Leigh Syndrome - IXC 109
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.
Ixchel’s main strategy is to identify novel mitochondrial activities and targets for FDA-approved drugs that have no previously characterized mitochondrial function. This ‘repurposing’ strategy decreases time to the clinic, as there is already a wealth of information regarding the pharmacokinetics (PK) and safety of existing drugs.
Ixchel repurposes known chemical compounds and generates IP protection including: novel indication (use) patents, orphan designation status, dosing patents, and formulation patents including mixtures of active compounds.
Leigh Syndrome is a severe mitochondrial disease of early childhood. IXC 109 uniquely extends longevity in the best mouse model of Leigh Syndrome
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FRIEDREICH'S ATAXIA - IXC 109