© 2017 Ixchel Pharma
Leigh Syndrome is an orphan neurodegenerative and myodegenerative disease, that strikes infants, that caused by inherited mutations in mitochondrial genes. IXC-109 improves longevity and functions in preclinical animal model of Leigh Syndrome
Ixchel holds exclusive intellectual property rights to a number of therapeutic drugs. These rights include an orphan designation and beneficial formulations for Friedreich’s ataxia and mitochondrial myopathy, IP rights to drugs and formulations in Leber’s hereditary Optic Neuropathy, and IP rights to modulators of the Shc molecule that target the causes of pediatric diabetes and obesity.
IXC-109 For Friedreich's ataxia. IXC-109 is a New Chemical entity (NCE) that dose-dependently increases frataxin and other mitochondrial proteins. IXC-103 is a repurposed drug molecule that increases frataxin and rescues Friedreich's cardiomyopathy in a mouse model.
Friedreich’s ataxia (FA) is a serious, crippling neurodegenerative and cardiodegenerative disease that leads to early death. This mitochondrial-inherited disease is considered orphan; it is also the most prevalent recessive ataxia, affecting 6,000 individuals in North America and 15,000 in Europe. There is no approved therapy for FA in the US or EU, hence there is a great unmet need.