Minoryx Therapeutics, a CataloniaBio & HealthTech member, has started a phase II clinical trial to evaluate the safety and efficacy of leriglitazone drug in pediatric patients with early-stage cerebral X-ALD (cALD). The first patient, a ten year-old-boy, was dosed at the Sant Joan de Déu Barcelona Children's Hospital.

The first results of the study, called NEXUS, are expected in 2021. In addition the European Medicines Agency (EMA) approved the proposed Pediatric Investigational Plan for leriglitazone for the treatment of X-ALD, allowing the company to file for an MAA in Europe based on the NEXUS trial, subject to positive results.

"cALD is a orphan neurodegenerative disorder characterized by rapid cerebral demyelination and inflammation of the brain, leading to death within a few years of diagnosis. There is an urgent need for alternative and less invasive treatments" says Dr Patricia Musolino, principal investigator of the study and Assistant Professor in Neurology at the Harvard Medical School.

Despite the current Covid-19 pandemic, the timelines of the other ongoing trials with leriglitazone have not been impacted (ADVANCE phase II/III in patients with adrenomyeloneuropathy and FRAMES phase II in patients with Friedreich’s Ataxia).

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Photo: Marc Martinell, co-founder and CEO of Minoryx, at Tecnocampus Mataró (Barcelona) - © Minoryx Therapeutics

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