FDA approves Phase III cerebral Adrenoleukodystrophy trial

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The US Food and Drug Administration (FDA) has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).

Patient recruitment is expected to start by the end of Q2 2023 with results anticipated by late 2025.

Marc Martinell, CEO of Minoryx, said: “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 option for patients suffering from this devastating orphan disease with a major unmet medical need.”

CALYX will enrol 40 adult male X-ALD patients with progressive cALD defined by the presence of gadolinium enhancing brain lesions. Adult cALD patients with gadolinium enhancing lesions have a rapid cognitive deterioration and survival of three to four 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 showed that after 24 weeks of treatment all patients were clinically stable and, radiologically, demonstrated disease arrest or lesion growth stabilisation. Radiological changes were similar to those attained with Hematopoietic Stem Cell Transplant (HSCT) or ex-vivo gene therapy.

“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. “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.”

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