Modality
ADC
MOA
CAR-T CD19
Target
APOC3
Pathway
NF-κB
SCDTTR AmyloidosisPBC
Development Pipeline
Preclinical
Jul 2019
→ Jul 2031
PreclinicalCurrent
NCT03045181
2,820 pts·TTR Amyloidosis
2019-07→2026-05·Not yet recruiting
NCT03375686
2,314 pts·TTR Amyloidosis
2021-05→2031-07·Active
5,134 total pts1 indication
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-05-111mo awayInterim· TTR Amyloidosis
2031-07-075.3y awayInterim· TTR Amyloidosis
Trial Timeline
Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
Preclinical
Not yet…
Preclinical
Active
Catalysts
Interim
2026-05-11 · 1mo away
TTR Amyloidosis
Interim
2031-07-07 · 5.3y away
TTR Amyloidosis
ActiveNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03045181 | Preclinical | TTR Amyloidosis | Not yet recr... | 2820 | PANSS |
| NCT03375686 | Preclinical | TTR Amyloidosis | Active | 2314 | OS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-1944 | Pfizer | NDA/BLA | APOC3 | |
| Doxacapivasertib | Eli Lilly | Phase 3 | APOC3 | |
| RHH-1546 | Roche | Phase 2/3 | C5 | |
| Datoglumide | AbbVie | Approved | CFTR | |
| AZN-6294 | AstraZeneca | Phase 2/3 | APOC3 | |
| Zanutinib | Bristol-Myers Squibb | Phase 2/3 | APOC3 | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| BII-4342 | Biogen | Phase 2/3 | CGRP | |
| ALN-5628 | Alnylam | Phase 1/2 | APOC3 | |
| INC-1582 | Incyte | Phase 1/2 | APOC3 |