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    Bringing new hope for people suffering from orphan CNS diseases

    At Minoryx, we are committed to making innovative treatments available for patients suffering from life threatening orphan diseases.

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    Minoryx
  • Orphan CNS diseases
    Leveraging solutions for patients 

    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.

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  • Our Science 
    Our 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.

    Read more
    Our Science 
    Our 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.

    Read more
  • Our programs
    Delivering innovative treatments
    Our wholly-owned lead asset, leriglitazone, is currently in clinical development for the treatment of a range of orphan CNS disorders with high unmet medical need.
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  • News
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    Minoryx raises €51 million to support Marketing Authorization Application and launch preparations for X-linked Adrenoleukodystrophy (X-ALD) therapy
    May 31st, 2022
    Additional data from Minoryx’s Phase 2/3 ADVANCE clinical trial presented at American Neurological Association (ANA) 2021
    November 3rd, 2021
    Minoryx publishes mechanism of action of leriglitazone in X-ALD in Science Translational Medicine journal
    June 3rd, 2021
    Minoryx Phase 2/3 ‘ADVANCE’ study results to be presented at the 2021 American Neurological Association (AAN) Annual Meeting
    April 12th, 2021
    Minoryx presents topline results from Phase 2/3 “ADVANCE” study demonstrating significant clinical benefit of leriglitazone in adrenomyeloneuropathy (AMN)
    January 26th, 2021
    Minoryx’s clinical candidate leriglitazone shows clinical benefit in a proof of concept Phase 2 study in Friedreich´s ataxia
    December 15th, 2020
    Minoryx’s preclinical data for leriglitazone in Friedreich’s Ataxia published in Neurobiology of Disease Journal
    November 16th, 2020
    European Investment Bank provides Minoryx with up to €25 million to support development of breakthrough therapies in orphan neurodegenerative diseases
    October 30th, 2020
    Minoryx Therapeutics and Sperogenix Therapeutics enter into an exclusive license agreement to develop and commercialize leriglitazone in mainland China, Hong Kong and Macau
    September 23rd, 2020
    Minoryx Therapeutics doses first patient with leriglitazone in registration-enabling cALD NEXUS trial
    May 12th, 2020
  • Contact
    Headquarters
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    Av. Ernest Lluch 32 - TCM3
    08302 Mataró
    (Barcelona) Spain
    info@minoryx.com 0034935441466+34 93 544 14 66
    Belgian R&D Site
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    Rue Auguste Piccard 48, I - Tech Incubator
    6041 Gosselies
    (Charleroi) Belgium
    info@minoryx.com 003278700162+32 787 00 162
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    by eMascaró