Modality
Gene Editing
MOA
WRNi
Target
Aβ
Pathway
DDR
NarcolepsyAML
Development Pipeline
Preclinical
~Aug 2013
→ ~Nov 2014
Phase 1
~Feb 2015
→ ~May 2016
Phase 2
~Aug 2016
→ ~Nov 2017
Phase 3
Feb 2018
→ Dec 2027
Phase 3Current
NCT03363228
2,223 pts·Narcolepsy
2022-01→2027-12·Recruiting
NCT04329743
601 pts·AML
2018-02→2025-01·Not yet recruiting
2,824 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-01-231.2y agoPh3 Readout· AML
2027-12-041.7y awayPh3 Readout· Narcolepsy
Trial Timeline
2018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q4
P3
Not yet…
P3
Recruit…
Catalysts
Ph3 Readout
2025-01-23 · 1.2y ago
AML
Ph3 Readout
2027-12-04 · 1.7y away
Narcolepsy
RecruitingNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03363228 | Phase 3 | Narcolepsy | Recruiting | 2223 | SRI-4 |
| NCT04329743 | Phase 3 | AML | Not yet recr... | 601 | OS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-7550 | Pfizer | Phase 3 | Aβ | |
| Sotovorutinib | Pfizer | Approved | Aβ | |
| ABB-1817 | AbbVie | Phase 3 | SHP2 | |
| BMY-7975 | Bristol-Myers Squibb | Phase 1 | MALT1 | |
| Bemalucimab | Bristol-Myers Squibb | Phase 1/2 | Aβ | |
| Datobrutinib | Sanofi | Phase 3 | Aβ | |
| GSK-4334 | GSK | Preclinical | CD47 | |
| DSN-791 | Daiichi Sankyo | NDA/BLA | Aβ | |
| AMG-2752 | Amgen | Preclinical | MDM2 | |
| REG-7737 | Regeneron | Preclinical | CD123 |