An expansive pipeline

We are focused on the rapid design and development of precision therapies for people with cancer and rare diseases.

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)1: KIT D816V
3Late stage development
»Indolent SM 2,3
5 - Commercial
5Commercial
»Advanced SM 2,4
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
1 - Discovery
1Discovery
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
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 January 8, 2024.

  • 1Also approved 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.
  • 2CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib in Greater China.
  • 3Approved 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.
  • 4Approved 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.


GIST = gastrointestinal stromal tumors. SM = systemic mastocytosis.

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