Minoryx Therapeutics Doses First Patient in Study for Rett Syndrome
Minoryx Therapeutics has taken a significant step in its mission to address rare neurological diseases by dosing the first patient in its Phase 2a TREE study, which investigates the safety and efficacy of leriglitazone for treating Rett syndrome.
This milestone marks the beginning of a rigorous clinical evaluation aimed at improving the lives of young patients suffering from this severe neurodevelopmental disorder.
The TREE study is being conducted at the Neurometabolic Disorders Unit of Hospital Sant Joan de Déu in Barcelona, under the leadership of Dr Ángeles García Cazorla. The trial involves 24 female patients between the ages of 5 and 12, who will receive either leriglitazone or a placebo over a period of 36 weeks.
Researchers will assess whether the drug can improve cognition, stabilise communication skills, enhance behaviour, and slow neuromuscular deterioration – all critical challenges faced by those living with Rett syndrome.
Minoryx has been building momentum with its innovative research in central nervous system (CNS) disorders. Following promising results in the NEXUS study, which evaluated leriglitazone in boys with cerebral adrenoleukodystrophy (cALD), the company is now expanding its focus to other orphan CNS diseases with high unmet medical need.
The CEO of Minoryx expressed enthusiasm about this latest development, stating that Rett syndrome is a devastating condition, and they are committed to exploring how leriglitazone can provide meaningful benefits for these young patients.
They went on to add that they look forward to collaborating with the excellent physicians at Hospital Sant Joan de Déu to further their understanding of the drug’s impact.
Leriglitazone’s potential is backed by a strong foundation of preclinical research. Studies have demonstrated its ability to counteract bioenergetic alterations in human Rett fibroblasts while also exhibiting anti-neuroinflammatory effects in Rett mouse models.
These findings suggest that leriglitazone may play a crucial role in addressing some of the core disease mechanisms of Rett syndrome.
Minoryx’s Chief Medical Officer echoed the company’s optimism, highlighting that the TREE study represents an important extension of their clinical work. They expressed that they are excited to initiate this study in female paediatric patients with Rett syndrome.
Furthermore, they added that Leriglitazone has already demonstrated clinical safety and efficacy in male paediatric patients, as well as in adult men and women in studies for X-ALD and Friedreich’s ataxia. They believe its mode of action aligns with key pathways involved in Rett syndrome, and they look forward to seeing how it performs in this population.
The TREE study is expected to run until the first half of 2026, with final results available after all patients complete the full 36-week treatment period followed by a 4-week follow-up.
Conclusion
With the launch of the TREE study, Minoryx Therapeutics is forging ahead in its mission to tackle Rett syndrome and other rare CNS disorders. Building on its past successes in related conditions, the company is hopeful that leriglitazone will offer a meaningful breakthrough for young patients who currently have limited treatment options.
As the study progresses, the global medical community will be watching closely, eager to see whether this promising therapy can help transform the standard of care for Rett syndrome.
News Credits: Minoryx doses first patient with leriglitazone in Rett syndrome study
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