News for Newbies. DR George Zavoico biotech expert.
As I've said before, Prana's approach to treating early Alzheimer's disease is unique. Its small-molecule, orally available drug, PBT2, appears to redistribute certain cations, zinc in particular, but copper and iron as well, away from beta-amyloid and back into the natural, intracellular compartments where they are normally sequestered. The cations promote precipitation and plaque formation, and interestingly, cation-driven protein precipitation of Huntingtin protein and alpha-synuclein contributes to the progression of Huntington's disease and Parkinson's disease, respectively.
Lately, Prana has been on a tear because topline results of a phase 2 Huntington's disease trial, called Reach2HD, are due in September or early October. With promising early results in Alzheimer's disease already in hand, positive results in Reach2HD would help validate the underlying hypothesis of the way PBT2 works. Imagine the value of a single, small molecule drug that may be effective in treating three debilitating neurodegenerative diseases.
Topline results of an ongoing phase 2 trial in Alzheimer's disease, called IMAGINE, are expected in March of next year. In July, the Australian authorities approved a proposal to commence a 12-month safety extension of IMAGINE that should provide longer-term safety results, as well as an indication of the durability of response to PBT2. The primary endpoint of IMAGINE is its effect on beta-amyloid deposits in the brain by imaging.