pp-127 Capsules
Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Indolent systemic mastocytosis (ISM); smouldering SM — adults with moderate-to-severe symptoms
Clinical stage: Phase 2/3
Modality: Small molecule
Mechanism / Target: Highly selective KIT D816V inhibitor — blocks constitutive KIT activation in mast cells; designed with CNS-sparing PK profile
Route / form: Oral capsule (once daily)
Differentiation: CNS-sparing design avoids cognitive adverse effects associated with avapritinib; targets largest mastocytosis population (ISM ~90% of SM cases)
Manufacturer #39164
Precision therapy company specialising in genomically defined cancers and rare diseases driven by kinase mutations. Leads in KIT/PDGFRA inhibitor development with FDA-approved and pipeline agents. Wholly owned CNS-sparing asset targeting mastocytosis unmet need.
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