Skyhawk's novel RNA-targeting small molecule for Huntington's Disease, SKY-0515, is now in its Phase 2/3 FALCON-HD clinical trial, having demonstrated a 72% reduction in HTT mRNA in its Phase 1 Healthy Volunteer MAD study
SKYHAWK invents novel small molecules that modify RNA expression, targeting some of the world's most intractable diseases.​
Our unique platform accelerates building RNA targeting small molecules across a range of fields including neurology, oncology and fibrotic diseases.
We have built collaborations with BMS, Biogen, Genentech, Ipsen, Merck, Sanofi, Takeda, Vertex and Merck KGaA, Darmstadt, Germany, that leverage our novel SKYSTAR® platform to target challenging diseases in a variety of therapeutic areas.
Small Molecules that Modify RNA Expression
Skyhawk
Latest News
Skyhawk Therapeutics Announces Positive First Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington's Disease
Skyhawk announced positive results from the first interim analysis of the Part C patient cohort in its Phase 1 clinical trial of SKY-0515, an investigational treatment for Huntington's disease (HD). At Day 84, patients receiving SKY-0515 demonstrate dose-dependent reductions of mHTT protein in blood including 62% at the 9mg dose. SKY-0515 has been generally well tolerated at both dose levels tested.
Our Collaborations
Skyhawk’s eight Pharma partnerships have sparked rapid investment in our proprietary platform.
Skyhawk’s Pharma collaborations have contributed biology insights, commercial perspectives and upfront payments of over $525 million to develop targeted drug candidates – driving growth in Skyhawk’s platform.
Now enrolling for our Falcon HD trial for treatment of Huntington's Disease
The trial aims to assess the potential of SKY-0515 to modulate RNA splicing and reduce the production of huntingtin (HTT) and PMS1 proteins, which are implicated in the pathology of Huntington's disease. Additional countries will be added to this trial soon.
Growing the
Pipeline for Patients
SKYHAWK is the clinic with SKY-0515, targeting Huntington's Disease. Our next two internal neurology programs, SKY-1300 and SKY-1500, address Spinocerebellar Ataxia Type 3, and Frontotemporal Dementia (FTD) respectively, each an indication with no approved therapeutic agents.
