Modality
ERT
MOA
RAS(ON)i
Target
CD123
Pathway
Lipid Met
RettLN
Development Pipeline
Preclinical
~Dec 2013
→ ~Mar 2015
Phase 1
~Jun 2015
→ ~Sep 2016
Phase 2
~Dec 2016
→ ~Mar 2018
Phase 3
~Jun 2018
→ ~Sep 2019
NDA/BLA
Dec 2019
→ Sep 2026
NDA/BLACurrent
NCT04971313
1,428 pts·LN
2019-12→2026-09·Not yet recruiting
1,428 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (1)
2026-09-145mo awayPh3 Readout· LN
Trial Timeline
2020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q4
NDA/BLA
Not yet…
Catalysts
Ph3 Readout
2026-09-14 · 5mo away
LN
Not yet recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04971313 | NDA/BLA | LN | Not yet recr... | 1428 | UPCR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| MRK-6781 | Merck & Co | Approved | TIGIT | |
| SNY-9073 | Sanofi | Phase 2 | B7-H3 | |
| REG-7737 | Regeneron | Preclinical | CD123 | |
| Tixarelsin | Vertex Pharma | Phase 1 | ALK | |
| Fixatuximab | Legend Biotech | Preclinical | CFTR | |
| ION-1378 | Ionis | NDA/BLA | TROP-2 | |
| 180-3597 | Innovent Bio | Preclinical | SHP2 | |
| Ivotuximab | Madrigal Pharma | Phase 2 | CD123 | |
| Zorizumab | Beam | Approved | CD123 | |
| CGO-2579 | CG Oncology | Approved | TIGIT |