Modality
Gene Editing
MOA
Anti-Aβ
Target
TYK2
Pathway
Epigenetic
PAH
Development Pipeline
Preclinical
~Mar 2021
→ ~Jun 2022
Phase 1
~Sep 2022
→ ~Dec 2023
Phase 2
Mar 2024
→ Jun 2027
Phase 2Current
NCT03987606
1,839 pts·PAH
2024-03→TBD·Recruiting
NCT07336027
451 pts·PAH
2025-04→2027-06·Completed
2,290 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2027-06-251.2y awayPh3 Readout· PAH
Trial Timeline
Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3
P2/3
Recruit…
P2/3
Complet…
Catalysts
Ph3 Readout
2027-06-25 · 1.2y away
PAH
RecruitingCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03987606 | Phase 2/3 | PAH | Recruiting | 1839 | DOR |
| NCT07336027 | Phase 2/3 | PAH | Completed | 451 | BodyWt |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxacilimab | Pfizer | Phase 2 | TYK2 | |
| LLY-8369 | Eli Lilly | Approved | Menin | |
| MRK-3732 | Merck & Co | Phase 1 | TYK2 | |
| ABB-8696 | AbbVie | Phase 3 | BCMA | |
| BAY-6035 | Bayer | Phase 1 | C5 | |
| Zenotinib | BioNTech | Phase 1 | CGRP | |
| COR-9566 | Corcept | Approved | TYK2 | |
| RLA-6817 | Relay Therapeutics | Phase 3 | TIGIT | |
| Lisoosocimab | Zealand Pharma | Approved | KIF18A | |
| PRA-419 | Praxis Precision | Approved | Cl18.2 |