Voyager Therapeutics (VYGR) Presents New Data Demonstrating Novel Candidate Therapeutic Antibodies Reduced Tau Pathology in Multiple Preclinical Models

Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy and neuroscience company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today presented data showing the use of novel antibodies targeting hyperphosphorylated forms of tau. Two therapeutic approaches were used to show that the spread of pathological tau can be impeded in rodent models: passive immunotherapy with novel antibodies and an intravenously delivered vectorized novel antibody using a blood-brain-barrier penetrating AAV capsid. The data were presented at the Alzheimer’s Association International Conference.

Tauopathies such as Alzheimer’s disease, progressive supranuclear palsy, and frontotemporal dementia are characterized by the progressive accumulation and/or propagation of neurofibrillary tangles associated with tau hyperphosphorylation in the brains of patients, leading to neuronal and synaptic loss, brain atrophy, and inflammation. Although current therapies are limited to symptom management and have a modest impact1, investigational therapeutics designed to disrupt the propagation of tau pathology may hold promise in the treatment of tauopathies.

“The Voyager team has maintained a long-standing focus on the development of novel approaches to disrupt the progression of tau pathology believed to be central to multiple neurodegenerative diseases,” said Todd Carter, Ph.D., Senior Vice President of Research at Voyager. “Our novel passive and vectorized anti-tau antibodies have important properties, including specific targeting of specific pathological epitopes of the tau protein that could offer a differentiated clinical profile compared to first generation approaches. We look forward to advancing research and discovery efforts for our tau program, potentially leading the way to vectorize select antibodies into gene therapy product candidates.”

Passive Antibody Research Highlights:

• 113 anti-tau antibodies were characterized based on specified biochemical and biophysical properties, including functional inhibition of filamentous tau seeding in vitro and high affinity and specificity for pathological forms of tau, and antibodies that met the desired target profile were selected for in vivo efficacy screening.
• Four novel antibodies targeting epitopes of the mid- and C-terminus tau domains were selected.
• Selected antibodies were demonstrated to block the seeding/propagation of filamentous tau in a P301S seeding-propagation tauopathy mouse model and demonstrated substantial reduction of induced tau pathology.

Vectorized Antibody Research Highlights:

• Vectorized antibody expression was detected in the hippocampus, cortex, and cerebrospinal fluid of mice as soon as two days after administration with sustained durability of expression more than six months post-dose.
• The vectorized antibody was observed to be well tolerated at all assessed doses, with robust efficacy observed in all P301S hippocampal seeding/propagation and P301S intrinsic mouse models of tauopathy.

These results build upon previous findings demonstrating distribution and expression of vectorized anti-tau antibodies in the mouse brain using a blood-brain barrier penetrant capsid administered intravenously.

Full results of both posters are available on the Investor page of the Voyager website.

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