Adrenomyeloneuropathy (AMN)
Minoryx Therapeutics works on the development of treatments for central nervous system disorders. Results are now published from the first and largest international study to include adult male X-linked adrenoleukodystrophy (X-ALD) patients for their lead candidate leriglitazone, assessing its efficacy and safety in male patients with adrenomyeloneuropathy (AMN). In its Phase 2/3 ADVANCE clinical trial, there were 116 randomized patients with 77 receiving leriglitazone and the other 39 a placebo. It was generally well tolerated, with clinically relevant differences in body sway measurements and positive trends for the Expanded Disability Status Scale (EDSS), the Severity Scoring system for Progressive Myelopathy (SSPROM), and quality of life. The EDSS measures neurological disability and the SSPROM measures the severity of myelopathy. An important result was it showed reduction in the progression of cerebral lesions and only those in the placebo group developed clinically progressive cerebral ALD (cALD). At week 96 in placebo patients with progression of cerebral lesions, the plasma biomarker neurofilament light chain had significantly increased levels, thus supporting the positive effect leriglitazone has on reducing axonal damage. Also at week 96, a significant increase in the Loes severity score was seen in the placebo group, this rates the severity of abnormalities found in MRI scans of the brain in order to assess disease progression and treatment effectiveness. Since the drug was found to have potential for benefit, it will now continue as an open label extension study where all participants, including those previously in the placebo group, will be given leriglitazone. This will allow further monitoring of the progression of myelopathy and cerebral lesion and the impact of treatment with leriglitazone.