Minoryx Therapeutics, a company specializing in the development of new drugs for orphan diseases, announces the start of its pivotal phase II/III clinical trial of MIN-102 for the treatment of adrenomyeloneuropathy (AMN), the most frequent phenotype of X-linked adrenoleukodystrophy (X-ALD).

The first patients were dosed at the University Hospital Vall d'Hebron (Barcelona) by Dr. Josep Gámez and at the Academic Medical Center (Amsterdam) by Dr Marc Engelen. In addition, recruitment was also initiated at the Institute of Genomic Medicine and Rare Disorders (Budapest) by Dr. Maria Molnar. More patients will be enrolled in several other European countries (United Kingdom, Germany, France, Italy and Poland) in the coming weeks and in the US by mid-2018.

The ADVANCE trial is a randomized, double-blind, placebo-controlled, potentially pivotal study with an open-label extension to determine the efficacy and safety of MIN-102. The trial aims to enroll more than 100 patients. The results of this potential registration enabling trial are expected at the end of 2020.

More information is available on Minoryx Therapeutics website.

Photo: Marc Martinell, Co-Founder and CEO of Minoryx - Minoryx Therapeutics