Idorsia initiates MODIFY, a Phase 3 registration study to assess lucerastat as a potential new treatment option for patients with Fabry disease - Seite 2

Martine Clozel, MD and Chief Scientific Officer, commented:
"Idorsia's preclinical data indicate that lucerastat has the potential to treat patients with Fabry disease, regardless of their specific gene mutation type. MODIFY will include patients who were never treated with enzyme or patients who stopped enzyme replacement therapy. In parallel to MODIFY, we will also run a pediatric study to assess lucerastat in children aged from 2 to 18 years. Lucerastat is therefore a potential new oral treatment option for a very broad spectrum of patients living with Fabry disease."

Guy Braunstein, MD and Head of Global Clinical Development, added:
"We have worked closely with patients during the development of the MODIFY protocol for lucerastat. We conducted an international patient survey to better understand the symptoms of patients with Fabry disease, and validated a patient reported outcome instrument to specifically assess Fabry neuropathic pain, in accordance with health authority guidance."

About the MODIFY study
MODIFY is a multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult patients with Fabry disease. The study aims to determine the effect of study treatment on neuropathic pain during 6 months of treatment, measured with Idorsia's validated Fabry disease pain instrument. At the end of the double-blind period, patients will be given the possibility to enter in an open label extension study. Approximately 108 patients are expected to be enrolled, randomized in a 2:1 ratio to either lucerastat or placebo. The study is expected to run for around 20 months.

Notes to the editor

About Fabry disease
Fabry disease is a rare, life threatening, genetic disorder involving a deficiency or dysfunction of alpha-galactosidase A (alpha-GalA) an enzyme that normally breaks down a fatty product known as globotriaosylceramide (Gb3) in the cells of the body. Over time, this may result in a build-up of Gb3 deposits throughout the body, particularly in the kidneys, heart and nervous system.

The symptoms range from neuropathic pain (primarily pain in the hands and feet), stomach, skin and eye problems, to hypertension, progressive kidney damage, cardiomyopathy and stroke. Since the symptoms are non-specific, Fabry disease is often undetected or misdiagnosed. As the disease is progressive, early diagnosis is essential to manage the symptoms as soon as possible and reduce the risk of developing serious complications.