Apilimod
As of the edition of 20 October 2026, Apilimod is a small molecule: of no established status; acts on PIKFYVE.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Status not established
- Kind
- Small molecule
- Registered trials
- 6, 0 stopped early
- Disease areas
- Digestive system; Nervous system; Nutrition and metabolism; Blood and lymph; Cancer; Immune system
US protection
No US approval on record.
With no US approval on record there are no listed US patents or exclusivities.
This is the US only.
Target
| Gene | Protein | UniProt |
|---|---|---|
| PIKFYVE | 1-phosphatidylinositol 3-phosphate 5-kinase | Q9Y2I7 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase not stated | 1 | 0 | 0 | 0 |
| Phase 1 | 1 | 1 | 0 | 0 |
| Phase 2 | 4 | 4 | 0 | 0 |
| All trials | 6 |
Who ran its trials
| Company | Role | Years |
|---|---|---|
| Madrigal Pharmaceuticals | Lead sponsor of trials | 2005 to now |
| Quince Therapeutics | Lead sponsor of trials |