Modality
Fusion Protein
MOA
JAK1/2i
Target
PD-L1
Pathway
Autophagy
SMAHeart Failure
Development Pipeline
Preclinical
~Jan 2014
→ ~Apr 2015
Phase 1
~Jul 2015
→ ~Oct 2016
Phase 2
~Jan 2017
→ ~Apr 2018
Phase 3
Jul 2018
→ Apr 2029
Phase 3Current
NCT03704654
2,043 pts·Heart Failure
2018-07→2029-04·Not yet recruiting
NCT07949491
1,072 pts·SMA
2025-12→2026-12·Active
NCT03460342
2,389 pts·SMA
2022-10→2025-12·Not yet recruiting
5,504 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (3)
2025-12-044mo agoPh3 Readout· SMA
2026-12-209mo awayPh3 Readout· SMA
2029-04-023.0y awayPh3 Readout· Heart Failure
Trial Timeline
Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2
P3
Not yet…
P3
Not yet…
P3
Active
Catalysts
Ph3 Readout
2025-12-04 · 4mo ago
SMA
Ph3 Readout
2026-12-20 · 9mo away
SMA
Ph3 Readout
2029-04-02 · 3.0y away
Heart Failure
ActiveNot yet recruiting|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03704654 | Phase 3 | Heart Failure | Not yet recr... | 2043 | UPDRS |
| NCT07949491 | Phase 3 | SMA | Active | 1072 | FEV1 |
| NCT03460342 | Phase 3 | SMA | Not yet recr... | 2389 | eGFR |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| ABB-7516 | AbbVie | Phase 3 | PD-L1 | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| Olpatinib | GSK | Preclinical | PD-L1 | |
| BAY-3684 | Bayer | Approved | PD-L1 | |
| Motavorutinib | Amgen | Phase 3 | CDK4/6 | |
| VRT-9481 | Vertex Pharma | Phase 1/2 | Menin | |
| Sovarelsin | BioMarin | Phase 2 | CFTR | |
| Peminaritide | Sarepta | NDA/BLA | PLK4 | |
| Niramavacamten | Hansoh Pharma | Phase 2 | SGLT2 | |
| Polatuximab | Nuvalent | Approved | PD-L1 |