Vigil Neuroscience, Inc. announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion on the Company's application for orphan drug designation for VGL101 for the treatment of CSF1R-related leukoencephalopathy, which includes adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). The Company is currently evaluating VGL101 in a Phase 2 proof-of-concept trial in patients with ALSP. VGL101 was previously granted orphan drug designation by the U.S. Food and Drug Administration for ALSP in July 2022.

The EMA may grant orphan drug designation based on a positive opinion issued by the COMP. The EMA's orphan drug designation is available to sponsors developing therapies that aim to treat or prevent rare, life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU, and for which no treatment is approved. Medicines that meet the EMA's orphan drug designation criteria qualify for certain benefits, such as reduced regulatory fees, protocol assistance, research grants, and, subject to obtaining and maintaining orphan medicine status, up to ten years of market exclusivity in the EU upon approval.

About the Phase 2 IGNITE Trial The Company's ongoing Phase 2 IGNITE trial is a global, open-label clinical trial evaluating VGL101 in approximately 15 patients with symptomatic ALSP who have a confirmed CSF1R gene mutation. As part of the protocol, patients will receive an intravenous (IV) infusion of VGL101 at 20 mg/kg or 40 mg/kg approximately every four weeks, for a treatment duration of one year. The primary objective of the IGNITE trial is to evaluate the safety and tolerability of VGL101.

Secondary objectives include evaluating the impact of VGL101 on magnetic resonance imaging (MRI) and its pharmacodynamics effect on fluid biomarkers in patients with symptomatic ALSP. Clinical efficacy outcome measures are also being collected as exploratory endpoints. In the fourth quarter of 2023, the Company expects to report interim 6-month data from the IGNITE trial in the first 6 patients who have received 20 mg/kg of VGL101.