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Minoryx Secures FDA Approval for Phase 3 Clinical Trial Targeting Cerebral Adrenoleukodystrophy

Friday, June 02, 2023

Minoryx Therapeutics Receives FDA Approval for Phase 3 Clinical Trial of Leriglitazone in Treating Cerebral Adrenoleukodystrophy (cALD)

Minoryx Therapeutics, a biotech company focused on developing therapies for central nervous system disorders, has announced that the US Food and Drug Administration (FDA) has granted approval for its Phase 3 clinical trial of leriglitazone. The trial, called CALYX, aims to evaluate the efficacy of leriglitazone in treating adult male patients with X-linked Adrenoleukodystrophy (X-ALD) who have cerebral Adrenoleukodystrophy (cALD).

The CALYX trial protocol has received both FDA and central institutional review board (IRB) approval. Patient recruitment is expected to begin by the end of the second quarter of 2023, with results anticipated by late 2025.

Minoryx CEO, Marc Martinell, expressed the company's dedication to providing therapeutic options for X-ALD patients and highlighted the significance of the Phase 3 trial in confirming the disease-modifying potential of leriglitazone. The trial will be funded through Minoryx's Series C financing, as well as proceeds from their strategic collaboration with Neuraxpharm in Europe.

The CALYX trial aims to enroll 40 adult male X-ALD patients with progressive cALD who have gadolinium-enhancing brain lesions. Pre-screening campaigns will be conducted at participating sites to identify eligible adult patients. The trial will utilize a placebo-controlled design with a 1:1 randomization. The primary endpoint will be either "time to death" or "bedridden with permanent ventilatory support." The trial will have an adaptive duration, with an initial efficacy read at 18 months and subsequent efficacy assessments at 27 and 36 months if necessary. The trial can be completed at any of these time points once statistical significance is achieved. Patients with cALD and gadolinium-enhancing lesions typically experience rapid cognitive deterioration and have a survival rate of 3 to 4 years.

The CALYX trial builds upon the positive results from the ADVANCE and NEXUS trials, as well as the ongoing compassionate use program, where leriglitazone has shown the ability to halt lesion progression. Leriglitazone's radiological changes have been comparable to those achieved through hematopoietic stem cell transplantation (HSCT) or ex-vivo gene therapy, indicating its potential clinical benefits for cALD patients.

CALYX will be conducted at selected centers in the United States and South America, with Dr. Ali Fatemi from the Kennedy Krieger Institute in Baltimore appointed as the Global Principal Investigator for the trial. Dr. Fatemi emphasized the urgent need for a treatment option that can slow or halt the progression of cALD, as the majority of patients, particularly adults, currently have no available treatment.

The FDA approval for the Phase 3 clinical trial represents an important milestone in the development of leriglitazone as a potential therapeutic option for patients with cerebral Adrenoleukodystrophy.

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