Modality
siRNA
MOA
CD3xCD20
Target
JAK2
Pathway
RNA Splicing
Huntington's
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
→ ~Aug 2021
Approved
Nov 2021
→ Mar 2030
ApprovedCurrent
NCT03124968
738 pts·Huntington's
2021-11→2028-12·Recruiting
NCT05261282
48 pts·Huntington's
2025-02→2030-03·Active
786 total pts1 indication
CompletedCurrentUpcoming
Catalysts (2)
2028-12-012.7y awayPh3 Readout· Huntington's
2030-03-013.9y awayPh3 Readout· Huntington's
Trial Timeline
Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2
Approved
Recruit…
Approved
Active
Catalysts
Ph3 Readout
2028-12-01 · 2.7y away
Huntington's
Ph3 Readout
2030-03-01 · 3.9y away
Huntington's
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03124968 | Approved | Huntington's | Recruiting | 738 | BodyWt |
| NCT05261282 | Approved | Huntington's | Active | 48 | PFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-1184 | Eli Lilly | NDA/BLA | C5 | |
| Zanuderotide | Novartis | Phase 1/2 | VEGF | |
| NVO-1361 | Novo Nordisk | Phase 3 | CD38 | |
| Zorizanubrutinib | Novo Nordisk | Phase 1 | BTK | |
| Pexamavacamten | Daiichi Sankyo | NDA/BLA | TIGIT | |
| GMA-5010 | Genmab | Phase 3 | JAK2 | |
| ITC-1028 | Intra-Cellular | Phase 2 | BCMA | |
| Teravorutinib | Illumina | Phase 2 | JAK2 | |
| AXS-4984 | Axsome | Preclinical | JAK2 | |
| Capirapivir | TG Therapeutics | Approved | JAK2 |