EPEN-14. A Phase 1, Safety and Dose Escalation Study of BXQ-350 in Patients with Advanced Solid Malignancies Demonstrates that BXQ-350 is Well Tolerated and Shows Signs of Potential Clinical Activity in Ependymoma Patients
| العنوان: | EPEN-14. A Phase 1, Safety and Dose Escalation Study of BXQ-350 in Patients with Advanced Solid Malignancies Demonstrates that BXQ-350 is Well Tolerated and Shows Signs of Potential Clinical Activity in Ependymoma Patients |
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| المؤلفون: | Olivier Rixe, John Morris, Robert Wesolowski, Emrullah Yilmaz, John Villano, Carolyn Muller, Trisha Wise-Draper, Vinay Puduvalli, Richard Curry III, Ray Takigiku, Gilles Tapolsky |
| المصدر: | Neuro-Oncology. 24:i41-i41 |
| بيانات النشر: | Oxford University Press (OUP), 2022. |
| سنة النشر: | 2022 |
| مصطلحات موضوعية: | Cancer Research, Oncology, Neurology (clinical) |
| الوصف: | The significance of a dysregulated sphingolipid metabolism in cancer, including brain tumors, has been demonstrated and several enzymes involved in sphingolipid metabolism are being investigated as novel therapeutic targets for adult and pediatric brain cancers. Saposin C (Sphingolipid Activator PrO[S]etIN) is a human protein encoded by the Psap gene and is an allosteric activator of several enzymes involved in sphingolipid/ceramide metabolism. BXQ-350 is a nanovesicle of Saposin C that has broad anticancer activity, selectively inducing apoptosis of cancer cells by lowering Sphingosine-1-Phosphate and increasing ceramides concentrations and inducing an anti-tumoral immune response. BXQ-350 was investigated in a Phase 1 dose-escalation safety study in cancer patients with advanced solid malignancies including brain tumors (NCT02859857). The primary objective of this single agent study was to describe the safety profile and to determine the maximum tolerated dose, or the biological effective dose, of BXQ-350 administered intravenously at escalating doses from 0.7 mg/kg up to 2.4 mg/kg. Multiple secondary parameters were included to characterize BXQ-350’s pharmacokinetic parameters, efficacy profile and potential biomarkers. This trial was performed at four US sites. Results indicate that BXQ-350 was safe and well-tolerated as no DLT was observed and an MTD was not reached. RANO or RECIST 1.1 criteria were used to evaluate tumor response. Analysis of plasma samples suggests that BXQ-350 modulates sphingolipid metabolism and impacts the immune system positively. Furthermore, potential signs of single agent activity were observed across tumor types, including in two ependymomas for which results will be presented. |
| تدمد: | 1523-5866 1522-8517 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_________::f5e8325c906f3dbc2dd9fc15f98c44afTest https://doi.org/10.1093/neuonc/noac079.151Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi...........f5e8325c906f3dbc2dd9fc15f98c44af |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 15235866 15228517 |
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