Modality
ERT
MOA
PI3Ki
Target
PCSK9
Pathway
Wnt
RCC
Development Pipeline
Preclinical
~Sep 2011
→ ~Dec 2012
Phase 1
~Mar 2013
→ ~Jun 2014
Phase 2
~Sep 2014
→ ~Dec 2015
Phase 3
~Mar 2016
→ ~Jun 2017
NDA/BLA
~Sep 2017
→ ~Dec 2018
Approved
Mar 2019
→ Oct 2031
ApprovedCurrent
NCT08928710
1,495 pts·RCC
2020-03→2027-05·Terminated
NCT08016001
1,704 pts·RCC
2019-03→2031-10·Terminated
NCT05545964
1,423 pts·RCC
2022-09→2027-07·Recruiting
4,622 total pts1 indication
CompletedCurrentUpcoming
Catalysts (3)
2027-05-121.1y awayPh3 Readout· RCC
2027-07-141.3y awayPh3 Readout· RCC
2031-10-195.6y awayPh3 Readout· RCC
Trial Timeline
2019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3Q42030Q2Q3Q42031Q2Q3Q4
Approved
Termina…
Approved
Termina…
Approved
Recruit…
Catalysts
Ph3 Readout
2027-05-12 · 1.1y away
RCC
Ph3 Readout
2027-07-14 · 1.3y away
RCC
Ph3 Readout
2031-10-19 · 5.6y away
RCC
RecruitingTerminated|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT08928710 | Approved | RCC | Terminated | 1495 | DAS28 |
| NCT08016001 | Approved | RCC | Terminated | 1704 | DOR |
| NCT05545964 | Approved | RCC | Recruiting | 1423 | NT-proBNP |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Tiramavacamten | Roche | Preclinical | SGLT2 | |
| NVS-5126 | Novartis | Phase 3 | SHP2 | |
| Zanusertib | AbbVie | Approved | PCSK9 | |
| Voxacilimab | AstraZeneca | Phase 1/2 | GLP-1R | |
| Capifutibatinib | Sanofi | Approved | PRMT5 | |
| Rimatenlimab | Novo Nordisk | Phase 2/3 | SGLT2 | |
| GIL-4361 | Gilead Sciences | Preclinical | PCSK9 | |
| REG-3155 | Regeneron | Phase 1/2 | MDM2 | |
| REG-2172 | Regeneron | Phase 1/2 | PCSK9 | |
| BMR-8377 | BioMarin | Phase 1/2 | PCSK9 |