Leriglitazone Meets Primary Endpoint in NEXUS Trial for Paediatric cALD
Minoryx Therapeutics and Neuraxpharm Group have announced a significant breakthrough in the treatment of cerebral adrenoleukodystrophy (cALD) in paediatric patients.
The companies reported that leriglitazone, an oral, once-daily treatment, successfully met the primary endpoint in the NEXUS trial – a key milestone offering renewed hope for patients and families affected by this devastating condition.
The 96-week NEXUS trial focused on evaluating the efficacy of leriglitazone in boys with cALD, a rare and life-threatening neurological disorder. All 20 patients in the study remained clinically stable throughout the trial.
Notably, 35% of patients achieved disease arrest, a figure substantially higher than the 10% typically expected based on natural history. Importantly, no treatment-related serious adverse events were reported, underscoring the treatment’s safety profile.
The Global Principal Investigator of the NEXUS trial emphasised the significance of the results, stating that Cerebral ALD in boys is a devastating disease both for the patients and their families, with treatment options being limited. The NEXUS results show that leriglitazone addresses a critical need for non-invasive treatments to arrest or slow down lesion growth.
Following the successful outcomes, Minoryx Therapeutics and Neuraxpharm Group plan to file for European Marketing Authorisation (MAA) by mid-2025. The CEOs of both companies shared their optimism about the trial results.
The CEO of Minoryx Therapeutics expressed his enthusiasm, remarking that they are pleased with the positive results from NEXUS, demonstrating that leriglitazone arrests brain lesions in children with cALD and clinical progression. These results are supported by evidence from other cALD studies, and they intend to submit the MAA to the EMA as soon as possible.
Echoing these sentiments, the CEO of Neuraxpharm Group added that cALD is a serious neurological disorder with devastating outcomes. The results from this trial are extremely encouraging, and they are committed to providing patients with an effective new treatment.
With these promising outcomes, Minoryx and Neuraxpharm have already begun compiling the regulatory documentation for submission to the European Medicines Agency. The full results of the NEXUS trial are set to be presented at upcoming neurology conferences, further highlighting this important development to the medical community.
In conclusion, the positive results of the NEXUS trial mark a significant step forward in addressing the urgent need for non-invasive treatment options for paediatric patients with cALD.
Leriglitazone’s ability to arrest lesion growth while maintaining clinical stability offers a much-needed ray of hope to families impacted by this devastating condition. As the companies move toward regulatory submission, the future looks brighter for young patients battling cALD.
News Credits: Leriglitazone meets primary endpoint in pivotal cALD trial
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