Yesterday Ionis Pharmaceutical presented new data from their Huntingtin lowering trial, IONIS-HTTRx. The data demonstrates a relationship between reduction of the disease-causing  Huntingtin protein and improved symptoms for people with early stage Huntington’s Disease.

After 3 months of treatment, the patients seems to benefit from the drug – now known as RG6042. This is very promising, considering that 3 months of treatment is a short period of time in the world of HD.

In an exploratory analysis, the degree of Huntingtin lowering correlated with improved scores on different measures. The improvement was seen in the ‘Stroop Word Reading Test’, the ‘Symbol Digit Modalities Test’, motor function and general functional capacity.

Read more: New Data from IONIS

– The substantial lowering of the mutant huntingtin protein and the good safety profile we observed in the study, give us hope that this new drug may have the potential to slow the progression of this devastating disease, said Dr. Sarah Tabrizi, the global lead investigator on the study.

Not ‘scientifically proved’ yet

It is important to stress that the results are exploratory, from a small number of patients in a study with few placebo patients. This means that the results are not ‘scientifically proved’.

The drug needs to be tested in a larger number of patients, over a longer period to know how it “actually” works over time.

It also needs to be compared against a bigger control (placebo) group in order to figure out the specific clinical benefits. The control group will receive a placebo drug, while the other group will receive the actual drug.

A promising future

With that being said, the results are promising. The Huntingtin lowering drug have shown to be safe and well-tolerated – which is a good start. Further, it has proven to lower the levels of the disease-causing Huntingtin and this lowering have shown to most likely improve symptoms in people with Huntington’s disease.

– I am very happy today, says Astri Arnesen, President of EHA.  – The news is very encouraging! We have indications that the drug reaches the brain and improves the condition for the patients – and that’s amazing!

– In my opinion we deserve to celebrate every victory on the road to efficient treatment. I want to raise the flag and be happy today knowing that there is still hard work ahead of us!