Patients Recruited for Phase 2 Study of Experimental Friedreich’s Ataxia Treatment

According to a press release from Barcelona-based biotechnology company Minoryx Therapeutics, the Company has completed enrollment for a phase 2 clinical study of its experimental Friedreich’s ataxia treatment, MIN-102 (generic name leriglitazone).

About Friedreich’s Ataxia

Friedreich’s ataxia is a rare, inherited progressive neurodegenerative disorder that ultimately severely impairs muscle coordination. The symptoms of the condition, which typically first present between 10 and 15 years of age, are caused by shortages of a protein called frataxin. Frataxin is found in mitochondria throughout the body, with especially high concentrations in the heart, spinal cord, and skeletal muscles.

The exact role of frataxin within cells is subject to further research, but the protein appears to play a role in the preparation of certain molecules used in the production of cellular energy. Insufficient frataxin levels are associated with significant cellular dysfunction.

The shortage of frataxin in Friedreich’s ataxia patients is caused by mutations to the gene FXM, which normally provides the instructions for producing the protein. Because these mutations are inherited in an autosomal recessive pattern, an individual will not develop Friedreich’s ataxia with only one mutated copy of the FXM gene. Typically, patients’ parents are “carriers,” displaying no symptoms and each carrying only one mutant form of the gene.

Initially, those affected may experience a general decline in coordination and balance. Eventually, patients may develop other sensory and motor dysfunction like speech, vision, and hearing problems. Many patients also develop hypertrophic cardiomyopathy — enlargement of the heart that can have life-threatening implications. Most affected require a wheelchair within ten years of the first symptoms.

Unfortunately, like for other progressive neurodegenerative conditions, there is no treatment that can slow or reverse the progression of Friedreich’s ataxia. Certain drugs may be employed to treat individual symptoms, and physical therapy can prolong patients’ use of their arms and legs.

About MIN-102 and the Upcoming Phase 2 Study

MIN-102 is an experimental PPARγ agonist, a class of anti-inflammatory drug that acts on peroxisome proliferator-activated receptors (PPARs). PPARs play important roles in the regulation of gene expression — by modifying how these receptors interpret the instructions of genes, researchers hope to mitigate the effects of the FXM mutations that cause Friedreich’s ataxia.

The phase 2 study will be a randomized, double-blind, placebo-controlled study primarily assessing MIN-102’s ability to limit disease progression compared to placebo over the course of a year. 39 patients from four countries in Europe will form the groups of the study, receiving either MIN-102 or placebo as a liquid-dissolved powder once a day. Disease progression will be assessed by examining high-tech imagining of the spinal cord.

According to Minoryx’s Chief Executive Officer Marc Martinell, the Company plans to complete treatment and reporting on data by the end of next year.

Friedreich’s ataxia can cause significant changes to a person’s way of life. Why is it important to develop treatments that slow the progression of chronic illnesses? Share your thoughts with Patient Worthy!

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email