Roche to licence Ionis Huntington's treatment after trial breakthrough

Ionis Pharmaceuticals on Monday said it had earned a $45m fee from Roche after the Swiss peer took up its option to licence its Huntington's disease treatment.

A key trial of the Ionis-HTT drug, which took place in the UK Germany and Canada, produced positive results, successfully lowering the level of the harmful Huntington protein in the nervous system.

The trial of 46 patients was led by Professor Sarah Tabrizi of the UCL Institute of Neurology and sponsored by Ionis.
Each patient received four doses of either IONIS-HTTRx or placebo, given by injection into the spinal fluid to enable it to reach the brain.

“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,” Tabrizi said.

Roche is now responsible for all IONIS-HTT development, regulatory and commercialization activities and costs, the companies said in a statement.