According to a story from BioSpace, the biotechnology company Minoryx Therapeutics recently announced that the company has received official approval from the Spanish Agency of Medicines and Medical Devices (AEMPS) to conduct a Phase 2 clinical trial. This trial will test the company’s lead investigational product candidate MIN-102 as a treatment for Friedreich’s ataxia, a rare neuromuscular disease. Minoryx is committed to the development of rare disease therapies.
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 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.
With the approval for the trial cleared recruitment is expected to begin in the few weeks. The starting trial site will be the Hospital La Paz in Madrid. Minoryx also plans to develop additional trial sites in Belgium, France, and Germany. The clinical trial is expected to include a total of 36 participants with Friedreich’s ataxia who are 12 years or older. Although current approaches can effectively help slow disease progression and control symptoms effectively, there is still no therapy that can halt progression completely.
As the company delves into this area of rare disease treatment, it has added two new members to its scientific advisory board: Dr. Massimo Pandolfo and Dr. Fanny Mochel. These experts will help facilitate the development of MIN-102 for Friedreich’s ataxia and other disorder affecting the central nervous system.