IONIS-HTTRx can reduce the level of the harmful huntingtin protein in the patients’nervous system. Credit: UCL.

UK-based University College London (UCL) scientists have reported positive results in a Phase l/lla trial of IONIS-HTTRx to treat Huntington’s disease.

A total of 46 patients with early Huntington’s disease were enrolled for the trial, which is led by UCL Institute of Neurology professor and IONIS-HTTRx global chief investigator Sarah Tabriziand sponsored by Ionis Pharmaceuticals.

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The trial was conducted across nine study centres in the UK, Germany and Canada. It found that IONIS-HTTRx successfully reduced the level of the harmful Huntington protein in the trial participant’s nervous system.

During the study, each patient received four doses of either IONIS-HTTRx or placebo, administered by injection into spinal fluid to enable it to reach the brain.

The dose of IONIS-HTTRx was also increased several times in line with the ascending-dose trial design.

“For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated.”

In addition, patient safety was observed throughout the study by an independent safety committee.

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Tabrizi said: “The results of this trial are of ground-breaking importance for Huntington’s disease patients and families.

“For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated.

“The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”

Based on the latest results, Ionis Pharmaceuticals partner Roche has already exercised its option to license IONIS-HTTRx and assumes responsibility for further development, regulatory activities, and commercialisation activities.

In June, Ionis announced that all patients in the newly completed trial would be allowed to take part in an open-label extension to receive IONIS-HTTRx.