According to a story from Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company’s phase 2 clinical trial. This trial is testing Minoryx’s lead experimental product candidate, leriglitazone, as a treatment for the rare disease Friedreich’s ataxia. Minoryx is committed to the development of new treatments for orphan diseases.
About Friedreich’s Ataxia
Friedreich’s ataxia (FRDA) is a neuromuscular genetic disorder which is characterized by abnormal gait due to the degeneration of nerve tissue in the spinal cord. The disorder is linked to a genetic mutation of the FXN gene. The degeneration of the nerves causes the spinal cord to become thinner and neurons begin to lose some of their myelin sheath, a fatty insulating layer that is protects these cells and is necessary for normal function. Symptoms of Friedreich’s ataxia include muscle weakness affecting the limbs, heart disorders, poor coordination, diabetes, high foot arches, vision and hearing problems, scoliosis, and slurred speech. Symptoms begin to appear at 7-10 years and the disorder progresses slowly overall. Management strategies for the disorder may include ACE inhibitors, surgery, speech and rehabilitation therapy, and orthoses. Life expectancy is around 50 years. Heart problems are the leading cause of death. To learn more about Friedreich’s ataxia, click here.
About The Clinical Trial and Leriglitazone
The initial dosing took a place at Hospital La Paz in Madrid, Spain. The clinical trial is planning to include around 36 Friedreich’s ataxia patients. These patients will be age twelve or older and the treatment period of the study will last one year.
Leriglitazone is an innovative orally available agonist of PPAR gamma, which research studies have shown is downregulated in Friedreich’s ataxia and a number of other diseases that impact the central nervous system. in pre-clinical evaluations, leriglitazone was capable of improving biogenesis and mitochondrial function as well as restoring energy production and improving the survival of nerve cells. The drug is also being tested in adrenomyeloneuropathy. The drug is also capable of crossing the blood-brain barrier.
If the clinical trial is able to yield positive results, leriglitazone could be the first disease modifying treatment for Friedreich’s ataxia.