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Minoryx Therapeutics is a private VC-backed company developing innovative therapies for the treatment of orphan CNS (central nervous system) diseases.

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, and currently, Minoryx 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.

The company was founded in 2011, raised more than €50m so far and has operations in Spain and Belgium.


Monsieur Marc Martinell, CEO Minoryx Therapeutics
Madame Marie Pussemier, Chargée RH & Administration
Phone : +32 787 00 162