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 mutant GIST
3 - Late-stage development
|
Avapritinib: PDGFRA
NDA/MAA submitted
|
3Late-stage development |
|
Avapritinib: KIT and PDGFRA 1
»Advanced 4L GIST
3 - Late-stage development
|
Avapritinib: KIT and PDGFRA
NDA/MAA submitted
|
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 |
|
Pralsetinib (BLU-667): RET 1
»Advanced 2L RET-fusion NSCLC
3 - Late-stage development
|
Pralsetinib (BLU-667): RET
NDA planned Q1 2020
|
3Late-stage development |
|
Pralsetinib: RET 1
»Advanced 1L RET-fusion NSCLC
0 - None
|
Pralsetinib: RET
Trial planned 2H 2019
|
0None |
|
Pralsetinib: RET 1
»Advanced EGFR-mutant NSCLC (+osimertinib)
0 - None
|
Pralsetinib: RET
Trial planned 2H 2019
|
0None |
|
Pralsetinib: RET 1
»Advanced 2L RET-mutant MTC
3 - Late-stage development
|
Pralsetinib: RET
NDA planned 1H 2020
|
3Late-stage development |
|
Pralsetinib: RET 1
»Other RET-altered solid tumors
2 - Early-stage development
|
Pralsetinib: RET
|
2Early-stage development |
|
Fisogatinib (BLU-554): FGFR4 1
»Advanced HCC
2 - Early-stage development
|
Fisogatinib (BLU-554): FGFR4
|
2Early-stage development |
|
Fisogatinib 1
»Advanced HCC (+CS-1001)
0 - None
|
Fisogatinib
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 Q1 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 August 1, 2019
- 1CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib 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 ongoing; phase 2 trial in patients with FOP planned Q4 2019.
- 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.