Modality
ERT
MOA
USP1i
Target
PSMA
Pathway
Tau
TTR AmyloidosisCF
Development Pipeline
Preclinical
~Nov 2011
→ ~Feb 2013
Phase 1
~May 2013
→ ~Aug 2014
Phase 2
~Nov 2014
→ ~Feb 2016
Phase 3
~May 2016
→ ~Aug 2017
NDA/BLA
Nov 2017
→ Apr 2030
NDA/BLACurrent
NCT03582151
1,272 pts·CF
2024-02→2030-04·Completed
NCT06859716
1,366 pts·CF
2017-11→2030-02·Not yet recruiting
2,638 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2030-02-173.9y awayPh3 Readout· CF
2030-04-134.0y awayPh3 Readout· CF
Trial Timeline
Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3
NDA/BLA
Not yet…
NDA/BLA
Complet…
Catalysts
Ph3 Readout
2030-02-17 · 3.9y away
CF
Ph3 Readout
2030-04-13 · 4.0y away
CF
CompletedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03582151 | NDA/BLA | CF | Completed | 1272 | BodyWt |
| NCT06859716 | NDA/BLA | CF | Not yet recr... | 1366 | PASI75 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| AZN-3724 | AstraZeneca | Preclinical | PSMA | |
| Doxacagene | Sanofi | Approved | PSMA | |
| SNY-2934 | Sanofi | Phase 3 | KRASG12D | |
| GSK-2051 | GSK | Phase 1 | PSMA | |
| AMG-9052 | Amgen | Phase 2/3 | CDK2 | |
| GIL-2445 | Gilead Sciences | Phase 2/3 | PSMA | |
| Datozumab | Regeneron | Phase 1 | B7-H3 | |
| BII-1564 | Biogen | Phase 2 | PSMA | |
| BGN-3859 | BeiGene | Approved | PSMA | |
| GMA-729 | Genmab | Phase 2 | BCL-2 |