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Archive - Mar 11, 2018

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Top-Line Data Demonstrate Significant Reductions of Disease-Causing Mutant Huntingtin Protein in People with Huntington's Disease

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), a leader in antisense therapeutics, announced, on March 1, 2018, the presentation of positive top-line data from a completed Phase 1/2 study of IONIS-HTTRx (RG6042) in people with early-stage Huntington's disease (HD) at the 13th Annual CHDI HD conference (http://chdifoundation.org/13th-annual-hd-therapeutics-conference/) (February 26-March 1) in Palm Springs, California. The data demonstrate that IONIS-HTTRx (RG6042) is the first drug in development to lower the disease-causing protein in people with HD. HD is a rare, progressive, neurodegenerative disease caused by genetic mutation in the huntingtin gene, which results in the production of a toxic protein, the mutant huntingtin (mHTT) protein, which gradually destroys neurons in the brain resulting in deterioration in mental abilities and physical control. Ionis designed IONIS-HTTRx (RG6042), a Generation 2+ antisense drug, to specifically reduce the production of all forms of the huntingtin protein, including normal and mHTT. "For nearly twenty years, I have seen many families devastated from losses to this progressive neurodegenerative disease. With IONIS-HTTRx (RG6042), the HD community has new hope for a therapy that can reduce the cause of HD, and therefore, may slow the progression and potentially prevent the disease in future generations, which is truly groundbreaking," said Dr. Sarah Tabrizi, Professor of Clinical Neurology, Director of the University College London's Huntington's Disease Centre and the global lead investigator on the study.