Modality
Gene Therapy
MOA
GLP-1ag
Target
IL-17A
Pathway
Complement
RettNarcolepsy
Development Pipeline
Preclinical
~Apr 2015
→ ~Jul 2016
Phase 1
~Oct 2016
→ ~Jan 2018
Phase 2
~Apr 2018
→ ~Jul 2019
Phase 3
Oct 2019
→ Jul 2029
Phase 3Current
NCT07842786
2,537 pts·Rett
2019-10→2029-07·Terminated
NCT03334905
2,330 pts·Rett
2020-11→2027-10·Not yet recruiting
4,867 total pts1 indication
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2027-10-141.5y awayPh3 Readout· Rett
2029-07-233.3y awayPh3 Readout· Rett
Trial Timeline
Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q4
P3
Termina…
P3
Not yet…
Catalysts
Ph3 Readout
2027-10-14 · 1.5y away
Rett
Ph3 Readout
2029-07-23 · 3.3y away
Rett
TerminatedNot yet recruiting|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT07842786 | Phase 3 | Rett | Terminated | 2537 | VA |
| NCT03334905 | Phase 3 | Rett | Not yet recr... | 2330 | HbA1c |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-8662 | Pfizer | NDA/BLA | CD19 | |
| Geliderotide | Novartis | NDA/BLA | IL-17A | |
| NVO-7840 | Novo Nordisk | Phase 2/3 | B7-H3 | |
| Tirasotorasib | Takeda | NDA/BLA | IL-17A | |
| GIL-9779 | Gilead Sciences | Phase 1 | IL-17A | |
| MRN-7601 | Moderna | Phase 2 | IL-13 | |
| ARG-1250 | Argenx | Phase 2 | C5 | |
| ILM-5680 | Illumina | Phase 2 | BET | |
| RVM-7089 | Revolution Medicines | Phase 3 | IL-17A | |
| CRS-184 | CRISPR Therapeutics | Phase 1/2 | AHR |