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 for leriglitazone is X-linked Adrenoleukodystrophy, a devastating neurodegenerative disease that exists in two forms: a chronic form, adrenomyeloneuropathy (AMN) and an acute form, cerebral ALD (cALD). Leriglitazone demonstrated robust preclinical proof-of-concept in relevant animal models of disease and successfully completed phase 1 clinical trials. Leriglitazone completed a phase 2/3 study in AMN in the EU and US showing a significant reduction of cerebral lesion progression and myelopathy symptoms and an open-label extension phase is still ongoing. The marketing authorization application (MAA) for adult male X-ALD patients is currently under review by the EMA. Additionally, a separate study in pediatric cALD is currently ongoing in EU. 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, hence, a phase 3 study in FRDA is under preparation.
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 for leriglitazone is X-linked Adrenoleukodystrophy, a devastating neurodegenerative disease that exists in two forms: a chronic form, adrenomyeloneuropathy (AMN) and an acute form, cerebral ALD (cALD). Leriglitazone demonstrated robust preclinical proof-of-concept in relevant animal models of disease and successfully completed phase 1 clinical trials. Leriglitazone completed a phase 2/3 study in AMN in the EU and US showing a significant reduction of cerebral lesion progression and myelopathy symptoms and an open-label extension phase is still ongoing. The marketing authorization application (MAA) for adult male X-ALD patients is currently under review by the EMA. Additionally, a separate study in pediatric cALD is currently ongoing in EU. 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, hence, a phase 3 study in FRDA is under preparation.
- NewsSee allMinoryx announces enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in US Phase 3 clinical trial, CALYXMinoryx gains FDA approval to initiate a Phase 3 clinical trial in patients with cerebral AdrenoleukodystrophyMinoryx presents positive interim results from NEXUS registration study for leriglitazone targeting pediatric ALD patients with cerebral adrenoleukodystrophy (cALD)The Lancet Neurology publishes results from Minoryx Therapeutics Phase 2/3 ADVANCE clinical trial of leriglitazone in X-linked AdrenoleukodystrophyLeriglitazone’s clinical proof of concept data in Friedreich’s Ataxia published in Neurology GeneticsMinoryx and Neuraxpharm announce a strategic alliance to provide a new therapy for rare CNS disease patients in EuropeMinoryx‘s Marketing Authorization Application for its lead candidate leriglitazone validated by EMA for orphan indication X-linked Adrenoleukodystrophy (X-ALD)Minoryx raises €51 million to support Marketing Authorization Application and launch preparations for X-linked Adrenoleukodystrophy (X-ALD) therapyAdditional data from Minoryx’s Phase 2/3 ADVANCE clinical trial presented at American Neurological Association (ANA) 2021Minoryx publishes mechanism of action of leriglitazone in X-ALD in Science Translational Medicine journal
