Modality
Small Molecule
MOA
IL-17i
Target
SMN2
Pathway
Tau
PsAHemophilia ABreast Ca
Development Pipeline
Preclinical
~Sep 2017
→ ~Dec 2018
Phase 1
Mar 2019
→ Dec 2029
Phase 1Current
NCT05894663
2,331 pts·Hemophilia A
2019-03→2029-12·Terminated
2,331 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2025-10-096mo agoPh1 Dose Esc· Breast Ca
2029-12-243.7y awayPh2 Data· Hemophilia A
Trial Timeline
2019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
P1/2
Termina…
Catalysts
Ph1 Dose Esc
2025-10-09 · 6mo ago
Breast Ca
Ph2 Data
2029-12-24 · 3.7y away
Hemophilia A
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05894663 | Phase 1/2 | Hemophilia A | Terminated | 2331 | PFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| SNY-2289 | Sanofi | Phase 2/3 | CD38 | |
| NVO-6275 | Novo Nordisk | Phase 1 | SMN2 | |
| Fixainavolisib | Takeda | Preclinical | CDK2 | |
| TAK-5300 | Takeda | Phase 1 | SMN2 | |
| GIL-2259 | Gilead Sciences | Approved | SMN2 | |
| REG-647 | Regeneron | Preclinical | SMN2 | |
| Elrarapivir | Vertex Pharma | NDA/BLA | PRMT5 | |
| Rimaglumide | Biogen | Phase 1/2 | BCL-2 | |
| ARG-3458 | Argenx | Preclinical | RET | |
| Nidaratamab | Krystal Biotech | Preclinical | RET |