Minoryx Therapeutics, a member of Catalonia.health, and the Neuraxpharm Group have announced the completion of recruitment for the global Phase 3 CALYX clinical trial, which is evaluating leriglitazone (NEZGLYAL®) in adult male patients with cerebral adrenoleukodystrophy (cALD), a rare neurodegenerative disease with no approved pharmacological treatment options. Top-line results from the trial are expected in early 2028 and will support a potential expansion of the approved indication in Europe and a regulatory approval application in the United States.
A global trial with 41 patients at centres of excellence
CALYX is a global, randomised, double-blind, placebo-controlled clinical trial that has recruited 41 adult male patients with cALD at selected centres of excellence across the United States, Europe, South America and India. The enrolled patients have gadolinium-enhancing brain lesions and are either not recommended for or have declined haematopoietic stem cell transplantation (HSCT). The trial has an adaptive duration, with an initial efficacy readout at 18 months and subsequent assessments at 27 and 36 months, if necessary.
In parallel, it is worth noting that in July 2026, the European Medicines Agency (EMA) issued a positive opinion from its Committee for Medicinal Products for Human Use (CHMP), recommending the granting of a marketing authorisation for NEZGLYAL® under exceptional circumstances for the treatment of boys aged 2 to 12 years with cALD and non-gadolinium-enhancing brain lesions. This recommendation was based on the results of the NEXUS trial and real-world evidence from compassionate use programmes, and represented the first European regulatory recognition for leriglitazone in an indication for which no pharmacological treatment had previously been approved. Final approval by the European Commission is now imminent, and Neuraxpharm, Minoryx’s strategic partner in Europe, is preparing for the product’s commercial launch.
Statements from company executives
Arun Mistry, Chief Medical Officer of Minoryx, highlighted that: “The completion of CALYX recruitment is an important milestone in confirming the disease-modifying potential of leriglitazone, expanding the EU authorisation and securing approval for leriglitazone in the US. With the results readout expected in early 2028, reaching this milestone allows us to begin preliminary planning for regulatory and commercial infrastructure in the US.”
“We are deeply grateful to the patients, families and global advocacy groups for supporting the trial and helping to advance a much-needed new therapeutic option for cALD,” said Sílvia Pascual, Vice President of Clinical Development at Minoryx.
Francisco Jurado, Chief Scientific Officer at Neuraxpharm, stressed that: “The completion of CALYX enrolment marks an important milestone in the development of leriglitazone and in generating clinical evidence in adult patients with cALD. Together with the anticipated European Commission approval later this month, this achievement highlights the continued progress of the clinical development programme and its potential to support future regulatory submissions across different cALD patient populations in Europe.”
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