Modality
Gene Therapy
MOA
PLK4i
Target
PCSK9
Pathway
PD-1/PD-L1
RCCALS
Development Pipeline
Preclinical
~Aug 2012
→ ~Nov 2013
Phase 1
~Feb 2014
→ ~May 2015
Phase 2
~Aug 2015
→ ~Nov 2016
Phase 3
~Feb 2017
→ ~May 2018
NDA/BLA
~Aug 2018
→ ~Nov 2019
Approved
Feb 2020
→ Mar 2029
ApprovedCurrent
NCT08287302
2,103 pts·ALS
2021-03→2029-03·Recruiting
NCT03667324
1,178 pts·RCC
2020-02→2025-03·Active
3,281 total pts2 indications
CompletedCurrentUpcoming
Catalysts (2)
2025-03-161.0y agoPh3 Readout· RCC
2029-03-163.0y awayPh3 Readout· ALS
Trial Timeline
2020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2
Approved
Active
Approved
Recruit…
Catalysts
Ph3 Readout
2025-03-16 · 1.0y ago
RCC
Ph3 Readout
2029-03-16 · 3.0y away
ALS
RecruitingActive|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08287302 | Approved | ALS | Recruiting | 2103 | DAS28 |
| NCT03667324 | Approved | RCC | Active | 1178 | Biomarker |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Zanusertib | AbbVie | Approved | PCSK9 | |
| AMG-2597 | Amgen | Phase 2/3 | CD38 | |
| GIL-4361 | Gilead Sciences | Preclinical | PCSK9 | |
| REG-2172 | Regeneron | Phase 1/2 | PCSK9 | |
| Riluinavolisib | Moderna | NDA/BLA | KIF18A | |
| BMR-8377 | BioMarin | Phase 1/2 | PCSK9 | |
| Capifutibatinib | Incyte | Phase 3 | PCSK9 | |
| INC-5849 | Incyte | Phase 2 | PCSK9 | |
| Talatapinarof | Samsung Biologics | Phase 1/2 | PCSK9 | |
| Liravorutinib | Biomea Fusion | Phase 1/2 | GPRC5D |