Modality
ERT
MOA
CGRPant
Target
SMN2
Pathway
Wnt
HCCWMPTSD
Development Pipeline
Preclinical
~Nov 2018
→ ~Feb 2020
Phase 1
May 2020
→ Nov 2029
Phase 1Current
NCT08288674
1,677 pts·WM
2022-09→2029-10·Active
NCT06271260
2,185 pts·WM
2020-05→2029-11·Recruiting
3,862 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2029-10-223.6y awayInterim· WM
2029-11-193.6y awayInterim· WM
Trial Timeline
Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
P1
Recruit…
P1
Active
Catalysts
Interim
2029-10-22 · 3.6y away
WM
Interim
2029-11-19 · 3.6y away
WM
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08288674 | Phase 1 | WM | Active | 1677 | SeizFreq |
| NCT06271260 | Phase 1 | WM | Recruiting | 2185 | MRD |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Terafutibatinib | Pfizer | Phase 1/2 | CD38 | |
| Sotovorutinib | Pfizer | Approved | Aβ | |
| ABB-3060 | AbbVie | Phase 2 | LAG-3 | |
| NVO-6275 | Novo Nordisk | Phase 1 | SMN2 | |
| TAK-5300 | Takeda | Phase 1 | SMN2 | |
| GIL-6239 | Gilead Sciences | Phase 2 | TNFα | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| REG-647 | Regeneron | Preclinical | SMN2 | |
| Tezecilimab | Regeneron | Approved | IL-23 | |
| INC-2432 | Incyte | Phase 2 | CD47 |