Global clinical trial testing Huntington’s pill to open in 3 new countries
The results, if positive, will support applications for regulatory approval
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A global clinical trial testing SKY-0515, Skyhawk Therapeutics’ daily oral therapy for Huntington’s disease, will soon start recruiting adults with the genetic condition in the U.S., Canada, and the U.K.
Regulatory authorities in all three countries have granted permission to open sites for the worldwide portion (004-WW, NCT07378644) of the pivotal FALCON-HD study, which began earlier this year. The Phase 2/3 trial is now recruiting participants ages 25 and older in Argentina, Brazil, Chile, and Georgia. This portion aims to enroll about 400 people with Huntington’s whose disease is confirmed through genetic testing.
“We are excited to now begin enrolling patients in our pivotal trial for Huntington’s in the United States, the United Kingdom and Canada,” Sergey Paushkin, Skyhawk‘s head of research and development, said in a company press release. Pivotal trials are those designed to produce robust results that, if positive, can be used to support applications seeking regulatory approvals of a therapy.
Another portion of this study (004-ANZ; NCT06873334) is being conducted at sites in New Zealand and Australia, and has completed recruitment — of a total 144 participants — ahead of schedule, per the developer.
A neurodegenerative disease, Huntington’s is caused by mutations in the gene that encodes the huntingtin protein. Taken as a once-daily pill, SKY-0515 is designed to reduce levels of both healthy and mutant forms of huntingtin, as well as another protein called PMS1 that is thought to help drive the disease.
10 countries now involved in global clinical trial of SKY-0515
FALCON-HD was launched after a first-in-human Phase 1/2 clinical trial (ACTRN12623001161617) in Australia suggested SKY-0515 had an adequate safety profile for further testing. Additionally, early data hinted that the experimental treatment might slow Huntington’s progression.
“Clinician and patient assessments done at twelve months of treatment [in the Phase 1/2 study] support what the SKY-0515’s compelling and consistent effects on critical biomarkers and [disease progression] scores demonstrate — the exciting possibility that SKY-0515 may offer Huntington’s patients a type of therapy they have long deserved, in a convenient daily pill,” Paushkin said.
Spurred by the promising early findings, Skyhawk launched FALCON-HD to evaluate the therapy’s safety and pharmacological properties, as well as its effectiveness. The trial will ultimately involve more than 500 adults with stage 2 or early-stage 3 Huntington’s.
Participants are being randomly assigned to daily treatment with either SKY-0515 — at one of three doses — or a placebo for up to 72 weeks, or nearly 1.5 years. Researchers will assess changes in blood levels of mutant huntingtin and brain volume. Validated measures of disease progression, including the Composite Unified Huntington’s Disease Rating Scale, will also be used for evaluation.
According to Skyhawk, “more than ten countries and more than twenty sites are now participating in the Company’s global pivotal trial for Huntington’s disease, with over 175 patients enrolled.”
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