- Orphan CNS diseases
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 no treatments currently 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 should address 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, for the treatment of X-linked Adrenoleukodystrophy (X-ALD) and Friedreich's Ataxia (FRDA), two rare orphan diseases of the CNS. Leriglitazone is also being evaluated in other undisclosed rare CNS disorders.
- Our focusAdrenoleukodystrophy
X-linked Adrenoleukodystrophy (X-ALD) is an orphan neurodegenerative disease caused by a mutation in the ABCD1 gene. X-ALD presents with two main neurologic phenotypes. The most common phenotype is adrenomyeloneuropathy (AMN), characterized by progressive severe motor dysfunction and which affects young adults. The acute form is cerebral ALD (cALD), characterized by brain neuroinflammation and early death, and which occurs both in children and adults.
X-ALD is characterized by central inflammatory demyelination in the spinal cord and brain, axonal degeneration and adrenal insufficiency.Our focusFriedreich's ataxia
Friedreich's ataxia (FRDA) is an orphan neurodegenerative genetic disease characterized by frataxin deficiency and resulting in mitochondrial dysfunction affecting the central nervous system and the heart. In the majority of cases, FRDA has an onset before the age of 25 and progressively impairs motor function, leading to ataxic gait, cardiac abnormalities and other medical co-morbidities.