Modality
siRNA
MOA
Anti-Aβ
Target
CD20
Pathway
Ferroptosis
Hemophilia A
Development Pipeline
Preclinical
~Jul 2017
→ ~Oct 2018
Phase 1
~Jan 2019
→ ~Apr 2020
Phase 2
~Jul 2020
→ ~Oct 2021
Phase 3
~Jan 2022
→ ~Apr 2023
NDA/BLA
Jul 2023
→ May 2027
NDA/BLACurrent
NCT03765634
2,233 pts·Hemophilia A
2024-10→2027-05·Active
NCT06522857
204 pts·Hemophilia A
2023-07→2026-07·Terminated
2,437 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-07-083mo awayPh3 Readout· Hemophilia A
2027-05-011.1y awayPh3 Readout· Hemophilia A
Trial Timeline
Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2
NDA/BLA
Termina…
NDA/BLA
Active
Catalysts
Ph3 Readout
2026-07-08 · 3mo away
Hemophilia A
Ph3 Readout
2027-05-01 · 1.1y away
Hemophilia A
ActiveTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03765634 | NDA/BLA | Hemophilia A | Active | 2233 | ORR |
| NCT06522857 | NDA/BLA | Hemophilia A | Terminated | 204 | PFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-8637 | Johnson & Johnson | NDA/BLA | CD20 | |
| LLY-8369 | Eli Lilly | Approved | Menin | |
| Lisolucimab | Novartis | Approved | CD20 | |
| ABB-8985 | AbbVie | Phase 2 | CD20 | |
| ABB-8696 | AbbVie | Phase 3 | BCMA | |
| BAY-6035 | Bayer | Phase 1 | C5 | |
| Zenotinib | BioNTech | Phase 1 | CGRP | |
| ARG-3265 | Argenx | Phase 1/2 | CD20 | |
| CRS-6525 | CRISPR Therapeutics | Phase 2 | CD20 | |
| RLA-6817 | Relay Therapeutics | Phase 3 | TIGIT |