Vigil Neuroscience announces interim data from IGNITE trial

Vigil Neuroscience announced interim data from the company’s Phase 2 IGNITE proof-of-concept clinical trial in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia, or ALSP. The interim data, representing the first six patients following six months of treatment with 20 mg/kg of iluzanebart, further support the favorable safety and tolerability profile as was previously seen in healthy volunteers. In addition, these data demonstrated clear target engagement and downstream pharmacological activity at 20 mg/kg consistent with the company’s previously reported Phase 1 data in healthy volunteers. Directionally supportive changes at six months on magnetic resonance imaging, or MRI, and neurofilament light, or NfL, biomarkers of disease progression in individual patients with ALSP were also observed. The company also reported findings from its ongoing natural history study, ILLUMINATE, which continued to provide critical insights on MRI and NfL biomarkers and supports soluble colony stimulating factor 1 receptor as a potential key biomarker of ALSP disease pathology. Key Highlights from Phase 2 IGNITE Interim Data: Favorable safety and tolerability profile, including no hematologic adverse events. Predictable pharmacokinetic and brain penetration profile consistent with Phase 1 data in healthy volunteers. Clear target engagement, based on sTREM2 levels, and downstream pharmacological activity, based on sCSF1R and osteopontin levels, at 20 mg/kg, consistent with Phase 1 data in healthy volunteers. Directionally supportive changes in both NfL and MRI measurements on ventricular volume and gray matter volume in individual patients. We believe the quality and consistency of the interim data further support the continuation of IGNITE without modification. Key Updates from Natural History Study ILLUMINATE: sCSF1R and NfL levels are remarkably altered in ALSP, supporting our hypothesis that these are key biomarkers of disease pathology. Totality of the data, including longitudinal progression observed on selected MRI measures and clinical endpoints, support engagement with regulatory authorities. We believe the quality and consistency of data in this interim analysis support chosen biomarkers for pharmacological activity. MRI measurements on ventricular volume and gray matter volume are emerging as key indicators of disease progression. Interim Montreal Cognitive Assessment, or MoCA, and Cortical Basal Ganglia Functional Scale data support use as clinical endpoints in ALSP at 12 months.