On April 20, 2020, Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced that its partner Roche, also known as Genentech in the United States, has completed enrollment for GENERATION HD1, a global Phase 3 study evaluating the efficacy and safety of tominersen (previously IONIS-HTTRx or RG6042), an investigational anti-sense therapy for people living with Huntington's disease (HD). "Completion of the enrollment of this Phase 3 study is an important landmark for the clinical development of tominersen and for families affected by Huntington's disease. While there is much work ahead of us, we are now closer to potentially providing a treatment for people living with this devastating disease. We are grateful to Huntington's disease patients, their families, and healthcare providers for their courage and resilience, particularly in the current challenging environment," said Brett P. Monia, PhD, Ionis' Chief Executive Officer. "At Ionis, knowing that sick people depend on us fuels our passion for discovering and delivering novel anti-sense medicines like tominersen, the first and only therapy in pivotal trials targeting the underlying cause of HD." GENERATION HD1 is evaluating the efficacy and safety of tominersen treatment administered once every two months (eight weeks) or every four months (16 weeks) over a period of 25 months, compared to placebo. The study has completed enrollment with 791 patients across approximately 100 sites around the world. HD is a devastating, and ultimately fatal, hereditary disease resulting in deterioration in mental abilities and physical control. Currently, there is no approved disease-modifying treatment for HD. There are approximately 3 to 10 per 100,000 people worldwide affected by HD. In the U.S. alone, there are approximately 40,000 people with symptomatic HD and more than 200,000 people at risk of having inherited the gene that causes HD.
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