BLU-808

BLU-808 Medication Report

Name: BLU-808 Name (English): BLU-808

Mechanism of Action: BLU-808 is an investigational, highly potent and selective oral inhibitor of wild-type KIT (tyrosine kinase). Wild-type KIT plays a central role in mast cell activation. By inhibiting KIT, BLU-808 aims to reduce mast cell activity.

Developer: Blueprint Medicines

Development Stage: Phase 2 clinical trials are planned or ongoing.

Potential Uses: BLU-808 is being investigated for the treatment of various mast cell disorders and allergic/inflammatory conditions, including:

• Chronic Spontaneous Urticaria
• Chronic Inducible Urticaria
• Allergic Rhinoconjunctivitis
• Allergic Asthma
• Mast Cell Activation Syndrome

Phase 1 Clinical Trial Data (Healthy Volunteers):

• BLU-808 was well-tolerated at all doses tested.
• No serious adverse events, discontinuations, or dose modifications due to adverse events, and no significant changes in laboratory measures were observed.
• Demonstrated dose-dependent serum tryptase reductions exceeding 80%, indicating mast cell target engagement.
• Exhibited a half-life supporting once-daily oral dosing and sustained target coverage across a range of doses.

Observed Adverse Events (Phase 1 Healthy Volunteer Trial):

• No treatment-emergent adverse events greater than Grade 1 were reported in the multiple-ascending dose cohorts (1-12 mg once daily).