Leber’s hereditary optic neuropathy (LHON) is a mitochondrial inherited disease that causes retinal cell degeneration and ultimate vision loss. IXC 201 is a repurposed compound that increases ATP synthesis by more than 100% in LHON mutant cells, and is thus a good candidate drug for the disease. IXC 201 also rescues blindness in a mouse model of LHON. Ixchel seeks partnerships to further develop IXC-201 from a preclinical package to clinical.
IXC 103 is a repurposed molecule that treats mitochondrial myopathy by increasing mitochondrial copy number and function in human cells. Ixchel holds a provisional patent for IXC 103 and is available for co-development or out-licensing. The repurposed nature of this drug provides expedited development from clinic to patient.
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LEBER'S OPTIC NEUROPATHY
IXC 203 is a repurposed molecule that is chemically distinct from IXC 201. IXC 203 increases ATP synthesis by more than 100% in LHON mutant cells, and is thus a good candidate drug for the disease. IXC 203 also rescues blindness in a mouse model of LHON. Ixchel seeks partnerships to further develop IXC 203 from preclinical package to clinical and beyond.
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.
Friedreich’s ataxia (FA) is a serious, crippling neurodegenerative and cardiodegenerative disease that leads to early death. This mitochondrial-inherited disease is considered orphan; however, 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.
The underlying molecular cause of FA is well-understood to be an inherited monogenic disease that causes a deficiency of frataxin protein. A deficiency of mitochondrial frataxin protein below 50% of normal patients is deemed the only cause of Friedreich’s ataxia. Frataxin is necessary for the proper formation of iron-sulfur clusters that are important for numerous cellular processes such as the regulation of inflammation and the prevention of neuro and cardio degeneration.