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 which exists in two forms: a chronic form, adrenomyeloneuropathy (AMN) and an acute form, cerebral ALD (cALD). Leriglitazone has demonstrated strong preclinical proof-of-concept in relevant animal models of disease and has successfully completed phase 1 clinical trials. Leriglitazone is currently in a registration enabling study in AMN in EU and US, a registration enabling study in cALD in EU and a proof of concept study in Friedreich Ataxia.
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 which exists in two forms: a chronic form, adrenomyeloneuropathy (AMN) and an acute form, cerebral ALD (cALD). Leriglitazone has demonstrated strong preclinical proof-of-concept in relevant animal models of disease and has successfully completed phase 1 clinical trials. Leriglitazone is currently in a registration enabling study in AMN in EU and US, a registration enabling study in cALD in EU and a proof of concept study in Friedreich Ataxia.
- NewsSee allMinoryx 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 trialMinoryx Therapeutics receives US FDA fast-track designation for leriglitazone in the treatment of X-ALDMinoryx Therapeutics receives Orphan Drug Designation from the European Commission for leriglitazone in the treatment of Friedreich’s AtaxiaMinoryx Therapeutics receives FDA Orphan Drug Designation for leriglitazone in Friedreich’s Ataxia
