An expansive pipeline
We are focused on the efficient design and development of medicines for people with mast cell disorders or solid tumors.
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
3 - Late stage development
|
|
3Late stage development |
|
BLU-808: Wild-type KIT
|
0None | |
|
»Chronic urticaria
2 - Early stage development
|
|
2Early stage development |
|
»Allergic rhinoconjunctivitis
2 - Early stage development
|
|
2Early stage development |
|
»Allergic asthma
1 - Discovery
|
|
1Discovery |
|
»Mast cell activation syndrome
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 |
|
AYVAKIT (avapritinib): PDGFRA
|
0None | |
|
»PDGFRA GIST
1,4
5 - Commercial
|
|
5Commercial |
|
BLU-956 (next gen): CDK2
|
0None | |
|
»HR+, HER2- breast cancer and CCNE1+ solid tumors
1 - Discovery
|
|
1Discovery |
|
Targeted protein degrader: CDK2
|
0None | |
|
»HR+, HER2- breast cancer and CCNE1+ solid tumors
1 - Discovery
|
|
1Discovery |
|
Targeted protein degrader: CDK4
|
0None | |
|
»HR+, HER2- breast cancer
1 - Discovery
|
|
1Discovery |
| Additional research | ||
|---|---|---|
| Additional research | Molecular target | Clinical phase |
| Additional research | ||
| Additional research |
Discovery
Early stage
development
Late stage
development
Commercial
|
test |
|
Programs: undisclosed
1 - Discovery
|
Programs: undisclosed
|
1Discovery |
ongoing or completed
planned
Updated as of May 1, 2025.
- 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.
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