Orion Corporation announces the completion of patient enrollment in the phase 3 REFALS trial, studying the effects of oral levosimendan (ODM-109) on respiratory function in patients with ALS.
“There remains an urgent need to provide novel therapies to improve muscle functions and quality of life in people living with ALS. The completion of patient enrollment is an important clinical milestone that takes us a step closer towards a potential new treatment option for patients with ALS“, said Christer Nordstedt MD, PhD, Senior Vice President, Research and Development, Orion Corporation. “We would like to thank all the patients living with ALS, their caregivers and dedicated researchers who made it possible to complete this phase of the study so rapidly. We expect the study to be completed in just over a year”, added Dr. Nordstedt.
REFALS is a Phase 3, double blind, randomized, placebo-controlled, study of oral levosimendan in patients with ALS. A total of 496 participants across 104 sites in the U.S., Canada, EU and Australia were enrolled for the study and are being treated with oral levosimendan 1-2 mg daily for 48 weeks. The primary outcome assessment of the study is slow vital capacity (SVC) in the supine position at 12 weeks. In addition, secondary assessments include the ALS functional rating scale and respiratory events through 48 weeks. The purpose of the trial is to demonstrate that orally administered levosimendan, by enhancing respiratory muscle function, can help maintain breathing capacity and hence preserve overall functionality of patients with ALS.
Levosimendan is a molecule originally developed by Orion for the treatment of acute decompensated heart failure as Simdax® i.v. formulation. Launched in 2000, Simdax® continues to be one of Orion's largest products by sales value and is sold in almost 60 countries.
ALS as a rare neurogenerative disease
ALS is a rare adult-onset neurodegenerative disease of upper and lower motor neurons resulting in progressive weakness, with death occurring from respiratory failure commonly as a result of diaphragmatic weakness typically within three to four years of diagnosis. Poor respiratory function is a major source of disability, fatigue, morbidity and mortality in ALS.
More information about the REFALS Phase III study: www.clinicaltrials.gov, Indentifier: NCT03505021
Prof. Paula Rytilä
Vice President and Chief Medical Officer
Tel. +358 (0)10 426 4416
Orionintie 1A, FI-02200 Espoo, Finland
Orion is a globally operating Finnish pharmaceutical company – a builder of well-being. Orion develops, manufactures and markets human and veterinary pharmaceuticals and active pharmaceutical ingredients. The company is continuously developing new drugs and treatment methods. The core therapy areas of Orion’s pharmaceutical R&D are central nervous system (CNS) disorders, oncology and respiratory diseases for which Orion develops inhaled Easyhaler® pulmonary drugs. Orion’s net sales in 2018 amounted to EUR 977 million and the company had about 3,200 employees at the end of the year. Orion’s A and B shares are listed on Nasdaq Helsinki.