On 10 January, a press release by Ferrer Internacional SA (Ferrer) announced that its anticipated free radical scavenger FAB122, an investigational oral formulation of edaravone, failed to achieve its primary efficacy and key secondary endpoints in the Phase III ADORE (ALS Declaration with Oral Edaravone) trial (NCT0178810) for amyotrophic lateral sclerosis (ALS). Edaravone reduces oxidative stress and elicits a cytoprotective impact on nerve cells, aiming to protect the neuronal cells from degeneration. This news is a disappointment to ALS patients across Europe, where there is a single approved treatment option in the form of riluzole, marketed by Sanofi as Rilutek, and as Teglutik that is marketed by Italfarmaco SA (France, Germany, Italy, Spain) and by Martindale Pharmaceuticals (UK); however, this therapy option has limited efficacy. Therefore, there is still a high unmet need for efficacious disease-modifying treatments for ALS.
The ADORE clinical trial, a double-blind, randomised, placebo-controlled Phase III study, aimed to investigate the efficacy and safety of 100mg edaravone (FAB122) once daily as an oral formulation in ALS patients over 48 weeks. Unfortunately, ADORE data indicates that the product did not demonstrate a significant benefit over placebo in slowing ALS progression, measured by change from baseline in the revised ALS Functional Rating Scale (ALSFRS-R) score after 48 weeks of daily oral edaravone dosing. Additionally, no improvement over placebo in long-term survival, measured by the Combined Assessment of Functional and Survival (CAFS) at 48 and 72 weeks for a subgroup of patients, was observed. Consequently, based on FAB122’s lack of efficacy, the company has also announced the conclusion of the open-label extension ADOREXT study (NCT05866926).
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Ferrer’s chief R&D officer Tatjana Naranda said in the press release, “We are disappointed with this outcome and would like to thank the people with ALS, caregivers, investigators, and clinical trial staff for their participation in the ADORE clinical trial. Although the results are not what we hoped for, our work to advance potentially transformative therapies in areas of high unmet need for people living with ALS will not stop.”
Historically, developing disease-modifying treatments for this rare and aggressive disease has proven challenging. The high failure rate in ALS clinical trials is often attributed to the disease’s unclear aetiology and complex pathophysiology. FAB122 is the third pipeline asset to recently fail a Phase III trial for ALS, following the failure of Cytokinetics‘s reldesemtiv in March 2023, and BrainStorm Cell Therapeutics’s NurOwn in September 2023. However, unlike Ferrer and Cytokinetics, BrainStorm Cell Therapeutics is still fighting for NurOwn and according to a December 26 press release, has acquired three new patents for NurOwn and NurOwn-Exosomes as a safeguard for NurOwn’s cell therapy, fostering hope for a strong market position upon potential approval, both positioning Brainstorm Cell Therapeutics favourably for monetisation and adding to the overall ALS treatment portfolio.
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