Modality
Gene Therapy
MOA
KIF18Ai
Target
BET
Pathway
Checkpoint
SMAMyelofibrosis
Development Pipeline
Preclinical
~Mar 2011
→ ~Jun 2012
Phase 1
~Sep 2012
→ ~Dec 2013
Phase 2
~Mar 2014
→ ~Jun 2015
Phase 3
~Sep 2015
→ ~Dec 2016
NDA/BLA
~Mar 2017
→ ~Jun 2018
Approved
Sep 2018
→ Jul 2029
ApprovedCurrent
NCT05244829
1,657 pts·SMA
2018-09→2025-05·Active
NCT05557733
1,311 pts·Myelofibrosis
2019-08→2029-07·Recruiting
NCT03557322
1,400 pts·SMA
2022-02→2027-03·Not yet recruiting
4,368 total pts2 indications
CompletedCurrentUpcoming
Catalysts (3)
2025-05-2810mo agoPh3 Readout· SMA
2027-03-1611mo awayPh3 Readout· SMA
2029-07-133.3y awayPh3 Readout· Myelofibrosis
Trial Timeline
Q3Q42019Q2Q3Q42020Q2Q3Q42021Q2Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q42028Q2Q3Q42029Q2Q3
Approved
Active
Approved
Recruit…
Approved
Not yet…
Catalysts
Ph3 Readout
2025-05-28 · 10mo ago
SMA
Ph3 Readout
2027-03-16 · 11mo away
SMA
Ph3 Readout
2029-07-13 · 3.3y away
Myelofibrosis
RecruitingActiveNot yet recruiting|StartCompletionToday
Trials (3)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT05244829 | Approved | SMA | Active | 1657 | Safety |
| NCT05557733 | Approved | Myelofibrosis | Recruiting | 1311 | EDSS |
| NCT03557322 | Approved | SMA | Not yet recr... | 1400 | Safety |
Competitors (10)
| Drug | Company | Phase | Target | MOA |
|---|---|---|---|---|
| PFE-7550 | Pfizer | Phase 3 | Aβ | |
| PFE-5767 | Pfizer | Phase 1/2 | BET | |
| RHH-1969 | Roche | Approved | BET | |
| Ribosacituzumab | Roche | Approved | Cl18.2 | |
| RHH-8482 | Roche | Phase 1 | BTK | |
| Pexatenlimab | Novartis | Phase 1/2 | GPRC5D | |
| NVS-5439 | Novartis | Preclinical | BET | |
| Ivocapivasertib | Bristol-Myers Squibb | Phase 1 | BET | |
| TAK-1836 | Takeda | Preclinical | EGFR | |
| Adagracapivasertib | BeiGene | NDA/BLA | BET |