Earlier this month Oncoceutics confirmed that the United States Patent and Trademark Office (USPTO) has issued patent to the company with an expiration date of January 29, 2036. This newly issued patent covers the composition of matter for ONC213, its di-salt formulation, and its use in the treatment of cancer. In addition to the claims related to ONC213, the patent also contains claims for millions of structurally-related imipridones.*
ONC213 is the fourth molecule in the company’s pipeline of “imipridone” family of anti-cancer small molecules that target G protein-coupled receptors, following ONC201, ONC206 and ONC212. As with other members of the imipridone class, ONC213 has very attractive chemical and biological properties including oral bioavailability, chemical stability and a large therapeutic index.
“We are delighted by the decision of the US Patent Office to grant Composition of Matter to the novel molecule ONC213,” noted Martin Stogniew, Ph.D., Chief Development Officer of Oncoceutics.
“This patent paves the way for future generations of imipridones to enter the clinic and eventually benefit the lives of patients,” Stogniew added.
Anti cancer activity
ONC213 has demonstrated anti-cancer activity and safety in various preclinical oncology models across hematological malignancies and solid tumors tested in the lab of Principal investigator Yubin Ge, Ph.D, Associate Professor of Oncology at the Barbara Ann Karmanos Cancer Institute and Wayne State University School of Medicine.
Results presented at the 60th American Society of Hematology Annual Meeting, held in San Diego, CA, December 1-4, 2018, demonstrated that ONC213 targets leukemic stem cells in patient-derived xenograft mouse models, is well tolerated and combines synergistically with Bcl-2 inhibitor Venetoclax.
ONC213 activity in AML cell lines and primary AML patient samples was first tested in vitro. ONC213 IC50s ranged from 91.7 nM to 2.4 µM in AML cell lines and primary acute myeloid leukemia (AML) patient samples, which are achievable in vivo based on results from a PK study in mice (a single dose of 50 and 100 mg/kg ONC213 resulted in peak plasma concentrations of 3.7 μM and 8 μM, respectively)
Ge recently received a grant from the Kids Without Cancer and the Children’s Hospital of Michigan Foundation to further determine the mechanism of action of ONC213 and enable biomarker selection for clinical studies.
“Our team at the Karmanos Cancer Institute is excited by the potential of ONC213 as a new type of cancer therapy,” noted Ge.
“We look forward to continuing development of this novel agent as it makes its way towards the clinic,” Ge added.
 Su Y, Li X, Edwards H, Polin L, Kushner J, et al. Venetoclax Synergistically Enhances the Antileukemic Activity of Imipridone ONC213, a Novel Imipridone ONC201 Analog, in Acute Myeloid Leukemia. Blood 2018 132:3936; doi: https://doi.org/10.1182/blood-2018-99-116997 [Article]
* Patent #10,266,533 entitled “7-BENZYL-4-(2-METHYLBENZYL)-2,4,6,7,8,9-HEXAHYDROIMIDAZO [1,2-A]PYRIDO [3,4-E]PYRIMIDIN-5(1H)-ONE, ANALOGS THEREOF, AND SALTS THEREOF AND METHODS FOR THEIR USE IN THERAPY”