Our Programs

Etavopivat

Sickle Cell Disease PKR activator

Our lead core product candidate, etavopivat, is an investigational agent that is designed to be a once-daily pill for the treatment of sickle cell disease. Etavopivat is a potent activator of pyruvate kinase-R (PKR), which is hypothesized to impact two pathways in the red blood cell.
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Olutasidenib

Acute Myeloid Leukemia IDH1m inhibitor

We have exclusively licensed olutasidenib, an investigational agent, to Rigel Pharmaceuticals to develop, manufacture and commercialize for any uses worldwide, including for the treatment of R/R AML.
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Olutasidenib

Glioma IDH1m inhibitor

Olutasidenib is an investigational agent designed to selectively inhibit mutated IDH1. In addition to our program in AML, we’re investigating olutasidenib in a Phase 1 clinical study for the treatment of gliomas due to favorable blood-brain barrier penetration and the high prevalence of mIDH1 in this indication.
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preclinical

Within our strategic focus of rare hematologic diseases and cancers, our research pipeline includes the most promising programs from our broad set of drug targets and chemical assets.