Cytokinetics has completed patient enrolment in the Phase II FORTITUDE-ALS trial of reldesemtiv (formerly CK-2127107) for the treatment of amyotrophic lateral sclerosis (ALS).
ALS is a progressive neurodegenerative disease that has a devastating effect on patients.
The trial features a double-blind, randomised, placebo-controlled, parallel group, dose ranging design.
Around 450 ALS patients from centres in the US, Canada, Europe, and Australia were randomised in 1:1:1:1 ratio to receive a twice-daily dose of either 150mg, 300mg or 450mg reldesemtiv or placebo for 12 weeks.
Primary efficacy endpoint of the FORTITUDE-ALS trial is the change from baseline in the percent predicted slow vital capacity (SVC) at 12 weeks.
Its secondary endpoints include slope of the change from baseline in the mega-score of muscle strength measured by hand-held dynamometry (HHD) and handgrip dynamometry, and incidence and severity of treatment-emergent adverse events (TEAEs).
The trial’s exploratory endpoints are the effect of reldesemtiv against placebo on self-assessments of respiratory function done at home by the patient, disease progression through quantitative measurement of speech production characteristics over time, and others.
FORTITUDE-ALS trial lead investigator Jeremy Shefner said: “There remains an urgent need in ALS to slow the decline of respiratory function, and to maintain stamina and muscle strength so that patients can sustain essential functionality for as long as possible.
“Fast skeletal muscle troponin activation may offer an important new approach to preserve strength in the presence of ongoing nerve loss and potentially complement current treatments for patients suffering from this dreadful disease.
“FORTITUDE-ALS is a large, international trial that was designed to answer key questions relating to this novel mechanism.”
Cytokinetics is currently developing reldesemtiv in collaboration with Astellas.
Reldesemtiv is a fast skeletal muscle troponin activator (FSTA) that has the potential to treat people living with diseases and conditions related to skeletal muscle weakness and/or fatigue.