Phase I/II study reports first drug to reduce disease-causing mutant huntingtin protein

IONIS-HTTRx (RG6042) is the first drug in development to be reported to significantly reduce disease-causing mutant huntingtin protein (mHTT) levels in early stage Huntington's disease (HD) patients. Ionis Pharmaceuticals (CA, USA) recently announced the Phase I/II results of IONIS-HTTRx (RG6042) at the 13th Annual CHDI HD conference (26 February –1 March, CA, USA). Roche (Basel, Switzerland), collaborators with Ionis Pharmaceuticals in developing antisense drugs for the treatment of HD since 2013, have subsequently released plans for a pivotal-stage study to determine the clinical efficacy and safety of IONIS-HTTRx (RG6042). Sarah Tabrizi, global lead investigator of the study and director of...