On September 10, 2019 Oncoceutics, Inc. reported a scientific publication in the Journal of Neuro-Oncology describing a complete response and other forms of clinical benefit in pediatric and adult H3 K27M-mutant glioma patients treated with ONC201 on expanded access protocols (Press release, Oncoceutics, SEP 10, 2019, View Source [SID1234558332]).

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This manuscript details radiographic findings and other clinical outcomes in eighteen H3 K27M-mutant glioma patients who were enrolled on expanded access protocols. These patients initiated ONC201 after prior radiation, and in many cases after also failing additional chemotherapy, a disease setting in neuro-oncology where no available therapies have been proven effective.

Overall findings include disease stabilization and radiographic regressions after initiation of ONC201 that are atypical for the disease course. Two adult and two pediatric cases are highlighted due to improvements in radiographic imaging of their disease, as well as disease-associated neurological symptoms. The most dramatic response was observed in an adult who initiated ONC201 at second recurrence following prior radiation and chemotherapy. After initiating ONC201, the patient’s tumor located in the thalamus and other sites in the brain completely regressed. These imaging findings were accompanied by improvements in headaches, nausea, and right-sided numbness caused by the disease.

Among the 14 patients with recurrent disease prior to initiation of ONC201, median progression-free survival was 14 weeks and median overall survival was 17 weeks. Three adults among the 14 recurrent patients remain on treatment progression-free with a median follow up of 41, 49.6, and 76.1 weeks. Among the 4 pediatric patients who initiated adjuvant ONC201 following radiation, two DIPG patients remain progression-free for at least 53 and 81 weeks and the other two progressed at 28.4 and 41.9 weeks.

"Expanded access to ONC201 was critical in allowing our H3 K27M-mutant patients to access the therapy while clinical trials were being initiated for this form of glioma that previously had no effective therapy," said Nicole Shonka, MD, Associate Professor of Internal Medicine at Nebraska Medical Center. "The complete response in the patient I treated is very striking, given the dismal prognosis of this type of grade IV glioma that is typically unresponsive to anything after initial radiation. We are eager to continue the evaluation of ONC201 in clinical trials to further establish the exciting results we saw in expanded access."