Modality
ERT
MOA
WRNi
Target
CFTR
Pathway
Amyloid
AMLALS
Development Pipeline
Preclinical
Apr 2017
→ Oct 2030
PreclinicalCurrent
NCT03884394
2,865 pts·AML
2024-11→2030-10·Completed
NCT04997092
2,447 pts·AML
2017-04→2028-04·Recruiting
5,312 total pts1 indication
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2028-04-222.1y awayInterim· AML
2030-10-244.6y awayInterim· AML
Trial Timeline
2017Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q4
Preclinical
Recruit…
Preclinical
Complet…
Catalysts
Interim
2028-04-22 · 2.1y away
AML
Interim
2030-10-24 · 4.6y away
AML
RecruitingCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03884394 | Preclinical | AML | Completed | 2865 | DAS28 |
| NCT04997092 | Preclinical | AML | Recruiting | 2447 | BodyWt |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| ABB-1817 | AbbVie | Phase 3 | SHP2 | |
| Datoglumide | AbbVie | Approved | CFTR | |
| BMY-7975 | Bristol-Myers Squibb | Phase 1 | MALT1 | |
| SNY-7254 | Sanofi | Phase 2 | CFTR | |
| SNY-5894 | Sanofi | Approved | CFTR | |
| GSK-4334 | GSK | Preclinical | CD47 | |
| AMG-2752 | Amgen | Preclinical | MDM2 | |
| REG-7737 | Regeneron | Preclinical | CD123 | |
| VRT-5853 | Vertex Pharma | Phase 3 | Aβ |