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

Idorsia Pharmaceuticals Ltd. / Idorsia initiates MODIFY, a Phase 3 registration study to assess lucerastat as a potential new treatment option for patients with Fabry disease . Processed and transmitted by Nasdaq Corporate Solutions. The issuer is solely responsible for the content of this announcement.

• Idorsia to host an investor webcast to discuss the Phase 3 program today at 14:00hrs CEST

             
Allschwil, Switzerland - May 16, 2018
Idorsia Ltd (SIX: IDIA) today announced that the first patient has been enrolled in a registration study to investigate the effect of lucerastat, as an oral monotherapy, for the treatment of adult patients with genetically confirmed Fabry disease, irrespective of their genetic mutation type.

MODIFY will recruit over 100 patients from 29 trial sites across 9 countries. Its primary endpoint is a reduction in neuropathic pain, described as feeling like burning, shocks or shooting, stabbing, tingling, and/or pins and needles primarily in the hands and feet. This major symptom is reported by many patients with Fabry disease as significantly impacting their daily activities and quality of life, despite existing treatment.

Dr. Derralynn Hughes, DPhil, FRCP, FRCPath and EU Coordinating Investigator, commented:
"Today's news is an important milestone for the Fabry research and patient communities that have contributed to the development of this study. Pain is a genuine and pressing unmet need of the Fabry patient population. Pain remains a significant burden for many patients, even for some of those who are already being treated with enzyme replacement therapy. Lucerastat represents an exciting potential new oral treatment option to address this."

Fabry disease is a rare, life threatening, inherited lysosomal storage disorder in which a particular lipid, called globotriaosylceramide (Gb3), accumulates in cells of many organs of the body. This build-up results in cellular dysfunction leading to a range of signs and symptoms from neuropathic pain (pain primarily in the hands and feet), stomach, skin and eye problems, to hypertension, progressive kidney damage, cardiomyopathy, and stroke. New treatment options are needed to treat the underlying mechanism of the disease and provide symptomatic relief.