An expansive pipeline
We are focused on the rapid design and development of precision therapies for people with cancer and rare diseases.
Our 2020 Blueprint
2
marketed products
4
pending
marketing
applications
marketing
applications
6
clinical-stage
therapeutic
candidates
therapeutic
candidates
8
research
programs
programs
Our “2020 Blueprint” strategy establishes a planned path to transform Blueprint Medicines, by the end of 2020, into a global commercial enterprise focused on delivering a portfolio of precision therapies to patients with cancer and rare diseases. See our press release.
Our portfolio
| Genetically defined cancers | ||
|---|---|---|
| Genetically defined cancers | Molecular target | Clinical phase |
| Genetically defined cancers | ||
| Genetically defined cancers |
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
|
test |
|
Avapritinib: PDGFRA 1
»Advanced PDGFRA Exon 18 mutant GIST
3 - Late-stage development
|
Avapritinib: PDGFRA
NDA planned 1H 2019
|
3Late-stage development |
|
Avapritinib: KIT and PDGFRA 1
»Advanced 4L GIST
3 - Late-stage development
|
Avapritinib: KIT and PDGFRA
NDA planned 1H 2019
|
3Late-stage development |
|
Avapritinib: KIT and PDGFRA 1
»Advanced 3L GIST
3 - Late-stage development
|
Avapritinib: KIT and PDGFRA
NDA planned 2020
|
3Late-stage development |
|
Avapritinib: KIT and PDGFRA 1
»Advanced 2L GIST
2 - Early-stage development
|
Avapritinib: KIT and PDGFRA
Trial planned 2H 2019
|
2Early-stage development |
|
BLU-667: RET 1
»Advanced 2L RET-fusion NSCLC
3 - Late-stage development
|
BLU-667: RET
NDA planned 1H 2020
|
3Late-stage development |
|
BLU-667: RET 1
»Advanced 1L RET-fusion NSCLC
0 - None
|
BLU-667: RET
Trial planned 2H 2019
|
0None |
|
BLU-667: RET 1
»Advanced EGFR-mutant NSCLC (+osimertinib)
0 - None
|
BLU-667: RET
Trial planned 2H 2019
|
0None |
|
BLU-667: RET 1
»Advanced 2L RET-mutant MTC
3 - Late-stage development
|
BLU-667: RET
NDA planned 1H 2020
|
3Late-stage development |
|
BLU-667: RET 1
»Other RET-altered solid tumors
2 - Early-stage development
|
BLU-667: RET
|
2Early-stage development |
|
BLU-554: FGFR4 1
»Advanced HCC
2 - Early-stage development
|
BLU-554: FGFR4
|
2Early-stage development |
|
BLU-554 1
»Advanced HCC (+CS-1001)
0 - None
|
BLU-554
Trial planned 2H 2019
|
0None |
|
4 WHOLLY OWNED PROGRAMS
»Undisclosed Targets
1 - Discovery
|
4 WHOLLY OWNED PROGRAMS
|
1Discovery |
| Rare diseases | ||
|---|---|---|
| Rare diseases | Molecular target | Clinical phase |
| Rare diseases | ||
| Rare diseases |
4 WHOLLY OWNED PROGRAMS
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
|
test |
|
Avapritinib: KIT D816V 1
»Advanced systemic mastocytosis
3 - Late-stage development
|
Avapritinib: KIT D816V
NDA planned 2020
|
3Late-stage development |
|
Avapritinib: KIT D816V 1
»Indolent and smoldering systemic mastocytosis
3 - Late-stage development
|
Avapritinib: KIT D816V
|
3Late-stage development |
|
BLU-782: ALK2 2
»Fibrodysplasia ossificans progressiva
2 - Early-stage development
|
BLU-782: ALK2
|
2Early-stage development |
| Cancer immunotherapy | ||
|---|---|---|
| Cancer immunotherapy | Molecular target | Clinical phase |
| Cancer immunotherapy | ||
| Cancer immunotherapy |
BLU-782: ALK2
Discovery
Early stage development
Late stage development
Regulatory submission
Approved
|
test |
|
UP TO 5 PROGRAMS 3
»Undisclosed targets
1 - Discovery
|
UP TO 5 PROGRAMS
|
1Discovery |
Updated as of February 26, 2019.
- 1CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, BLU-554 and BLU-667 in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains all rights in the rest of the world.
- 2Phase 1 trial in healthy volunteers.
- 3In collaboration with Roche. Blueprint Medicines has U.S. commercial rights for up to two programs. Roche has worldwide commercialization rights for up to three programs and ex-U.S. commercialization rights for up to two programs.
Definitions: 1L, first-line; 2L, second-line; 3L, third-line; 4L, fourth-line; GIST, gastrointestinal stromal tumors; HCC, hepatocellular carcinoma; MTC, medullary thyroid cancer; NSCLC, non-small cell lung cancer.