The United States Patent and Trademark Office (USPTO) has issued patent #9,688,679 for the use of ONC201 to treat all leukemias, to Oncoceutics, a clinical-stage drug discovery and development company with a novel class of compounds that selectively target G protein-coupled receptors for oncology. The patent includes treatment options for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).
The anti-cancer small molecule ONC201 is the founding member of a novel class of anti-cancer compounds called imipridones. Currently in Phase II clinical trials in multiple advanced cancers the imipridone family has demonstrated anti-cancer activity and safety in preclinical models and ongoing clinical trials. This including a trial for AML and MDS being conducted as part of a development alliance between Oncoceutics and the University of Texas MD Anderson Cancer Center.
ONC201 upregulates the integrated stress response (ISR) and acts as a dual inactivator of Akt/ERK, leading to TRAIL gene activation.
Since the discovery of ONC201 as a p53-independent inducer of TRAIL gene transcription, preclinical studies have determined that ONC201 has anti-proliferative and pro-apoptotic effects against a broad range of tumor cells but not normal cells. To date, the mechanism of action of ONC201 involves engagement of PERK-independent activation of the integrated stress response, leading to tumor upregulation of DR5 and dual Akt/ERK inactivation, and consequent Foxo3a activation leading to upregulation of the death ligand TRAIL.
The first-in-human clinical trial of ONC201 in advanced aggressive refractory solid tumors confirmed that ONC201 is exceptionally well-tolerated. These trials established the recommended phase II dose of 625 mg administered orally every three weeks defined by drug exposure comparable to efficacious levels in preclinical models. 
The new patent issued by United States Patent and Trademark Office (USPTO) is the sixth issued on ONC201, and continues to expand the IP protection the company has received covering the compound?s use in cancer. These patents, combined with issued patents around the compound?s formulation and use in combination with other therapies, as well as FDA regulatory designations that ONC201 has received, provides ONC201 with a robust suite of intellectual property.
?We are delighted that the USPTO has recognized these indications as novel uses of ONC201 and granted the appropriate patent protections,? said Martin Stogniew, Ph.D., Chief Development Officer of Oncoceutics.
?The company?s current patent estate gives Oncoceutics more than 15 years of patent protection for the vast majority of human cancers, including tumor types where ONC201 is currently in Phase II trials,? Stogniew added.
Last editorial review: July 1, 2017
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