Modality
Cell Therapy
MOA
Menini
Target
Cl18.2
Pathway
Wnt
PNHSMA
Development Pipeline
Preclinical
~Sep 2014
→ ~Dec 2015
Phase 1
~Mar 2016
→ ~Jun 2017
Phase 2
~Sep 2017
→ ~Dec 2018
Phase 3
Mar 2019
→ Aug 2031
Phase 3Current
NCT03841642
2,327 pts·SMA
2019-03→2031-08·Active
NCT06143040
909 pts·PNH
2025-05→2026-02·Terminated
3,236 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-02-032mo agoPh3 Readout· PNH
2031-08-105.4y awayPh3 Readout· SMA
Trial Timeline
2019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
P3
Active
P3
Termina…
Catalysts
Ph3 Readout
2026-02-03 · 2mo ago
PNH
Ph3 Readout
2031-08-10 · 5.4y away
SMA
ActiveTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03841642 | Phase 3 | SMA | Active | 2327 | UPDRS |
| NCT06143040 | Phase 3 | PNH | Terminated | 909 | PFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| JNJ-2735 | Johnson & Johnson | Phase 2/3 | Cl18.2 | |
| Ivorelsin | Eli Lilly | Approved | JAK1 | |
| Ribosacituzumab | Roche | Approved | Cl18.2 | |
| Adagrazumab | Novartis | Phase 2/3 | Cl18.2 | |
| Zorimavacamten | Bristol-Myers Squibb | Preclinical | Cl18.2 | |
| NVO-2974 | Novo Nordisk | NDA/BLA | PARP | |
| Doxafotisoran | Daiichi Sankyo | Phase 1 | Cl18.2 | |
| Olpafutibatinib | Immunocore | Approved | SMN2 | |
| PRA-419 | Praxis Precision | Approved | Cl18.2 | |
| CRB-3801 | Caribou Bio | Preclinical | Cl18.2 |