A Phase 2 Study of Bezuclastinib (CGT9486), an Oral, Selective, and Potent KIT D816V Inhibitor, in Adult Patients with Advanced Systemic Mastocytosis (Advsm)

Systemic mastocytosis (SM) is characterized by excessive accumulation of mast cells in bone marrow and other extracutaneous tissues encompassing a spectrum of variants ranging from non-advanced to advanced disease (Shomali et al, 2018). Advanced systemic mastocytosis (AdvSM) is an aggressive and life-threatening form of SM with an overall survival from <6 months to ~3-4 years depending on subtype (Gotlib et al, 2018; NCCN Systemic Mastocytosis, Version 2.2019; Pardanani, 2019; Rossignol et al, 2019; Shomali et al, 2018). The three distinct subtypes of AdvSM include aggressive systemic mastocytosis (ASM), SM with an associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL) (NCCN Systemic Mastocytosis, Version 2, 2019; Pardanani, 2019; Shomali et al, 2018).