Roche, Io­n­is her­ald a big win in ear­ly-stage Hunt­ing­ton’s dis­ease tri­al, demon­strate dis­ease mod­i­fy­ing po­ten­tial

Io­n­is $IONS and its part­ners at Roche have un­veiled the bio­mark­er da­ta from a Phase I/II study that re­cent­ly drove the phar­ma gi­ant to fol­low through with a $45 mil­lion pay­ment to op­tion rights to IO­N­IS-HTT Rx (RG6042).

Re­searchers said Thurs­day evening they tracked a mean re­duc­tion of 40% in mu­tant hunt­ingtin pro­tein at the two high­est dos­es of the ther­a­py. Some pa­tients saw up to 60% re­duc­tions. And that, they add, is a first for any Hunt­ing­ton’s drug. It’s that tox­ic pro­tein that lays waste to neu­rons in the brain, caus­ing a se­vere neu­rode­gen­er­a­tive dis­ease marked by de­clin­ing mus­cle co­or­di­na­tion and cog­ni­tive abil­i­ties, which typ­i­cal­ly slow­ly kills pa­tients over a course of 20 years.

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