Modality
ERT
MOA
FXIai
Target
BET
Pathway
Ferroptosis
Pompe
Development Pipeline
Preclinical
~Feb 2020
→ ~May 2021
Phase 1
Aug 2021
→ Mar 2027
Phase 1Current
NCT03868537
2,500 pts·Pompe
2021-08→2026-11·Active
NCT08301381
2,994 pts·Pompe
2025-07→2027-03·Terminated
5,494 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-11-047mo awayPh2 Data· Pompe
2027-03-2412mo awayPh2 Data· Pompe
Trial Timeline
Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2
P1/2
Active
P1/2
Termina…
Catalysts
Ph2 Data
2026-11-04 · 7mo away
Pompe
Ph2 Data
2027-03-24 · 12mo away
Pompe
ActiveTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03868537 | Phase 1/2 | Pompe | Active | 2500 | FEV1 |
| NCT08301381 | Phase 1/2 | Pompe | Terminated | 2994 | CfB |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5767 | Pfizer | Phase 1/2 | BET | |
| RHH-1969 | Roche | Approved | BET | |
| NVS-5439 | Novartis | Preclinical | BET | |
| NVS-6360 | Novartis | Approved | CD38 | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| AZN-1715 | AstraZeneca | Phase 3 | AHR | |
| Ivocapivasertib | Bristol-Myers Squibb | Phase 1 | BET | |
| BMY-9931 | Bristol-Myers Squibb | Phase 1 | GPRC5D | |
| NVO-7877 | Novo Nordisk | Preclinical | GPRC5D | |
| TAK-8730 | Takeda | Phase 2 | VEGF |