Modality
siRNA
MOA
SOS1i
Target
RET
Pathway
Autophagy
Pompe
Development Pipeline
Preclinical
~Jun 2013
→ ~Sep 2014
Phase 1
~Dec 2014
→ ~Mar 2016
Phase 2
~Jun 2016
→ ~Sep 2017
Phase 3
Dec 2017
→ Aug 2028
Phase 3Current
NCT04058715
2,518 pts·Pompe
2023-06→2028-07·Completed
NCT05642419
2,015 pts·Pompe
2017-12→2028-08·Not yet recruiting
4,533 total pts1 indication
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2028-07-102.3y awayPh3 Readout· Pompe
2028-08-182.4y awayPh3 Readout· Pompe
Trial Timeline
Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q4
P3
Not yet…
P3
Complet…
Catalysts
Ph3 Readout
2028-07-10 · 2.3y away
Pompe
Ph3 Readout
2028-08-18 · 2.4y away
Pompe
CompletedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04058715 | Phase 3 | Pompe | Completed | 2518 | DAS28 |
| NCT05642419 | Phase 3 | Pompe | Not yet recr... | 2015 | SRI-4 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-2901 | Pfizer | Phase 2 | AHR | |
| RHH-5389 | Roche | Preclinical | RET | |
| MRK-1380 | Merck & Co | Preclinical | SHP2 | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| Terarelsin | AbbVie | Preclinical | SOS1 | |
| BMY-8678 | Bristol-Myers Squibb | Approved | RET | |
| 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 |