Amyl Raises Funds to Develop Therapies for Amyloidosis

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by Marisa Wexler MS |

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Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance therapeutic candidates for familial amyloid polyneuropathy (FAP) and other forms of amyloidosis.

“The ClariTY platform offers the unique opportunity to generate therapeutic candidates able to target all amyloid fibril deposits, a key cause of amyloidosis,” Pierre Vandepapelière, MD, PhD, Amyl’s CEO, chief medical officer, and cofounder, said in a press release. “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.”

Amyloidosis comprises a group of diseases characterized by the production of misfolded proteins that form abnormal clumps, called amyloid fibrils, that gradually accumulate and damage different body tissues. In FAP, amyloid fibrils made up of a protein called transthyretin tend to accumulate mainly in peripheral nerves — those found outside the brain and spinal cord — leading to the onset of a condition called peripheral neuropathy.

Blocking the abnormal aggregation of proteins into amyloid fibrils is being explored as a potential therapeutic strategy in many forms of amyloidosis, but most therapies in development are designed to target one particular protein that forms amyloid fibrils in a single disease.

With its ClariTY platform, Amyl is hoping to design new therapies that can target different types of amyloid fibrils by focusing on a structural feature, called the amyloid fold, that is found in many proteins that form these fibrils.

These treatment candidates are designed to prevent the accumulation and spread of amyloid fibrils, while also helping the body eliminate existing protein aggregates from different tissues, according to the company. 

“The ClariTY platform provides a unique and powerful tool for treating amyloidosis, for which treatment options are very limited today,” Karine Goraj, PhD, Amyl’s chief science officer, said. “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.”

This round of funding was led by an international group of investors, including the Belgian private-public fund Noshaq.

“Our equity investment in Amyl represents a significant opportunity to participate in the development of what we believe is an exciting technology for the treatment of Amyloidosis,” said Gaetan Servais, CEO of Noshaq.

Vandepapelière added: “We are excited to have raised this Series A financing from a syndicate of high-quality and international investors.”