Philadelphia, PA (November 3, 2017) – Oncoceutics, Inc. announced today that the first patient has been treated in a Phase II clinical trial of ONC201 for patients with recurrent high-grade gliomas that contain a specific mutation called the histone H3 K27M mutation. The trial is led by Andrew Chi, MD, PhD, Chief of Neuro-oncology and co-Director of the Brain Tumor Center at New York University (NYU) School of Medicine and NYU Langone Health, and will enroll approximately 40 patients with gliomas with the H3 K27M mutation.
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 the mutation 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.
The decision to pursue a clinical trial focused on patients with the H3 K27M mutation in their tumor was made based on a number of findings in both clinical and pre-clinical studies demonstrating that ONC201 is effective against tumors with the mutation. The clinical findings include results from a 22-year-old patient with a recurrent high-grade glioma containing the H3 K27M mutation who was treated with ONC201 at the Dana Farber Cancer Institute. At enrollment, this patient had two malignant lesions in her brain that have decreased by 96% in response to ONC201 over time, and she remains on therapy after more than 1.5 years. In addition, preclinical efficacy and mechanistic studies have shown that H3 K27M gliomas cells are extremely sensitive to ONC201; this data will be reported at the upcoming 2017 Annual Meeting of the Society of Neuro-Oncology in San Francisco.
“The previously reported outlier response to ONC201 in a patient with H3 K27M glioma combined with the preclinical results that we are seeing in my lab make me excited to offer ONC201 to our patients with this molecularly-defined disease,” said Dr. Chi. “This disease has been resistant to any drug studied in the clinic to date. ONC201 may be able to change this record and allow precision medicine to address this patient population that is now understood on a molecular level but lacks an actionable therapeutic option. Additionally, the lack of side effects and ease of administration as an infrequent oral agent that obviates the need of hospital-based procedures are features of significant value.”
“This clinical trial could change medical practice for a molecularly-defined group of patients,” said Wolfgang Oster, MD, PhD, Chief Executive Officer and Chairman at Oncoceutics. “Based on the results we have seen in patients with the H3 K27M tumor mutation who have received ONC201, as well as the preclinical data generated by Dr. Chi, we believe that the clonal and persistent nature of this mutation in gliomas offers a unique opportunity to eliminate these devastating tumors.”
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.
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