H. Lundbeck A/S (Lundbeck) today announced that amlenetug has received Fast Track designation from the United States (US) Food and Drug Administration (FDA).

This designation is based on the outcome of AMULET, the phase II trial, which was presented in March 2024 at the International Conference on Alzheimer's and Parkinson's Diseases and related neurological disorders (AD/PD) in Lisbon, Portugal.

With this designation, Lundbeck will benefit from all FDA Fast Track Designation features, including rolling potential reviews, and intensive FDA guidance on an efficient drug development program.

"We are pleased that amlenetug has received Fast Track Designation for the potential treatment of Multiple System Atrophy. This is a step forward in our commitment to address significant unmet needs in this devastating disease," said Johan Luthman, EVP and Head of Research & Development at Lundbeck.

Amlenetug has also been granted Orphan Drug Designation (ODD) for the potential treatment of MSA by US FDA in April 2024 and by EMA in May 2021 and the SAKIGAKE designation from the Ministry of Health, Labor and Welfare (MHLW) in Japan in March 2023. The FDA grants ODD to investigational therapies intended for treatment, prevention, or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation.

About amlenetug
Amlenetug is a human monoclonal antibody (mAb) that recognizes and binds to all major forms of extracellular α-synuclein and thereby intended to prevent uptake and inhibit seeding of aggregation. Amlenetug has an active Fc region, which may increase immune-mediated clearance of α- synuclein /mAb complexes through microglia mediated uptake.

Amlenetug is being developed by Lundbeck under a joint research and licensing agreement between Lundbeck and Genmab A/S.

About the MASCOT trial
MASCOT (NCT06706622) is a phase III, interventional, randomized, double-blind, parallel-group, placebo-controlled, optional open-label extension trial that will be conducted in North America, Europe, and Asia.

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 will be delivered as an intravenous infusion every four weeks.

About the AMULET trial
AMULET trial (NCT05104476) was a phase II, randomized, double-blind, placebo-controlled clinical trial of amlenetug as a potential treatment for patients with MSA. A total of N=61 MSA patients were randomized 2:1 to either amlenetug or placebo and treated between 48 to 72 weeks, followed by an ongoing 96 weeks open-label extension period offering all participants to receive treatment with amlenetug.

The primary objective was to evaluate the efficacy of amlenetug on clinical progression in patients with MSA, aiming at showing a slowing in clinical progression in the active treatment arm compared to placebo on a 5% significance level evaluated 1-sided as well as safety and tolerability. The secondary objectives included evaluation of amlenetug on patient's functioning, disease severity and other aspects of MSA.

Amlenetug was delivered as an intravenous infusion every four weeks.

About MSA
MSA is a rapidly progressing rare condition of the nervous system 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 the patients typically live for 6 to 9 years after symptom onset.

In a person with MSA, an abnormal build-up of the protein alpha-synuclein is thought to be responsible for damaging areas of the brain that control balance, movement, and the body's normal functions. 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 may 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.

References

1 - Fast Track | FDA

2 - NHS : Multiple system atrophy - NHS (www.nhs.uk)

Contacts

About H. Lundbeck A/S
Lundbeck is a biopharmaceutical company focusing exclusively on brain health. With more than 70 years of experience in neuroscience, we are committed to improving the lives of people with neurological and psychiatric diseases.

Brain disorders affect a large part of the world's population, and the effects are felt throughout society. With the rapidly improving understanding of the biology of the brain, we hold ourselves accountable for advancing brain health by curiously exploring new opportunities for treatments.

As a focused innovator, we strive for our research and development programs to tackle some of the most complex neurological challenges. We develop transformative medicines targeting people for whom there are few or no treatments available, expanding into neuro-specialty and neuro-rare from our strong legacy within psychiatry and neurology.

We are committed to fighting stigma and we act to improve health equity. We strive to create long term value for our shareholders by making a positive contribution to patients, their families and society as a whole.

Lundbeck has approximately 5,500 employees in more than 50 countries and our products are available in more than 80 countries. For additional information, we encourage you to visit our corporate site www.lundbeck.com and connect with us via LinkedIn.