IONIS-HTT Rx (RG6042) Top-Line Data Demonstrate Significant Reductions of Disease-Causing Mutant Huntingtin Protein in People with Huntington's Disease

Phase 1/2 study first to demonstrate disease-modifying potential Up to ~60% reduction (mean ~40%) in the mutant huntingtin protein (mHTT) observed in the cerebral spinal fluid (CSF) of patients treated for three months with IONIS-HTT Rx (RG6042) at the two highest doses tested with mHTT levels continuing to decline at the last measurement mHTT reductions of 40-60% in CSF correspond to an estimated 55%-85% reduction in the cortex of the brain, where mHTT is highly expressed, based on preclinical data mHTT reductions observed in the Phase 1/2 study exceeded reductions that produced disease benefit in animal models of HD Roche is working to quickly advance IONIS-HTT Rx (RG6042) to a pivotal study Conference call and webcast tomorrow, March 2nd, 11:00 a.m. ET CARLSBAD, Calif., March 1, 2018 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), a leader in antisense therapeutics, today announced 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. The data demonstrate that IONIS-HTTRx (RG6042) is the first drug in development to lower the disease-causing protein in people with HD.

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