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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