FDA Grants Fast Track Designation to Amlenetug As Treatment for Multiple System Atrophy
The designation is based on the AMULET phase 2 trial, which was the first-in-human study of the drug.
The FDA granted fast track designation to amlenetug as a potential new treatment for multiple system atrophy (MSA). Recently, the MASCOT phase 3 (NCT06706622) trial was initiated to assess efficacy and safety of the drug for MSA.1
The designation is based on the AMULET (NCT03611569) phase 2 trial, which was the first-in-human study of the drug. | Image Credit: monticellllo | stock.adobe.com
"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," Johan Luthman, executive vice president and head of research & development at Lundbeck, said in a news release.1
The designation is based on the AMULET (NCT03611569) phase 2 trial, which was the first-in-human study of the drug. In the study, investigators aimed to determine the safety, tolerability, pharmacokinetics, and target engagement of various doses of the investigational drug. The study included healthy patients who were non-Japanese and Japanese and patients with Parkinson disease.1,2
Patients were aged 18 to 55 years with a body mass index of 18 to 32 km/m2 (for non-Japanese patients) and 18 to 26 km/m2 (Japanese patients.) They also had a clinic diagnosis of Parkinson disease and a minimum of 3 months of stable symptoms prior to the study. Further, patients who were on treatment needed to be on a stable dose for a minimum of 3 months.2
The primary outcome included number of individuals with treatment-emergent adverse events (TEAEs), with secondary outcomes of area under the concentration curve from 0 to day 84, maximum observed plasma concentration of the drug, and clearance of the drug.2
A total of 59 healthy individuals were included in part A of the study. Fifteen patients with Parkinson disease were assigned to part B. A single dose of amlenetug was safe and well tolerated in both parts of the study. In part A, there were 31 patients who reported 64 TEAEs, and 10 patients in part B reported 33. They were mostly mild and considered unrelated to the treatment, with the most common being lumbar punctures/puncture site pain, headaches, and common infections.3
There were no trends of increasing TEAEs for either cohort. In part A, TEAEs considered to be possibly or probably related to treatment included headache, hypoesthesia, dizziness, hyperhidrosis, and migraine. In part B, TEAEs that were possible or probably included headache, nausea, orthostatic hypotension, balance disorder, and vomiting.3
The MASCOT trial is a randomized, double-blind, phase 3 trial that will build on the results from the AMULET trial. The objective includes showing a consistent trend for amlenetug in slowing clinical progression of MSA, despite not meeting the primary end point.4
In April 2024, the FDA also granted amlenetug orphan drug designation for the potential treatment of MSA.1
