CAMBRIDGE, Mass. – Vigil Neuroscience, a biotechnology company harnessing the power of microglia for the treatment of neurodegenerative diseases, announced today the enrollment of the first subject in a natural history study dedicated to patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). ALSP is a rare, genetically defined fatal neurodegenerative disease with strong mechanistic and biochemical associations to microglia dysfunction.
“With great sense of responsibility, we have initiated the first ever prospective natural history study in ALSP, designed by patients, advocates and physicians aiming to advance the understanding of this devastating neurodegenerative disease,” said Spyros Papapetropoulos, Vigil’s Chief Medical Officer. “This study is a key component in the development plan of our lead clinical candidate, VGL101, as a potential treatment option for ALSP patients.”
This observational, multicenter study will prospectively follow patients with ALSP to understand the natural course of the disease, evaluate imaging and soluble biomarkers, and provide insights into potential endpoints and patient populations for future interventional trials with VGL101, a fully human monoclonal antibody agonist targeting triggering receptor expressed on myeloid cells 2 (TREM2). The study is expected to enroll participants at sites across the United States, Canada, and Europe.
“There is a high unmet medical need in ALSP, for which there are currently no approved treatments,” said Dr David Lynch, from the Institute of Neurology, University College London, UK. “This study is designed to enable us to identify and evaluate relevant biomarkers as well as potential clinical outcome measures to further advancements in developing new treatments for ALSP patients.”
Details of the study and study centers can be found on clinicaltrials.gov (identifier: NCT05020743). More information about ALSP can be found on ALSPinfo.com.
VGL101, Vigil’s lead product candidate, is a fully human monoclonal antibody agonist targeting human triggering receptor expressed on myeloid cells 2 (TREM2), which is responsible for maintaining microglial cell function. TREM2 deficiency is believed to be a driver of certain neurodegenerative diseases. VGL101 is in development for the treatment of rare microgliopathies, such as adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), as well as other neurodegenerative diseases for which TREM2 and/or microglia deficiency is believed to be a key driver of disease pathway.
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a rare, inherited, autosomal dominant neurological disease with high penetrance. It is caused by a mutation to the CSF1R gene and affects an estimated 10,000 people in the US, with similar prevalence in Europe and Japan. The disease generally presents itself in the fourth decade of life, is diagnosed through genetic testing and established clinical/radiologic criteria and is characterized by cognitive dysfunction, neuropsychiatric symptoms, and motor impairment. These symptoms typically exhibit rapid progression with a life expectancy of approximately seven years on average after diagnosis, causing significant patient and caregiver burden. There are currently no approved therapies for the treatment of ALSP, underlining the high unmet need in this rare indication.
About Vigil Neuroscience
Vigil Neuroscience is a microglia-focused therapeutics company treating both rare and common neurodegenerative diseases by restoring the vigilance of microglia, the sentinel cells of the brain’s immune system. We are utilizing the tools of modern neuroscience drug development across multiple therapeutic modalities in our efforts to rapidly develop precision-based therapies to improve the lives of patients and their families. www.vigilneuro.com
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