Adrenomyeloneuropathy (AMN)
Minoryx Therapeutics, a biotech company focussed on the development of therapeutics for disorders of the central nervous system (CNS), have recently announced results from a Phase 2/3 clinical trial of leriglitazone for the treatment of adrenomyeloneuropathy (AMN) in male patients. The clinical trial was conducted on 96 patients over approximately 2 years, and it was found that early-symptomatic AMN patients who received leriglitazone experienced a clinically meaningful difference in the ability to complete a ‘6-minute walk test’ compared to patients who received a placebo. In addition, patients who received leriglitazone also did well on the ‘sway test’, which together with the results of the walk test indicate reduced loss of control of balance and body posture. These patients also experienced reduced progression on both the Expanded Disability Status Scale and the Severity Score System for Progressive Myelopathy compared to patients who received placebo. Leriglitazone treatment also had the effect of reducing the progression of cerebral lesions. Following the trial, around 90% of patients elected to keep taking the treatment, and data will continue to be collected.
This study by Minoryx Therapeutics demonstrates promise for the use of leriglitazone for the treatment of AMN, and the company is currently preparing for discussions with regulatory authorities regarding how AMN patients can gain access to this drug in future.