Skyhawk's novel RNA-targeting small molecule for Huntington's disease, SKY-0515, is now in its Phase 2/3 FALCON-HD worldwide clinical trial, having demonstrated a 62% reduction in HTT protein, cUHDRS improvement at 3, 6 and 9 months, and continued safety, in its ongoing Phase 1C patient 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 Releases Twelve-Month cUHDRS Subcomponent Results from its Phase 1/2 Clinical Trial of SKY-0515 in Huntington’s Disease
Twelve-month findings showed favorable and consistent trends for participants receiving SKY-0515 across all four cUHDRS subcomponents, including Total Functional Capacity (TFC), Total Motor Score (TMS), Symbol Digit Modalities Test (SDMT), and Stroop Word Reading Test (SWRT)
Skyhawk also releases Clinician and Patient Global Impression (CGI and PGI) improvement of disease survey data, which shows that at twelve months no clinician or patient assessed disease progression – all saw either disease stabilization, or improvement
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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 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 in the clinic with SKY-0515, targeting Huntington's Disease. Our next three internal neuro programs, SKY-0925, SKY-2100, and SKY-4200 address rare-neuro indications with significant unmet needs and limited therapeutic options.
