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Pipeline
Leveraging our strong heritage of research and development, we’ve built our clinical development pipeline by selecting the most promising drug targets and chemical assets that align with our strategic focus on rare hematologic diseases and cancers. Our pipeline consists of product candidates in multiple disease areas, including sickle cell, thalassemia and prostate cancer.
Sickle Cell Disease
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.
Learn moreAcute Myeloid Leukemia
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.
Learn moreGlioma
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.
Learn moreWe’ve exclusively licensed programs to Rigel Pharmaceuticals, Boehringer Ingelheim and Celgene—now Bristol Myers Squibb—based on molecules we discovered. We’re also actively evaluating other external opportunities for innovation consistent with our strategy.