Modality
ERT
MOA
PCSK9i
Target
Menin
Pathway
RNA Splicing
ADHD
Development Pipeline
Preclinical
~Nov 2019
→ ~Feb 2021
Phase 1
May 2021
→ Aug 2027
Phase 1Current
NCT05049921
359 pts·ADHD
2021-05→2027-08·Terminated
359 total pts1 indication
Phase 2
Phase 3
NDA/BLA
Approved
CompletedCurrentUpcoming
Catalysts (1)
2027-08-061.3y awayInterim· ADHD
Trial Timeline
Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3
P1
Termina…
Catalysts
Interim
2027-08-06 · 1.3y away
ADHD
Terminated|StartCompletionToday
Trials (1)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05049921 | Phase 1 | ADHD | Terminated | 359 | OS |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| LLY-8369 | Eli Lilly | Approved | Menin | |
| NVS-1475 | Novartis | Phase 2 | Menin | |
| MRK-8368 | Merck & Co | Phase 3 | ALK | |
| Ribofutibatinib | AstraZeneca | Phase 2/3 | SOS1 | |
| AZN-4015 | AstraZeneca | Preclinical | BCMA | |
| Polazasiran | Amgen | Phase 2 | LAG-3 | |
| Cevitinib | Regeneron | Phase 3 | FGFR | |
| VRT-9481 | Vertex Pharma | Phase 1/2 | Menin | |
| Olpainavolisib | Moderna | Phase 1 | Menin | |
| INC-1261 | Incyte | Phase 1/2 | PRMT5 |