First patient dosed in phase 1/2a trial of Huntington’s, spinocerebellar ataxia therapy

Key takeaways:

• The trial will evaluate VO659, an antisense oligonucleotide for Huntington’s disease and spinocerebellar ataxia types 1 and 3.
• Preclinical data showed reductions in mutant huntingtin protein.

Vico Therapeutics announced that the first patient has been dosed in a phase 1/2a study evaluating VO659, an investigational antisense oligonucleotide for Huntington’s disease and spinocerebellar ataxia types 1 and 3.

According to a release from Vico, the multicenter, open-label basket study will assess the safety and tolerability of multiple ascending doses of intrathecally administered VO659 in an estimated 71 individuals diagnosed with early manifest Huntington’s or mild to moderate spinocerebellar ataxia type 1 (SCA1) or type 3 (SCA3). The investigational therapy is designed to target the CAG repeat expansion that causes these diseases.

In preclinical studies, VO659 demonstrated significant and dose-dependent reductions of mutant huntingtin protein and improved motor function in vivo, as well as allele-preferential reductions of the protein in Huntington’s patient cell models. Similar reductions in mutant ATXN1 and mutant ATXN3 also were reported in patient cell models of SCA1 and SCA3, per the release.

“We are encouraged by the continued progress of our development program and very pleased to announce the first patient dosed in this phase 1/2a study of VO659 in [Huntington’s disease], SCA1 and SCA3,” Scott Schobel, MD, chief medical officer at Vico, stated in the release. “VO659 is the first allele-preferential [antisense oligonucleotide] in clinical development with broad application to all CAG repeat expansion diseases.”