Modality
ERT
MOA
FGFRi
Target
C5
Pathway
Epigenetic
PAHWMADHD
Development Pipeline
Preclinical
~Nov 2014
→ ~Feb 2016
Phase 1
~May 2016
→ ~Aug 2017
Phase 2
~Nov 2017
→ ~Feb 2019
Phase 3
May 2019
→ Jun 2028
Phase 3Current
NCT06895585
633 pts·PAH
2019-05→2028-06·Active
NCT03576366
949 pts·WM
2024-09→2026-05·Terminated
1,582 total pts2 indications
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (3)
2026-04-172w awayAdCom· ADHD
2026-05-131mo awayPh3 Readout· WM
2028-06-142.2y awayPh3 Readout· PAH
Trial Timeline
Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3
P3
Active
P3
Termina…
Catalysts
AdCom
2026-04-17 · 2w away
ADHD
Ph3 Readout
2026-05-13 · 1mo away
WM
Ph3 Readout
2028-06-14 · 2.2y away
PAH
ActiveTerminated|StartCompletionToday
Trials (2)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT06895585 | Phase 3 | PAH | Active | 633 | BodyWt |
| NCT03576366 | Phase 3 | WM | Terminated | 949 | FEV1 |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-1956 | Eli Lilly | Phase 1/2 | BCMA | |
| LLY-1184 | Eli Lilly | NDA/BLA | C5 | |
| RHH-1546 | Roche | Phase 2/3 | C5 | |
| BAY-6035 | Bayer | Phase 1 | C5 | |
| ARG-1250 | Argenx | Phase 2 | C5 | |
| SRP-9822 | Sarepta | Phase 3 | SOS1 | |
| Elratapinarof | Halozyme | Phase 2 | C5 | |
| HAL-1232 | Halozyme | Approved | C5 | |
| Rimainavolisib | Illumina | Phase 2 | PD-1 | |
| Elrainavolisib | Roivant Sciences | Preclinical | Nectin-4 |