Minoryx has announced enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in the US Phase III clinical trial, CALYX. As part of CALYX, Minoryx is conducting an extensive pre-screening MRI-based campaign aimed at identifying eligible adult X-linked Adrenoleukodystrophy (X-ALD) patients with cALD.
In June 2023, the US Food and Drug Administration (FDA) approved CALYX’s trial of leriglitazone to treat adult male X-ALD patients with cALD. Now, the first patients have been enrolled in CALYX Phase III. This additional small-scale trial has been agreed with the FDA prior to US approval for X-ALD.
“CALYX has been designed as a Phase III trial to confirm the disease modifying potential of leriglitazone that we detected in the previous ADVANCE and NEXUS trials,” said Arun Mistry, CMO, Minoryx. “The initiation of enrollment will enable Minoryx to take the final steps towards US approval of leriglitazone for treatment of X-ALD patients.”
“Minoryx has begun an extensive pre-screening program aimed at identifying additional eligible patients. This will push the Phase III CALYX trial forward and bring leriglitazone to patients as quickly as possible,” said Sílvia Pascual, VP Clinical Development, Minoryx. “In patients with cALD, appearance of gadolinium-enhancing lesions typically precedes a phase with pronounced lesion growth and rapid clinical deterioration. Minoryx therefore wants to encourage adult X-ALD patients to participate in the pre-screening program to have cALD diagnosed as early as possible.”
US Phase III clinical trial, CALYX
CALYX is a double-blind, randomised, placebo-controlled Phase III clinical trial that will recruit 40 adult male patients with cALD across selected centres of excellence in the US and South America. The primary endpoint measuring survival, and secondary endpoints include the Loes score, major functional disabilities, activities of daily living and major neurocognitive impairment. Centres in the US are already enrolling patients and results are anticipated by early 2026.
“The X-ALD patient community is very excited to have a study commencing in adult cALD where few options are currently available,” said Kathleen O’Sullivan-Fortin, Co-Founder, ALD Connect. “This study will help increase the understanding of cALD and raise disease awareness among both patients and physicians.”
Dual US and EU approval approach
In Europe, review of the Marketing Authorization Application (MAA) is currently ongoing at EMA. Neuraxpharm, a CNS specialist company and Minoryx’s strategic partner in Europe, is preparing for commercial launch of leriglitazone.
Minoryx is having to take two approaches for approval of leriglitazone, with the MAA currently ongoing with the EMA on the basis of previous trials, and Minoryx’s partnership with Neuraxpharm preparing for European commercial launch. This dual-approach for US and European approval could see X-ALD patient access at different times on both sides of the Atlantic.