Modality
Cell Therapy
MOA
PD-L1i
Target
WRN
Pathway
Complement
ThymomaSMAWilms
Development Pipeline
Preclinical
~Feb 2014
→ ~May 2015
Phase 1
~Aug 2015
→ ~Nov 2016
Phase 2
Feb 2017
→ Nov 2025
Phase 2Current
NCT03264682
225 pts·SMA
2017-07→2025-11·Not yet recruiting
NCT04024495
114 pts·Wilms
2017-02→TBD·Active
339 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2025-11-224mo agoPh2 Data· SMA
Trial Timeline
2017Q2Q3Q42018Q2Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q4
P2
Active
P2
Not yet…
Catalysts
Ph2 Data
2025-11-22 · 4mo ago
SMA
ActiveNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03264682 | Phase 2 | SMA | Not yet recr... | 225 | PASI75 |
| NCT04024495 | Phase 2 | Wilms | Active | 114 | 6MWD |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-1085 | Pfizer | Preclinical | FXIa | |
| Rilulemzoparlimab | Novartis | Phase 2/3 | KRASG12D | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| BMY-3371 | Bristol-Myers Squibb | Phase 3 | WRN | |
| SNY-9600 | Sanofi | NDA/BLA | B7-H3 | |
| Lisorapivir | Takeda | Preclinical | WRN | |
| Motainavolisib | Amgen | NDA/BLA | CD47 | |
| BNT-8090 | BioNTech | Phase 2 | AuroraA | |
| Miriglumide | Alnylam | Phase 3 | CD3 |