Modality
Nanobody
MOA
C5i
Target
RET
Pathway
RNA Splicing
SLESCD
Development Pipeline
Preclinical
~Feb 2013
→ ~May 2014
Phase 1
~Aug 2014
→ ~Nov 2015
Phase 2
~Feb 2016
→ ~May 2017
Phase 3
~Aug 2017
→ ~Nov 2018
NDA/BLA
Feb 2019
→ Feb 2028
NDA/BLACurrent
NCT05508493
2,453 pts·SCD
2019-02→2026-04·Recruiting
NCT05141294
963 pts·SLE
2020-06→2028-02·Completed
3,416 total pts2 indications
Approved
CompletedCurrentUpcoming
Catalysts (2)
2026-04-213w awayPh3 Readout· SCD
2028-02-251.9y awayPh3 Readout· SLE
Trial Timeline
2019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2
NDA/BLA
Recruit…
NDA/BLA
Complet…
Catalysts
Ph3 Readout
2026-04-21 · 3w away
SCD
Ph3 Readout
2028-02-25 · 1.9y away
SLE
RecruitingCompleted|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05508493 | NDA/BLA | SCD | Recruiting | 2453 | EDSS |
| NCT05141294 | NDA/BLA | SLE | Completed | 963 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| RHH-5389 | Roche | Preclinical | RET | |
| Fixanesiran | AbbVie | Preclinical | RET | |
| BMY-8678 | Bristol-Myers Squibb | Approved | RET | |
| DSN-2247 | Daiichi Sankyo | NDA/BLA | EZH2 | |
| VRT-1576 | Vertex Pharma | Phase 2/3 | RET | |
| ARG-3458 | Argenx | Preclinical | RET | |
| Rimabrutinib | Madrigal Pharma | Phase 1/2 | GPRC5D | |
| Nidaratamab | Krystal Biotech | Preclinical | RET | |
| XEN-577 | Xenon Pharma | Phase 2/3 | C5 | |
| Fixabrutinib | Verve | Phase 1 | RET |