ONC201 Trial Begins for Endometrial and Breast Cancer

In In The News by Barbara Jacoby

Oncoceutics, Inc. announced today that the first patient has been treated in a clinical trial of ONC201 for patients with certain types of advanced endometrial and breast cancer. The Phase II trial is led by Alexandra Zimmer, MD, Assistant Research Physician at the Women’s Malignancies Branch at the National Cancer Institute (NCI) Center for Cancer Research, part of the National Institutes of Health (NIH). The study will enroll up to 94 adult patients using ONC201 as a single-agent and will require tumor biopsies to enable direct evaluation of the activity of ONC201 within the tumor (Trials.cancer.gov Identifier#NCT03394027).

Early results from ongoing clinical trials with ONC201 in high grade gliomas has shown promising single-agent activity that is related to the ability to target dopamine receptors. This study will extend the evaluation of ONC201 beyond glioma to include additional tumor types. This is the first clinical trial to launch out of a consortium of multiple investigators across different divisions at the NIH who are working with Oncoceutics to create, understand, and translate its novel class of therapies called imipridones to address unmet medical needs in oncology.

Endometrial and breast cancers have emerged as tumors that are sensitive to ONC201 in preclinical models through unique mechanisms of action that will be investigated in this clinical trial. Preclinical findings from a team of NCI investigators led by Stan Lipkowitz, MD, PhD, Chief of the Women’s Malignancies Branch in the NCI’s Center for Cancer Research, have shown that ONC201 has unique deleterious effects on the mitochondria of breast cancer cells. These findings are described in a recent publication (Greer et al., Oncotarget, In Press). This Phase II study continues the effort to understand the mechanism of ONC201, and to provide therapies to patients with metastatic breast cancer that are in need of new treatments.

Independent work led by Victoria Bae-Jump, MD, PhD, at UNC Lineberger Comprehensive Cancer Center, has found that dysregulated expression of dopamine receptors targeted by ONC201 induce tumor cell death in endometrial cancer. These findings were disclosed as an oral presentation at the recent annual meeting of the Society of Gynecological Oncology meeting in New Orleans.

“Unlike many other cancers, endometrial cancer has not gained targeted therapy treatment options despite the clear need for patients who have failed chemotherapy,” said Dr. Bae-Jump. “Our recent work shows that endometrial cancer cells harbor altered expression of dopamine receptors that can be targeted by ONC201, creating the potential for an actionable molecular target for this disease.”

For Patients

Patients interested in enrolling please contact NCI’s toll-free number 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615) and/or the Web site: https://trials.cancer.gov.

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.