Oncoceutics, Inc. announced publication of a research article in the journal Molecular Cancer, which describes the discovery of ONC201 as a selective lead compound with a novel chemical structure and novel mechanism of action that warranted clinical testing.
In an attempt to identify inducers of tumor cell death that spare normal cells and overcome therapeutic resistance, a small molecule library that represents 140,000 compounds was screened to identify compounds that fulfill certain molecular criteria to enable this profile. Using a series of in vitro and in vivo assays, ONC201 was the only compound to show significant apoptosis in tumor cells without harming normal cells by employing pathways that are highly conserved within the immune-oncology armamentarium. The uniqueness of its biological activity is similarly mirrored in its molecular structure, which possesses a pharmacophore that is unique among 63 million publicly indexed molecules. This study cumulatively indicates that ONC201 is a selectively active antitumor agent that possesses a novel mechanism of action that includes, but extends well beyond, the induction of the TRAIL gene, and it highlights the therapeutic potential of this novel class of anticancer drugs.
“Even during the discovery process, it was immediately apparent that ONC201 has a clearly differentiated mechanism that translates to an uncommonly favorable safety and efficacy profile relative to many cancer drugs that are on the market,” said Joshua Allen, PhD, VP-Research & Development of Oncoceutics.
“The majority of conventional cancer therapies and even more modern targeted therapies have significant toxicities that can limit their use in patients. This article describes the discovery process that identified ONC201 as a mutation-agnostic and non-toxic anti-cancer agent that kills cancer cells and that may be an option when other therapies are no longer effective,” commented Wafik El-Deiry, MD, PhD, FACP, Professor of Oncology, Co-Program Leader of Molecular Therapeutics, Deputy Cancer Center Director for Translational Research at Fox Chase Cancer Center, Scientific Founder of Oncoceutics and senior author of the article.
The link to the journal article is available here.
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