Minoryx announces enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in US Phase 3 clinical trial, CALYX

As part of CALYX, Minoryx is conducting an extensive pre-screening MRI-based campaign aimed at identifying eligible adult X-ALD patients with cerebral Adrenoleukodystrophy (cALD)

Mataró, Barcelona, Spain, November 16, 2023 - Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the first patients have been enrolled in its US Phase 3 clinical trial (CALYX) of lead candidate leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD), an orphan indication with no alternative therapeutic options.

“CALYX has been designed as a Phase 3 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 3 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.”

CALYX is a double-blind randomized placebo-controlled phase 3 clinical trial that will recruit 40 adult male patients with cALD across selected centers 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. Centers 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.”

In Europe, review of the Marketing Authorization Application (MAA) is currently ongoing at EMA. Neuraxpharm, a leading CNS specialist company and Minoryx’s strategic partner in Europe, is preparing for commercial launch of leriglitazone.

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