Vigil Neuroscience (VIGL) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Company overview and strategy

• Focuses on developing microglia-targeted therapeutics for rare and common neurodegenerative diseases by restoring microglia function, with TREM2 identified as a key regulator.

• Differentiates through a precision-based approach, targeting indications with strong genetic and mechanistic links to microglia dysfunction, reducing translational risk.

• Pipeline includes both a fully human monoclonal antibody and a first-in-class small molecule TREM2 agonist, enabling targeting of both rare and common diseases.

• Strategic partnership with Sanofi raised $40 million at a 100% premium, extending cash runway into 2026 and validating the small molecule program.

• Plans to release full phase I small molecule data and final ALSP program analysis in the first half of next year.

ALSP program and clinical progress

• ALSP is a rare, devastating neurodegenerative disease caused by CSF1R mutations, leading to microglia dysfunction and rapid progression.

• No approved therapies exist, and the company is first to enter this space, addressing a significant unmet need.

• TREM2 activation is hypothesized to compensate for CSF1R deficiency, supported by preclinical and clinical evidence.

• Phase II IGNITE study uses a fully human monoclonal antibody, with interim data showing slowing of disease progression in key biomarkers.

• Ongoing engagement with the FDA has opened the door to accelerated approval, with a focus on biomarker-driven endpoints.

Biomarker strategy and regulatory engagement

• MRI and soluble CSF1R are key biomarkers, with MRI changes correlating strongly with disease progression and cognition.

• The ILLUMINATE natural history study provides critical data for synthetic control arms and regulatory discussions.

• FDA has encouraged further biomarker data and alignment on analysis before final data release, supporting the accelerated approval pathway.

• Patient identification efforts, including ALSP aware, have increased disease awareness and genetic testing, revealing higher prevalence than previously estimated.