Idorsia to present long-term lucerastat data and kidney biopsy results at WORLDSymposium

Core Insights - Idorsia Ltd announces the presentation of long-term results from the lucerastat treatment at the 22nd Annual WORLDSymposium™, highlighting its potential as a first-in-class oral substrate reduction therapy for Fabry disease [1][7] Study Overview - The MODIFY study was a multicenter, double-blind, randomized, placebo-controlled trial involving 118 patients to assess the efficacy and safety of lucerastat as an oral monotherapy for Fabry disease [3] - The study did not meet its primary endpoint of reducing neuropathic pain but showed significant reductions in plasma and urinary Gb3 levels compared to placebo, sustained over time in the open-label extension [4][14] Long-term Efficacy - An interim analysis of the open-label extension revealed a notable reduction in the rate of eGFR decline among patients treated with lucerastat, suggesting a potential disease-modifying effect [5] - Patients with impaired renal function at baseline experienced a marked attenuation of kidney function loss, indicating lucerastat's promise in long-term organ protection [5][14] Kidney Biopsy Sub-study - A kidney biopsy sub-study evaluated Gb3 accumulation in key kidney cell types in male patients with classic Fabry disease who received lucerastat for at least two years, providing insights into its impact on renal disease biology [2] Patient Population and Disease Background - Fabry disease is a rare, X-linked lysosomal storage disorder caused by mutations in the GLA gene, leading to the accumulation of Gb3 and progressive damage across multiple organ systems [6][8] - Recent studies suggest a higher prevalence of Fabry disease than previously estimated, with over 21,000 diagnosed patients expected across the US, EU5, and Japan by 2034 [9] Current Treatment Landscape - Current treatment options for Fabry disease include enzyme replacement therapies and oral chaperone therapy, which have limitations, highlighting the unmet need for a well-tolerated, oral, disease-modifying therapy [10][11]