Skyhawk Therapeutics Reports Positive Results for SKY-0515 in Huntington's Disease Phase I Trial, Demonstrating Up to 72% Reduction in Huntingtin Protein mRNA
Friday, July 12, 2024
Skyhawk Therapeutics, Inc. has announced positive results from Parts A and B of its Phase 1 clinical trial of SKY-0515 for Huntington's disease (HD). SKY-0515, a novel small molecule RNA splicing modulator developed by Skyhawk's RNA splicing platform, achieved an average reduction of 72% in HTT mRNA levels at a daily oral dose of 9 mg. The drug was well tolerated across all tested doses.
SKY-0515 is designed to lower levels of HTT and PMS1 proteins, key drivers of HD pathology and somatic CAG repeat expansion. This dual-target approach is expected to offer greater therapeutic benefits compared to targeting HTT alone, addressing an unmet need in the treatment landscape for Huntington's disease, which currently lacks disease-modifying therapies.
Professor Ed Wild from University College London commented on the potential of reducing both HTT and PMS1 levels, highlighting the significance for patients. Huntington's disease affects over 40,000 individuals in the United States and is characterized by inherited neurodegeneration.
Skyhawk Therapeutics, emphasized SKY-0515's effectiveness in reducing HTT mRNA and potentially inhibiting PMS1 protein, noting its favorable safety profile in the study. The Phase 1 trial has progressed to include patients with early-stage Huntington's disease, with recruitment ongoing and pivotal data expected in mid-2025.
Skyhawk Therapeutics, expressed optimism about SKY-0515's advancements and its potential impact on disease management. The Phase 1 trial aims to evaluate the drug's safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers and early-stage HD patients across its three parts: single-dose evaluation in healthy adults (Part A), multiple-dose escalation (Part B), and ongoing patient-focused evaluation (Part C). This comprehensive approach aims to provide critical insights into SKY-0515's therapeutic potential.
Source: globenewswire.com
