Receiving a cancer diagnosis is terrifying. Cancer is responsible for one out of every four deaths in the United States1 and a patient’s fight with cancer can last for years. Thankfully, doctors and scientists have made large advances in cancer treatment in the past decade. Advanced surgical techniques, new options for radiation therapy, and more effective chemotherapies are helping more people beat cancer faster. But many of these newer treatment options still have serious side effects and can damage the patient’s healthy cells. The smartest minds in cancer research are trying to find treatments that can effectively fight cancer without the damaging side effects.
Oncoceutics, Inc. believes that it has found a solution. Building on a new class of safe and effective cancer fighting compounds called imipridones, Oncoceutics has developed a first-in-class drug, ONC201, that shows great potential in advanced cancers. Oncoceutics is running multiple Phase II trials that are targeting a wide range of cancers, including brain tumors, endometrial cancer and other solid and liquid cancers. Previous Phase I studies have demonstrated that ONC201 is well-tolerated and effective against advanced cancers.
How does ONC201 work?
G protein-coupled receptors (GPCRs) are the largest family of receptors that reside on the surface of human cells. These complex receptors mediate many important physiological functions by binding to extracellular ligands and controlling many signaling pathways inside the cell. Dysregulation of GPCRs have been linked to many human diseases, and more than a third of all marketed drugs engage one or more of these receptors.
However, surprisingly few GPCR-targeted therapies have been developed for oncology, despite evidence that they play an important role in the survival of cancer cells. Furthermore, it’s been clinically proven that we can target GPCRs safely without toxic effects to normal cells. This represents a significant opportunity for new therapeutics that lack the damaging side effects of currently available chemotherapies and targeted agents.
Imipridones are a novel class of anti-cancer compounds developed by Oncoceutics, that selectively target GPCRs. The first imipridone to reach the clinical stage is ONC201. The small molecule kills cancer cells through selective targeting of the GPCR Dopamine Receptor D2 (DRD2), which fuels tumor growth and is overexpressed on cancer cells relative to normal cells. By targeting DRD2, ONC201 offers a new method to disrupt critical pathways that have been proven to be effective in fighting cancer.
Oncoceutics completed a successful Phase I study in solid tumors and has underway a number of Phase I/II and Phase II clinical programs in both solid and hematological malignancies. Among these indications, the company focuses on brain tumor patients with a certain mutation called H3 K27M.
The H3 K27M mutation has emerged as a prevalent genetic mutation in aggressive midline gliomas that involve specific parts of the brain, including the thalamus, pons, or spinal cord. In 2016, the World Health Organization categorized any tumor that contains the H3 K27M mutation as highest grade (IV) because the mutation confers such a dismal prognosis. Beyond palliative radiation, no medical therapy has been shown to provide clinical benefit for patients with this mutation in their tumor. ONC201 is able to penetrate the blood-brain-barrier effectively and seems to show efficacy in clinical trials for gliomas with the H3 K27M mutation. Oncoceutics is currently conducting clinical trials targeting H3 K27M-mutant gliomas both in pediatric and adult patients.
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 completed a successful Phase I study in solid tumors and has underway a number of Phase I/II and Phase II clinical programs in both solid and hematological malignancies.