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 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 journalMinoryx Phase 2/3 ‘ADVANCE’ study results to be presented at the 2021 American Neurological Association (AAN) Annual MeetingMinoryx presents topline results from Phase 2/3 “ADVANCE” study demonstrating significant clinical benefit of leriglitazone in adrenomyeloneuropathy (AMN)Minoryx’s clinical candidate leriglitazone shows clinical benefit in a proof of concept Phase 2 study in Friedreich´s ataxiaMinoryx’s preclinical data for leriglitazone in Friedreich’s Ataxia published in Neurobiology of Disease JournalEuropean Investment Bank provides Minoryx with up to €25 million to support development of breakthrough therapies in orphan neurodegenerative diseases