Modality
mRNA
MOA
CGRPant
Target
Cl18.2
Pathway
RAS/MAPK
MCCSMA
Development Pipeline
Preclinical
~Apr 2016
→ ~Jul 2017
Phase 1
~Oct 2017
→ ~Jan 2019
Phase 2
~Apr 2019
→ ~Jul 2020
Phase 3
~Oct 2020
→ ~Jan 2022
NDA/BLA
Apr 2022
→ Aug 2027
NDA/BLACurrent
NCT05042665
36 pts·SMA
2022-04→2026-08·Recruiting
NCT03958391
2,899 pts·SMA
2022-11→2027-08·Terminated
2,935 total pts1 indication
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-08-155mo awayPh3 Readout· SMA
2027-08-111.4y awayPh3 Readout· SMA
Trial Timeline
Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3
NDA/BLA
Recruit…
NDA/BLA
Termina…
Catalysts
Ph3 Readout
2026-08-15 · 5mo away
SMA
Ph3 Readout
2027-08-11 · 1.4y away
SMA
RecruitingTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05042665 | NDA/BLA | SMA | Recruiting | 36 | CfB |
| NCT03958391 | NDA/BLA | SMA | Terminated | 2899 | HAM-D |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| Terafutibatinib | Pfizer | Phase 1/2 | CD38 | |
| Sotovorutinib | Pfizer | Approved | Aβ | |
| Mavutenlimab | Johnson & Johnson | Phase 3 | Cl18.2 | |
| JNJ-2735 | Johnson & Johnson | Phase 2/3 | Cl18.2 | |
| Ribosacituzumab | Roche | Approved | Cl18.2 | |
| Adagrazumab | Novartis | Phase 2/3 | Cl18.2 | |
| ABB-3060 | AbbVie | Phase 2 | LAG-3 | |
| Zorimavacamten | Bristol-Myers Squibb | Preclinical | Cl18.2 | |
| Doxafotisoran | Daiichi Sankyo | Phase 1 | Cl18.2 | |
| GIL-6239 | Gilead Sciences | Phase 2 | TNFα |