Alzheimer’s candidate off to a good start

BERWYN, Pa.—Mid-July was go-time for this year’s Alzheimer’s Disease International Conference (AAIC 2017 London), and among the presenters was QR Pharma Inc., a Phase 2 biopharmaceutical company advancing novel therapies for Alzheimer’s disease, Parkinson’s disease and other neurodegenerative conditions. The company’s presentation, “Reducing APP through Translational Inhibition can Prevent or Treat Alzheimer’s disease in Down Syndrome,” focused on its work with Posiphen in Down syndrome.

Studies were conducted by investigators from The University of California campuses in San Diego (Mobley Laboratory) and Los Angeles (John Laboratory) assessing Posiphen’s effects in healthy vs. diseased Down syndrome cells and mice.

Down syndrome is also known as trisomy 21, for the three copies of chromosome 21 that are a hallmark of the disease. This chromosome contains the amyloid precursor protein (APP) gene, which plays a role in the brain’s production of amyloid plaques. In addition to an extra copy leading to high levels of APP, having three copies of chromosome 21 is a well-known risk factor in the development of early-onset Alzheimer’s disease. As a result, a majority of adult Down syndrome patients present with signs of Alzheimer’s by middle age, with dementia symptoms developing in their 70s.

“The observed development of Alzheimer’s disease in adults with Down syndrome has been an area of increased study as the life expectancy of adults with Down syndrome has extended. The data generated in these studies demonstrates Posiphen’s ability to downregulate elevated APP levels in well-understood in-vitro and in-vivo disease models of Down syndrome,” Richard Fitzgerald, QR Pharma’s chief financial officer, said in a press release.

Posiphen works to decrease levels of APP and its fragments as well as restoring vesicle transport and neurotransmitter release, and as such, could help prevent or treat Alzheimer’s disease in patients with Down syndrome or Alzheimer’s alone. It is a “small, hydrophobic, orally available molecule that enters the brain readily,” QR Pharma notes on its website, and is “the only drug ever described that inhibits more than one neurotoxic aggregating protein.” Compared to standard approaches, Posiphen inhibits synthesis of APP, tau and α-Synuclein, aiming to block the formation of the toxic proteins rather than eliminating them after the fact.

The recent AAIC presentation noted that Posiphen demonstrated efficacy in preclinical rodent models of Alzheimer’s disease, Parkinson’s disease, traumatic brain injury and glaucoma. In an open IND clinical trial in 120 healthy volunteers, QR Pharma was able to establish pharmacokinetics and maximum tolerated dose, and saw preliminary proof of concept in four patients with mild cognitive impairment. A Phase 2 clinical study of Posiphen in early Alzheimer’s patients is still ongoing.

“The data suggests that Posiphen should be tested in a preventive setting in Down syndrome individuals, as they carry a substantial risk of early onset of Alzheimer’s disease,” stated Dr. Claudine Bruck, director of the board for QR Pharma.

“These studies provide further confirmation of the neuroprotective effects of Posiphen,” said Dr. Maria Maccecchini, president and CEO of QR Pharma. “They add to a deep data set supporting the unique activity and potentially broad application of Posiphen in preventing as well as treating abnormal cellular environments and normalizing function.”

This isn’t the only conference that’s seen QR Pharma present promising data on Posiphen in Alzheimer’s models. In March the company made a presentation, “Treating Acute and Chronic Neurodegeneration by Inhibiting Neurotoxic Aggregating Proteins,” at the 13^th AD/PD International Conference in Vienna. As noted in a press release, “The presentation highlights studies conducted by investigators from Columbia University characterizing the effects of Posiphen in a transgenic model of AD and from UCSF showing the effects of Posiphen in a transgenic model of PD. Posiphen administered daily orally to transgenic APP/PS1 mice preserved spatial working memory and brain function; while administered daily intraperitoneally to transgenic alpha-synuclein mice it restored gut motility and coordination.”