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  • Leriglitazone
    Clinical phase
    Indication
    Preclinical
    Phase 1
    Proof of Concept
    Registration Enabling
    NDA/MAA
    X-ALD+
    Friedreich's Ataxia+
    Other CNS Diseases+
    AMN
    cALD

    Minoryx’s lead program is a selective, differentiated PPAR gamma agonist (leriglitazone) being developed for the treatment of X-linked Adrenoleukodystrophy (X-ALD); it has the potential to treat both the severe cerebral form of ALD (cALD) and the chronic form, adrenomyeloneuropathy (AMN), as well as other orphan CNS diseases, such as Friedreich's Ataxia (FRDA).

    Leriglitazone was evaluated in a double-blind, placebo-controlled phase 2/3 clinical study (ADVANCE) in adult AMN patients in EU and US, overall showing a significant effect on reducing progression of cerebral lesions and progression of myelopathy symptoms. An open-label extension is ongoing to generate long term safety and efficacy data. The marketing authorization application (MAA) for adult male X-ALD patients is under review by the EMA, and Minoryx is currently in discussions with the FDA to define the next steps for its US approval path. is under discussions with regulatory authorities on the approval path for AMN population. A separate study in pediatric cALD patients is currently ongoing in EU (NEXUS) to further assess the impact of leriglitazone on the progression of cerebral lesions.

    Leriglitazone is also being investigated for other orphan CNS diseases, and a proof of concept study in patients with FRDA (FRAMES) was completed showing clinical benefit in this population. The design of the confirmatory phase 3 study in this population is now under discussion with regulatory authorities.

    More info
    Focus
    Mechanism of Action in X-ALD
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    Focus
    Mechanism of Action in FRDA
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  • 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