Modality
Degrader
MOA
JAK1/2i
Target
PLK4
Pathway
Sphingolipid
T2DPNH
Development Pipeline
Preclinical
~May 2014
→ ~Aug 2015
Phase 1
~Nov 2015
→ ~Feb 2017
Phase 2
~May 2017
→ ~Aug 2018
Phase 3
~Nov 2018
→ ~Feb 2020
NDA/BLA
May 2020
→ Jan 2027
NDA/BLACurrent
NCT03912777
2,718 pts·PNH
2020-05→2027-01·Recruiting
NCT05024864
1,525 pts·PNH
2020-10→2025-10·Not yet recruiting
4,243 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-10-255mo agoPh3 Readout· PNH
2027-01-1610mo awayPh3 Readout· PNH
Trial Timeline
Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027
NDA/BLA
Recruit…
NDA/BLA
Not yet…
Catalysts
Ph3 Readout
2025-10-25 · 5mo ago
PNH
Ph3 Readout
2027-01-16 · 10mo away
PNH
RecruitingNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03912777 | NDA/BLA | PNH | Recruiting | 2718 | ORR |
| NCT05024864 | NDA/BLA | PNH | Not yet recr... | 1525 | UPCR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Fixanesiran | AbbVie | Preclinical | RET | |
| Motavorutinib | Amgen | Phase 3 | CDK4/6 | |
| VRT-9481 | Vertex Pharma | Phase 1/2 | Menin | |
| Bemanesiran | BioNTech | Phase 3 | PLK4 | |
| Sovarelsin | BioMarin | Phase 2 | CFTR | |
| Pexaosocimab | United Therapeutics | NDA/BLA | PLK4 | |
| Polalucimab | Jazz Pharma | Preclinical | PLK4 | |
| Niramavacamten | Hansoh Pharma | Phase 2 | SGLT2 | |
| ILM-2412 | Illumina | Phase 3 | PLK4 | |
| ROI-3353 | Roivant Sciences | Phase 1/2 | PLK4 |