Amyl Therapeutics

Amyl Therapeutics, a preclinical-stage biotechnology company headquartered in Liège, Belgium, was founded in November 2020 by Dr. Pierre Vandepapelière and Dr. Florent Gros. Dr. Vandepapelière, who serves as CEO and CMO, brings over 30 years of experience in vaccine and biological therapeutics development from roles at GSK and as CEO of Imcyse. Dr. Gros, the company's Chairman, has a background that includes experience at Novartis. The leadership team also includes Chief Scientific Officer Dr. Karine Goraj.

The company is focused on developing treatments for a range of diseases caused by the misfolding and aggregation of amyloid proteins, such as neurodegenerative conditions like Alzheimer's and Parkinson's, as well as systemic amyloidosis. The business operates in the biotechnology and pharmaceutical market, developing therapeutic candidates for which it will likely seek partnerships with larger pharmaceutical companies for late-stage clinical development, manufacturing, and commercialization. Its business model centers on raising capital through equity financing and non-dilutive grants to fund research and development through the preclinical and early clinical stages.

Amyl Therapeutics' core technology is its Pan-Amyloid Immunotherapy (PAI) platform, previously known as ClariTY. This platform develops novel therapies designed to universally target a common structural feature shared across all types of toxic amyloid aggregates, including amyloid-beta, tau, and alpha-synuclein. This approach differs from many existing treatments that target only a single type of misfolded protein. The therapeutic candidates aim to both clear existing toxic aggregates and inhibit the formation of new ones, offering potential for treatment and prevention. The company states its molecules are being engineered for enhanced brain penetration and a reduced risk of side effects like ARIA (amyloid-related imaging abnormalities) that have been concerns with other antibody treatments. The company's initial focus is on amyloidosis, with plans to start a Phase I trial in 2025, before expanding into Alzheimer's and Parkinson's disease. A lead program is focused on Down Syndrome-associated Alzheimer’s Disease (DS-AD), which qualifies for orphan drug designation.

Since its founding, Amyl Therapeutics has secured significant funding. In June 2021, it closed a Series A round of €18.3 million, consisting of equity and non-dilutive funding. In November 2023, the company announced an additional €11.1 million, comprising a €5 million extension to its Series A from investors including Merieux Equity Partners and Noshaq, and €6.1 million in non-dilutive financing from the Walloon region. This capital is intended to advance its lead candidates to strong preclinical proof-of-concept and prepare for clinical development.

Keywords: Pan-Amyloid Immunotherapy, neurodegenerative diseases, amyloidosis, Alzheimer's treatment, Parkinson's treatment, protein misfolding diseases, preclinical biotechnology, ClariTY platform, amyloid fibrils, Florent Gros, Pierre Vandepapelière, systemic amyloidosis, Liège biotech, amyloid-beta, tau protein, alpha-synuclein, orphan drug designation, Down Syndrome-associated Alzheimer’s Disease, biologics development, monoclonal antibodies alternative