Hematologic Malignancies
Acute myeloid leukemia, myelodysplastic syndromes, and chronic myelomonocytic leukemia are prominent areas of interest, with additional attention to relapsed and refractory disease and mutation-defined blood cancers.
- IDH1-mutated AML
- IDH2-mutated AML
- MDS/AML
- CMML
Research activity also includes treatment settings for newly diagnosed and previously treated hematologic malignancies.
Neuro-Oncology and Neurologic Disorders
The sponsor supports studies in glioma and other brain tumors, with a clear focus on IDH-mutant disease, alongside selected neurologic conditions affecting development and movement.
- IDH1-mutant glioma
- IDH2-mutant glioma
- Grade 2 glioma
- Parkinson disease
- KCNT1-related developmental and epileptic encephalopathy
These trials reflect interest in both oncologic and rare neurologic indications.
Solid Tumors and Molecularly Defined Cancers
Clinical research spans several solid tumors defined by actionable alterations, including cholangiocarcinoma, colorectal cancer, non-small cell lung cancer, and gastric or gastroesophageal-junction cancer.
- IDH1-mutated cholangiocarcinoma
- Metastatic colorectal cancer
- NSCLC
- MSI-H/dMMR gastric cancer
- MTAP-deleted solid tumors
- RAS/MAPK-mutant malignancies
There is also interest in chondrosarcoma and other advanced solid tumor settings.
Metabolic Disease
The sponsor maintains a separate research stream in type 2 diabetes mellitus, including patients whose glycaemic control remains inadequate despite established treatment.
- Type 2 diabetes
- Glycaemic control
- Combination oral therapy
This area extends the portfolio beyond oncology into chronic metabolic care.
Targeted Molecular Oncology
A substantial portion of the portfolio centers on molecularly selected cancers driven by IDH1, IDH2, KRAS, BRAF, MTAP, and related pathway alterations.
- IDH-mutant malignancies
- RAS/MAPK-altered tumors
- MTAP-deleted cancers
- Biomarker-defined solid and hematologic tumors
These studies cover both monotherapy and combination treatment approaches across oncology indications.
