Alector (ALEC) Cantor Global Healthcare Conference 2025 summary

Company overview and strategy

• Focused on curing neurodegenerative disorders using three approaches: removing misfolded proteins, replacing damaged or missing proteins, and repairing immune/nerve cells.

• Pipeline includes two late-stage programs for protein replacement and multiple preclinical programs targeting misfolded proteins.

• Progranulin loss is a universal risk factor for several neurodegenerative diseases; drugs aim to restore or elevate progranulin levels.

• Brain carrier (shuttle) platform enables delivery of various drug modalities, including antibodies, enzyme replacement, and siRNA.

• Plans to introduce two new drugs to the clinic by 2026.

Lead program: FTD and latozinemab

• Latozinemab elevates progranulin by blocking its degradation, restoring levels in FTD patients with progranulin mutations.

• Phase II INFRONT-2 showed normalization of progranulin, 48% slowdown in cognitive decline, and positive biomarker changes.

• Phase III INFRONT-3 is a two-year, placebo-controlled study with 106 symptomatic and 16 pre-symptomatic patients, using CDR-Sum of Boxes and plasma progranulin as co-primaries.

• High confidence in achieving statistical significance for both primary endpoints; even partial clinical benefit may support approval.

• Drug is well tolerated, with no serious adverse effects reported.

Biomarkers and trial design

• Key biomarkers: progranulin, volumetric MRI, GFAP, neurofilament light chain (NFL), and proteomics.

• GFAP normalizes with treatment but does not quantitatively correlate with disease severity.

• NFL stabilization observed in phase II; not always a consistent marker for drug response.

• Trial includes both early and more severe FTD patients, as well as pre-symptomatic carriers to assess prevention potential.