Oncoceutics Release: ONC201 Phase I Trial Results Reported In Clinical Cancer Research

Philadelphia, PA (March 22, 2017) – Oncoceutics, Inc. announced the publication of an original research article in the journal Clinical Cancer Research entitled “First-in-human Clinical Trial of Oral ONC201 in Patients with Refractory Solid Tumors” that describes the Phase I trial of the company’s lead compound at the Rutgers Cancer Institute of New Jersey. The trial, led by Mark Stein, MD, a medical oncologist and member of the Phase I/Investigational Therapeutics Program at Rutgers Cancer Institute, demonstrated that the company’s lead compound ONC201 is well-tolerated at plasma concentrations that meet or exceed the targeted therapeutic threshold and is biologically active in advanced cancer patients when orally administered at the recommended phase II dose (RP2D) of 625 mg every 3 weeks.

ONC201 selectively binds to and antagonizes DRD2, a member of the dopamine receptor family and of the superfamily of G protein-coupled receptors (GPCRs) that is dysregulated in many cancers. GPCRs represent therapeutic targets that have largely been untapped in oncology despite evidence showing that selective inhibition of DRD2 has the potential to be a promising novel therapeutic concept in oncology. The completion of the first-in-human clinical trial enables Phase II studies that are now underway for ONC201. Its validation as a viable clinical drug candidate also enables the development of a family of a novel class of GPCR-targeting anti-cancer agents called imipridones, derived from the founding compound ONC201.

Highlights of the first-in-human trial include the following: no adverse events > Grade 1 and possibly attributed to study drug occurred; PK exceeded target parameters, and pharmacodynamic assays demonstrated biological activity, including induction of caspase-cleaved keratin 18 and prolactin as serum biomarkers of apoptosis and DRD2 antagonism, respectively. Most importantly, radiographic regression of several individual metastatic lesions was observed along with prolonged stable disease (> 27 weeks) in prostate and endometrial cancer patients.

“I am delighted that Rutgers Cancer Institute of New Jersey had the opportunity to introduce the first imipridone into the clinic,” said Rutgers Cancer Institute’s Dr. Stein, “providing patients who have exhausted their therapeutic options with a novel treatment for cancers that have no available therapies.”

Wafik El-Deiry, MD, PhD, Deputy Cancer Center Director of the Fox Chase Cancer Center, and in whose lab ONC201 was discovered added: “We are excited with the successful completion of the Phase I study, translating the potential of both ONC201 and the impridone family to clinically benefit our patients. I thank the investigators in this study and Oncoceutics for sharing our vision for this drug and for being pioneers in the clinical phase.”

“This first clinical paper on ONC201 is exciting in many ways,” said J. Silvio Gutkind, PhD, Professor, Department of Pharmacology, Moores Cancer Center, University of California San Diego. “Most particularly, it opens the door to targeting GPCRs in cancer, a well-established target class for modern drug development that has yet to be exploited in oncology. The field of oncology has benefitted many times from introductions of novel drug classes that involve new mechanisms to tackle difficult-to-treat cancers. I applaud the investigators for putting a stake in the ground to claim the introduction of a new class of compounds that has the potential to enrich the armamentarium of drugs and cancer targets that are available for oncologists to treat their patients.”

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 has 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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