ClivalLundbeck’s Amlenetug Gains Japanese Orphan Drug Status for MSA Treatment

H. Lundbeck A/S (Lundbeck), a global pharmaceutical company, announced that the Ministry of Health, Labor, and Welfare (MHLW) in Japan has granted Orphan Drug Designation (ODD) to its investigational drug, amlenetug, a potential new treatment option targeting multiple system atrophy (MSA).

The ODD in Japan adds to other important designations: the SAKIGAKE designation by Japan’s MHLW in March 2023, the Orphan Drug Designation (ODD) by the US FDA in April 2024, and by EMA in May 2021.

“We are pleased to receive Orphan Drug Designation for amlenetug in Japan. This designation highlights the potential of amlenetug as a treatment option for people living with multiple system atrophy. We are hopeful of the potential for amlenetug to slow the clinical progression of this devastating disease and look forward to advancing its development through the MASCOT trial”, said Johan Luthman, EVP and head of research & development at Lundbeck.

Lundbeck has recently initiated, MASCOT, a phase III trial to assess efficacy and safety of amlenetug in the treatment of MSA.

The MASCOT trial will open for enrollment in North America, Europe, Asia and Australia.
 
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 has an active Fc region, which may increase immune-mediated clearance of a- 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.

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.

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 people living 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 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.

H. Lundbeck A/S is a global pharmaceutical company specialized in neurological and psychiatric disorders. For more than 70 years, the company have been at the forefront of neuroscience research. The company are dedicated to advancing brain health and transforming lives

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