Lundbeck has announced that the final patient has been randomised ahead of schedule in its global phase 3 Mascot trial, which is evaluating amlenetug as a potential treatment for multiple system atrophy, a rare and fatal neurodegenerative disease.
The company said the early completion reflects strong engagement from the international MSA community and highlights the urgent need for new therapeutic options. The multicentre study is running across North America, Europe, Asia and Australia and is designed to assess whether amlenetug can slow clinical disease progression.
Professor Günter Höglinger, Lead Investigator of the Mascot trial, said: “Completing randomization of a global Phase 3 trial in MSA is a significant achievement for the field.” He added: “Advancing the development of a potential treatment for this underserved disease addresses a critical unmet need for people living with MSA.”
Amlenetug is a monoclonal antibody targeting the α-synuclein protein in the brain, with the aim of inhibiting its spread between cells. Lundbeck believes that by addressing a key underlying cause of MSA, the therapy could become a first-in-class option. The compound previously showed encouraging results in a phase 2 study.
Johan Luthman, Executive Vice President and Head of Research and Development at Lundbeck, said: “With no currently available treatments to slow the clinical progression of MSA, the urgency for innovation is exceptionally high.” He continued: “Successfully randomizing the last patient into the trial sets us on a strong trajectory to hopefully bring a much-needed treatment to people living with this devastating disease.”
Amlenetug has received orphan drug designation in the EU, Japan and the US, as well as fast track and SAKIGAKE designations. With randomisation complete, participants will continue through the double-blind treatment period followed by an optional open-label extension.
