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
| Genomically defined cancers | ||
|---|---|---|
| Genomically defined cancers | Molecular target | Clinical phase |
| Genomically defined cancers | ||
| Genomically defined cancers |
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
|
test |
|
Avapritinib: PDGFRA
|
5Approved | |
|
»PDGFRA GIST
1,2,3
5 - Approved
|
|
5Approved |
|
Pralsetinib: RET
|
3Late-stage development | |
|
»RET+ NSCLC
1,2,4,5
5 - Approved
|
|
5Approved |
|
»EGFR+ NSCLC (+osimertinib)
1,2,4
0 - None
|
|
0None |
|
»RET+ MTC
1,2,4
3 - Late-stage development
|
NDA
|
3Late-stage development |
|
»RET+ thyroid cancer
1,2,4
4 - Regulatory submission
|
NDA
|
4Regulatory submission |
|
»Other RET-altered solid tumors
1,2,4
2 - Early-stage development
|
|
2Early-stage development |
|
Fisogatinib: FGFR4
|
2Early-stage development | |
|
»Advanced HCC
2
2 - Early-stage development
|
|
2Early-stage development |
|
»Advanced HCC (+CS1001)
2
2 - Early-stage development
|
|
2Early-stage development |
|
BLU-945: EGFR+ T790M/C797S triple mutant
|
0None | |
|
»EGFR+ NSCLC
1
1 - Discovery
|
|
1Discovery |
|
Research program: EGFR+ C797S double mutant
|
0None | |
|
»EGFR+ NSCLC
1
1 - Discovery
|
|
1Discovery |
|
Research programs: 2 undisclosed targets
1 - Discovery
|
Research programs: 2 undisclosed targets
|
1Discovery |
| Rare diseases | ||
|---|---|---|
| Rare diseases | Molecular target | Clinical phase |
| Rare diseases | ||
| Rare diseases |
Research programs: 2 undisclosed targets
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
|
test |
|
Avapritinib: KIT
|
3Late-stage development | |
|
»Advanced SM
2
3 - Late-stage development
|
NDA
|
3Late-stage development |
|
»Indolent SM
2
3 - Late-stage development
|
|
3Late-stage development |
|
BLU-263: KIT
|
0None | |
|
»Indolent SM
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 |
|
Research program: MAP4K1 6
1 - Discovery
|
Research program: MAP4K1
|
1Discovery |
|
Research programs: 3 undisclosed targets 6
1 - Discovery
|
Research programs: 3 undisclosed targets
|
1Discovery |
ongoing or completed
planned
Updated as of September 25, 2020.
- 1Unresectable or metastatic disease.
- 2CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan.
- 3Approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Received conditional marketing authorization in Europe for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
- 4In collaboration with Roche. Blueprint Medicines and Roche have co-exclusive rights to develop and commercialize pralsetinib in the U.S., and Roche has exclusive rights to develop and commercialize pralsetinib outside the U.S., excluding the CStone territory.
- 5Received accelerated approval in the U.S. for the treatment of adults with metastatic RET fusion-positive NSCLC. Continued approval may be contingent on a confirmatory trial. The proposed indication for the MAA is locally advanced or metastatic RET fusion-positive NSCLC previously treated with platinum-based chemotherapy.
- 6In collaboration with Roche. Blueprint Medicines has U.S. commercial rights for up to two programs. Roche has worldwide commercialization rights for up to two programs and ex-U.S. commercialization rights for up to two programs.
GIST = gastrointestinal stromal tumors. HCC = hepatocellular carcinoma. MAA = marketing authorization application. MTC = medullary thyroid cancer. NDA = new drug application. NSCLC = non-small cell lung cancer. SM = systemic mastocytosis.
Not for promotional use.