Minoryx, clutching data from failed trial, knocked back by EMA but vows to seek rerun of review

The European Medicines Agency (EMA) has swatted away Minoryx Therapeutics’ attempt to bring a rare disease drug candidate to market on the strength of a clinical trial that missed its primary endpoint. But, undeterred, Minoryx and partner Neuraxpharm are seeking a re-examination of the regulatory rejection.

Barcelona-based Minoryx compared the molecule, the PPAR gamma agonist leriglitazone, to placebo in people with adrenomyeloneuropathy, a form of X-linked adrenoleukodystrophy (X-ALD). The study found the drug candidate was no better than placebo at improving the distance covered during the six-minute walk test at week 96, causing the phase 2/3 clinical trial to miss its primary endpoint.

Minoryx focused on secondary endpoints when it first broke news of the failure, and continued to put the data on cerebral lesions and myelopathy symptoms front and center as it raised 51 million euros ($55 million), used the trial to seek EMA approval and allied with Neuraxpharm to commercialize leriglitazone in Europe.

The positive take on the data ran into regulatory reality Friday, when the EMA’s Committee for Medicinal Products for Human Use recommended against granting conditional approval to leriglitazone in patients aged two years and older with cerebral adrenoleukodystrophy (cALD), a severe form of X-ALD.

In response, Minoryx and Neuraxpharm reiterated their belief in the evidence and set out their plans to seek reexamination of the decision in cALD. The companies “remain committed” to bringing leriglitazone “to the broader ALD patient community,” Marc Martinell, CEO of Minoryx, said in a statement.

Minoryx is already working to generate more data in cALD, kicking off a 40-subject phase 3 trial last year as part of its push to clear the bar for authorization set by the FDA, but results are now expected in early 2026. As such, near-term hopes of getting leriglitazone to market rest on the EMA re-examination, a process that enabled Mirati Therapeutics to overturn a decision last year.