Date: 2018-09-05
Type of
information: update on patient enrollment
phase: 2-3
Announcement: update on patient enrollment
Company: Minoryx Therapeutics (Spain)
Product: MIN-102 ( hydroxypioglitazone -5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride)
Action
mechanism:
- PPAR agonist. MIN-102 is a novel, orally bioavailable and selective PPAR gamma agonist. This metabolite from pioglitazone showed a superior brain penetration and safety profile, allowing PPAR gamma engagement above the level that can be safely achieved with pioglitazone and other glitazones. It showed robust preclinical proof of concept in several animal models. In X-ALD, mutations on ABCD1 trigger a cascade of events leading to mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination and axonal degeneration. MIN-102, through its PPAR gamma activity, prevents such dysfunctions, thus it has the potential to treat both adrenomyeloneuropathy (AMN) and cerebral ALD (cALD).
Disease: patients with the AMN (adrenomyeloneuropathy) phenotype of X-linked adrenoleukodystrophy (X-ALD)
Therapeutic
area: Rare diseases - Genetic diseases
Country: France, Germany, Hungary, The Netherlands, Spain, UK, USA
Trial
details:
- The ADVANCE trial is a randomized, double-blind, placebo-controlled 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 and promising in vivo efficacy. The primary outcome is to evaluate the efficacy of MIN-102 on the progression of AMN in male patients, as determined by a motor function test. The trial aims to enroll more than 100 patients and results are expected at the end of 2020. (NCT03231878)
Latest
news:
- • On September 5, 2018, Minoryx Therapeutics announced 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).
- • On January 4, 2018, Minoryx Therapeutics announced the initiation of treatment of the first two patients in the ADVANCE trial, a pivotal 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 patients were dosed at the Vall d’Hebron University Hospital (Barcelona, Spain) by Dr. Josep Gámez and at the Academic Medical Center (Amsterdam, The Netherlands) by Dr Marc Engelen. Recruitment was also initiated at the Institute of Genomic Medicine and Rare Disorders (Budapest, Hungary) by Dr. Maria Molnar. The ADVANCE trial will be initiated in several other European countries (United Kingdom, Germany, France, Italy and Poland) in the coming weeks and in the US by mid-2018.
- The trial was designed based on input from Minoryx’s scientific advisory board, comprised of internationally renowned EU and US clinical experts in X-ALD, (including Dr. Patrick Aubourg, Dr. Marc Engelen, Dr. Florian Eichler and Dr. Gerald Raymond). Additional advice was also obtained from major patient advocacy groups. The design and endpoints were finalized following regulatory interactions with the European Medicines Agency (EMA) and the FDA.
Is
general: Yes
