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.
- 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 X-ALD (ADVANCE) in the EU and US showing a significant reduction of cerebral lesion progression and a reduction of incidences of cerebral lesions and myelopathy symptoms. Additionally, a separate study in paediatric cALD (NEXUS) is currently ongoing in EU and after 24 weeks of treatement, all evaluable patients in NEXUS were clinically stable and radiologically demonstrated disease arrest or lesion growth stabilization. Finnaly, a phase 3 study in adult male patients with progressive cALD (CALYX) is currently recruiting in the US. The marketing authorization application (MAA) for adult male X-ALD patients is currently under review by the EMA.
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.
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 X-ALD (ADVANCE) in the EU and US showing a significant reduction of cerebral lesion progression and a reduction of incidences of cerebral lesions and myelopathy symptoms. Additionally, a separate study in paediatric cALD (NEXUS) is currently ongoing in EU and after 24 weeks of treatement, all evaluable patients in NEXUS were clinically stable and radiologically demonstrated disease arrest or lesion growth stabilization. Finnaly, a phase 3 study in adult male patients with progressive cALD (CALYX) is currently recruiting in the US. The marketing authorization application (MAA) for adult male X-ALD patients is currently under review by the EMA.
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.
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