Modality
siRNA
MOA
SOS1i
Target
BCL-2
Pathway
Amyloid
ALS
Development Pipeline
Preclinical
~Aug 2014
→ ~Nov 2015
Phase 1
~Feb 2016
→ ~May 2017
Phase 2
~Aug 2017
→ ~Nov 2018
Phase 3
~Feb 2019
→ ~May 2020
NDA/BLA
~Aug 2020
→ ~Nov 2021
Approved
Feb 2022
→ Oct 2031
ApprovedCurrent
NCT08445847
1,451 pts·ALS
2022-02→2031-10·Not yet recruiting
1,451 total pts1 indication
CompletedCurrentUpcoming
Catalysts (2)
2026-07-083mo awayBTD· ALS
2031-10-025.5y awayPh3 Readout· ALS
Trial Timeline
2022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
Approved
Not yet…
Catalysts
BTD
2026-07-08 · 3mo away
ALS
Ph3 Readout
2031-10-02 · 5.5y away
ALS
Not yet recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08445847 | Approved | ALS | Not yet recr... | 1451 | UPDRS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-2901 | Pfizer | Phase 2 | AHR | |
| RHH-8550 | Roche | Approved | BCL-2 | |
| MRK-1380 | Merck & Co | Preclinical | SHP2 | |
| Terarelsin | AbbVie | Preclinical | SOS1 | |
| Doxarapivir | Bristol-Myers Squibb | Phase 3 | KRASG12C | |
| DSN-6862 | Daiichi Sankyo | Preclinical | IL-23 | |
| GIL-2011 | Gilead Sciences | Preclinical | VEGF | |
| REG-647 | Regeneron | Preclinical | SMN2 | |
| Rimaglumide | Biogen | Phase 1/2 | BCL-2 | |
| Cevisacituzumab | Alnylam | Preclinical | MALT1 |