Modality
ASO
MOA
C5i
Target
SHP2
Pathway
PD-1/PD-L1
PompeCrohn'sCKD
Development Pipeline
Preclinical
~Sep 2018
→ ~Dec 2019
Phase 1
~Mar 2020
→ ~Jun 2021
Phase 2
~Sep 2021
→ ~Dec 2022
Phase 3
~Mar 2023
→ ~Jun 2024
NDA/BLA
Sep 2024
→ May 2027
NDA/BLACurrent
NCT05710052
1,820 pts·Pompe
2024-09→2027-05·Not yet recruiting
1,820 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (1)
2027-05-241.1y awayPh3 Readout· Pompe
Trial Timeline
Q42025Q2Q3Q42026Q2Q3Q42027Q2
NDA/BLA
Not yet…
Catalysts
Ph3 Readout
2027-05-24 · 1.1y away
Pompe
Not yet recruiting|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05710052 | NDA/BLA | Pompe | Not yet recr... | 1820 | DOR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| NVS-5126 | Novartis | Phase 3 | SHP2 | |
| MRK-1380 | Merck & Co | Preclinical | SHP2 | |
| ABB-1817 | AbbVie | Phase 3 | SHP2 | |
| AZN-8281 | AstraZeneca | Phase 1 | SHP2 | |
| AZN-8478 | AstraZeneca | NDA/BLA | SHP2 | |
| DSN-2247 | Daiichi Sankyo | NDA/BLA | EZH2 | |
| MRN-7409 | Moderna | NDA/BLA | SHP2 | |
| 180-3597 | Innovent Bio | Preclinical | SHP2 | |
| Tezelucimab | Ascendis Pharma | Preclinical | SHP2 | |
| Rimabrutinib | Madrigal Pharma | Phase 1/2 | GPRC5D |