Amylyx Pharmaceuticals has begun dosing patients in the Phase II CENTAUR clinical trial of its therapeutic candidate, AMX0035, to treat those with amyotrophic lateral sclerosis (ALS).
AMX0035 is an oral formulation comprising a synergistic combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA) to minimise neuronal death and inflammation.
The randomised, double-blind, multi-centre, placebo-controlled Phase II trial will assess the safety and efficacy of the therapeutic candidate in 132 subjects over 24 weeks.
CENTAUR is currently being performed in partnership with the ALS Association, ALS Finding a Cure and the Massachusetts General Hospital (MGH), and will be supervised by MGH’s Neurological Clinical Research Institute (NCRI) and the North-east ALS Consortium (NEALS).
The trial is set to be performed at 25 NEALS member medical centres in the US, and is designed to allow trial access, easy visits to trial sites and patient engagement, as well as to measure functional and biochemical changes.
Massachusetts General Hospital physician and trial principal investigator Sabrina Paganoni said: “The CENTAUR trial builds on promising results with AMX0035 in preclinical studies and clinical experience with the drug’s two components.
“The team at NCRI worked closely with Amylyx to design an innovative trial that will evaluate AMX0035’s safety and efficacy, advance our understanding of several biomarkers, and provide insights into ALS disease biology.”
The CENTAUR trial will employ the Accurate Test of Limb Isometric Strength (ATLIS) technology designed to quantitatively monitor changes in muscle strength of the patients.
Changes in disease pathology will also be evaluated during the trial using PET imaging of neuroinflammation and blood-based markers of neurodegeneration.
The firm also intends to allow CENTAUR subjects to participate in an open-label extension study after completion of the trial’s treatment period.