Amylyx Pharmaceuticals, Inc. ((AMLX)) announced an update on their ongoing clinical study.
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Amylyx Pharmaceuticals, Inc. is conducting a Phase 1 clinical trial titled Phase 1, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Evaluate Safety, Tolerability, PK and PD of Antisense Oligonucleotide AMX0114 Administered to Adult Participants With Amyotrophic Lateral Sclerosis. The study aims to assess the safety and tolerability of AMX0114, an antisense oligonucleotide, in adults with amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease.
The intervention being tested is AMX0114, administered via intrathecal injection. This drug is designed to reduce levels of calpain-2, a protein linked to neuronal degeneration, with the goal of slowing neuron injury and death in ALS patients.
The study follows a randomized, sequential intervention model with quadruple masking, meaning that participants, care providers, investigators, and outcomes assessors are blinded. The primary purpose is treatment-focused, aiming to evaluate the drug’s safety profile.
The study began on April 7, 2025, with its primary completion and estimated completion dates yet to be announced. The most recent update was submitted on August 4, 2025. These dates are crucial for tracking the study’s progress and potential data release.
The clinical trial’s progress could influence Amylyx’s stock performance and investor sentiment, as successful outcomes may enhance the company’s market position in the ALS treatment landscape. Competitors in the neurological treatment space will also be closely monitoring these developments.
The study is currently ongoing, with further details available on the ClinicalTrials portal.