The FDA has given Amylyx the green light to launch its Phase I LUMINA study of AMX0114. Image credit: Vitawin / Shutterstock

The US Food and Drug Administration (FDA) has lifted a clinical hold on Amylyx Pharmaceuticals’ Phase I trial of AMX0114 in amyotrophic lateral sclerosis (ALS).

The lift allows Amylyx to open US sites for screening, enrolment, and dosing in the Phase I LUMINA study (NCT06665165). The multicentre, multiple ascending dose trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in 48 ALS patients. Patients will be randomised on a 3:1 ratio to receive AMX0114 or placebo by intrathecal administration once every four weeks for up to four doses.

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LUMINA will also assess change from baseline in calpain-2 levels, neurofilament light (NfL) levels, and other pharmacodynamic biomarkers of ALS.

The FDA hold came after Amylyx asked the agency to approve the first-in-human studies for AMX0114—an investigational antisense oligonucleotide (ASO) targeting calpain-2. According to a November 2024 press release, as part of the review, the FDA limited the drug to a 12.5mg starting dose but after requesting more information, the FDA put a clinical hold on the therapy in the US.

The announcement comes as Amylyx expects to initiate the Phase I LUMINA clinical trial in Canada at the beginning of 2025. The company anticipates early cohort data from LUMINA in 2025.

Chief medical officer of Amylyx, Dr. Camille Bedrosian said: “Calpain-2 is a well-established target in several neurological diseases and a protease known to cleave many substrates including neurofilament, tau, and TDP43 proteins. In addition to being considered an essential protein in the process of axonal degeneration, calpain-2 has been repeatedly linked to neurofilament biology in published studies. We are dedicated to investigating therapies to potentially treat people living with ALS, and we are excited to work with study investigators and clinical trial sites across North America to enrol patients in LUMINA.”

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The trial comes after Amylyx’s previous efforts in the disease led to a market withdrawal of Relyvrio (sodium phenylbutyrate and taurursodiol) after the therapy failed to beat placebo at improving patients’ physical function in a Phase III trial. The therapy was approved based on data from the Phase II CENTAUR study which included an open-label extension (OLE) long-term follow-up. The Phase III failure and drugs market departure saw a 70% cull of Amylyx’s workforce and an 85% drop in the company’s stock price. The drug generated $381m in sales in 2023, according to the company’s financials.