Orion has enrolled the first patient in the REFALS trial, a Phase III clinical study of levosimendan (ODM-109) for the treatment of symptoms of amyotrophic lateral sclerosis (ALS), also known as motor neurone disease (MND), that causes the death of neurons controlling voluntary muscles.
The randomised, double-blind, placebo-controlled, parallel-group, multicentre trial is designed to examine the ability of orally administered levosimendan to maintain the breathing capacity and overall functioning of ALS patients.
The trial is expected to enrol a total of 450 patients at around 100 clinical sites in Europe, North America and Australia.
During the trial, the patients will be treated for around one year after administering the drug for the first time.
Primary endpoints of the trial are supine slow vital capacity (SVC) starting from baseline at 12 weeks and pulmonary assessment.
The trial’s secondary endpoints include combined assessment of ALS functional rating scale (revised) (ALSFRS-R) function and survival, time to respiratory event, change from baseline in respiratory function of ALSFRS-R at 48 weeks, among others.
Orion is expected to invest nearly €60m in the trial over a period of around three years.
Depending upon the results of the trial, Orion will file for marketing authorisation of levosimendan in the US and Europe.
Levosimendan is a molecule developed by Orion to treat acute decompensated heart failure.
Orally administered levosimendan has already received an orphan drug designation both in the US and in the European Union (EU).