Advertisment
Lundbeck to present pipeline developments for amlenetug in multiple system atrophy at MDS Congress 2025
H. Lundbeck A/S (Lundbeck) today announced that pipeline developments regarding amlenetug, a novel investigational molecule for the potential treatment of Multiple System Atrophy (MSA), will be presented at the 2025 International Congress of Parkinson’s Disease and Movement Disorders® in Honolulu, Hawaii (Oct 5-9).
Amlenetug is a human monoclonal antibody (mAb), which binds to pathological aggregations of the protein α-synuclein in the brain, thereby inhibiting its spreading to nearby brain cells. Amlenetug holds promise as a first-in-class therapy, with the potential to slow clinical disease progression for patients with MSA, a condition for which no approved treatments currently exist.
At the congress, Lundbeck will share Insights into the design of the Phase 3 MASCOT trial and its use of innovative Bayesian progression modeling methods.1,2 Rare diseases, such as MSA, face unique challenges due to limited prior data available to guide the design of drug development programs and smaller patient populations to enroll in trials.6,7
“MSA is a neurodegenerative disease with no currently available treatments to slow its relentless progression,” said Johan Luthman, EVP and Head of Research and Development at Lundbeck. “Amlenetug seeks to address this unmet need by targeting the underlying biology of MSA and delaying disease progression. The MASCOT trial has therefore been designed based on innovative methodologies, including Bayesian progression modeling and adaptive elements, to effectively evaluate its impact on this debilitating neurological disorder.”
This dynamic approach utilizes all available data over a long treatment period, enabling assessment of potential slowing of clinical disease progression overall. This is in contrast to traditional clinical trial design, which focuses on isolated time points.
The MASCOT trial is ongoing in many regions, including the EU, US and Japan, where the Bayesian progression modelling framework has been discussed with regulatory authorities in support of marketing registration. This further underscores the innovative nature of Lundbeck’s approach and the critical unmet need in this disease area.
During the congress, Lundbeck will also showcase valuable insights from patients and caregivers who participated in the Phase 2 AMULET trial. These perspectives, captured through embedded patient experience interviews, have been instrumental in informing the design of the Phase 3 MASCOT trial and in shedding light on the substantial burden the disease places on patients and their families.3
Lundbeck’s scientific presentations at the 2025 International Congress of Parkinson’s Disease and Movement Disorders®:
| Exploring MSA: Bridging clinical insight, experiences of people living with MSA, and the path toward targeted treatment | Scientific Symposium | Wednesday, Oct 811:45-12:45 HST |
| A randomized, double-blind phase 3 trial of amlenetug versus placebo in patients with MSA: The MASCOT study1 | ePosterLotte Kjærsgaard | Sunday, Oct 5 12:48 HST |
| Assessing disease progression in MSA: Development of a Bayesian Progression Model (BPM)2 | ePosterJonas Wiedemann | Sunday, Oct 5 13:21 HST |
| Incorporating patient and care partner feedback on the protocol for a clinical trial assessing progression in MSA3 | ePosterBeatrice Yang | Sunday, Oct 5 12:57 HST |
About Multiple System Atrophy
MSA is a rapidly progressing rare condition that causes damage to nerve cells in the brain. MSA is seriously debilitating and places a high disease burden on patients. Symptoms of MSA usually start between 55 and 60 years of age, and patients typically live for 6 to 9 years after symptom onset.9
In a person with MSA, an abnormal build-up of the protein α-synuclein is thought to be responsible for damaging the areas of the brain that control balance, movement, and the body’s normal functions.10 The symptoms of MSA are wide-ranging and include muscle control problems, similar to those of Parkinson’s disease.9 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 and are accompanied by reduced capacity to live independently. Death is often due to respiratory problems. Although there are many different possible symptoms of MSA, not everyone who is affected will experience all of them. There is currently no cure for MSA and no available treatment to slow its progression.9
