Ionis and Roche have licensed together for the development of IONIS-HTTRx, a breakthrough drug in the treatment of Huntington’s disease.
Huntington’s disease is a rare neurological condition that attacks the brain and results in a loss of physical and mental function. Currently, there is a grim prognosis for the disease, with treatment limited to symptom management. To learn more about Huntington’s disease, click here.
However, IONIS-HTTRx is the first (and only) drug to demonstrate efficacy in treating the underlying biological cause of Huntington’s. It works to reduce the production of the mutant and toxic huntingtin protein (mHTT), so it is much closer to a cure than the current treatment regimen. Initial trial results proved this function of the drug.
Furthermore, IONIS-HTTRx created a result that was dose-dependent: the first time that this has been achieved. Furthermore, the drug was well-tolerated and overall safe for the 46 patients it was administered.
Now, Ionis will be working quickly to conduct a larger clinical study to test if the drug can slow disease progression. Hopefully, the drug will continue to prove successful in patients that desperately need a cure.
To learn more about IONIS-HTTRx from PharmaPhorum, click here!