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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 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.

    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 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.

    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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    The Lancet Neurology publishes results from Minoryx Therapeutics Phase 2/3 ADVANCE clinical trial of leriglitazone in X-linked Adrenoleukodystrophy
    January 20th, 2023
    Leriglitazone’s clinical proof of concept data in Friedreich’s Ataxia published in Neurology Genetics
    November 29th, 2022
    Minoryx and Neuraxpharm announce a strategic alliance to provide a new therapy for rare CNS disease patients in Europe
    November 10th, 2022
    Minoryx‘s Marketing Authorization Application for its lead candidate leriglitazone validated by EMA for orphan indication X-linked Adrenoleukodystrophy (X-ALD)
    September 14th, 2022
    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
  • Contact
    Headquarters
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    Av. Ernest Lluch 32
    08302 Mataró
    (Barcelona) Spain
    info@minoryx.com 0034935441466+34 93 544 14 66
    Belgian R&D Site
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    Ave. Jean Mermoz 32
    6041 Gosselies
    (Charleroi) Belgium
    info@minoryx.com 0032023420803+32 (0)2 342 08 03
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    by eMascaró