Vigil Neuroscience (VIGL) Goldman Sachs 45th Annual Global Healthcare Conference summary

Scientific and clinical focus

• Developing novel microglia-targeted therapies for rare and common neurodegenerative diseases, with initial focus on TREM2 as a key modulator.

• Employing both monoclonal antibody and small molecule TREM2 agonists, each positioned for specific indications.

• Precision medicine approach targets diseases with clear genetic links to microglia dysfunction.

• Experienced management team has delivered on all milestones in four years.

ALSP program and clinical progress

• Iluzanebart (TREM2 agonist antibody) is in phase II for ALSP, a primary microgliopathy with aggressive progression and no approved treatments.

• Natural history study (ILLUMINATE) is ongoing to understand disease progression and identify biomarkers for accelerated approval.

• Interim phase II data show good safety, tolerability, and encouraging biomarker changes, including increased soluble CSF1R and slowed disease progression in some patients.

• Patient engagement with FDA and introduction of ALSPAware, a no-cost genetic testing program, support community needs and regulatory alignment.

• Recent prevalence data suggest ALSP is more common than previously thought, with significant opportunity for impact.

Biomarker and regulatory strategy

• Biomarkers such as soluble TREM2, CSF1R, and NfL are central to both natural history and interventional studies.

• Strong correlations between cognitive decline and MRI volumetric changes support use of imaging as a potential surrogate endpoint.

• FDA is open to MRI as a biomarker for accelerated approval, pending further data.