RARE Daily

Amyl Raises $22 million to Develop Novel Therapies for Amyloidosis

June 3, 2021

Belgian biotech Amyl Therapeutics closed a $22 million (€18.3 million) series A financing to advance its amyloid fibrils specific ClariTY technology platform for the treatment of all forms of amyloidosis. 

Photo: Pierre Vandepapelière, co-founder, CEO, and chief medical officer

The funding round comprises $10.3 million (€8.6 million) in equity led by Noshaq, a regional Belgian private-public fund, with support from Merieux Participations, Sambrinvest and other private investors; and $11.6 million (€9.7 million) in non-dilutive funding from the DG06, a key policy-design and implementing body for research and innovation policy in the Walloon region of Belgium.

Amyl Therapeutics uses a novel mechanism to bind to the amyloid fold of toxic protein aggregates of various types, including antibody light chain (AL) or transthyretin (TTR) aggregates, which accumulate in peripheral organs to cause systemic amyloidosis diseases, but also amyloid-β (Aβ), Tau, and α-synuclein aggregates, which accumulate in the brain to cause Alzheimer’s and Parkinson’s diseases. Therapies based on the ClariTY technology platform are designed to prevent the further accumulation of aggregates and clear existing aggregates from the peripheral organs and the brain, while also blocking further cell-to-cell spread of misfolded proteins. These therapies may offer a breakthrough approach to treat protein misfolding diseases.

“The ClariTY platform offers the unique opportunity to generate therapeutic candidates able to target all amyloid fibril deposits, a key cause of amyloidosis,” said Pierre Vandepapelière, co-founder, CEO, and chief medical officer. “We believe novel therapies with this profile will represent a major advance in the treatment of diseases caused by amyloid fibrils and will have a significant competitive advantage.”

Amyl will use the series A funding to progress the discovery and preclinical development of therapeutic candidates that are able to target multiple misfolded proteins implicated in both progressive peripheral and neurodegenerative rare diseases. With the funds raised Amyl Therapeutics expects to generate strong preclinical proof of concept for its lead therapeutic candidates and to set up a production process as part of its clinical readiness.

Amyloidosis is a group of diseases characterized by an endogenous production of misfolded proteins that aggregate as insoluble amyloid fibrils and form deposits in various organs and tissues, resulting in organ dysfunction, serious morbidity and even death. More than 30 of such proteins have been identified as amyloid precursors in humans. These are grouped into localized forms, and systemic ones with the most common types of systemic amyloidosis being “light chain” (AL) amyloidosis, hereditary and old age transthyretin (ATTR) amyloidosis, and inflammatory amyloidosis with protein A (AA).

There are currently between 30,000 and 45,000 patients suffering from AL amyloidosis in the United States and Europe, which is the most common form of the disease, affecting about 70 percent of all patients diagnosed with systemic amyloidosis. At present, there is no effective treatment for established amyloidosis diseases.

“By developing therapeutic solutions that could not only stop progression but also remove existing amyloid deposits in organs, we hope to be part of the solution to treat a large populations of Amyloidosis patients,” said Karine Goraj, chief scientific officer of Amyl.

Following the closing of the financing, Vandepapelière, Florent Gros, Amyl co-founder and chairman, Valérie Calenda, managing partner at Merieux Equity Partners, Kenneth Buckfire, president of Miller Buckfire & Co., and Amel Tounsi, investment manager at Noshaq, will join the Board of Directors.

Author: Rare Daily Staff

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