Oncoceutics Announces FDA Acceptance of Investigational New Drug Application for Phase I Trial of ONC206

In Clinical Trials by Barbara Jacoby

Oncoceutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application for ONC206, allowing the first-in-human trial of the compound. ONC206 is the first of the family of drug candidates, which we call “imipridones”, that possess the same core chemical structure as ONC201.

The first clinical trial for ONC206 in adults with primary central nervous system neoplasms will be conducted at the National Cancer Institute (NCI) under the leadership of Mark Gilbert, MD, Chief of the Neuro-Oncology Branch, Center for Cancer Research (CCR) and Brett Theeler, MD, Clinical Collaborator at the NCI Neuro-Oncology Branch, CCR.

Dr. Gilbert has previously demonstrated that ONC206 exerts therapeutic effects in preclinical models for neuro-oncology indications that are commonly recognized as refractory to standard of care. ONC206 has been studied extensively by several other research groups and showed antitumor activity in different tumor types. Preclinical studies suggest that the properties of ONC206 include penetrance of the blood brain barrier, feasibility of oral administration, demonstration of a well-tolerated safety profile, and selective antagonization of dopamine receptor D2 (DRD2). While DRD2 is targeted by both ONC206 and ONC201, the receptor pharmacology of ONC206 is unique and distinct, which may offer therapeutic options beyond those of ONC201. ONC206 was very well tolerated in GLP toxicology studies in Sprague-Dawley rats and beagle dogs at therapeutic and exaggerated doses that did not reveal any dose-limiting toxicities.

ONC206 is protected by issued composition of matter patents in the USA and Europe.

“We are pleased by the FDA’s acceptance of our IND application for ONC206 that provides further validation of our overall development program,” said Martin Stogniew, PhD, Chief Development Officer of Oncoceutics. “The introduction of another imipridone into clinical trials represents a major milestone for the company and demonstrates the therapeutic value of the new class of imipridone compounds.”

“DRD2 has emerged as a viable novel target in neuro-oncology and controls diverse signaling pathways. However, druggable ligands to target DRD2 have remained elusive. We are encouraged to see the different biological effects exerted by ONC201 and ONC206 via dopamine receptor activated pathways, including their synergistic cooperation that clearly demonstrates the multi-functionality of this G protein-coupled receptor. The common features of both compounds that include high penetrance of the blood brain barrier, convenient oral administration and excellent tolerability is rewarding for our patients, in particular children,” said Sabine Mueller, MD, PhD, Head of Clinical Programs, DIPG Center of Expertise Zurich and Adjunct Associate Professor, University of California, San Francisco.

“We plan to evaluate the safety and clinical activity of ONC206 in patients with central nervous system malignancies,” said Dr. Gilbert. “This clinical trial will incorporate biomarker information derived from our molecular studies of ONC206 in an effort to enable future patient selection.”

About Oncoceutics

Oncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds, called “imipridones,” that selectively target G protein-coupled receptors for oncology. The first lead compound to emerge from this program is ONC201, an orally active small molecule DRD2 antagonist, the first one that exhibits a highly specific bi-topic binding to the receptor. The company is supported by grants from the NCI, FDA, The Musella Foundation, and a series of private and public partnerships