© 2017 Ixchel Pharma

Therapeutic R&D

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.

Repurposed Drugs

Gino Cortopassi

(530) 304-6810



Mitochondrial myopathy is a disease in which genetic defects cause inhibited mitochondrial function that leads to a number of muscular problems in affected patients. These symptoms include excessive muscle weakness, muscle wasting, and exercise intolerance. Limited mitochondrial function can also affect the heart or muscles that promote lung function, thus causing severe health consequences. Mitochondrial diseases are, by nature, orphan in their occurrence and there is no FDA-approved treatment for mitochondrial myopathy.

Ixchel has developed the repurposed drug, IXC 103, that addresses the underlying cause of mitochondrial myopathy by increasing mitochondrial copy number and function in patient cells. This drug is FDA-approved to treat non-mitochondrial myopathy indications, and is thus a great candidate for further development and treatment for this indication.




Leber’s hereditary optic neuropathy (LHON) is a mitochondrial inherited disease that causes retinal cell degeneration and total blindness by 30 years of age. The genetic cause of LHON is due to mutations affecting the mitochondrial complex I protein. LHON affects 5,000 individuals in the US and 13,000 in Europe; this disease is thus considered orphan in its prevalence. There is no approved therapy for Leber’s hereditary optic neuropathy in the US, nor is there any effective treatment for the disease in either the US or EU.

Ixchel Pharma has identified two repurposed molecules that increase ATP expression in LHON deficient cells: IXC 201 and IXC 203. These compounds are FDA-approved to treat non-LHON indications; IXC 201 and IXC 203 are currently in the development stage for LHON indication.

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.