Minoryx is focused on the discovery and development of novel treatments for severe, orphan diseases of the central nervous system (CNS) with high unmet medical need.
- Our ScienceOur leading program is a novel, selective PPAR gamma agonist
We have discovered and developed a novel, selective PPAR gamma agonist, leriglitazone, which is currently in clinical development for multiple orphan CNS disorders. The lead indication is X-linked Adrenoleukodystrophy, a severe neurodegenerative disease that exists in two forms: a chronic form, adrenomyeloneuropathy (AMN) and an acute form, cerebral ALD (cALD).
The European Commission (EC) has granted marketing authorization approval under exceptional circumstances for NEZGLYAL (leriglitazone) for the treatment of Cerebral Adrenoleukodystrophy (cALD) in males with Adrenoleukodystrophy (ALD) aged 2 to 12 years with non-Gadolinium (Gd) enhancing lesions (i.e. Gd negative) in brain Magnetic Resonance Imaging (MRI), with a Neurological Functional Score (NFS) of 0 or 1.
Leriglitazone has shown preclinical proof-of-concept in animal models of disease by modulating pathways leading to neuroinflammation, demyelination, mitochondrial dysfunction, oxidative stress, and axonal degeneration. In clinical trials, it has shown clinical benefit in both paediatric cALD patients in the NEXUS clinical trial and adult cALD patients in the ADVANCE trial. Results from NEXUS demonstrate that paediatric cALD patients are clinically and radiologically stable after over 96 weeks of treatment or at a visit prior to Haematopoietic Stem Cell Transplantation (HSCT). Data from ADVANCE showed that leriglitazone reduced cALD progression. More than 170 patients with cALD have received treatment to date. Additionally, a phase 3 study in adult male patients with progressive cALD (CALYX) is currently ongoing..
Leriglitazone offers a strong potential for indication expansion into other CNS diseases. In this regard, a proof of concept study in Friedreich's Ataxia (FRDA) showed clinical benefit in this population and by the end of 2026 we will obtain read-out from our phase 2 trial (TREE) in Rett syindrome.
Our Science Our leading program is a novel, selective PPAR gamma agonistWe have discovered and developed a novel, selective PPAR gamma agonist, leriglitazone, which is currently in clinical development for multiple orphan CNS disorders. The lead indication is X-linked Adrenoleukodystrophy, a severe neurodegenerative disease that exists in two forms: a chronic form, adrenomyeloneuropathy (AMN) and an acute form, cerebral ALD (cALD).
The European Commission (EC) has granted marketing authorization approval under exceptional circumstances for NEZGLYAL (leriglitazone) for the treatment of Cerebral Adrenoleukodystrophy (cALD) in males with Adrenoleukodystrophy (ALD) aged 2 to 12 years with non-Gadolinium (Gd) enhancing lesions (i.e. Gd negative) in brain Magnetic Resonance Imaging (MRI), with a Neurological Functional Score (NFS) of 0 or 1.
Leriglitazone has shown preclinical proof-of-concept in animal models of disease by modulating pathways leading to neuroinflammation, demyelination, mitochondrial dysfunction, oxidative stress, and axonal degeneration. In clinical trials, it has shown clinical benefit in both paediatric cALD patients in the NEXUS clinical trial and adult cALD patients in the ADVANCE trial. Results from NEXUS demonstrate that paediatric cALD patients are clinically and radiologically stable after over 96 weeks of treatment or at a visit prior to Haematopoietic Stem Cell Transplantation (HSCT). Data from ADVANCE showed that leriglitazone reduced cALD progression. More than 170 patients with cALD have received treatment to date. Additionally, a phase 3 study in adult male patients with progressive cALD (CALYX) is currently ongoing..
Leriglitazone offers a strong potential for indication expansion into other CNS diseases. In this regard, a proof of concept study in Friedreich's Ataxia (FRDA) showed clinical benefit in this population and by the end of 2026 we will obtain read-out from our phase 2 trial (TREE) in Rett syindrome.
- NewsSee allEuropean Commission grants marketing authorisation for NEZGLYAL® (leriglitazone), the first pharmacological treatment approved for cerebral Adrenoleukodystrophy (cALD), a rare neurodegenerative diseaseMinoryx and Neuraxpharm complete enrolment in leriglitazone pivotal Phase 3 trial (CALYX) in adult patients with cerebral Adrenoleukodystrophy (cALD)NEZGLYAL® (leriglitazone) receives a positive CHMP opinion for the treatment of cerebral Adrenoleukodystrophy (cALD), a rare neurodegenerative diseaseLeriglitazone Marketing Authorization Application submitted for treatment of cerebral adrenoleukodystrophy has been validated by European Medicines AgencyMinoryx receives a EUR 26.9m grant within the framework of the European IPCEI Med4Cure project for the continued development of leriglitazone in lethal orphan CNS diseasesResults from NEXUS interim analysis published in eClinicalMedicine, part of THE LANCET DiscoveryScienceMinoryx announces dosing of the first patient with leriglitazone in the phase 2a study in Rett syndrome (TREE study)Leriglitazone has met the primary endpoint in NEXUS, the pivotal trial for pediatric patients with cerebral AdrenoleukodystrophyLeriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophy in compassionate-use study published in BrainUpdate on Regulatory Review of NEZGLYAL® (leriglitazone) in the EU