Positive interim results announced in phase 2 trial of leukoencephalopathy drug

Vigil Neuroscience Inc. has announced positive interim data from a phase 2 proof-of-concept clinical trial of a monoclonal antibody to treat those suffering from adult-onset leukoencephalopathy with axonal spheroids and pigmented glia.

Interim data from the IGNITE study, analyzing six participants after 6 months of treatment with 20 mg/kg of iluzanebart (VGL101), found favorable safety, tolerability and pharmacokinetic profiles as well as positive downstream pharmacological activity based on sCSF1R and osteopontin levels at a dose of 20 mg/kg, both consistent with previously reported phase 1 data in healthy volunteers, according to a company release.

The researchers also observed positive changes at 6 months on MRI and neurofilament light (NfL) biomarkers of disease progression in individual patients with ALSP.

The open-label proof-of-concept trial will eventually evaluate iluzanebart in approximately 15 patients with symptomatic ALSP who also have a confirmed CSF1R gene mutation. Participants will receive an IV infusion of iluzanebart at 20 mg/kg or 40 mg/kg approximately every 4 weeks for 1 year.

"The positive interim results from our phase 2 IGNITE trial represent the first clinical data reported from an interventional study in patients with ALSP and reaffirm our belief in the potential of iluzanebart as a novel treatment option,” Ivana Magovčević-Liebisch, PhD, JD, president and CEO of Vigil, said in the release.

Vigil expects to release additional data from the IGNITE trial, including results for enrollees in both the 20 mg/kg and 40 mg/kg cohorts at 6 months, by the third quarter of 2024.

Editor's Note: this article was updated to correct the spelling of Vigil's president and CEO.