Update on Regulatory Review of leriglitazone in the EU

A re-examination for cerebral adrenoleukodystrophy (cALD) will be requested following the negative CHMP opinion on X-linked adrenoleukodystrophy (X-ALD)

Barcelona, Spain and Düsseldorf, Germany - 26 January, 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 central nervous system (CNS) disorders, today announce that the EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended not to grant marketing authorization for leriglitazone as a treatment of X-ALD.

Minoryx and Neuraxpharm are seeking a re-examination for conditional approval for patients with cerebral adrenoleukodystrophy (cALD), which is characterized by demyelinating brain lesions which can become rapidly progressive, leading to acute neurological decline and death in three to four years.

Minoryx and Neuraxpharm strongly believe that leriglitazone has a positive benefit / risk balance in patients with cALD. Data from ADVANCE1, in adult male patients with ALD, showed that leriglitazone reduces the progression of lesions and the development of progressive cALD. This is now supported by 24 week data from NEXUS2, in male pediatric patients with early stage cALD, showing a reduction in lesion progression comparable to Hematopoietic Stem Cell Transplantation (HSCT)-based approaches. These findings are being validated through confirmatory long term follow-up from ongoing trials NEXUS and CALYX3, in adult patients with progressive cALD. The companies believe that leriglitazone could be a lifesaving treatment for these patients in a disease without pharmacological treatment options available. This unmet need underscores the critical and pressing nature of this appeal.

Marc Martinell, CEO, Minoryx said: “We are obviously disappointed with the Committee’s decision, but we strongly believe that leriglitazone provides clinically meaningful benefits for patients by reducing the development of progressive cALD and stabilizing lesion progression. Consequently, we are going to request re-examination of leriglitazone for conditional marketing authorisation for treatment of patients with cALD. Furthermore, we continue to generate additional evidence from the two ongoing confirmatory trials (CALYX and NEXUS) and we remain committed to bringing this new therapeutic option to the broader ALD patient community. We are enormously grateful for their continued support.”

Dr. Jörg-Thomas Dierks, CEO, Neuraxpharm said: “Leriglitazone has the potential to transform the lives of those suffering from cALD. In partnership with Minoryx. we remain committed to obtaining approval for leriglitazone as we strongly believe that these patients and their families deserve a broadly accessible and effective treatment option. This is a cruel and fast-moving disease which is devastating for patients and their families. In such an acute and fatal disease, with no other treatment options, this therapy could be critically important for patients.”

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