An experimental product, AMX0035 has sodium phenylbutyrate (PB) and taurursodiol (TURSO; ursodoxicoltaurine) active agents.
These complementary agents were merged in a co-formulation to reduce the death of neurons and their dysfunction.
AMX0035 acts on the endoplasmic reticulum and pathways of mitochondrial-dependent neuronal degeneration in ALS, as well as various neurodegenerative ailments.
Named PHOENIX or A35-004, the randomised, international, placebo-controlled Phase III trial will assess the safety and efficacy of AMX0035 in ALS patients.
Combined analysis of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score progression over a period of 48 weeks and survival will be the trial’s primary efficacy goal.
Variation in slow vital capacity (SVC), ventilation-free survival rates, quality of life subject-reported outcome analysis and various other measures will comprise secondary efficacy outcomes.
The trial will be carried out at around 65 centres in Europe and the US in alliance with the Treatment Research Initiative to Cure ALS (TRICALS) and Northeast ALS Consortium (NEALS), respectively.
It will enrol nearly 600 subjects within two years of symptom development.
Amylyx Global Clinical Operations head Erin Whitney said: “We’re grateful for the opportunity to work with TRICALS and NEALS, two of the largest ALS research organisations in the world, on a trial of this scale.
“We are excited by the potential of these data to add to our growing body of evidence for AMX0035 in ALS and for this study to provide an opportunity for access to AMX0035 to those living with ALS in the United States and Europe.”
The company has recently filed a new drug application with the US Food and Drug Administration for AMX0035 and intends to obtain marketing authorisation from the European Medicines Agency by this year-end.
In December 2019, Amylyx announced that AMX0035 slowed disease progression in the Phase II CENTAUR trial for treating ALS.