Minoryx focuses its research on orphan CNS diseases, a large group of rare diseases affecting the central nervous system and usually characterized by progressive neurodegeneration. Most of these diseases are genetic and besides the CNS they may affect a wide range of organs. They are usually severe and devastating diseases, dramatically debilitating and often life-threatening, hence there is a high unmet medical need.

Some general pathways might be recognized in all different pathogenic cascades of these diseases, including oxidative stress, free radical formation, inflammation, mitochondrial dysfunction and energy imbalance. Several of the emerging therapies currently under development aim to target only one of these affected pathological cascades.

Conversely, PPAR gamma agonists, simultaneously act on multiple pathways through modulation of genes that are key to counteract oxidative stress, stimulate mitochondrial biogenesis, decrease inflammation and prevent neuronal death and demyelination.

The beneficial effects of PPAR gamma agonists in CNS disorders, both orphan and non orphan, have been demonstrated in multiple preclinical studies including Multiple Sclerosis, Parkinson’s Disease, Alzheimer’s Diseases, Huntington’s Disease, Friedreich’s Ataxia, Amyotrophic Lateral Sclerosis, Stroke and Traumatic Brain Injury. However, clinical studies failed to show beneficial effects due to insufficient modulation of PPARγ within the CNS.

There is not a pharmacological treatment for X-linked Adrenoleukodystrophy (X-ALD) the leading orphan CNS indication that Minoryx is pursuing with MIN-102, a PPARγ agonist able to adequately modulate the receptor within the CNS.

Additionally, results obtained to date suggest that this drug candidate has great potential for extending its use into other CNS disorders.

Adrenoleukodystrophy

X-linked Adrenoleukodystrophy (X-ALD) presents two main neurologic phenotypes. The most serious is cerebral ALD (cALD), characterized by brain neuroinflammation and early death, and occurring both in children and adults. The most common is adrenomyeloneuropathy (AMN), characterized by progressive severe motor dysfunction and affecting young adults.

Children and adult forms of X-ALD are characterized by central inflammatory demyelination in the brain, axonal degeneration and adrenal insufficiency.