• Philadelphia, PA (November 13, 2017) – Oncoceutics, Inc. today announced that five clinical and preclinical presentations on ONC201 and ONC206 for the treatment of high grade gliomas will be presented at the 22nd Annual Scientific Meeting and Education Day of the Society of Neuro-Oncology, held November 16th-19th in San Francisco.

    The series of presentations describe DRD2 as a novel therapeutic concept for neuro-oncology that is addressed by ONC201 and other imipridones, such as ONC206. Results will be presented that demonstrate the effectiveness and mechanism of ONC201 in biomarker-defined gliomas, including the H3 K27M mutation. Efficacy results will include preclinical models and clinical evidence of activity in recurrent glioma patients. These results formed the rationale for an ongoing clinical program that is dedicated to this molecularly-defined patient population.

    The H3 K27M mutation has emerged as a prevalent genetic mutation in aggressive midline gliomas that involve specific parts of the brain, including the thalamus, pons, or spinal cord. In 2016, the World Health Organization categorized any brain tumor that contains the H3 K27M mutation as the highest grade (IV) because its confers such a dismal prognosis. Beyond palliative radiation, no medical therapy has been shown to provide clinical benefit for patients with this mutation in their tumor.

    Summaries and presentation information are provided below.

    Presentation Type: Oral presentation (ACTR-51)

    Title: Clinical Evaluation of the Imipridone ONC201 in Recurrent Glioblastoma: Predictive and Pharmacodynamic Biomarker Analyses

    Presenter: Isabel Arrillaga-Romany, MD, PhD Associate Clinical Director, Neuro-Oncology, MGH Cancer Center

    Date, Time, and Location: November 17th, 4:45pm, Yerba Buena Salons 9


    • 17 recurrent, bevacizumab-naïve, IDH1/2 WT glioblastoma patients received 625mg ONC201 every three weeks in the first arm of a Phase II clinical trial.
    • All 15 patients with available archival tumor tissue had expression of DRD2.
    • Patients who experience clinical benefit exhibited low intratumoral DRD5 expression, including the patients with the strongest response who also had the H3 K27M mutation


    Presentation Type: Oral presentation (EXTH-42)

    Title: K27M mutant gliomas are selectively killed by ONC201, a small molecule inhibitor of

    dopamine receptor D2

    Presenter: Andrew Chi, MD, PhD, Chief of neuro-oncology at the Laura and Isaac Perlmutter Cancer Center and co-director of the NYU Langone Brain Tumor Center

    Date, Time, and Location: November 17th, 8:06pm, Golden Gate C2


    • Patient-derived H3 K27M mutant glioma cells are selectively sensitive to ONC201 in vitro.
    • DRD2 expression was increased and DRD5 expression was decreased in H3 K27M gliomas.
    • Forms rationale for ongoing investigations of ONC201 clinical activity in recurrent H3 K27M glioma.


    Presentation Type: Oral presentation (EXTH-43)

    Title: Differentiated pharmacology of the imipridone ONC201, the first selective DRD2/3 antagonist in clinical neuro-oncology

    Presenter: Joshua Allen, PhD, VP of Research and Development at Oncoceutics

    Date, Time, and Location: November 17th, 2:25pm, Yerba Buena Salons 7


    • ONC201 is highly specific for DRD2 and DRD3, which differentiates ONC201 from other dopamine receptor antagonists.
    • Unique residues on the DRD2 receptor govern ONC201’s activity
    • Receptor pharmacology explains the unique selectivity, safety, and anti-cancer activity of ONC201 observed in clinical trials.


    Presentation Type: Poster (DDIS-08)

    Title: The small molecule imipridone ONC201 is active in glioblastoma with DRD2 pathway dysregulation

    Presenter: Varun Prabhu, PhD, Associate Director of Research and Development at Oncoceutics

    Date, Time, and Location: November 18th, 5-7pm Golden Gate AB


    • DRD2 is overexpressed in glioblastoma, and DRD2 antagonism induces tumor cell apoptosis.
    • Expression of DRD5, a D1-like dopamine receptor that counteracts downstream DRD2 signaling, has a significant inverse correlation with ONC201 efficacy in cancer cell lines.
    • DRD5 was found to be involved in acquired resistance and innate tumor cell sensitivity to ONC201.


    Presentation Type: Poster (DDIS-06)

    Title: ONC206, an imipridone family member, suppresses glioma stem cell maintenance

    Presenter: Jinkyu Jung, PhD, Research Fellow on the Preclinical Translational Research Team at the National Cancer Institute’s Neuro-Oncology Branch

    Date, Time, and Location: November 18th, 5-7pm, Golden Gate AB


    • ONC206 is an analog of ONC201 that exhibits potent, selective DRD2 antagonism and depletes patient-derived glioma stem cells (GSCs).
    • ONC206 down-regulated protein expression of oncogenic stem cell markers in the GSCs and prevented tumor sphere formation.


    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 recently 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 significant grants over the last two years, from institutions such as 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 and collaborations between Oncoceutics and leading cancer research institutions, including The University of Texas MD Anderson Cancer Center, the NIH/NCI, Harvard and the Fox Chase Cancer Center.  The company has established a robust intellectual property position, including several issued patents.


    Contact press@oncoceutics.com for more information.