BREAKING: Skyhawk Therapeutics has just announced promising interim results from its Phase 1 clinical trial of SKY-0515 for treating Huntington’s disease. The trial, which includes patients with early-stage Huntington’s, shows a remarkable 62% reduction in mutant huntingtin (mHTT) protein at the 9mg daily oral dose by Day 84.
This urgent development highlights the potential of SKY-0515 as a groundbreaking treatment for a condition that currently affects over 40,000 symptomatic patients in the United States alone, with hundreds of thousands more globally facing this devastating disease. The drug has demonstrated excellent brain penetration and a favorable safety profile, raising hopes for those suffering from Huntington’s disease.
“SKY-0515 is reducing mHTT protein to the most impressive extent we’ve seen so far in patients,” stated Ed Wild, Professor of Neurology at University College London. He emphasized that the clinical and biomarker data show no safety concerns at any tested dose. This news represents a significant milestone in the fight against Huntington’s disease, a hereditary condition that currently has no approved therapies to slow or halt its progression.
Skyhawk’s Head of R&D, Sergey Paushkin, echoed this sentiment:
“The strength of SKY-0515’s biomarker response after just 84 days of treatment underscores its potential as a transformative therapy for HD.”
The ongoing Phase 2/3 FALCON-HD trial, currently enrolling participants in Australia and New Zealand, aims to evaluate the efficacy and safety of SKY-0515 in a larger cohort. This trial will assess various dose levels over a treatment period of at least 12 months, further investigating the drug’s ability to modulate RNA splicing and reduce both mHTT and PMS1 proteins—key drivers of Huntington’s pathology.
As SKY-0515 continues to show positive results, the implications for patients are profound. The prospect of an orally administered treatment could be a game-changer, offering hope to those who have long faced a grim prognosis.
Skyhawk Therapeutics is committed to accelerating the development of novel therapies for rare neurological diseases, with plans to introduce additional treatments into clinical trials by the end of 2027. The first of these is set to initiate trials in mid-2026.
As the clinical landscape shifts, the medical community and patients alike are closely monitoring these developments. This is a critical moment for Huntington’s disease research, and the world is watching. For ongoing updates, stay tuned as we follow Skyhawk’s journey towards potentially transformative therapies.
