Modality
ASO
MOA
CAR-T CD19
Target
VEGF
Pathway
Complement
GISTSCDPNH
Development Pipeline
Preclinical
~Dec 2014
→ ~Mar 2016
Phase 1
~Jun 2016
→ ~Sep 2017
Phase 2
~Dec 2017
→ ~Mar 2019
Phase 3
~Jun 2019
→ ~Sep 2020
NDA/BLA
Dec 2020
→ Jun 2030
NDA/BLACurrent
NCT08045119
526 pts·GIST
2022-03→2030-06·Terminated
NCT05352206
1,238 pts·PNH
2020-12→2027-06·Recruiting
1,764 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (2)
2027-06-171.2y awayPh3 Readout· PNH
2030-06-144.2y awayPh3 Readout· GIST
Trial Timeline
Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3
NDA/BLA
Recruit…
NDA/BLA
Termina…
Catalysts
Ph3 Readout
2027-06-17 · 1.2y away
PNH
Ph3 Readout
2030-06-14 · 4.2y away
GIST
RecruitingTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08045119 | NDA/BLA | GIST | Terminated | 526 | OS |
| NCT05352206 | NDA/BLA | PNH | Recruiting | 1238 | DAS28 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-1546 | Roche | Phase 2/3 | C5 | |
| Zanuderotide | Novartis | Phase 1/2 | VEGF | |
| Datoglumide | AbbVie | Approved | CFTR | |
| TAK-8730 | Takeda | Phase 2 | VEGF | |
| GIL-2011 | Gilead Sciences | Preclinical | VEGF | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| Bemazumab | Regeneron | Phase 1 | VEGF | |
| BII-4342 | Biogen | Phase 2/3 | CGRP | |
| Pemicapivasertib | Legend Biotech | Phase 3 | VEGF | |
| Sematapinarof | Legend Biotech | NDA/BLA | VEGF |