Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces that the first patient in the TREE study has been dosed with leriglitazone. TREE is a phase 2a clinical study assessing the safety and efficacy of lead candidate leriglitazone, a novel, brain-penetrant and selective PPAR gamma agonist, to treat patients with Rett syndrome.
The TREE study will enrol 24 female patients aged 5-12 y who will be dosed with leriglitazone or placebo for 36 weeks. The study will be carried out at the Neurometabolic Disorders Unit of the Hospital Sant Joan de Déu, Barcelona, Spain under the direction of Dr. Ángeles García Cazorla, Head of Research in Neurology and Head of Neurometabolic Unit.
In preclinical models, treatment with leriglitazone resulted in recovery of the bioenergetic alterations in human Rett fibroblasts. In a Rett mouse model, leriglitazone exerted an anti-neuroinflammatory effect, resulting in the amelioration of general condition and exploratory activity. Based on these preclinical findings, the TREE study is designed to show improved cognition with stabilization/gain of communication skills, improved behaviour and delaying neuromuscular worsening resulting in improved motor skills.
Following the positive results in the study NEXUS in boys with cerebral adrenoluekodystrophy (cALD) we will pursue additional orphan CNS indications with high unmet medical need,”
Marc Martinell, CEO, 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."
“We are excited to have initiated the TREE study. Leriglitazone has a mode of action relevant to the pathways associated with Rett syndrome,” said Arun Mistry CMO, Minoryx. “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.”
“The preclinical data for leriglitazone in Rett models is very encouraging as leriglitazone demonstrates effect on neuroinflammation and mitochondrial function which are hallmarks of Rett syndrome.” said Dr. Ángeles García Cazorla, Head of Research in Neurology and Head of the Neurometabolic Disorders Unit, Hospital Sant Joan de Déu. “Patients with Rett syndrome have complex clinical pictures where a new treatment that could provide improvements in cognition, behaviour and communication skills would help not just the patient to have an improved quality of life but also the family too.
The results from the TREE study are expected during the first half of 2026 when all patients have concluded 36 weeks of treatment and 4 weeks of follow-up.
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