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Roche exercises option to develop Ionis' drug for Huntington's disease

Ionis Pharmaceuticals Inc. said Roche Holding AG exercised its option to license IONIS-HTTRx following the completion of a phase 1/2a study of the treatment in people with Huntington's disease.

Roche is taking over all development and commercial activities of the drug. Roche paid a $45 million license fee to Ionis for exercising the option. The transaction is subject to clearances under the Hart-Scott-Rodino Act.

IONIS-HTTRx is the first therapy in clinical development to target the underlying cause of Huntington's by reducing the production of the toxic mutant huntingtin protein. Huntington's disease is a rare genetic neurodegenerative disease resulting in deterioration in mental abilities and physical control. About 30,000 individuals in the U.S. have symptomatic Huntington's disease.

The safety and tolerability profile of the therapy observed in the study supports continued development. "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," said Sarah Tabrizi, global lead investigator on the phase 1/2a study.