Minoryx doses first patient with leriglitazone in Rett syndrome study

Phase 2a TREE study assesses safety and efficacy of leriglitazone

Minoryx Therapeutics has dosed the first patient in its phase 2a TREE study, which evaluates the safety and efficacy of leriglitazone in treating Rett syndrome. The trial involves 24 female patients aged 5-12 years, receiving leriglitazone or placebo for 36 weeks.

Conducted at the Neurometabolic Disorders Unit of Hospital Sant Joan de Déu, the study aims to show improved cognition, stabilised communication skills, better behaviour and delayed neuromuscular worsening. Dr Ángeles García Cazorla leads the study.

“Following the positive results in the study NEXUS in boys with cerebral adrenoleukodystrophy (cALD), we will pursue additional orphan CNS indications with high unmet medical need,” said Marc Martinell, CEO of Minoryx. “Rett syndrome is one such indication, and we look forward to collaborating with the team of excellent physicians from Hospital Sant Joan de Déu in Barcelona.”

Preclinical studies showed leriglitazone’s recovery effects on bioenergetic alterations in human Rett fibroblasts and its anti-neuroinflammatory effect in Rett mouse models. Based on these findings, the TREE study will assess improvements in cognition, behaviour, communication skills and motor skills.

Minoryx’s Chief Medical Officer, Arun Mistry, said: “We are excited to have initiated the TREE study. Leriglitazone has a mode of action relevant to the pathways associated with Rett syndrome. Thus far we have clinical safety and efficacy data in male paediatric patients, adult men and adult women from studies in X-ALD and Friedreich’s ataxia and the TREE study expands to paediatric female patients with Rett syndrome.”

Results from the TREE study are expected in the first half of 2026 after all patients complete 36 weeks of treatment and 4 weeks of follow-up.