Date: 2017-01-18
Type of information: Nomination
Compound: scientific advisory board
Company: Minoryx Therapeutics (Spain)
Therapeutic area: Rare diseases
Type agreement: nomination
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Details:
- • On January 18, 2017, Minoryx Therapeutics announced that, Dr. Patrick Aubourg, Dr. Marc Engelen, Dr. Florian Eichler and Dr. Gerald Raymond, four renowned clinical experts in X-linked adrenoleukodystrophy (X-ALD) joined its scientific advisory board. The disease-specific knowledge of these clinical experts will further complement and strengthen the existing scientific advisory board consisting of Dr. David Eckland (chair), Dr. Joan Aymamí and Dr. Xavier Badia.
- Dr. Patrick Aubourg is professor of pediatrics at the Medical University Paris-Sud (France), head of the Pediatric Neurology Department at the Hospital Bicêtre (Le Kremlin Bicêtre, France) and director of Inserm Research Unit UMR1169 at the Medical University Paris-Sud/Paris Saclay University (France). He is a member of several networks aiming to develop new therapeutic approaches in neurodegenerative diseases, in particular in the field of leukodystrophies.
- Dr. Florian Eichler is associate professor of neurology at Harvard Medical School (USA), assistant in neurology at Massachusetts General Hospital, director of the Leukodystrophy Clinic and director of the Center for Rare Neurological Diseases. Dr. Eichler studies monogenetic lipid metabolism disorders of the nervous system at the Massachusetts General Hospital and Harvard Medical School. His research focuses on the genetics of peroxisomal disorders, lipid metabolism and spatial aspects of nuclear magnetic resonance spectroscopy. Current projects include analyzing metabolic changes seen in the brain by MR measures and determining the neurotoxicity of newly discovered atypical sphingolipids.
- Marc Engelen received his MD from the University of Amsterdam (The Netherlands) in 2002. He trained as a neurologist and subsequently specialized in pediatric neurology at the Academic Medical Center (AMC) in Amsterdam. He obtained his PhD on adrenoleukodystrophy in 2012. He is currently a member of the medical staff in the Department of Neurology and the Department of Pediatrics. He has a special interest in peroxisomal disorders; the AMC was recently designated the national expert center for this group of diseases. In 2014 he received a Veni grant from NWO (The Netherlands Organisation for Scientific Research) to study the natural history of adrenoleukodystrophy and identify predictive biomarkers in the onset of cerebral ALD.
- Dr. Gerald Raymond received his medical degree from the University of Connecticut (USA). He trained in pediatrics at Johns Hopkins University and in neurology at Massachusetts General Hospital. He completed his training in clinical genetics at Harvard University. Dr. Raymond is a pediatrician, a geneticist and a neurologist with a special interest in developmental and neurogenetic disorders affecting children and adults. He is currently working at the University of Minnesota Masonic Children’s Hospital. Dr. Raymond’s work focuses on the diagnosis, care and treatment of all aspects of ALD and other peroxisomal disorders. Recently, he has advanced newborn screening of ALD and clinical trials on the natural history and treatment of all aspects of ALD.
- David Eckland has been head of metabolic disease clinical research at GSK. His experience ranges from preclinical stages to post marketing studies. He has worked in senior positions (managing director of Takeda Europe R&D, and R&D director, Ark Therapeutics), where he developed pioglitazone, among other drugs. Dr Eckland has covered many therapeutic areas, including oncology, rheumatology, cardiovascular, urology and metabolic disease. He has supervised numerous phase IV clinical trials, interacting with commercial and business development colleagues. He has also lectured on clinical trial design at University College, London.
- Minoryx Therapeutics’ lead candidate, MIN-102 targets this most prevalent peroxisomal disorder, X-linked adrenoleukodystrophy (X-ALD), a rare and chronically debilitating life threatening neurodegenerative disease, currently with no available treatment. MIN-102 is a differentiated PPAR gamma agonist with a superior profile for central nervous system related diseases and excellent in-vivo efficacy. It has shown robust preclinical proof of concept in multiple animal models. Phase I studies have been initiated based on these results. A phase II/III trial in adult AMN patients has been planned. This is expected to be launched during the first half of 2017.
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