Gastrointestinal stromal tumor
Research activity in gastrointestinal stromal tumor centers on targeted treatment strategies for patients with advanced disease, including combinations with sunitinib and evaluation of tumor control in GIST.
- Targeted therapy for GIST
- Sunitinib-based treatment combinations
- Advanced solid tumor management
The sponsor’s work in this area reflects a focus on molecularly directed options for gastrointestinal stromal tumor treatment.
Advanced systemic mastocytosis
In advanced systemic mastocytosis, the clinical portfolio addresses disease control, safety, and tolerability of bezuclastinib, with attention to identifying an active systemic exposure range and an optimal treatment dose.
- Bezuclastinib in AdvSM
- Safety and tolerability assessment
- Dose selection in mast cell disorders
This area is centered on therapeutic development for a rare, mast cell-driven hematologic condition.
Nonadvanced systemic mastocytosis
Studies in nonadvanced systemic mastocytosis focus on symptomatic disease management across indolent and smoldering forms, with comparisons against placebo and continued evaluation of oral bezuclastinib.
- Indolent systemic mastocytosis
- Smoldering systemic mastocytosis
- Oral treatment evaluation
The therapeutic emphasis includes symptom-related disease control and characterization of pharmacokinetic and pharmacodynamic profiles in nonadvanced disease.


