What is it?
Huntington disease (HD) is a neurodegenerative genetic disease causing the death of brain cells. First named by American physician George Huntington in 1872, it is believed HD was brought to Australia in 1842 by women from Somerset, England.
HD affects the ganglia and front lobe, the parts of the brain responsible for motor control, coordination, cognition, emotions, cognition and personality. Similar to Alzheimer’s disease, the brains of people with HD will shrink as the disease progresses over time.
HD is caused by a mutation in the Huntington gene, first identified in 1993, which causes the gene to be longer than it should. The child of someone with Huntington’s disease has a 50% chance of inheriting the disease.
Typically once a person has been diagnosed with Huntington’s disease, the average life expectancy is 15-25 years. A diagnosis of Huntington disease is usually made after a series of neurological, psychological and blood tests, and based on family history. Symptoms usually appear between the ages of 30 and 50.
How common is HD?
It is believed about 60,000 Europeans, 30,000 Americans, 2000 Australians have Huntington disease, although the number at risk of developing the disease is much higher.
What is the limit of existing therapy?
There is no cure for Huntington disease although there are several treatments which can help alleviate the symptoms. However, some current drugs may produce side effects including insomnia, nausea, depression and involuntary contractions.
What is Prana’s progress?
The results of the Reach2HD Phase 2 clincial trial investigating PBT2 as a treatment foor Huntington disease were released in February 2014.
The primary endpoint of the study was met. In this study, PBT2 was safe and well tolerated.
The effects of PBT2 were also tested on cognition, motor performance, behaviour and functional capacity, of which cognition was pre-specified as the main efficacy outcome.
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 (p<0.001) and 26 weeks (p=0.042).
Prana now plans to meet with the US regulator, the Food and Drug Administration, and other regulatory agencies to discuss the next steps in the clinical development of PBT2 as a treatment for Huntington’s disease.
Prana has a library of 1000 novel MPACs available for screening and assessment for therapeutic applications in neurodegenerative diseases.