Therapy Areas: Devices
First Patients in Phase II Clinical Trial of AMX0035 for Treatment of ALS Elect to Participate in Open Label Extension
9 April 2018 - - Cambridge, Massachusetts-based neurodegenerative disease treatment developer Amylyx Pharmaceuticals, in collaboration with The ALS Association, ALS Finding a Cure, the Northeast ALS Consortium, and the Massachusetts General Hospital Neurological Clinical Research Institute, have announced that the first patients to complete the CENTAUR phase II clinical trial have elected to continue treatment in an open label extension study of AMX0035, the company said.
All patients who complete the main phase of the study will be eligible for this extension and will receive AMX0035 with no placebo comparator.
The CENTAUR (Combination of Phenylbutyrate and Tauroursodeoxycholic Acid) trial is a randomized, double-blind, placebo-controlled Phase II clinical trial enrolling 132 ALS patients nationwide. The trial's primary objectives are to evaluate the safety and tolerability of AMX0035 and to assess the drug's impact on disease progression over the 24-week study period.
The CENTAUR trial is the first known drug trial to measure patient muscle strength using an innovative device called ATLIS (Accurate Test of Limb Isometric Strength), and will include assessments of blood and imaging based biological markers of ALS to confirm target engagement.
AMX0035 is an oral combination of two small molecules, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Each compound has shown efficacy in several cellular and animal models of ALS, and when individually tested have both met endpoints of safety, tolerability, and demonstrated preliminary signs of efficacy.
In preclinical trials, a synergistic effect between the two compounds has been demonstrated, suggesting that the combination may have improved efficacy compared to the individual agents.
Amylyx Pharmaceuticals is a clinical-stage pharmaceutical company currently developing a novel therapeutic for Amyotrophic Lateral Sclerosis and Alzheimer's disease. The company's therapeutic, AMX0035, targets the neuroinflammation and nerve cell death that characterize these diseases.


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