New Data from IONIS-HTT Rx Phase 1/2 Study Demonstrates Correlation Between Reduction of Disease-causing Protein and Improvement in Clinical Measures of Huntington's Disease

First drug to demonstrate lowering of mutant huntingtin, the disease-causing protein, in people with Huntington's disease CARLSBAD, Calif., April 24, 2018/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, today presented top-line data from the Phase 1/2 study of IONIS-HTTRx (RG6042) in people with early stage Huntington's disease (HD) at the 70thAmerican Academy of Neurology (AAN) meeting in Los Angeles, California. Results from exploratory analyses of data from the study demonstrated correlations between reductions in mutant huntingtin (mHTT), the disease-causing protein, and improvements in clinical measures of Huntington's disease.

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