New Phase 1 Study Begins Testing Promising Alzheimer’s Treatment
Key Takeaways
- Voyager Therapeutics has begun a phase 1 trial for its anti-tau antibody VY-TAU01.
- VY-TAU01 aims to inhibit the spread of the tau protein implicated in Alzheimer's disease.
- Initial tau PET imaging data from the study is expected by 2026.
Did You Know?
Introduction to the Study
Voyager Therapeutics has begun a new phase 1 study for its anti-tau antibody VY-TAU01, a potential treatment for Alzheimer’s disease (AD). This investigational drug aims to stop the spread of pathological tau, a protein closely linked to the progression and cognitive decline seen in AD patients.
Study Design and Goals
The ongoing study is a single-ascending dose (SAD), double-blind, placebo-controlled trial conducted at a single center. Approximately 48 healthy volunteers will participate, divided into multiple cohorts. The main goal is to gather preliminary data that will shape a future phase 1b trial, focusing on patients with early AD.
How VY-TAU01 Works
VY-TAU01 is a recombinant, humanized IgG4 monoclonal antibody administered intravenously. Unlike other anti-tau antibodies, it targets a specific region of the tau protein, known as the C-terminal epitope. Preclinical studies have shown promising results, with VY-TAU01 significantly slowing down the spread of tau in animal models.
Timeline and Expectations
Initial tau PET imaging data from this study are expected in the second half of 2026. This data will help researchers understand VY-TAU01’s impact on pathological tau. Voyager plans to start the phase 1b trial in 2025, pending successful outcomes from the current study.
Specific Advantages of VY-TAU01
Voyager Therapeutics believes VY-TAU01 stands out due to its targeted epitope. During the 2022 Alzheimer’s Association International Conference (AAIC), data indicated that intravenously delivered anti-tau antibodies could inhibit more than 70% of pathological tau spreading in mice.
Company Statements
According to Toby Ferguson, MD, PhD, chief medical officer at Voyager, the start of this clinical trial marks a significant milestone. “Alzheimer disease remains an area with tremendous unmet patient need, despite recent advances. We are encouraged by our preclinical data demonstrating the ability of VY-TAU01 to significantly slow tau spreading, and we look forward to evaluating its therapeutic potential in the clinic,” said Dr. Ferguson.
Previous Studies on Anti-Tau Antibodies
In the analysis presented at AAIC 2022, Voyager evaluated a pool of 728 anti-tau antibodies. Out of these, four were selected for their promising characteristics. These antibodies showed robust reductions in tau pathology in treated mice, particularly AB01, which demonstrated the highest efficacy.
Why Focus on Tau Instead of Amyloid-ß?
Historically, amyloid-ß-targeting therapies for AD have shown limited success, prompting a shift towards tau protein, the main component of neurofibrillary tangles in AD. Most tau-targeting therapies under trial are immunotherapies that can target tau both inside and outside cells.
Important Considerations in Antibody Selection
When choosing antibodies for clinical trials, factors such as the choice of epitope, antibody subclass, charge, patient population, and action mechanism are critically evaluated. These considerations ensure the selection of the most promising candidates for further investigation.
Future Prospects
The ongoing development of VY-TAU01 underscores the potential in targeting tau proteins for AD treatment. As research progresses, it offers a beacon of hope for millions affected by Alzheimer's, aiming to bring a much-needed breakthrough in the treatment of this debilitating disease.
