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news.Australian firm Prana Biotechnology has reported results of its Reach2HD Phase II clinical trial investigating PBT2 as a treatment for Huntington disease.
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The Phase II trial met its primary safety endpoint and achieved statistically significant improvement in a measure of executive function (cognition), which included part of the trial’s main efficacy outcome.
The double-blind, placebo-controlled trial was conducted by Huntington Study Group at research sites in the US and Australia.
In the trial, a total of 109 individuals with Huntington disease were enrolled and were randomly assigned to receive daily doses of either PBT2 250mg, PBT2 100mg, or placebo for 26 weeks.
As of now, the company intends to advance PBT2 into a confirmatory Phase III clinical trial that could allow PBT2 to be approved for the treatment of Huntington disease.
Around 95% (104 of 109) of participants completed the trial on their assigned dose, and there were no substantial differences in adverse events across the two PBT2 dose groups and the placebo group.
The company claims that only one of the ten reported serious adverse events was deemed by the clinical site investigator to be related to drug treatment.
The effects of PBT2 were tested on cognition, motor performance, behaviour and functional capacity, of which cognition was pre-specified as the main efficacy outcome.
The company said that there was a statistically significant improvement in performance on the Trail Making Test Part B, in the PBT2 250mg group compared to placebo at both 12 and 26 weeks.
As per the data secured from an earlier trial, PBT2 has improved executive function in Alzheimer’s disease patients, the Reach2HD trial included a plan to evaluate the effects of PBT2 on an Executive Function Composite z-score that included the Trail Making Test Part B.
According to the company, significant improvement was observed in the z-score in a pre-specified analysis of Reach2HD participants with early stage Huntington disease, as measured by their Total Functioning Capacity score.
Image: A montage of three images of single striatal neurons transfected with a disease-associated version of huntingtin, the protein that causes Huntington’s disease. Photo: courtesy of Leevanjackson