As reported by producer Minoryx; the pharmaceutical leriglitazone, used to treat early stage cerebral ALD (cALD) has just announced its plans to continue onto its Phase 2 Nexus trial without delay from COVID-19. The neurological disorder takes patients lives within years of onset, and the current treatment options are extremely invasive ematopoietic stem cell transplants. This innovative treatment option, already in late stages of development, could potentially offer the first safe and effective option available for cALD patients, and the current approval is enabling speedier trials. They have just begun treatment on their first patient in Barcelona, a ten year old boy.
cALD
Cerebral ALD (cALD) is the acute form of x-linked adrenoleukodystrophy, X-ALD. X-ALD is a peroxisomal disease caused by a genetic mutation on the ABCD1 gene. Because it is linked to the x-chromosome, it primarily affects males, though some females express symptoms later in life. The neurological disease is degenerative, progressively accumulating v chain fatty acids in the tissues. The disease results in severe inflammation in the brain and rapid cerebral demyelination, which will take patients lives within a few years.
The New Treatment Option: Leriglitzaone
Leriglitazone is a novel drug, developed by Minroyx Therapetuics, a biotech company that works in clinical trials for novel therapies targeting orphan CNS diseases that have no treatment options. They work to fill gaps for unmet needs by this community. Their drug is a brain penetrant, available orally. The drug is thought to potentially treat an array of related CNS disorders, including X-ALD, cALD, and Freidreich’s Ataxia. The disease has undergone trials already for adrenomyeloneuropathy (AMN) and Friedreich’s Ataxia.
The studies principal investigator from Harvard Medical School, Dr. Patricia Musolino said to ALA Group, “With newborn screening currently being implemented, it is now possible to diagnose patients with early stage cALD, an orphan neurodegenerative disorder characterized by rapid cerebral demyelination and inflammation of the brain, leading to death within a few years of diagnosis.” With this proper diagnosis, now there needs to be a treatment to meet the needs of those diagnosed.
The Nexus Trial
The NEXUS clinical trial is entering Phase 2 in which 13 patients with cALD will receive the treatment who will have symptoms and the cerebral MRI lesions tracked to evaluate the treatments safety and effectiveness. If the trial passes, the manufacturer has made a PIP agreement which gives the drug two extra years of market exclusivity, after the standard 10 given to all innovative orphan drugs. The pending approval by the European Medicines Agency (EMA), set to be enacted if the NEXUS trial proves successful, will enable the young patients to have a more accessible and quicker registration for the medicine. The first patient has been enrolled in the NEXUS trials now, which is expected to finish in 2021, and is not thought to be delayed by COVID-19 restrictions.
