H. Lundbeck A/S announced that the last patient has been randomized in MASCOT (NCT06706622), a global Phase 3 clinical trial evaluating amlenetug in people with multiple system atrophy (MSA), a rapidly progressing and fatal neurodegenerative disease for which no approved treatments currently exist. Randomization was completed earlier than anticipated, underscoring the broad engagement across the global MSA community and Lundbeck?s commitment to bringing innovation to patients with high unmet medical need. The multicenter MASCOT trial is currently ongoing across North America, Europe, Asia, and Australia to evaluate the potential of amlenetug to slow the clinical disease progression of MSA.
Amlenetug is designed to target the a-synuclein protein in the brain and inhibit its spread to nearby brain cells. By addressing a key underlying cause of MSA, amlenetug holds the potential to become a first-in-class therapy for this rare disorder. Amlenetug has received Orphan Drug Designation in the EU, Japan, and the U.S., as well as Fast Track designation in the U.S. and SAKIGAKE designation in Japan.
With randomization now complete, the MASCOT trial will continue as planned, with participants progressing through the double-blind treatment period followed by the optional open-label extension. Amlenetug is an investigational compound that is not approved for marketing by any regulatory authority worldwide, and the efficacy and safety of amlenetug have not been established. MSA is a rapidly progressing rare condition that causes damage to nerve cells in the brain.
In a person with MSA, an abnormal build-up of the protein a-synuclein is thought to be responsible for damaging the areas of the brain that control balance, movement, and the body's normal functions. MSA is seriously debilitating and places a high disease burden on patients. There is currently no cure for MSA and no available treatment to slow its clinical progression.
Symptoms of MSA usually start between 55 and 60 years of age, and the typical time to death is 8.6 years after symptom onset. Although there are many different possible symptoms of MSA, not everyone who is affected will experience all of them. The symptoms of MSA are wide-ranging and include muscle control problems, similar to those of Parkinson's disease.
Many different functions of the body can be affected, and symptoms including urinary incontinence, frequent falling, and unintelligible speech occur within 3 years of disease onset. MSA is accompanied by reduced capacity to live independently, and death is often due to respiratory problems. Amlenetug is a human monoclonal antibody (mAb) that recognizes and binds to all major forms of extracellular a-synuclein and thereby intended to prevent uptake and inhibit seeding of aggregation. Amlenetug is being developed by Lundbeck under a joint research and licensing agreement between Lundbeck and Genmab A/S. MASCOT (NCT06706622) is a Phase 3 interventional, randomized, double-blind, parallel-group, placebo-controlled, optional open-label extension trial that will be conducted in North America, Europe, Asia and Australia.
The trial comprises 2 parts: A double-blind period where participants are randomized to receive either high or low doses of amlenetug, or placebo for 72 weeks, followed by an open-label extension period where all participants enrolled in the trial are offered treatment with amlenetug. The aim of the trial is to evaluate the efficacy, safety, and tolerability of amlenetug in patients with MSA. Amlenetug is delivered as an intravenous infusion every four weeks.
