Publication date: Aug 11, 2019
Recently, we talked with Scott Schobel, MD, MSc, clinical science leader for Roche’s Huntington Disease Program, about the latest clinical data that the company presented at the American Academy of Neurology (AAN) annual meeting held in Philadelphia, PA
Roche is developing an antisense drug (RG6042) to treat Huntington’s disease, in partnership with Ionis Pharmaceuticals. In theory, the drug should stop the synthesis of the Huntingtin protein and the therapy specifically targets the root cause of the condition.
Huntington disease is a hereditary, neurodegenerative disorder due to mutations in the Huntingtin gene. Symptoms usually appear in the 4th, or 5th decade of life and can dramatically impact the patient and their family. The person will develop a plethora of symptoms, that have a physical (weight loss, involuntary chorea movements, poor coordination, and difficulty walking, talking and swallowing), cognitive (difficulty focusing, planning, remembering, decisions making; impaired insight) and emotional (depression, apathy, irritability, anxiety, obsessive behaviour) impact.
Currently, there is no approved treatment to delay the progression of the disease not properly address its pathophysiology.
At the AAN annual meeting, data from Roche’s ongoing, open-label study was presented involving RG6042.
Dr. Schobel said, -RG6042 is a short strand of DNA that is made in the test tube that can bind to the target mRNA, which is the substance that produces the Huntingtin protein that causes the disease.”
The 9-month open label data presented at AAN showed the drug to be safe. -RG6042 in both the Q4 week and Q8 week arm was well tolerated across the arms,” said Dr. Schobel, adding, -We now have the knowledge that we can sustainably lower the protein in CSF –
which is right in line with our pre-clinical, PK/PD model predictions.”
- Huntington’s Disease Explained
- Huntington’s Disease Treatment Market Adopts Innovation to Stay Competitive Forecast 2026
- Patient from Leeds Teaching Hospital becomes first in UK on global Huntington’s Disease trial