US trial with leriglitazone to commence in mid-2023 providing an FDA-approved route to US market whilst EMA evaluates European Marketing Authorization Application (MAA)
Mataró, Barcelona, Spain, May 31, 2023 - Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the US Food and Drug Administration (FDA) approval of its Phase 3 clinical trial (CALYX) of lead candidate leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).
The CALYX protocol has received both FDA and central IRB approval. Significant preparations for the trial commencement have been completed and patient recruitment is expected to start by the end of Q2 2023 with results anticipated by late 2025.
“Minoryx is focused on bringing therapeutic options to X-ALD patients and we now have an agreed route to the US market with a Phase 3 trial designed to confirm the disease modifying potential of leriglitazone.” said Marc Martinell, CEO, Minoryx and added: “CALYX will be funded from the Series C financing together with proceeds from our European strategic collaboration with Neuraxpharm. We look forward to initiating this trial which could provide an important therapeutic optionfor patients suffering from this devastating orphan disease with a major unmet medical need.”
CALYX will enroll 40 adult male X-ALD patients with progressive cALD defined by the presence of gadolinium enhancing brain lesions. A pre-screening campaign will be initiated across participating sites in order to identify adult patients with brain lesions. The trial is placebo controlled with 1:1 randomization, and the primary endpoint is “time to death” or “bedridden with permanent ventilatory support”. The trial has an adaptive duration with an initial pre-specified efficacy read at 18 months and if needed subsequent efficacy assessments at 27 and 36 months, respectively with the option to complete the study at any of the three time points once statistical significance is reached. Adult cALD patients with gadolinium enhancing lesions have a rapid cognitive deterioration and survival of 3-4 years.
The design of CALYX builds upon the results from the ADVANCE and NEXUS trials and from the on-going compassionate use program where leriglitazone has demonstrated the ability to halt lesion progression. The NEXUS interim analysis (recently disclosed at AAN1 conference) showed that after 24 weeks of treatment all patients were clinically stable and, radiologically, demonstrated disease arrest or lesion growth stabilization. Radiological changes were similar to those attained with Hematopoietic Stem Cell Transplant (HSCT) or ex-vivo gene therapy, hence it is expected that leriglitazone could provide a comparable clinical benefit to cALD patients.
CALYX will be conducted across selected centers of excellence in the US and South America and Minoryx has appointed Dr Ali Fatemi at the Kennedy Krieger Institute in Baltimore, US as the Global Principal Investigator for CALYX.
“There is a major unmet medical need for a treatment that can halt or slow the disease progression in cALD as the majority of patients, and particularly adults, do not have any treatment option” said Dr Ali Fatemi, Global Principal Investigator of CALYX, and added: “Leriglitazone results are so far very encouraging and with CALYX the objective is to confirm the clinical benefit of leriglitazone and provide cALD patients with a treatment that can arrest or slow-down their neurological decline and prolong their lives”.