This is an experimental drug - and things may rapidly change.
This page is current as of 1/26/24, but everything can change - contact us if you definitely need to know.


Onc-201 is an oral, well tolerated experimental drug used to treat a serious rare pediatric and young adult brain tumor: H3K27M diffuse midline glioma, including DIPG. This type of tumor has no approved treatments and average survival is only 9 months. Onc-201 has been shown to have very few safety related side effects in over 350 patients, and in a small trial of this specific brain tumor type showed at least a doubling of expected survival time with many patients still doing well, and a patient benefit rate of over 50%, which is unheard of in this tumor type.

Clinical Trials

There are a few clinical trials going on with this drug. Click here to see them.

Compassionate Use in the USA

(Added 1/26/2024) Statement from Chimerix on counterfeit Onc-201 and new ways to access Onc-201 for those who were using the counterfeit Onc-201

We used to fund and run the compassionate use program in the USA for Onc-201, but we ran out of funding and sadly the program had to close. A smaller expanded access program might be open now. See https://oncoceutics.com/expanded-access/ for details!.

Access outside of the USA

France now supplies Onc-201 for free for those who need it!
The DMG-ACT trial is now open in Europe and Austrailia! (as well as all over the USA!)

Latest News about Onc-201

Clinical efficacy of ONC-201 in H3K27M mutated DMG is driven by disruption of integrated metatbolic and epigenetic pathways (Added 8/26/23). This peer reviewed paper discussed the results of two clinical trials showing that Onc-201 (by itself) doubles survival in these patients! Hopefully will do much better in combinations!(We helped fund this project!)

We had a webinar about combination therapy for DIPG / DMG which is based on Onc-201.

Chimerix Announces Positive ONC201 Data in Recurrent H3 K27M-mutant Diffuse Midline Glioma to Be Presented at the Society for Neuro-Oncology Annual Meeting

There was an FDA ODAC meeting on June 20, 2019 to discuss the development process of Onc-201.
Here are some important files from that meeting:

Our privacy / cookie policy has changed.
Click HERE to read it!