IDH1 positive Solid Tumors

Sept. 06, 2024 -- 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 clinical and preclinical data from studies evaluating naxitamab and GD2-SADA, respectively, in neuroblastoma. The results are summarized in poster presentations scheduled to be presented September 6 – 7, 2024, at the American Academy of Cancer Research (“AACR”) Special Conference in the Advances in Pediatric Cancer Research in Toronto, Canada. Naxitamab maintains disease control in patients with refractory/relapsed high-risk neuroblastoma: 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, during poster session B. This study analyzed the disease control rate with a 12-week minimum of stable disease from the start of naxitamab treatment, based on data from a prespecified interim analysis of Trial 201 (NCT03363373). Patients with refractory/relapsed high-risk neuroblastoma and residual disease in the bone and/or bone marrow who were treated with naxitamab in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”) achieved a disease control rate of 63%, with the results suggesting consistent disease control irrespective of baseline Curie score. “Naxitamab’s ability to maintain disease control provides an important measure for those who are most at risk for disease progression,” said Vignesh Rajah, MBBS, DCH, MRCP (UK), Chief Medical Officer. “The results reflect our deep commitment to advancing the science and therapeutic management of neuroblastoma.” High-affinity binding of GD2-SADA to Tb-DOTA: A poster titled “GD2-SADA, a bispecific fusion protein that forms self-assembling and disassembling (“SADA”), GD2-avid tetramers with high affinity for chelated radiolanthanides” (poster # A075) will be presented today, September 6, 2024, during poster session A. The study demonstrated tight binding interactions between GD2-SADA and DOTA-chelated terbium, a metal in the same lanthanide family as lutetium with multiple medical isotopes of potential benefit in diagnosis and therapy. Building on previous in vitro findings, the study also characterized the self-assembly and disassembly of GD2-SADA, a dynamic equilibrium that permits high avidity binding of non-radiolabeled GD2-SADA tetramers to GD2-expressing tumors and the renal clearance of disassembled monomers during the first step of pre-targeted radiotherapy (“PRIT”). In a preclinical model of neuroblastoma, also presented in the poster, the chelated radioisotope is administered during the second step of PRIT and binds to GD2-SADA on tumor cells, delivering cytotoxic radiation with minimal off-target exposure. The results have informed ongoing PK/PD modeling and the initial dosing in Trial 1001 (NCT05130255), a first-in-human Phase 1 trial of GD2-SADA PRIT with 177Lu-DOTA in adolescent and adult patients with GD2-positive solid tumors, and with Trial 1002 planned for pediatric patients with high-risk neuroblastoma. “The data highlight the importance of continued innovation in the treatment of high-risk neuroblastoma, an aggressive and relentless tumor, and the most common extracranial solid tumor in children,” said Dr Rajah. “We remain committed to unlocking the full potential of naxitamab and our novel SADA PRIT technology platform in our mission of improving the lives of patients.” 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. Researchers at MSK, including Dr. Nai-Kong Cheung, developed the SADA technology for radioimmunotherapy, which is exclusively licensed by MSK to Y-mAbs. Dr. Cheung has intellectual property rights and interests in technology, and as a result of this licensing arrangement, MSK has institutional financial interests in the technology. 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. GD2-SADA is a bispecific fusion protein that tightly binds to the glycolipid GD2 and Lutetium 177 (Lu 177)-DOTA, a chelated or “caged” radionuclide. In the first step of pre-targeted radiotherapy, non-radiolabeled GD2-SADA tetramers are infused and bind to GD2-expressing solid tumors, while unbound GD2-SADA protein disassembles into low molecular weight monomers that are removed by the kidney. The second infusion delivers the “radioactive payload,” which binds directly to GD2-SADA on tumor cells for localized irradiation. GD2-SADA PRIT with Lutetium 177-DOTA is currently being investigated in adults and adolescents in Trial 1001 (NCT05130255). 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. The content above comes from the network. if any infringement, please contact us to modify.