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Clinical efficacy of the antitumor agent ONC201 in GBM

抗肿瘤药物ONC201治疗GBM的临床疗效

基本信息

批准号：
9405071
负责人：
Wolfgang Oster
金额：
$ 22.47万
依托单位：
ONCOCEUTICS, INC.
依托单位国家：
美国
项目类别：
财政年份：
2015
资助国家：
美国
起止时间：
2015-04-01 至 2018-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9405071
关键词：
Advanced Malignant Neoplasm Adverse effects Animal Model Animals Apoptosis Authorization documentation Biological Markers Blood - brain barrier anatomy Brain Neoplasms Cancer Patient Canis familiaris Capital Cell Line Cell model Cells Chemicals Clinic Clinical Clinical Data Clinical Protocols Clinical Trials Data Development Diagnostic radiologic examination Dose Drug Targeting Drug usage Enrollment Ensure Excipients Feedback Formulation Funding Future Genes Glioblastoma Human Image In Vitro Institution Investments Laboratories Libraries Ligands Malignant Neoplasms Manuals Medical Modeling Mutate New Agents Newly Diagnosed Normal Cell Oral Patient Selection Patients Pharmaceutical Preparations Phase Phase II Clinical Trials Phosphotransferases Privatization Probability Process Property Radio Rattus Recurrence Refractory Reproducibility Research Personnel Resistance Safety Sampling Schedule Secure Serum Markers Statistical Data Interpretation TNFRSF10B gene TNFSF10 gene Testing Therapeutic Time Toxicology Translating Work analytical method anticancer activity antitumor agent antitumor effect arm base cancer cell capsule clinical application clinical development clinical efficacy commercialization efficacy evaluation improved in vivo killings man manufacturing process nanomolar neoplastic cell novel novel anticancer drug novel therapeutics permissiveness phase 1 study phase II trial pre-clinical predictive marker public health relevance receptor research clinical testing response response biomarker screening small molecule standard of care success targeted treatment therapy resistant transcription factor tumor unnecessary treatment

项目摘要

DESCRIPTION (provided by applicant): ONC201 is a clinical-stage first-in-class small molecule with demonstrated anticancer activity against aggressive refractory/recurrent glioblastoma multiforme (GBM) tumors in vitro, ex vivo, and in vivo. This novel compound imparts its antitumor effects through a unique mechanism of action that involves indirect dual inactivation of Akt and ERK in tumor but not normal cells. Several academic investigators at premier institutions have demonstrated the optimal preclinical profile of ONC201 in a multitude of models. These findings have prompted Oncoceutics to rapidly translate this product into the clinic to treat advanced cancer patients. The first-in-man trial is a phase I/II study that will include patients with GBM is scheduled to begin in early 2014 at MGH and has been reviewed by the FDA as part of the accepted IND. Following completion of the dose-escalation phase I portion of the trial supported by Oncoceutics, the phase II trial can begin to evaluate the monoagent efficacy of ONC201 in patients with advanced GBM. In this fast-track application, we propose to develop an automated manufacturing process to provide the clinical supply of drug product followed by evaluation of the clinical efficacy of the resulting product in patients with GBM. Phase/Aim #1: Optimize ONC201 formulation and produce GMP product for GBM trial; Phase/Aim #2: Evaluate clinical efficacy of oral ONC201 in GBM. These studies will enable a development path for ONC201 to treat GBM, which is otherwise unattainable based on investor feedback that indicates a clear need for pilot clinical data in the indication to enable private support. This proposal seeks to make a novel anticancer agent available to patients in dire need of treatment options, will push the product toward commercialization, sufficiently derisk private investments to enable subsequent development, and will advance the field forward by enabling clinical evaluation of a novel therapeutic mechanism.