Phase 3 Trial is Underway for Huntington’s Disease

Ionis Pharmaceuticals and Roche have been collaborating on a drug for Huntington’s Disease which will treat the root cause of the condition. Currently, there are no such approved treatments, meaning no cure for this fatal diagnosis.

Huntington’s Disease is a hereditary neurodegenerative disorder which causes deterioration of the patient’s physical and mental abilities. It affects approximately 30,000 people in the United States. However, there are an additional 200,000 people who are at genetic risk.

Thanks to collaboration, Ionis Pharmaceuticals and Roche have been able to announce progress in their Phase 3 clinical trial. They have just released that the first patient has officially enrolled in their Phase 3 study investigating RG6042.

The Trial

The goal of RG6042 is to reduce the body’s production of the mutant huntingtin protein which is toxic. Ionis believes it could significantly reduce the effects of the disease.

This Phase 3 study is only one of the projects Ionis is working on this year. The company hopes to advance three more studies into clinical trials in 2019. One of their notable past developments was SPINRAZA for spinal muscular atrophy (SMA). SPINRAZA has since been commercialized for all forms of the condition.

Hopefully, we will see this Phase 3 study for Huntington’s Disease progress similarly, with RG6042 becoming a therapeutic option for those living with this devastating condition. The initiation of enrollment for the Phase 3 study has already brought hope to many families affected by Huntington’s Disease.

You can read more about this Phase 3 study and the collaboration between Ionis Pharmaceuticals and Roche here.


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