Amyl Tx raises €8.25M to develop universal neurodegeneration drug

Why it matters: Amid a resurgence in venture capital funding for European biotechs, Amyl Tx has raised money to complete key tasks on its path to clinical development. The biotech will use the cash to finalize development of its clinical candidate ready for GMP manufacturing and collect preclinical in vivo efficacy and safety data to validate its platform.

Big picture: Biogen and Eisai’s Leqembi and Eli Lilly’s Kisunla have won approvals in Alzheimer’s disease based on evidence that they improve outcomes by removing amyloid-beta from the brain. Yet the drugs have limited efficacy and research shows proteins other than amyloid-beta are implicated in the disease, fueling work on molecules against tau and alpha-synuclein and explorations of combination therapies. 

• Amyl Tx has predicted that efforts to combine Leqembi and Kisunla with antibodies against tau and alpha-synuclein will face challenges. One drug may interfere with expression of the protein targeted by the other antibody, and combinations could cause safety and immunogenicity issues. 

What they’re saying: In a statement, Pierre Vandepapelière, co-founder, CEO and CMO of Amyl Tx, said: “Our results already demonstrate that our fusion protein combines the therapeutic strengths of three monoclonal antibodies while addressing many key limitations in brain delivery and safety.”

How it works: Amyl Tx has found a structure, specifically a cross-beta sheet, that is shared by amyloid-beta, tau and alpha-synuclein fibrils. Starting with a fusion protein that engages the structure, the biotech has modified the molecule to cross the blood-brain barrier and persist longer in the body.

• In preclinical tests, the drug candidate was as effective as Leqembi at triggering the clearance of amyloid-beta fibrils and inhibited the aggregation of tau monomers. It also showed reduced propensity for amyloid-related imaging abnormalities, adverse events associated with existing Alzheimer’s drugs.
• The involvement of amyloid aggregates in more than 30 disorders, including Parkinson’s disease, supports Amyl Tx’s vision for applying its platform to a range of neurodegenerative diseases.  

Yes, but: Amyl Tx has yet to test its lead candidate in humans. Clinical trials may fail to replicate positive signs that Amyl Tx has seen in mice and in vitro tests, causing the biotech’s program to join the long list of unsuccessful Alzheimer’s projects. 

What’s next: Amyl Tx is extending its laboratory facilities in Liège, Belgium. With its research activities expanding, the eight-person company plans to double the size of its R&D team.