Amylyx Pharmaceuticals, Inc. Receives U.S. FDA Fast Track Designation for AMX0114 for the Treatment of Amyotrophic Lateral Sclerosis

Published: 2025-06-03 08:35:43 ET AMLX

Published on 06/03/2025 at 08:35

Amylyx Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide (ASO) targeting cal pain-2 for the treatment of people living with Amyotrophic lateral sclerosis (ALS). The FDA's Fast Track designation is designed to facilitate the development and expedited review of therapies that are intended to treat serious and life-threatening conditions and demonstrate the potential to address an unmet medical need. A therapy that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA, as well as Priority Review if relevant criteria continue to be met.

Amylyx designed AMX0114 to target cal pain-2, a calcium-activated protease. Peer-reviewed research has demonstrated that overactive cal pain-2 activity may be an important driver of disease progression in ALS and other neurodegenerative diseases by executing the degeneration of axons, the long tubular neuronal segments which carry signals from neurons to the muscle or other neurons. In preclinical studies, AMX0114 showed improved neuronal survival and reductions in extracellular neurofilament light (NfL) levels across multiple disease models.

In April 2025, the Company announced the first participant was dosed in LUMINA, a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose Phase 1 clinical trial designed to evaluate the safety and biological activity of AMX0114 in people living with ALS. LUMINA will also assess broadly researched ALS biomarkers, including changes from baseline in NfL levels. Approximately 48 participants will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks, for up to four doses.

Amylyx expects early cohort data from LUMINA in 2025. AMX0114 is an investigational antisense olig onucleotide (ASO) targeted cal pain-2 (CAPN2) for the potential treatment of ALS. The U.S. Food and drug Administration (FDA) has grant AMX0114 Fast Track designation for the treatment of ALS.

In preclinical studies, treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in CAPN2 mRNA and cal pain-2 protein levels in disease-relevant cell models of axonal degeneration. This translated to improved neuronal survival, including in a model of TDP-43 ALS, and reductions in extracellular Neurofilament light (Nf LUMINA) levels across multiple disease models and paradigms of neuronal injury. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death.

More than 90% of people with ALS have sporadic disease, showing no clear family history. The Phase 1 LUMINA clinical trial (NCT066) is the Phase 1 LUMINA clinical study (NCT066) in the Phase 1 LUMINA trial (NCT066). The Phase 1 LUMINA trial of AMX0114 is the Phase 1 LUMina clinical trial (NCT067) in the Phase 1 LumINA clinical trial (NfL) in the Phase 1 LUINA clinical trial (NCT 066) in the Phase 1LUMINA clinical trial (Nf LUMINA).

The Phase 1 LUMina clinical study (NCT067) is the first patient in ALS. The Phase 1 LUMina trial of AMX0114 in the Phase 1 LUMina trial (NfL) is the Phase 1 LumINA trial (NfL) trial (NfL) and the Phase 1 LUMINA study (NfL) trial of AMX0114) in people living with ALS.