PTI-125 is an oral, small molecule drug developed by the Austin, Texas-based company in collaboration with other partners. It has been shown to significantly improve Alzheimer’s neurodegeneration in animal models of the disease and in post mortem brain tissue from Alzheimer’s patients, including receptor dysfunction, neuroinflammation, tau protein modification and insulin resistance, as well as plaques and tangles that are hallmarks of the disease.
PTI-125 works by binding to a particular site on filamin A (FLNA), a protein critical to beta amyloid’s toxicity. Beta amyloid1-42 (A42) exerts its toxic effects, causing the tangles and plaques found in the brains of people with Alzheimer’s.
A42’s toxic signaling requires the help of FLNA. The effect of PTI-125 on this process was the focus of the study, “PTI-125 reduces amyloid-related Alzheimer’s pathogenesis by targeting filamin A,” that appeared in Alzheimer’s & Dementia. In this article, researchers demonstrated that PTI-125 prevented amyloid-related Alzheimer’s damage. PTI-125 also had a powerful anti-inflammatory effect in animal models.
The NIH grant will allow Pain Therapeutics to begin evaluating PTI-125 in humans. The agency’s National Institute on Aging awarded the $1.7 million grant after a competitive, in-depth evaluation of PTI-125 for scientific and technical merit. The NIH review process relies on input from academics, clinical and industry experts in Alzheimer’s.
“We are once again grateful to the NIH for its support of our research program around PTI-125,” Remi Barbier, chairman, president and CEO of Pain Therapeutics, said in a press release. “NIH has long been a champion of innovative new science that stands up to rigorous, peer-reviewed evaluation and that has potential to benefit human health in areas of unmet needs, such as Alzheimer’s disease.”
The grant precedes an investigational new drug application the company expects to submit for PTI-125 to the U.S. Food and Drug Administration later this year.