Modality
Gene Therapy
MOA
USP1i
Target
KIF18A
Pathway
Tau
PompeHeart FailureTTR Amyloidosis
Development Pipeline
Preclinical
~Feb 2018
→ ~May 2019
Phase 1
~Aug 2019
→ ~Nov 2020
Phase 2
Feb 2021
→ Aug 2027
Phase 2Current
NCT05101601
88 pts·TTR Amyloidosis
2021-02→2027-08·Completed
88 total pts1 indication
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2027-08-051.3y awayPh3 Readout· TTR Amyloidosis
Trial Timeline
2021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3
P2/3
Complet…
Catalysts
Ph3 Readout
2027-08-05 · 1.3y away
TTR Amyloidosis
Completed|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05101601 | Phase 2/3 | TTR Amyloidosis | Completed | 88 | PFS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Niranesiran | Johnson & Johnson | Phase 2 | KIF18A | |
| SNY-2934 | Sanofi | Phase 3 | KRASG12D | |
| AMG-9052 | Amgen | Phase 2/3 | CDK2 | |
| Datozumab | Regeneron | Phase 1 | B7-H3 | |
| Riluinavolisib | Moderna | NDA/BLA | KIF18A | |
| GMA-729 | Genmab | Phase 2 | BCL-2 | |
| Pexanaritide | Innovent Bio | Preclinical | SGLT2 | |
| ROI-9675 | Roivant Sciences | NDA/BLA | HER2 | |
| NUV-2032 | Nuvalent | Preclinical | KIF18A | |
| DNL-9555 | Denali | Phase 1/2 | KIF18A |