Vico Therapeutics has started patient dosing in an expanded cohort under a twice-yearly regimen for its Phase I/IIa clinical trial of VO659 in Huntington’s disease (HD), spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1).
Encompassing several European countries, the study is designed to evaluate VO659, an antisense oligonucleotide therapy targeting the CAG repeat expansion associated with multiple polyglutamine diseases.
VO659 aims to address nine polyglutamine disorders, including HD, SCA3 and SCA1.
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Earlier results indicated a 38% reduction in cerebrospinal fluid (CSF) mutant HTT (mHTT) and a 2.5% reduction in CSF neurofilament light chain (Nf-L) after four months of dosing with VO659 in HD patients.
The current study evaluates the tolerability, safety, pharmacodynamic, and pharmacokinetic effects of six-month dosing intervals over 12 months following intrathecal administration.
According to Vico Therapeutics, several participants have received doses so far, with the treatment showing tolerability and good safety, as well as no serious adverse events to date.
Vico Therapeutics CEO Micah Mackison said: “I am very encouraged by our earlier results showing a significant reduction of mHTT directly in the central nervous system with a favourable CSF Nf-L profile early in the time course.
“The twice annual patient-friendly dosing regimens will enable us to assess longer-term safety and pharmacodynamic effects, taking advantage of VO659’s long half-life.”
The clinical trial is proceeding under an approved clinical trial application in Europe.
Vico Therapeutics has secured approval from the US Food and Drug Administration (FDA) for its investigational new drug (IND) application and intends to start US-based trials for VO659 within the year.
At CHDI’s 21st Annual HD Therapeutics Conference in Palm Springs, California, Vico Therapeutics’ chief scientific officer Nicole Datson will present new data on the mechanism of action of VO659. Additionally, chief medical officer Scott Schobel will provide an update on the ongoing Phase I/IIa trial.
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