Modality
ERT
MOA
IL-23i
Target
IL-17A
Pathway
RNA Splicing
Urothelial CaPompe
Development Pipeline
Preclinical
~Jul 2016
→ ~Oct 2017
Phase 1
~Jan 2018
→ ~Apr 2019
Phase 2
~Jul 2019
→ ~Oct 2020
Phase 3
Jan 2021
→ Mar 2027
Phase 3Current
NCT08010690
2,242 pts·Pompe
2021-02→2026-08·Terminated
NCT04744494
2,115 pts·Pompe
2021-01→2027-03·Not yet recruiting
4,357 total pts1 indication
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (4)
2026-03-241w agoAdCom· Pompe
2026-07-284mo awayNDA· Urothelial Ca
2026-08-255mo awayPh3 Readout· Pompe
2027-03-0711mo awayPh3 Readout· Pompe
Trial Timeline
2021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2
P3
Not yet…
P3
Termina…
Catalysts
AdCom
2026-03-24 · 1w ago
Pompe
NDA
2026-07-28 · 4mo away
Urothelial Ca
Ph3 Readout
2026-08-25 · 5mo away
Pompe
Ph3 Readout
2027-03-07 · 11mo away
Pompe
TerminatedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08010690 | Phase 3 | Pompe | Terminated | 2242 | MRD |
| NCT04744494 | Phase 3 | Pompe | Not yet recr... | 2115 | 6MWD |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Geliderotide | Novartis | NDA/BLA | IL-17A | |
| GSK-6983 | GSK | Phase 2 | ALK | |
| Motarapivir | GSK | Preclinical | AuroraA | |
| Tirasotorasib | Takeda | NDA/BLA | IL-17A | |
| GIL-9779 | Gilead Sciences | Phase 1 | IL-17A | |
| Zenotapinarof | Vertex Pharma | Preclinical | CD38 | |
| Bemasotorasib | Exelixis | Phase 2 | CD38 | |
| RVM-7089 | Revolution Medicines | Phase 3 | IL-17A | |
| Tirasotorasib | Viking Therapeutics | Phase 1 | CFTR | |
| Adagrarapivir | Ideaya Bio | Phase 1/2 | IL-17A |