Huntington's disease drug promising in mid-stage study

By Reuters Staff

(Reuters) - Australia-based Prana Biotechnology Ltd said today its experimental drug to treat Huntington's disease met the main goals of safety and tolerability in a mid-stage study.

The Reach2HD Phase 2 double-blind, placebo-controlled study was conducted by the Huntington Study Group at research sites in the United States and Australia. The study enrolled 109 individuals with Huntington's disease who were randomly assigned to receive the drug PBT2 (100 or 250 mg) or placebo for 26 weeks

Huntington's disease is characterized by a loss in coordination and cognitive decline due to a degeneration of nerve cells in certain parts of the brain.

The company said the primary endpoints of safety and tolerability with the drug were met.

For secondary endpoint, they report statistically significant improvement in a measure of executive function in subjects given the higher dose (p=0.042). PBT2 250 mg was also associated with a favourable signal in functional capacity.

"Preliminary evidence suggests PBT2 250 mg reduced atrophy of brain tissue in areas affected in Huntington disease, seen in a pilot imaging sub-study," the company said.

Prana said there were no substantial differences in adverse events in patients treated with the drug, and those on placebo. Only one of the 10 serious adverse events was related to the treatment, the company said.

Dr Ira Shoulson, Professor of Neurology at Georgetown University and Chair of the Huntington Study Group, who was not involved in the trial and acts as an advisor to Prana, said: "This is the first time we have observed dose-related slowing in functional decline over a six month period of treatment, which taken together with the safety reassurance, will provide genuine optimism for the Huntington disease community to support a larger confirmatory trial of PBT2 in Huntington disease."

Based on these findings, Prana said it would start Phase 3 study of the drug to treat Huntington's disease. The company is also testing the drug in a mid-stage study for Alzheimer's disease.

Dr Rudy Tanzi, Professor of Neurology at Harvard Medical School and Prana's Chief Scientific Advisor, said, "the observation of significant improvement in executive function with PBT2 in this clinical trial for Huntington disease and the previously reported Alzheimer's trial, suggests a common mechanism for neurodegeneration in these diseases based on metal interactions. In my opinion, these findings significantly elevate the potential for PBT2 as an effective therapy for both Huntington disease and Alzheimer's disease."

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