Clinical Trials – ONC201 for Neuroendocrine Cancer (including Pheo/Para)


What is ONC201?

A company called Oncoceutics is developing a novel class of safe and effective cancer therapies called imipridones. Imipridones have a unique three-ring core structure and selectively target G protein-coupled receptors (GPCRs), the largest class of membrane receptors and a common target of approved drugs that are underexploited in oncology. Despite being historically uncommon as targets in oncology, GPCRs control an array of critical prosurvival and stress signaling pathways that are often dysregulated in human cancer to favor cancer cell survival and propagation.

The ability of imipridones to target GPCRs with a high degree of selectivity represents a novel opportunity in oncology that generates remarkably safe and effective therapeutics. ONC201, the founding member of this novel class of therapies, is an orally active, safe, and selective antagonist of the GPCR Dopamine Receptor D-2 (DRD2). Antagonism of DRD2 has been shown to cause downstream activation of the integrated stress response and inactivation of Ras signaling, both of which have been proven clinically effective in oncology and have resulted in approved therapeutics such as bortexomib and cetuximab. ONC201 represents the first clinical opportunity to engage these proven signaling pathways by selectively targeting DRD2, which is overexpressed or otherwise dysregulated in many types of cancer.

What Makes this different from other NET treatments?

This looks like a different type of targeted approach to treating Neuroendocrine Cancer whereas others have been focused on somatostatin receptor-based treatments such as somatostatin analogues (octreotide and lanreotide) and Lu177 (Lutathera) PRRT. Afinitor (Everolimus) and Sutent (Sunitinib) also use different pathways as their mechanism of action.

The Clinical Trial for Neuroendocrine Cancer?

The purpose of this study is to learn if a new drug, ONC201 can make tumors become smaller or go away completely. Investigators also want to learn if ONC201 can prevent new deposits of cancer from appearing in new places in participants (metastases). A phase 2 study of ONC201 in Pheochromocytoma/Paraganglioma and other neuroendocrine tumors will determine whether inhibition of DRD2 (a member of the dopamine receptor family) is safe in neuroendocrine cancers including Pheochromocytoma/Paraganglioma.

ONC201 is an investigational (experimental) agent and has a favorable safety profile in phase 1 and early phase 2 clinical trials in advanced cancers. One of the secondary aims is to determine efficacy of ONC201 by reduction in dose of anti-hypertensive medications, especially Pheochromocytoma/Paraganglioma.

There are also clinical trials of ONC201 in other cancers including multiple myeloma, leukemia, lymphoma, endometrial cancer, and other solid tumors.

Further Reading:

1. Oncoceutics websiteclick here

2. The clinical trial document – click here

3. Chimerix Announces Publication of Positive ONC201 Data from Phase 2 Study in Neuroendocrine Tumors in Clinical Cancer Research (looks like just for Pheo/Para)


I am not a doctor or any form of medical professional, practitioner or counsellor. None of the information on my website, or linked to my website(s), or conveyed by me on any social media or presentation, should be interpreted as medical advice given or advised by me. 

Neither should any post or comment made by a follower or member of my private group be assumed to be medical advice, even if that person is a healthcare professional.   

Please also note that mention of a clinical service, trial/study or therapy does not constitute an endorsement of that service, trial/study or therapy by Ronny Allan, the information is provided for education and awareness purposes and/or related to Ronny Allan’s own patient experience. This element of the disclaimer includes any complementary medicine, non-prescription over the counter drugs and supplements such as vitamins and minerals.


Whenever I post about a trial or study, some people get excited without understanding that these new treatments and capabilities can very often take years to come to fruition and it’s also possible that clinical trials can be halted, or that national approval agencies will not approve the final product.  Plus, not everyone will be eligible, so always check the exclusion and inclusion criteria in the relevant clinical trials document  Please bear that in mind when reading studies/clinical trials posted on RonnyAllan.NET

Thanks for reading.


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