Oncoceutics Release: Efficacy Results For ONC201 In Recurrent Glioblastoma Published

Philadelphia, PA (May 30, 2017) – Oncoceutics, Inc. announced that results from the first 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 Oncotarget. The study, conducted at Massachusetts General Hospital (MGH) and the Dana Farber Cancer Institute (DFCI), demonstrated that ONC201 given as a single agent showed evidence of activity against recurrent glioblastoma and was well tolerated.

The median overall survival for the 17 patients in this first cohort was approximately ten months, which compares favorably to the median overall survival indicated in metanalyses in the literature describing outcomes for patients with recurrent glioblastoma. In addition, two of the 17 patients had particularly favorable outcomes. One patient experienced an 82% average reduction in her two tumor lesions, and these lesions have stayed reduced for more than seven months. A second patient, whose tumor was surgically removed prior to initiating ONC201, has remained disease-free without any tumor recurrence. Both of these patients have taken ONC201 therapy for thirteen and fourteen months respectively, and remain on therapy today.

All 17 patients in this first cohort had histologically-confirmed glioblastoma that had recurred one or more times after receiving standard-of-care chemotherapy and radiation, and they received 625mg of ONC201 once every three weeks. Overall, the treatment was well tolerated. Only 2 treatment-emergent adverse events occurred on study, and both were manageable. There were no treatment discontinuations due to toxicity.

As previously announced, this study (NCT02525692) has been expanded to enroll an additional thirty-six patients on a weekly dosing schedule of ONC201. In addition to weekly dosing, the expanded clinical trial includes a cohort of patients who will receive ONC201 before and after surgical removal of their tumor, facilitating molecular analyses of tumor specimens and allowing for an examination of the penetrance and mechanism of action of ONC201 directly in patients’ tumors. Both cohorts of the trial are supported by a Small Business Innovation Research (SBIR) grant from the National Cancer Institute of the National Institutes of Health.

In addition, because of the unique genetics of the patient who experienced an 82% reduction in tumor – a specific mutation in a specific histone H3 gene that has been recently discovered to be prevalent in younger patients with diffuse midline glioma – Oncoceutics has made available an expanded access program that will provide ONC201 to select patients with recurrent brain tumors that exhibit a histone H3 mutation. (This study is listed on www.clinicaltrials.gov under NCT03134131.)

"Although it is a single patient, this response is highly encouraging. No therapy has proven to be effective at inducing a sustained response in a recurrent K27M mutant midline glioma," said Andrew Chi, MD, PhD, Co-Director of the Brain Tumor Center at NYU Langone Medical Center. "We are excited about the possibility that ONC201 may have activity against these highly malignant tumors and have begun testing ONC201 against patient-derived K27M mutant gliomas in our lab."

“Some of the earliest observations with ONC201 pointed to the potential of this compound to address central nervous system malignancies,” said Joshua Allen, Vice President of Research and Development at Oncoceutics. “It is profoundly gratifying to see preclinical findings translate into clinical benefit for patients with a devastating disease like glioblastoma. We are eagerly evaluating these clinical observations in the laboratory and in additional clinical studies so that we can continue to learn how this compound, and other imipridones, can make maximum impact in oncology.”

About Oncoceutics

Oncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds that selectively target G protein-coupled receptors for oncology. The first lead compound to result from this program is ONC201, an orally active DRD2 small molecule antagonist that is well-tolerated and effective against advanced cancers. The company completed a successful Phase I study in solid tumors and has begun additional Phase I/II and Phase II clinical programs in both solid and hematological malignancies. Oncoceutics and collaborative groups have received more than $7 million in grants over the last two years, including grants from the National Cancer Institute, the U.S. Food and Drug Administration, the Pennsylvania Department of Health, and The Musella Foundation. In addition, outside interest in the company’s portfolio has resulted in several R&D alliance agreements between Oncoceutics and leading comprehensive cancer centers, including The University of Texas MD Anderson Cancer Center and the Fox Chase Cancer Center. The company has established a robust intellectual property position, including several issued patents.

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