Clinical Studies
Skyhawk is advancing RNA-targeted small molecule therapies into the clinic, with a focus on diseases of high unmet need.
SKY-0515 — FALCON-HD Trial
Huntington's Disease
About the Trial
The FALCON-HD trial is a global Phase 2/3 study evaluating SKY-0515, an oral small molecule designed to reduce mutant huntingtin (HTT) protein in patients with Huntington's Disease. SKY-0515 works by modifying the splicing of the HTT mRNA, reducing the production of the toxic mutant protein.
Phase 1 data demonstrated a 62% reduction in mutant HTT protein levels in cerebrospinal fluid, along with clinically meaningful improvements in cUHDRS at 3, 6, and 9 months of treatment.
Key Results
- 62% reduction in mutant HTT protein (CSF)
- cUHDRS improvement at 3, 6 and 9 months
- Continued safety in ongoing Phase 1C patient study
- Oral, once-daily small molecule administration
- TGA (Australia) and global regulatory approvals obtained
Trial Information
- Identifier
- FALCON-HD
- Phase
- Phase 2/3
- Indication
- Huntington's Disease
- Route
- Oral
- Target
- HTT mRNA splicing
- Status
- Now enrolling worldwide
Interested in participating in the FALCON-HD trial?
If you or a loved one has Huntington's Disease and would like to explore eligibility for the FALCON-HD trial, complete our patient inquiry form and our team will follow up with information about enrollment sites near you.
See if you're eligible