Amylyx: FDA Lifts Hold on ALS Trial

 Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Phase 1 clinical trial of AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for people living with amyotrophic lateral sclerosis (ALS). With the clinical hold lifted, Amylyx is now working to open U.S. sites for screening, enrollment, and dosing.

The announcement comes as Amylyx expects to initiate the Phase 1 LUMINA clinical trial in Canada in the beginning of 2025. LUMINA is a multicenter, randomized, placebo-controlled, multiple ascending dose trial evaluating the safety and biological activity of AMX0114.

LUMINA will also assess ALS biomarkers, including change from baseline in neurofilament light (NfL) levels. Approximately 48 people living with ALS will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks for a total of up to 4 doses.

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