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
Regulatory submission
Approved
test
AYVAKIT® (avapritinib): KIT
3Late-stage development
»Advanced SM 1,2
5 - Approved
5Approved
»Indolent SM 1
4 - Regulatory submission
4Regulatory submission
Elenestinib (BLU-263): KIT
0None
»Indolent SM
3 - Late-stage development
3Late-stage development
Wild-type KIT research program
0None
»Mast cell disorders
1 - Discovery
1Discovery
Lung cancer
Lung cancer Molecular target Clinical phase
Lung cancer
Lung cancer
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
GAVRETO® (pralsetinib): RET
0None
»RET+ NSCLC 1,3,4
5 - Approved
5Approved
BLU-945: EGFR
0None
»EGFR+ NSCLC 5
2 - Early-stage development
2Early-stage development
BLU-525: EGFR
0None
»EGFR+ NSCLC 5
1 - Discovery
1Discovery
BLU-451: EGFR exon 20 insertions
0None
»EGFR+ NSCLC
2 - Early-stage development
2Early-stage development
Breast cancer
Breast cancer Molecular target Clinical phase
Breast cancer
Breast cancer
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
BLU-222: CDK2
0None
»ER+/HER2- breast cancer
2 - Early-stage development
2Early-stage development
Additional genomically
defined cancers
Additional genomically
defined cancers
Molecular target Clinical phase
Additional genomically
defined cancers
Additional genomically
defined cancers
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
AYVAKIT: PDGFRA
5Approved
»PDGFRA GIST 1,6
5 - Approved
5Approved
GAVRETO: RET
3Late-stage development
»RET+ thyroid cancer 1,3,7
5 - Approved
5Approved
»Other RET+ solid tumors 1,3
3 - Late-stage development
3Late-stage development
BLU-222: CDK2
0None
»CDK2-vulnerable cancers
2 - Early-stage development
2Early-stage development
Cancer immunotherapy
Cancer immunotherapy Molecular target Clinical phase
Cancer immunotherapy
Cancer immunotherapy
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
BLU-852: MAP4K1
0None
»Advanced cancers 3
1 - Discovery
1Discovery
Undisclosed research program
0None
»Advanced cancers 3
1 - Discovery
1Discovery
Research
Research Molecular target Clinical phase
Research
Research
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
test
Multiple undisclosed research programs
1 - Discovery
Multiple undisclosed research programs
1Discovery

ongoing or completed

planned

Updated as of January 23, 2023.

  • 1CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib and pralsetinib in Greater China.
  • 2Approved 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.
  • 3In collaboration with Roche.
  • 4Received U.S. accelerated approval for adults with metastatic RET fusion-positive NSCLC. Received conditional marketing authorization in Europe for adults with advanced RET fusion-positive NSCLC not previously treated with a RET inhibitor.
  • 5Zai Lab has exclusive rights to develop and commercialize BLU-945 and BLU-525 in Greater China.
  • 6Approved 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.
  • 7Received U.S. accelerated approval for advanced or metastatic RET-mutant medullary thyroid cancer and RET fusion-positive thyroid cancer.


GIST = gastrointestinal stromal tumors. NSCLC = non-small cell lung cancer. SM = systemic mastocytosis.

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