, selectively binds to GD2-expressing tumors and Lutetium 177 (Lu 177)-DOTA, facilitating targeted radiotherapy. This innovative approach is currently under clinical investigation in adolescents and adults.

Y-mAbs remains dedicated to developing and commercializing advanced cancer therapies, leveraging technologies like the SADA PRIT platform and bispecific antibodies. Their product pipeline includes DANYELZA®, the first FDA-approved treatment for high-risk neuroblastoma in the bone or bone marrow following partial or stable response to prior treatments.

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