An expansive pipeline

We are focused on the efficient design and development of medicines for people with mast cell disorders or solid tumors.

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Rapidly advancing pipeline

Mast cell disorders
Mast cell disorders Molecular target Clinical phase
Mast cell disorders
Mast cell disorders
Discovery
Early stage
development
Late stage
development
Commercial
test
AYVAKIT® (avapritinib): KIT D816V
3Late stage development
»Indolent SM 1,2
5 - Commercial
5Commercial
»Advanced SM 1,3
5 - Commercial
5Commercial
Elenestinib (next gen): KIT D816V
0None
»Indolent SM
2 - Early stage development
2Early stage development
BLU-808: Wild-type KIT
0None
»Chronic urticaria
2 - Early stage development
2Early stage development
Undisclosed mast cell
targets/modalities
1 - Discovery
Undisclosed mast cell
targets/modalities
1Discovery
Solid tumors
Solid tumors Molecular target Clinical phase
Solid tumors
Solid tumors Undisclosed mast cell
targets/modalities
Discovery
Early stage
development
Late stage
development
Commercial
test
AYVAKIT (avapritinib): PDGFRA
0None
»PDGFRA GIST 1,4
5 - Commercial
5Commercial
BLU-222: CDK2
0None
»HR+, HER2- breast cancer
2 - Early stage development
2Early stage development
»Other CDK2 vulnerable cancers
2 - Early stage development
2Early stage development
BLU-956 (next gen): CDK2
0None
»HR+, HER2- breast cancer
1 - Discovery
1Discovery
Targeted protein degrader: CDK2
0None
»HR+, HER2- breast cancer
1 - Discovery
1Discovery
Targeted protein degrader: undisclosed
1 - Discovery
Targeted protein degrader: undisclosed
1Discovery
Additional research
Additional research Molecular target Clinical phase
Additional research
Additional research Targeted protein degrader: undisclosed
Discovery
Early stage
development
Late stage
development
Commercial
test
Programs: undisclosed
1 - Discovery
Programs: undisclosed
1Discovery

ongoing or completed

planned

Updated as of August 1, 2024.

  • 1CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib in Greater China.
  • 2Approved in the U.S. for adults with indolent SM. Approved in Europe (AYVAKYT®) for adults with indolent SM with moderate to severe symptoms inadequately controlled on symptomatic treatment.
  • 3Approved in the U.S. for adults with advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). Approved in Europe (AYVAKYT) for adults with ASM, SM-AHN or MCL, after at least one systemic therapy.
  • 4Approved in the U.S. for adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Approved in Europe (AYVAKYT) for adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.


GIST = gastrointestinal stromal tumors. SM = systemic mastocytosis.

Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation.

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