Skyhawk Therapeutics to Present Additional Positive Topline Data from Parts A and B of Phase 1 Clinical Trial of SKY-0515 at European Huntington's Disease Network and Enroll-HD 2024

The presentation highlights additional positive topline results from SAD/MAD study and preclinical data of SKY-0515, a novel small molecule designed to reduce both HTT and PMS1 proteins, two key drivers of Huntington's disease

BOSTON, Sept. 11, 2024 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate critical RNA targets, today announced that additional positive topline data from Parts A and B of the company's Phase 1 clinical trial of SKY-0515 will be presented at the European Huntington's Disease Network and Enroll-HD 2024 meeting to be held from September 12-14, 2024 in Strasbourg, France.

SKY-0515 demonstrated an average huntingtin (HTT) mRNA reduction of 72% at a daily oral dose of 9mg and was generally well tolerated at all doses tested in Parts A and B of its Phase 1 clinical trial.  Given these positive results, Part C of the Company's Phase 1 trial is now enrolling patients with Huntington's, and topline data are expected in Q2 2025. 

Details of the data presentation are as follows:

"We are pleased to be presenting additional clinical data which further differentiate SKY-0515 at a meeting dedicated to advancing treatments for Huntington's disease, a rare hereditary neurodegenerative disease for which there are no approved disease-modifying therapies," said Clint Musil, CEO at Skyhawk. "Lowering HTT and stabilizing somatic expansion via PMS1 are two important therapeutic approaches being pursued in HD research. We look forward to sharing our data with leading HD researchers and clinicians as we believe they underscore the potential of SKY-0515 to achieve both these important clinical objectives."

About SKY-0515 Phase 1 Clinical Study 
SKY-0515 is currently being evaluated in a Phase 1 clinical trial. The Phase 1 clinical trial is a first-in-human trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics, specifically blood biomarker modulation activity, of SKY-0515 in healthy volunteers and individuals with early-stage Huntington's disease (HD). The trial is separated into three parts. Parts A and B evaluated SKY-0515 in healthy volunteers.

Part A was a double-blind placebo-controlled single ascending dose study in healthy adult volunteers. In Part A, five cohorts of participants were dosed with escalating single doses of SKY-0515 ranging from 1mg to 16mg or placebo. Additionally, the influence of food on the pharmacokinetics of SKY-0515 was examined in a dedicated cohort.

Part B was a double-blind placebo-controlled multiple ascending dose study in healthy adult volunteers. In Part B, three cohorts of participants were randomized to receive multiple ascending doses of SKY-0515 ranging from 1mg to 9mg or placebo administered daily from days 1 to 14 (inclusive). Dose levels of SKY-0515, identified in Parts A and B, will be evaluated in Part C.

Part C is a double-blind placebo-controlled parallel design study of two dose levels of SKY-0515 and placebo in individuals with early-stage HD (HD-ISS Stage 1, 2, or mild Stage 3) preceded by an observational period lasting a minimum of 28 days, which aims to evaluate pharmacodynamic parameters such as mutant HTT protein and mRNA. Recruitment for Part C has begun, and topline data are expected in Q2 2025.

Additional information for the SKY-0515 Phase 1 study, including participating sites and eligibility criteria, can be found at the Australian New Zealand Clinical Trials Registry.

About Skyhawk Therapeutics
Skyhawk Therapeutics is a clinical-stage biotechnology company focused on the discovery and development of novel small molecule therapies designed to modulate critical RNA targets and revolutionize patient treatment for some of the world's most intractable diseases. Skyhawk's discovery expertise is rooted in its proprietary drug discovery platform, which assesses, identifies, and tests RNA splicing targets and small molecules across a broad range of therapeutic areas and disease states. Skyhawk has built collaborations with multiple pharma partners that leverage Skyhawk's novel platform across disease areas including neurodegenerative disease, autoimmune disease, and oncology. For more information visit www.skyhawktx.com.

Skyhawk Contacts
Kyle Dow, VP Corporate Development
[email protected] 

Investor Contact
Anne Marie Fields
Precision AQ
[email protected]
332-213-1956

SOURCE Skyhawk Therapeutics