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Exciting results in drug trial for rare disease Friedreich’s ataxia

A drug company has announced positive results in the first global trial of a drug for a rare neuromuscular disease Friedreich’s ataxia (FA) which included patients from the UCLH Ataxia Centre for the UK.

Reata Pharmaceuticals said FA patients who took omaveloxolone (Omav) for 48 weeks in a phase 2 trial named MOXIe had a statistically significant improvement in their symptoms compared to those receiving a placebo.

The company said the drug has the potential to be the first approved treatment for FA. It said that, subject to discussions with regulators, it now plans to apply for approval to market the drug internationally.

Regulators may first require researchers to carry out one further stage of the trial to test the drug.

FA is an inherited, debilitating, neurodegenerative disorder that is typically diagnosed during adolescence and can ultimately lead to premature death. Patients with FA experience progressive loss of coordination, muscle weakness, and fatigue, which commonly progresses to motor incapacitation and wheelchair reliance.

Symptoms generally occur in children, with patients requiring a wheelchair by their teens or early 20s, and affects around 22,000 people globally. Currently there are no approved treatments for the condition.

The MOXIe trial recruited 103 patients with FA at 11 study sites in the US, Europe and Australia, and studied the impact of Omav on patients’ scores on the modified Friedreich’s Ataxia Rating Scale (mFARS) relative to placebo after 48 weeks of treatment.

The UCLH patients included in the trial were seen at the NIHR UCLH Clinical Research Facility at the Leonard Wolfson Experimental Neurology Centre.

Scores on the mFARS scale – which measures FA disease progression – were the primary outcome researchers looked at. Full trial results – including secondary outcomes – will be released by Reata soon.

Prof Paola Giunti, head of the London Ataxia Centre at UCLH, said: “We are excited by these promising results of the trial and hope this will lead to the development of a much-needed approved treatment for Friedreich’s ataxia. I am so grateful to all the people with FA who have taken part in this trial. We are also proud to have contributed to the preclinical study conducted in my laboratory showing the beneficial effects of omav on our cell models.”

President and Chief Executive Officer of Reata Warren Huff said the trial results “bring us closer to our goal of providing an urgently needed therapy to patients with FA.”

Sue Millman, CEO of Ataxia UK, said: "People with Friedreich’s ataxia have been waiting many years for the possibility of a treatment whilst their condition deteriorates. Ataxia UK is delighted that there is now something on the horizon which gives hope that a treatment may soon be available."