Mataró, Barcelona Spain – Minoryx Therapeutics, a company specializing in the development of new drugs for orphan diseases, today announces the dosing of its first US patient as part of its ongoing phase 2/3 clinical trial of MIN-102 for the treatment of adrenomyeloneuropathy (AMN). The trial enrolls adult male patients affected by AMN, the most frequent phenotype of X-linked adrenoleukodystrophy (X-ALD).

The first US patient was dosed at the Massachusetts General Hospital (MassGeneral) under the supervision of Dr. Florian Eichler. Two additional US sites will be initiating recruitment shortly: the Kennedy Krieger Institute in Baltimore, Maryland (a John Hopkins Medical Institution), with Dr. S. Ali Fatemi, and the School of Medicine of the Stanford University in California, with Dr. J.Sampson.

The first European patients were dosed at the end of 2017 and in early 2018, at the University Hospital Vall d’Hebron (Barcelona, Spain) and at the Academic Medical Center (Amsterdam, The Netherlands). Recruitment is progressing rapidly in Europe: further participating centers include the Institute of Genomic Medicine and Rare Disorders (Budapest, Hungary), Hôpital de la Pitié-Salpétrière (Paris, France), Universität Leipzig, Klinik und Poliklinik für Neurologie (Leipzig, Germany), Istituto Neurologico Carlo Besta (Milan, Italy) and the National Hospital for Neurology and Neurosurgery (London, United Kingdom).

The ADVANCE trial is a randomized, double-blinded, placebo-controlled, potentially pivotal study with an open-label extension to determine the efficacy and safety of MIN-102, a novel, orally bioavailable and selective PPAR gamma agonist with a superior profile for central nervous system-related diseases. The primary outcome is to evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients, as determined by a motor function test. The trial aims to enroll more than 100 patients; results are expected at the end of 2020.

“We are proud to be the first hospital in the United States to be involved in this important clinical trial for X-ALD patients,” said Dr. Eichler from MassGeneral. “We are looking forward to enrolling further patients and eagerly await results.”

“We have achieved an important milestone in our trial with the first patient in the US dosed with MIN-102,” said Dr. Uwe Meya, chief medical officer of Minoryx. “We are delighted that patient enrollment is already at over 50 per cent, a significant result for a clinical study in a rare disease such as X-ALD.”