Leriglitazone meets primary endpoint in pivotal cALD trial
Promising trial results support marketing authorisation application
Minoryx Therapeutics and Neuraxpharm Group have announced that leriglitazone has met the primary endpoint in the NEXUS trial for paediatric patients with cerebral adrenoleukodystrophy (cALD).
The companies plan to file for European Marketing Authorization by mid-2025.
“Cerebral ALD in boys is a devastating disease both for the patients and their families. Treatment options are limited,” said Patricia Musolino, Global Principal Investigator of NEXUS. “The NEXUS results show that leriglitazone addresses a critical need for non-invasive treatments to arrest or slow down lesion growth.”
The 96-week trial evaluated once-daily oral leriglitazone in pediatric patients with cALD. All 20 patients remained clinically stable, with 35% meeting the arrested disease criteria, significantly higher than the 10% expected from natural history.
No treatment-related serious adverse events were reported.
“We are pleased with the positive results from NEXUS, demonstrating that leriglitazone arrests brain lesions in children with cALD and clinical progression,” said Marc Martinell, CEO of Minoryx. “These results are supported by evidence from other cALD studies. We intend to submit the MAA to the EMA as soon as possible.”
Dr Jörg-Thomas Dierks, CEO of Neuraxpharm, added, “cALD is a serious neurological disorder with devastating outcomes. The results from this trial are extremely encouraging, and we are committed to providing patients with an effective new treatment.”
Based on these successful results, Minoryx and Neuraxpharm have started compiling the regulatory file for the European Union submission.
The full results of the NEXUS trial will be presented at upcoming neurology conferences.