Amlenetug receives FDA Fast Track Designation

Potential treatment for multiple system atrophy shows promise

Lundbeck’s investigational drug, amlenetug, has been granted Fast Track Designation by the US Food and Drug Administration (FDA) as a potential treatment for multiple system atrophy (MSA).

This decision follows promising results from the AMULET phase 2 trial presented in March 2024.

Amlenetug, a human monoclonal antibody, targets all major forms of extracellular α-synuclein, aiming to prevent its uptake and aggregation.

Lundbeck has also begun the MASCOT phase 3 trial to further assess the efficacy and safety of amlenetug for treating MSA.

Dr Johan Luthman, EVP and Head of Research & Development at Lundbeck, expressed optimism about the FDA’s Fast Track Designation for amlenetug. He stated that it reflects Lundbeck’s commitment to addressing unmet needs in treating this devastating disease.

Amlenetug received Orphan Drug Designation (ODD) from the US FDA in April 2024, the EMA in May 2021 and SAKIGAKE designation from Japan’s Ministry of Health Labor and Welfare in March 2023. The FDA grants ODD to therapies for rare diseases affecting fewer than 200,000 people in the US.

The MASCOT trial, an interventional phase 3 study, involves randomising participants to receive high or low doses of amlenetug or placebo for 72 weeks, followed by an open-label extension period.

The goal is to evaluate amlenetug’s efficacy, safety and tolerability in MSA patients.

The AMULET phase 2 trial involved 61 MSA patients who received amlenetug or placebo over 48 to 72 weeks, followed by a 96-week open-label extension.

The primary objective was to show a slowing in clinical progression in the active treatment arm compared to placebo.

Secondary objectives included assessing amlenetug’s impact on patient functioning, disease severity and other MSA aspects. Amlenetug is administered intravenously every four weeks.