Isabel Arrillaga-Romany, MD, PhD of @MGHNeuroOnc @Oncoceutics answers what data has come out of the trial?
Oncoceutics, Inc. announced that results from the second and third cohort of the Phase II trial for patients with recurrent glioblastoma treated with the company’s lead imipridone, ONC201, were recently published in the journal Neuro-Oncology. This manuscript represents the third publication among a series of reports demonstrating durable radiographic regressions with ONC201 in patients with H3 K27M-mutant glioma. The study (NCT02525692) also demonstrated that weekly oral administration of ONC201 given as a single agent was well-tolerated, achieved therapeutic intratumoral concentrations, and intratumoral pharmacodynamic activity in adult recurrent glioblastoma patients.
ONC201 achieved intratumoral concentrations that exceeded preclinical efficacy thresholds and induced intratumoral biomarkers of pharmacodynamic signaling and apoptosis indicating target engagement. The radiographic regression of multi-focal, multi-recurrent disease with ONC201 administration in an adult recurrent H3 K27M-mutant glioblastoma patient remained durable for >1.5 years. No treatment modifications or discontinuations due to toxicity were observed, including those who underwent re-resection.