• Statistically significant difference in arrested disease in leriglitazone treated patients when compared to the natural history of the disease
   
• Submission of European marketing authorization application (MAA) for leriglitazone expected mid-2025.

Barcelona, Spain and Düsseldorf, Germany - 11 December 2024 - Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders and Neuraxpharm Group (Neuraxpharm), a leading European specialty pharmaceutical company focused on the treatment of CNS disorders, today announced that the NEXUS trial has been completed and that the primary endpoint has been met. The parties intend to file for European Marketing Authorization of leriglitazone in pediatric and adult cerebral Adrenoleukodystrophy (cALD) patients by mid-2025.

The NEXUS trial was a 96-week, pivotal, open-label study designed to evaluate the efficacy and safety of once-daily oral dosing of leriglitazone in pediatric patients with cALD. The primary endpoint assessed the proportion of patients that had clinical and radiological arrested disease at week 96 or at a visit prior to hematopoietic stem cell transplant (HSCT). The evaluable population in the study consisted of 20 patients treated for a minimum of 24 weeks. All 20 patients remained clinically stable whilst on treatment and 7 out of 20 patients (35%) met the arrested disease criteria, which is significantly greater than the 10% self-arrested patients that would be expected from natural history (p<0.05). 

Leriglitazone was well tolerated in all children, there were no treatment-related serious adverse events nor discontinuations due to treatment related adverse events. 

The full results of the NEXUS trial will be presented at upcoming neurology conferences.

“Cerebral ALD in boys is a devastating disease both for the patients and their families. Treatment options are limited, physicians and families of patients have constantly been searching for better tools and therapies to manage cALD in boys.” said Patricia Musolino, Global Principal Investigator of Nexus. “The NEXUS results attest that leriglitazone addresses a critical unmet need for non-invasive treatments that can be administered at the moment brain lesions are identified to arrest or slow down lesion growth.”

“We are very pleased with the positive results from NEXUS demonstrating that leriglitazone not only arrests the demyelinating brain lesions in children with cALD but also arrests clinical progression. These results are further supported by evidence from the cALD related endpoints of ADVANCE1 in adult patients and the findings reported in cALD patients treated under compassionate use1” said Marc Martinell, CEO, Minoryx. ”Together with our partner, Neuraxpharm, we intent to submit the MAA to the EMA for this life-threatening condition as soon as possible.”

Dr. Jörg-Thomas Dierks, CEO, Neuraxpharm said; “cALD is a very serious neurological disorder with devastating outcomes for patients and their families. The results from this trial are extremely encouraging and we are fully committed to working with Minoryx to swiftly provide patients with an effective new treatment for this otherwise fatal and cruel disease.”

Based on the successful results from NEXUS, Minoryx and Neuraxpharm have begun to compile the regulatory file for MAA submission in the European Union.