Phase III MODIFY study of lucerastat fails to meet primary endpoint in Fabry disease.

Lucerastat was well tolerated and researchers saw a consistent reduction of plasma Gb3, confirming the pharmacological activity of lucerastat. Despite this biological effect, no reduction in neuropathic pain was observed after six months of treatment, based on the patient reported outcome tool. In addition, most patients chose to continue in the open-label extension study and in a few weeks, the company expect to see more results that will inform its decision on the future of lucerastat.

MODIFY was a multicenter, double-blind, randomized, placebo?controlled, parallel-group study to determine the efficacy and safety of lucerastat as an oral monotherapy in adult patients with Fabry disease. MODIFY determined the effect of study treatment on neuropathic pain during 6 months of treatment, measured with Idorsia’s validated Fabry disease pain instrument. 118 patients were randomized in a 2:1 ratio to either lucerastat or placebo. At the end of the double-blind period, 107 patients entered into an ongoing open label extension study (NCT03737214), which is investigating the long-term safety and tolerability of lucerastat oral therapy and to further evaluate its clinical efficacy on renal and cardiac function, in adult patients with Fabry disease over a period of up to a further 48 months.