Modality
siRNA
MOA
USP1i
Target
BCL-2
Pathway
T-cell
SLETTR AmyloidosisMDS
Development Pipeline
Preclinical
~Jun 2018
→ ~Sep 2019
Phase 1
~Dec 2019
→ ~Mar 2021
Phase 2
Jun 2021
→ Nov 2031
Phase 2Current
NCT06861752
2,200 pts·TTR Amyloidosis
2021-06→2031-11·Active
2,200 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-05-081mo awayPh1 Dose Esc· TTR Amyloidosis
2031-11-245.7y awayPh2 Data· TTR Amyloidosis
Trial Timeline
Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
P2
Active
Catalysts
Ph1 Dose Esc
2026-05-08 · 1mo away
TTR Amyloidosis
Ph2 Data
2031-11-24 · 5.7y away
TTR Amyloidosis
Active|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06861752 | Phase 2 | TTR Amyloidosis | Active | 2200 | CfB |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-8550 | Roche | Approved | BCL-2 | |
| SNY-2934 | Sanofi | Phase 3 | KRASG12D | |
| AMG-9052 | Amgen | Phase 2/3 | CDK2 | |
| Datozumab | Regeneron | Phase 1 | B7-H3 | |
| Rimaglumide | Biogen | Phase 1/2 | BCL-2 | |
| ARG-4339 | Argenx | Phase 2 | BCL-2 | |
| HAL-996 | Halozyme | Phase 1/2 | BCL-2 | |
| Pexanaritide | Innovent Bio | Preclinical | SGLT2 | |
| ROI-9675 | Roivant Sciences | NDA/BLA | HER2 | |
| Bemaderotide | Revolution Medicines | Phase 3 | BCL-2 |