our programs

Glioma

Olutasidenib

There are certain solid tumors that afflict individuals with a mutation of the enzyme IDH1. Our investigational agent is designed to inhibit mutated IDH1 in patients with glioma (a type of primary brain tumor), chondrosarcoma (a slow-growing bone cancer affecting connective tissue or cartilage) and intrahepatic cholangiocarcinoma (a malignancy of liver bile ducts).

Gliomas are one of the most common types of primary brain tumors. They are aggressive and difficult to treat.1 As with most primary brain tumors, the exact cause of gliomas is unknown.2 More than 70% of individuals with gliomas present with an IDH1 mutation, but treatment options for this group are limited.3 IDH1 mutations also occur in approximately 40% of chondrosarcomas and approximately 20% of intrahepatic cholangiocarcinomas.4

There remains significant unmet medical need for this patient population.

>70 %

gliomas with IDH1 mutations

~40 %

chondrosarcoma cases with IDH1 mutations

~20 %

intrahepatic cholangiocarcinoma cases with IDH1 mutations

Therapeutic potential of olutasidenib.

IDH1 is a natural enzyme that is part of the normal metabolism of all cells. Mutations of IDH1 can produce excessive amounts of the oncometabolite 2-hydroxyglutarate (2-HG), which impairs stem cell differentiation and promotes cancer cell growth and progression in solid tumors.

Olutasidenib is an oral and selective small-molecule investigational agent designed to selectively bind to and inhibit mutated IDH1 enzymes.

Development Status

We’re evaluating olutasidenib in an exploratory Phase 1 trial for glioma.

Learn more about our Phase 1 glioma trial

 

1 Ostrom, Q. T., Gittleman, H., Truitt, G., Boscia, A., Kruchko, C., & Barnholtz-Sloan, J. S. (2018). CBTRUS statistical report: Primary brain and other central nervous system tumors diagnosed in the United States in 2011–2015. Neuro-Oncology, 20(4): iv1-iv86. Retrieved from link.
2 Mayo Clinic. (2020). Glioma. Retrieved from link.
3 Yan, H., Parsons, W., Jin, G., McLendon, R., Rasheed, B. A., Yuan, W., Kos, I., Batinic-Haberle, I., Jones, S., Riggins, G. J., Friedman, H., Friedman, A., Reardon, D., Herndon, J., Kinzler, K. W., Velculescu, V. E., Vogelstein, B., & Bigner, D. D. (2009). IDH1 and IDH2 mutations in gliomas. The New England Journal of Medicine, 360(1): 765-773. Retrieved from link.
4 Molenaar, R. J., Maciejewski, J. P., Wilmink, J. W., & van Noorden, C. J. F. (2018). Wild-type and mutated IDH1/2 enzymes and therapy responses. Oncogene, 27(15): 1949-1960. Retrieved from link.