Modality
ERT
MOA
CAR-T CD19
Target
GIP-R
Pathway
RNA Splicing
MGPAH
Development Pipeline
Preclinical
Nov 2022
→ Sep 2028
PreclinicalCurrent
NCT05795962
1,764 pts·PAH
2022-11→2025-04·Recruiting
NCT08547622
259 pts·PAH
2024-01→2028-09·Terminated
2,023 total pts1 indication
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-04-2411mo agoInterim· PAH
2028-09-082.4y awayInterim· PAH
Trial Timeline
Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
Preclinical
Recruit…
Preclinical
Termina…
Catalysts
Interim
2025-04-24 · 11mo ago
PAH
Interim
2028-09-08 · 2.4y away
PAH
RecruitingTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05795962 | Preclinical | PAH | Recruiting | 1764 | eGFR |
| NCT08547622 | Preclinical | PAH | Terminated | 259 | Biomarker |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-5094 | Johnson & Johnson | Phase 2/3 | GIP-R | |
| RHH-1546 | Roche | Phase 2/3 | C5 | |
| RHH-3592 | Roche | Phase 1/2 | GIP-R | |
| Datoglumide | AbbVie | Approved | CFTR | |
| AZN-5978 | AstraZeneca | Phase 2/3 | GIP-R | |
| Ivotuximab | GSK | NDA/BLA | GIP-R | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| Elrarapivir | Regeneron | Phase 1 | GIP-R | |
| BII-4342 | Biogen | Phase 2/3 | CGRP | |
| Capifutibatinib | Intra-Cellular | Phase 3 | GIP-R |