Orion has completed the clinical phase 3 REFALS trial evaluating the efficacy and safety of oral levosimendan in the treatment of ALS (amyotrophic lateral sclerosis).
The primary endpoint of the trial was to show a statistically significant difference between levosimendan and placebo in slow vital capacity (SVC) in supine position at 12 weeks compared to the baseline. An important secondary endpoint was to show a statistically significant difference between levosimendan and placebo in patient functionality measured by ALSFRS-R Scale (Revised Amyotrophic Lateral Sclerosis Functional Rating Scale) in combination with survival at 48 weeks compared to the baseline.
These pre-specified endpoints were not met in the trial. No new safety concerns were identified for levosimendan in this study. The REFALS phase 3 trial data will be presented at a future scientific meeting.
“We would like to express our warmest thanks to the patients, carers and investigators for participating in the REFALS trial. The results were not what we wished and particularly we are disappointed on behalf of the ALS patients and their caregivers. There are very limited treatment options for ALS at the moment and there is a high unmet need for new innovative therapies. The REFALS trial still provides great resources to aid ongoing ALS research”, says professor Outi Vaarala, Senior Vice President, Head of Research and Development at Orion.
About REFALS and REFALS-ES
REFALS was 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 492 patients were being treated with levosimendan 1-2 mg or placebo daily up to 48 weeks. The primary outcome assessment of the study was slow vital capacity (SVC) in the supine position at 12 weeks. In addition, secondary assessments included the ALS functional rating scale and respiratory events through 48 weeks. The purpose of the trial was to demonstrate that levosimendan by enhancing respiratory muscle function, can help maintain breathing capacity and hence preserve overall functionality of patients with ALS.
REFALS-ES provided an opportunity for all applicable patients completing the REFALS study to receive levosimendan treatment for as long as it is clinically required. Due to the results of REFALS study, the REFALS-ES study continuation is under review.
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Outi Vaarala, Senior Vice President, Head of Research and Development
tel. +358 50 966 3472
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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, Finnish heritage rare diseases and respiratory diseases for which Orion develops inhaled Easyhaler® pulmonary drugs. Orion's net sales in 2019 amounted to EUR 1,051 million and the company had about 3,300 employees at the end of the year. Orion's A and B shares are listed on Nasdaq Helsinki.