Ferrer reports that the Phase III ADORE clinical trial (EudraCT 2020-003376-40 /NCT05178810) with an oral formulation of edaravone (FAB122) in patients with amyotrophic lateral sclerosis (ALS) did not meet the key primary or secondary endpoints.
The ADORE clinical trial is a multicenter, international, double-blind, randomized, placebo-controlled Phase III study to investigate the efficacy and safety of 100 mg of an oral formulation of edaravone (FAB122) once daily, in patients with ALS, for 48 weeks. Participants were randomly assigned to receive either PNF122 or placebo at a 2-to-1 ratio, while continuing to receive their usual standard treatment for ALS. The study was carried out with the support of TRICALS, the largest European research initiative to find a cure for ALS.
The ADORE data show that the product did not show a significant benefit over placebo in ALS patients in slowing disease progression, as measured from the change in baseline score on the ALSFRS-R scale, after 48 weeks of daily dosing with the oral formulation of edaravone. No improvement over placebo in long-term survival, as measured by CAFS at 48 weeks and 72 weeks for the subgroup of patients, was also not observed. The results of the study also concluded that the product has been shown to be safe and well tolerated.
Tatjana Naranda, Chief R&D Officer at Ferrer, said: "We are disappointed with this result and would like to thank the people with ALS, caregivers, researchers and study staff for their participation in the ADORE clinical trial. Although the results are not what we expected, our research and work to advance potentially transformative therapies in areas of high unmet clinical need for people with ALS will not stop."
Prof. Leonard H. van den Berg, President of TRICALS, Professor of Neurology at University Medical Center Utrecht, The Netherlands, and Principal Investigator of the clinical trial, says: "We at TRICALS expected a different outcome from the ADORE study and are disappointed that this is not the positive outcome we all wanted. We will continue to do our best to find a treatment for ALS as soon as possible. We want to thank all the patients who dedicated their time and effort to this trial and Ferrer for the good collaboration that we hope will be repeated in the future".
The company also announces the completion of the clinical trial extension (Open label extension), called ADOREXT (EudraCT 2022-003050-32/ NCT05866926), due to the lack of efficacy of the oral formulation of edaravone (FAB122).
Amyotrophic Lateral Sclerosis (ALS)1, the most common neuromotor disease, is a neurodegenerative disorder of the motor neurons of the brain and spinal cord, progressively causing paralysis, until death, usually 2 to 5 years after diagnosis. ALS is a rare disease with a multifactorial etiology whose pathogenic mechanism is not yet precisely understood. ALS typically appears in people between 40 and 70 years of age, being more frequent in men than in women, and a distinction is made between two types: familial ALS, which affects 10-15% of patients, and sporadic ALS, which affects 85-90% of them.
1 Masrori P, Van Damme P. Amyotrophic lateral sclerosis: a clinical review. Eur J Neurol. 2020; 27(10):1918-1929.
