Modality
ERT
MOA
SGLT2i
Target
FLT3
Pathway
Sphingolipid
ALSADPKD
Development Pipeline
Preclinical
~May 2014
→ ~Aug 2015
Phase 1
~Nov 2015
→ ~Feb 2017
Phase 2
~May 2017
→ ~Aug 2018
Phase 3
Nov 2018
→ Apr 2031
Phase 3Current
NCT07960590
118 pts·ALS
2022-01→2025-11·Completed
NCT03055018
2,580 pts·ALS
2018-11→TBD·Active
NCT07296031
1,536 pts·ADPKD
2019-06→TBD·Active
+1 more trial
6,477 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-11-115mo agoPh3 Readout· ALS
2031-04-255.1y awayPh3 Readout· ADPKD
Trial Timeline
Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3
P3
Active
P3
Active
P3
Not yet…
P3
Complet…
Catalysts
Ph3 Readout
2025-11-11 · 5mo ago
ALS
Ph3 Readout
2031-04-25 · 5.1y away
ADPKD
ActiveCompletedNot yet recruiting|StartCompletionToday
Trials (4)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07960590 | Phase 3 | ALS | Completed | 118 | ACR20 |
| NCT03055018 | Phase 3 | ALS | Active | 2580 | eGFR |
| NCT07296031 | Phase 3 | ADPKD | Active | 1536 | ACR20 |
| NCT05745977 | Phase 3 | ADPKD | Not yet recr... | 2243 | HAM-D |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-8168 | Johnson & Johnson | Phase 2/3 | FLT3 | |
| JNJ-8232 | Johnson & Johnson | NDA/BLA | MALT1 | |
| Bemalucimab | Bristol-Myers Squibb | Phase 1/2 | Aβ | |
| BAY-3308 | Bayer | Phase 1 | ALK | |
| Sotorapivir | Moderna | Approved | FLT3 | |
| MRN-8225 | Moderna | Preclinical | TNFα | |
| Motazanubrutinib | Biogen | Preclinical | CD38 | |
| ALN-5628 | Alnylam | Phase 1/2 | APOC3 | |
| Nidaratamab | Exelixis | Phase 3 | FLT3 | |
| Zanumavacamten | Iovance | Phase 2 | FLT3 |