Modality
Small Molecule
MOA
CDK4/6i
Target
CFTR
Pathway
Sphingolipid
NarcolepsyLGSEpilepsy
Development Pipeline
Preclinical
~Mar 2015
→ ~Jun 2016
Phase 1
~Sep 2016
→ ~Dec 2017
Phase 2
~Mar 2018
→ ~Jun 2019
Phase 3
Sep 2019
→ Dec 2031
Phase 3Current
NCT04940795
192 pts·LGS
2022-01→2026-04·Active
NCT08329637
1,222 pts·Narcolepsy
2019-09→2031-12·Not yet recruiting
1,414 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-04-172w awayPh3 Readout· LGS
2031-12-195.7y awayPh3 Readout· Narcolepsy
Trial Timeline
Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
P3
Not yet…
P3
Active
Catalysts
Ph3 Readout
2026-04-17 · 2w away
LGS
Ph3 Readout
2031-12-19 · 5.7y away
Narcolepsy
ActiveNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT04940795 | Phase 3 | LGS | Active | 192 | OS |
| NCT08329637 | Phase 3 | Narcolepsy | Not yet recr... | 1222 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-5767 | Pfizer | Phase 1/2 | BET | |
| Nidasacituzumab | Eli Lilly | Approved | CFTR | |
| RHH-1969 | Roche | Approved | BET | |
| Datoglumide | AbbVie | Approved | CFTR | |
| Ivocapivasertib | Bristol-Myers Squibb | Phase 1 | BET | |
| Zorimavacamten | Bristol-Myers Squibb | Preclinical | Cl18.2 | |
| SNY-7254 | Sanofi | Phase 2 | CFTR | |
| SNY-5894 | Sanofi | Approved | CFTR | |
| Fixatenlimab | GSK | Phase 1/2 | JAK1 | |
| VRT-4938 | Vertex Pharma | Preclinical | HER2 |