The last patient in Lundbeck’s phase 3 clinical trial, MASCOT, has been randomised ahead of schedule in a study evaluating amlenetug as a potential treatment for multiple system atrophy (MSA).
The phase 3 trial will continue with a double-blind period where participants are randomised to receive either high or low doses of amlenetug, or placebo, for 72 weeks. This is followed by an optional open-label extension period where all participants will be offered treatment with amlenetug.
The multicentre phase 3 trial, which is currently ongoing across North America, Europe, Asia and Australia, aims to demonstrate that amlenetug has the potential to slow the clinical disease progression of MSA.
By addressing a key underlying cause of MSA by targeting the α-synuclein protein in the brain to inhibit its spread to nearby brain cells, amlenetug could become a first-in-class therapy for this rare disorder.
Professor Günter Höglinger, Lead Investigator of the MASCOT trial, said: “Completing randomisation of a global phase 3 trial in MSA is a significant achievement for the field.
“Advancing the development of a potential treatment for this underserved disease addresses a critical unmet need for people living with MSA.”
MSA causes debilitating damage to nerve cells in the brain. The symptoms are wide-ranging and include muscle control problems, with an average survival time after symptom onset of just over eight and a half years.
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.
“Successfully randomising 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 been given Orphan Drug Designation in the EU, Japan and the US, Fast Track designation in the US and SAKIGAKE designation in Japan.