Skyhawk Therapeutics Expands Global Phase 2/3 Trial of SKY-0515 for Huntington’s Disease After New Regulatory Approvals

Skyhawk Therapeutics has received additional regulatory approvals to expand its global Phase 2/3 FALCON-HD clinical program for SKY-0515, an investigational oral treatment for Huntington's disease.

The company announced that the US Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application. At the same time, regulatory authorities in Canada and the United Kingdom have also accepted Clinical Trial Applications (CTAs), allowing the company to begin patient enrollment in all three countries.

These approvals mark an important step as Skyhawk moves its lead Huntington's disease therapy into a larger global pivotal trial.

Patient Enrollment Begins Across Three Countries

With these regulatory clearances in place, Skyhawk is now enrolling patients in the United States, the United Kingdom, and Canada for the Phase 2/3 FALCON-HD study.

According to Sergey Paushkin, Head of Research and Development at Skyhawk Therapeutics, the company believes the latest clinical findings support the continued development of SKY-0515.

He said that patient and clinician assessments after 12 months of treatment, together with improvements seen in important biomarkers and clinical outcome measures, suggest that SKY-0515 could become the kind of convenient daily treatment many Huntington's disease patients have been waiting for.

SKY-0515 Continues to Show Encouraging Safety Profile

Skyhawk reported that SKY-0515 has demonstrated strong penetration into the central nervous system while maintaining a favorable safety profile across all dose levels studied so far.

The company also stated that the treatment has generally been well tolerated during clinical development, supporting its advancement into larger studies.

About the FALCON-HD Phase 2/3 Trial

The FALCON-HD clinical program is designed as a randomized, double-blind, placebo-controlled, dose-ranging study evaluating the safety, efficacy, and pharmacodynamic effects of SKY-0515.

The program consists of two major studies.

The first study, FALCON-HD 004-ANZ, enrolled 144 patients with Stage 2 and early Stage 3 Huntington's disease across Australia and New Zealand. Enrollment for this portion of the study has already been completed.

The second study, FALCON-HD 004-WW, is significantly larger and plans to enroll up to 400 additional patients across more than 40 clinical sites worldwide. Several study sites have already started treating patients.

Researchers will evaluate how well SKY-0515 slows disease progression while continuing to monitor safety and tolerability throughout treatment.

Earlier Clinical Study Helped Build Confidence

The ongoing Phase 2/3 program builds on results from Skyhawk's earlier Phase 1/2 clinical trial.

That first-in-human study evaluated the safety, tolerability, pharmacokinetics, pharmacodynamics, and early clinical activity of SKY-0515 in both healthy volunteers and people living with early-stage Huntington's disease.

The trial included three parts.

The first two parts focused on healthy volunteers, while the third part enrolled patients with Stage 1, Stage 2, and mild Stage 3 Huntington's disease.

Participants received treatment over an initial 84-day period followed by a blinded 12-month extension study in which all participants continued receiving active therapy at either a low or high dose.

Enrollment in the Phase 1/2 program has now been completed.

How SKY-0515 Works

SKY-0515 is an investigational oral small-molecule RNA splicing modifier developed using Skyhawk's proprietary SKYSTAR platform.

The therapy is designed to reduce both mutant huntingtin (mHTT) protein and PMS1 protein, two important biological targets linked to Huntington's disease.

By targeting RNA before harmful proteins are produced, the company hopes the treatment may slow disease progression rather than simply managing symptoms.

Why Huntington's Disease Still Needs Better Treatments

Huntington's disease is a rare inherited neurological disorder that gradually damages nerve cells in the brain.

The disease affects more than 40,000 people in the United States, while hundreds of thousands of individuals are impacted globally through family history and genetic risk.

Patients experience worsening movement problems, cognitive decline, and psychiatric symptoms over time.

Although several therapies help manage symptoms, there are currently no approved treatments proven to slow or stop the progression of Huntington's disease.

This continues to leave a major unmet medical need for patients and their families.

Skyhawk Continues Expanding Its RNA Medicine Pipeline

Beyond SKY-0515, Skyhawk Therapeutics is continuing to build its pipeline of RNA-targeting medicines using its proprietary SKYSTAR platform.

The company says it is developing several additional therapies for rare neurological diseases that currently have no approved disease-modifying treatments.

Skyhawk expects multiple new development programs from its pipeline to enter clinical testing by the end of 2027.

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