Modality
mRNA
MOA
CFTRmod
Target
FGFR
Pathway
Amyloid
HSPBCNarcolepsy
Development Pipeline
Preclinical
~Oct 2016
→ ~Jan 2018
Phase 1
~Apr 2018
→ ~Jul 2019
Phase 2
Oct 2019
→ Nov 2029
Phase 2Current
NCT05030620
2,281 pts·HS
2025-12→2025-03·Completed
NCT05194058
1,752 pts·Narcolepsy
2019-10→2029-11·Active
4,033 total pts2 indications
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-03-201.0y agoPh3 Readout· HS
2029-11-053.6y awayPh3 Readout· Narcolepsy
Trial Timeline
Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
P2/3
Active
P2/3
Complet…
Catalysts
Ph3 Readout
2025-03-20 · 1.0y ago
HS
Ph3 Readout
2029-11-05 · 3.6y away
Narcolepsy
ActiveCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05030620 | Phase 2/3 | HS | Completed | 2281 | SeizFreq |
| NCT05194058 | Phase 2/3 | Narcolepsy | Active | 1752 | DAS28 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-4358 | Eli Lilly | Phase 3 | B7-H3 | |
| LLY-1592 | Eli Lilly | NDA/BLA | FGFR | |
| Doxarasimod | Merck & Co | NDA/BLA | FGFR | |
| Gelinaritide | AbbVie | Preclinical | FcRn | |
| AZN-7403 | AstraZeneca | Phase 2/3 | FGFR | |
| Cevitinib | Regeneron | Phase 3 | FGFR | |
| Darafutibatinib | BioNTech | Preclinical | PRMT5 | |
| ALN-8757 | Alnylam | Preclinical | TIGIT | |
| Ivotuximab | Madrigal Pharma | Phase 2 | CD123 | |
| Tixatapinarof | Recursion | Approved | FGFR |