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 EU and US showing significant reduction of cerebral lesion progression and myelopathy symptoms. and an open-label extension phase is still ongoing. 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 EU and US showing significant reduction of cerebral lesion progression and myelopathy symptoms. and an open-label extension phase is still ongoing. 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 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 diseasesMinoryx Therapeutics and Sperogenix Therapeutics enter into an exclusive license agreement to develop and commercialize leriglitazone in mainland China, Hong Kong and MacauMinoryx Therapeutics doses first patient with leriglitazone in registration-enabling cALD NEXUS trial