EMA Validates Marketing Application for Leriglitazone in Cerebral Adrenoleukodystrophy Treatment

In an important step for patients with cerebral adrenoleukodystrophy (cALD), Minoryx Therapeutics, a biotech company specializing in treatments for rare central nervous system disorders, and Neuraxpharm Group, a prominent European pharmaceutical firm, have submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for their lead candidate, leriglitazone (NEZGLYAL®). This submission targets the treatment of both pediatric and adult male patients with cALD, a severe form of X-linked adrenoleukodystrophy (X-ALD).

The application, now validated and under review by the Committee for Medicinal Products for Human Use (CHMP), is supported by data from several studies, notably the NEXUS study. This pivotal, open-label study lasted 96 weeks and focused on the efficacy and safety of daily oral doses of leriglitazone in children with cALD. The study's primary endpoint was achieved, showing that 35% of patients met the criteria for arrested disease, a figure significantly higher than the expected 10% from natural progression. Overall, all 20 evaluated patients remained stable under treatment, and the drug was generally well tolerated with no adverse events causing discontinuation.

Additional backing for the MAA comes from the ADVANCE study, a double-blind, placebo-controlled trial involving 116 participants in Europe and the U.S. This study demonstrated leriglitazone's ability to reduce the incidence and progression of brain lesions in adults. Data from an ongoing compassionate use program for both adult and pediatric patients also reinforces the submission.

Marc Martinell, CEO of Minoryx, expressed optimism about leriglitazone's potential, highlighting that it could become the first pharmacological treatment for this debilitating orphan disease if approved. The drug addresses a significant unmet medical need in treating cALD, a condition lacking effective pharmacological options.

Arun Mistry, Chief Medical Officer at Minoryx, emphasized the importance of the clinical development program and compassionate use data, underscoring leriglitazone’s efficacy across different age groups. He also conveyed the company's commitment to collaborating with the EMA throughout the evaluation process to ensure the therapy reaches those in need.

Neuraxpharm's CEO, Dr. Jörg-Thomas Dierks, hailed the EMA's validation of the MAA as a crucial advancement in providing an essential treatment to patients. Given the severe impact of cALD and the scarcity of effective treatments, he expressed hope for a favorable outcome from the CHMP.

Minoryx is continuing the development of leriglitazone with ongoing trials such as the CALYX trial for adult patients with cALD and the TREE study for pediatric Rett syndrome patients. Both studies are actively recruiting participants.

Leriglitazone, branded as NEZGLYAL®, is a novel oral PPAR gamma agonist designed by Minoryx. It has shown a promising safety and efficacy profile in clinical trials and has secured orphan drug status for X-ALD from both the FDA and EMA. Furthermore, it has received Fast Track and Rare Pediatric Disease designations from the FDA.

Minoryx Therapeutics, founded in 2011, is headquartered in Spain and focuses on developing novel therapies for rare CNS diseases with high unmet medical needs. The company has raised over €120 million in funding, supporting its mission to deliver new treatments for challenging neurological conditions.

Neuraxpharm Group, with a broad presence across Europe and beyond, is dedicated to addressing CNS disorders. The group continues to innovate and expand its product portfolio to meet patient needs worldwide.