A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU 285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis with Symptoms Inadequately Controlled with Standard Therapy

Indolent Systemic Mastocytosis (ISM)

Part 1 •The RP2D in patients with ISM. Part 2 •Mean change in ISM-SAF TSS, from Baseline to C7D1. Part 3 •The long-term safety and efficacy of avapritinib.

• Change in measures of mast cell burden: serum tryptase, KIT D816V allele burden in blood, bone marrow mast cells. • Change in best supportive care usage. • Change in MC-QoL, PGIS, SF-12, PGIC, and EQ-5D-5L. • Safety and tolerability of avapritinib, as assessed by AEs, vital signs, electrocardiograms, and laboratory tests. • Pharmacokinetics of avapritinib (Part 1 and 2 only)

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