Modality
ERT
MOA
JAK1/2i
Target
CFTR
Pathway
Complement
MG
Development Pipeline
Preclinical
~Sep 2015
→ ~Dec 2016
Phase 1
~Mar 2017
→ ~Jun 2018
Phase 2
Sep 2018
→ Apr 2029
Phase 2Current
NCT04725933
1,468 pts·MG
2018-09→2027-01·Recruiting
NCT03450253
1,349 pts·MG
2021-10→2029-04·Recruiting
2,817 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (3)
2026-06-102mo awayConference· MG
2027-01-2410mo awayPh2 Data· MG
2029-04-063.0y awayPh2 Data· MG
Trial Timeline
Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2
P2
Recruit…
P2
Recruit…
Catalysts
Conference
2026-06-10 · 2mo away
MG
Ph2 Data
2027-01-24 · 10mo away
MG
Ph2 Data
2029-04-06 · 3.0y away
MG
Recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04725933 | Phase 2 | MG | Recruiting | 1468 | ORR |
| NCT03450253 | Phase 2 | MG | Recruiting | 1349 | HAM-D |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| Datoglumide | AbbVie | Approved | CFTR | |
| SNY-7254 | Sanofi | Phase 2 | CFTR | |
| SNY-5894 | Sanofi | Approved | CFTR | |
| Motavorutinib | Amgen | Phase 3 | CDK4/6 | |
| VRT-9481 | Vertex Pharma | Phase 1/2 | Menin | |
| Sovarelsin | BioMarin | Phase 2 | CFTR | |
| Peminaritide | Sarepta | NDA/BLA | PLK4 | |
| Fixatuximab | Legend Biotech | Preclinical | CFTR |