Leriglitazone halts progression of fatal neurodegenerative disorder

Brain scan

Leriglitazone has been shown to halt disease progression in adult patients with early cerebral adrenoleukodystrophy (cALD) in a compassionate-use study.

cALD is a fatal neurodegenerative disorder that is characterised by growing, demyelinating brain lesions, with death occurring typically within three to four years of onset.

The study was led by Fanny Mochel, Professor at the Hôspital Universitaire La Pitié-Salpêtrière (Paris, France), as part of an early-access programme through the French national drug agency (ANSM).

Thirteen adult male patients with progressive cALD were treated, all of whom were either awaiting hematopoietic stem cell transplantation (HSCT) – the only treatment option – or ineligible for HSCT.

Among the 13 patients treated with leriglitazone, the disease stabilised clinically and in radiological tests in 10 patients through up to two years of follow-up.

Five patients that had originally presented with gadolinium enhancement (active neuroinflammation) lesions in the corticospinal tract that usually lead to poor HSCT outcomes all became gadolinium negative. Importantly, the lesion load was reduced in four of these patients.

Moreover, plasma neurofilament light chain levels (a biomarker for neurodegeneration) stabilised in all 10 patients and correlated with lesion load.

The two patients who continued to deteriorate were both over 60 years of age with prominent cognitive impairment at baseline, while one patient died from Covid-19 a few months after initiating treatment.

Minoryx to launch Phase III trial

“This cohort study shows that leriglitazone can halt neuroinflammation and disease progression in adult patients with cALD at early disease stages, as evidenced by clinical, radiological, and biological stability for up to two years of treatment,” said Professor Mochel. “All five patients who initiated treatment while on the waiting list for stem cell transplant have now been taken off, on the basis of the positive results observed with leriglitazone.”

In consultation with the US Food and Drug Administration, Minoryx has launched a Phase III clinical trial (CALYX5) in patients with adult progressive cALD that have a profile similar to those treated at Hôspital Universitaire La Pitié-Salpêtrière.

“The results are very encouraging and support the efficacy of leriglitazone that we have observed in our clinical trials, ADVANCE3 and NEXUS4,” said Marc Martinell, CEO, Minoryx. “In these studies in adult and paediatric patients with cALD, leriglitazone is able to reduce further lesion development and stabilise patients clinically and radiologically.”

Diana Spencer, Senior Digital Content Editor, DDW

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