Modality
Cell Therapy
MOA
IL-13i
Target
CDK4/6
Pathway
Checkpoint
SMAADHD
Development Pipeline
Preclinical
~Jun 2018
→ ~Sep 2019
Phase 1
Dec 2019
→ Apr 2029
Phase 1Current
NCT04933373
56 pts·ADHD
2021-03→2027-08·Not yet recruiting
NCT05947756
1,046 pts·SMA
2019-12→2029-04·Not yet recruiting
1,102 total pts2 indications
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (3)
2025-10-046mo agoPh1 Dose Esc· SMA
2027-08-281.4y awayInterim· ADHD
2029-04-213.1y awayInterim· SMA
Trial Timeline
Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2
P1
Not yet…
P1
Not yet…
Catalysts
Ph1 Dose Esc
2025-10-04 · 6mo ago
SMA
Interim
2027-08-28 · 1.4y away
ADHD
Interim
2029-04-21 · 3.1y away
SMA
Not yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04933373 | Phase 1 | ADHD | Not yet recr... | 56 | UPCR |
| NCT05947756 | Phase 1 | SMA | Not yet recr... | 1046 | LiverFat |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Voxaderotide | Johnson & Johnson | NDA/BLA | PI3Kα | |
| RHH-7975 | Roche | Phase 3 | FXIa | |
| NVS-8902 | Novartis | Preclinical | TROP-2 | |
| Gozesotorasib | Bristol-Myers Squibb | NDA/BLA | HER2 | |
| BMY-4586 | Bristol-Myers Squibb | Preclinical | KRASG12C | |
| AMG-1919 | Amgen | Phase 2/3 | CDK2 | |
| Motavorutinib | Amgen | Phase 3 | CDK4/6 | |
| MRN-5715 | Moderna | NDA/BLA | CDK4/6 | |
| ROI-3353 | Roivant Sciences | Phase 1/2 | PLK4 | |
| Elralucimab | Axsome | Phase 2 | CDK4/6 |