Voyager Therapeutics (VYGR) investor relations material

Key scientific and clinical updates

• Focus for 2024 is on advancing two tau-directed assets: a C-terminal antibody and a gene therapy tau knockdown, with key tau PET imaging data expected by year-end and a pivotal readout for a similar asset (BIIB080) mid-year.

• First clinical use of newly discovered blood-brain barrier-penetrant capsids, with two programs entering the clinic: tau knockdown gene therapy and a Neurocrine-partnered Friedreich's ataxia program.

• Additional data on the shuttle platform, derived from the capsid discovery platform, will be presented, representing three major inflection points for the year.

Tau as a disease driver and therapeutic target

• Human genetics do not strongly implicate tau in Alzheimer's, but clinical and pathological data suggest tau progression is closely linked to cognitive decline.

• Amyloid abnormalities precede tau accumulation, but the spread of tau correlates with disease staging and progression.

• Reducing pathological tau, rather than amyloid, may be more directly related to slowing or preventing dementia.

Development strategy and regulatory engagement

• Ongoing, constructive interactions with the FDA, including pre-IND and Type C meetings, to ensure safe and effective study design for tau knockdown gene therapy.

• Initial clinical goal is to demonstrate tau reduction in the brain using PET imaging, following the precedent set by BIIB080.

• IND filing for tau knockdown gene therapy planned for Q2, with clinical entry in the second half of the year.