Modality
Gene Therapy
MOA
BiTE
Target
WRN
Pathway
JAK/STAT
MelanomaSchizophreniaRett
Development Pipeline
Preclinical
May 2019
→ Jun 2027
PreclinicalCurrent
NCT05549733
1,971 pts·Melanoma
2021-07→2027-06·Terminated
NCT04944188
41 pts·Rett
2019-05→2026-12·Recruiting
2,012 total pts2 indications
Phase 1
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-12-169mo awayInterim· Rett
2027-06-071.2y awayInterim· Melanoma
Trial Timeline
Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3
Preclinical
Recruit…
Preclinical
Termina…
Catalysts
Interim
2026-12-16 · 9mo away
Rett
Interim
2027-06-07 · 1.2y away
Melanoma
RecruitingTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05549733 | Preclinical | Melanoma | Terminated | 1971 | EFS |
| NCT04944188 | Preclinical | Rett | Recruiting | 41 | MRD |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-3251 | Eli Lilly | Phase 2 | MDM2 | |
| MRK-3745 | Merck & Co | Phase 2 | WRN | |
| Zorisotorasib | AstraZeneca | Phase 1 | WRN | |
| BMY-3371 | Bristol-Myers Squibb | Phase 3 | WRN | |
| Zanutinib | Bristol-Myers Squibb | Phase 2/3 | APOC3 | |
| Lisorapivir | Takeda | Preclinical | WRN | |
| Nirafutibatinib | Bayer | Phase 2 | WRN | |
| Bemarelsin | Daiichi Sankyo | Preclinical | GLP-1R | |
| DSN-7360 | Daiichi Sankyo | Approved | SGLT2 | |
| BII-8315 | Biogen | NDA/BLA | CDK2 |