Modality
Gene Therapy
MOA
GLP-1ag
Target
PLK4
Pathway
Proteasome
PAH
Development Pipeline
Preclinical
~Nov 2017
→ ~Feb 2019
Phase 1
~May 2019
→ ~Aug 2020
Phase 2
~Nov 2020
→ ~Feb 2022
Phase 3
~May 2022
→ ~Aug 2023
NDA/BLA
Nov 2023
→ Jul 2028
NDA/BLACurrent
NCT03410549
2,302 pts·PAH
2025-06→2026-02·Active
NCT07565348
146 pts·PAH
2023-11→2028-07·Terminated
2,448 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-02-072mo agoPh3 Readout· PAH
2028-07-132.3y awayPh3 Readout· PAH
Trial Timeline
Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3
NDA/BLA
Termina…
NDA/BLA
Active
Catalysts
Ph3 Readout
2026-02-07 · 2mo ago
PAH
Ph3 Readout
2028-07-13 · 2.3y away
PAH
ActiveTerminated|StartCompletionToday
Trials (2)
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-8662 | Pfizer | NDA/BLA | CD19 | |
| NVO-7840 | Novo Nordisk | Phase 2/3 | B7-H3 | |
| MRN-7601 | Moderna | Phase 2 | IL-13 | |
| Bemanesiran | BioNTech | Phase 3 | PLK4 | |
| ARG-1250 | Argenx | Phase 2 | C5 | |
| Peminaritide | Sarepta | NDA/BLA | PLK4 | |
| Pexaosocimab | United Therapeutics | NDA/BLA | PLK4 | |
| Polalucimab | Jazz Pharma | Preclinical | PLK4 | |
| ILM-5680 | Illumina | Phase 2 | BET | |
| ILM-2412 | Illumina | Phase 3 | PLK4 |