Minoryx focuses on orphan diseases of the central nervous system (CNS), a large group of rare diseases often characterized by neurodegeneration and for which there are currently no treatments available. These diseases are usually chronic, progressively debilitating and often life-threatening, with high unmet medical need.

Some orphan CNS diseases are of genetic origin, but they can also be triggered by external unknown factors and may affect other organs in addition to the brain. Several complex, interplaying pathways are contributing to the neurodegenerative process: mitochondrial dysfunction, oxidative stress, and neuro-inflammation all play an important role in neurodegeneration, independent of the underlying cause of disease. Research into novel effective treatments, like leriglitazone, addresses this complexity, rather than focus on a single pathway approach.

PPAR gamma controls multiple genes that are central to mitochondrial biogenesis, oxidative stress, inflammation, and myelination. The potential beneficial effects of PPAR gamma agonists in CNS disorders have been demonstrated preclinically in several orphan and non-orphan indications such as Multiple Sclerosis, Parkinson’s Disease, Alzheimer’s Diseases, Huntington’s Disease, Amyotrophic Lateral Sclerosis, Stroke and Traumatic Brain Injury. However, to date clinical studies failed to show beneficial effects of PPAR gamma agonists in these indications, due to insufficient target engagement in the CNS.

Minoryx is developing leriglitazone, a differentiated and selective brain penetrant PPAR gamma agonist able to reach the required CNS exposure in humans. Minoryx is developing leriglitazone for the treatment of X-linked Adrenoleukodystrophy (X-ALD) and Friedreich's Ataxia (FRDA), two rare orphan diseases of the CNS with high unmet medical need. The marketing authorization application (MAA) for leriglitazone as a treatment for adult male X-ALD patients is under review by the EMA. Leriglitazone is also being evaluated in other rare CNS disorders.