Novel Huntington’s Therapy to Enter Clinical Development

Ionis Pharmaceuticals, Inc. — formerly Isis Pharmaceuticals — announced the U.S. Food and Drug Administration (FDA) has approved its Orphan Drug Designation application for IONIS-HTT_Rx as a potential treatment of Huntington’s disease (HD).

Ionis’ IONIS-HTT_Rx is a generation 2.0+ antisense drug developed to treat HD by reducing the production of all forms of the huntingtin (HTT) protein. As such, it is the first therapy targeting the direct cause of HD to enter clinical development. Ionis is partnering with Roche to develop antisense drugs to treat HD, combining Ionis’ proprietary antisense technology and expertise with Roche’s scientific knowledge in the development of neurodegenerative therapeutics. IONIS-HTT_Rx has also received Orphan Drug Designation by the European Medicines Agency as an HD treatment.

“HD is a rare genetic neurological disease in which patients experience deterioration of both mental abilities and physical control. Although the toxic protein produced from the huntingtin (HTT) gene in HD patients has been a target of interest for many years, IONIS-HTT_Rx is the first therapy to enter clinical development that is designed to treat the underlying cause of this fatal disease. The granting of Orphan Drug Designation in both the US and Europe highlights the significant need for a drug that could transform the treatment of HD,” C. Frank Bennett, PhD, Ionis Pharmaceuticals’ senior vice president of Research, said in a press release.

The Orphan Drug Act gives economic incentives to encourage the development of therapies for diseases that affect less than 200,000 people in the U.S. This designation provides companies with seven years of market exclusivity if the drug is approved for use in the U.S., tax credits for clinical expenses, an FDA-user fee exemption and FDA assistance in clinical trial design.

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