Friedreich’s Ataxia
Friedreich’s ataxia (FRDA) is a rare disease which is caused by a frataxin deficiency. This deficiency results in the degeneration of nerves within the spinal cord. This nerve damage causes a loss of coordination, strength, and muscle control. In just 10 to 15 years after diagnosis, most patients can no longer stand.
Unfortunately, the disease is progressive with no known cure. This means the only option patients have is symptomatic treatment.
FRAMES
Leriglitazone (MN-102) is a selective gamma agonist developed by Minoryx Therapeutics. Researchers believe that it could become an effective treatment for Friedreich’s Ataxia. Preclinical studies and a Phase 1 investigation have both produced promising results.
The treatment targets the PPARγ/PGC1a pathways which other studies have indicated are downregulated in the disease. Targeting these pathways therefore could have disease modifying potential. In the preclinical models, MN-102 successfully was able to upregulate the PGC1a pathway. Additionally, it increased neuron survival, restored energy production, and improved biogenesis and mitochondrial function.
The Phase 1 investigation showed that MN-102 was able to effectively cross the blood/brain barrier, was well tolerated, and engaged the desired pathways at the same level that it did in the preclinical investigations.
Minoryx has just announced that the very first patient has been dosed in a Phase 2 study of MN-102 called FRAMES. FRAMES is a placebo-controlled, double-blind investigation that aims to assess the safety and efficacy of MN-102.
The very first dosing occurred at a site in Madrid, Spain. Other trial sites are located in Germany, France, and Belgium. These sites are currently enrolling. The trial aims to enroll 36 Friedreich’s ataxia patients in total. All participants must be at least 12 years old and will receive treatment for a full year.
The principal investigator of this study is Alexandra Durr from Paris, France.
Other Targets
In addition to Friedreich’s ataxia, MN-102 has shown promise as a therapy for other central nervous system (CNS) diseases. For instance, the treatment is currently in a Phase 2/3 study for adrenomyeloneuropathy (AMN), which is a form of X-linked Adrenoleukodystrophy. This study has completed enrollment (116 AMN patients). A quarter of the participants have already received MN-102 for 12 months.
The therapy has been given Orphan Drug Designation in both the US and the EU for X-ALD.
Hopefully, this investigation for AMN and the FRAMES trial for Friedreich’s ataxia will continue to show the efficacy of MN-102 and we will see a new treatment option become available for this patient population soon.
You can read more about the FRAMES trial here.
