News Article

Aphios Pockets $2.4M SBIR Grant for Preclinical Alzheimer Disease Candidate
Date: Jan 24, 2012
Source: Genetic Engineering News ( click here to go to the source)

Featured firm in this article: Aphios Corporation of Woburn, MA



Aphios has received a Phase 2 SBIR grant from the NIA amounting to $2.4 million over two years. The money will cover development of the company's candidate for treating Alzheimer disease (AD), which is currently in early clinical development.

"Aphios is developing APH-0703, a potent protein kinase C (PKC) modulator that activates the alpha-secretase pathway, which can dramatically enhance the generation of soluble amyloid precursor protein (sAPP), diminishing plaques and cognitive deficits associated with Alzheimer disease," explains Trevor P. Castor, Ph.D., president and CEO, Aphios.

"We are developing novel formulations of APH-0703 based on Aphios' proprietary hydrophobic-based formulation and patented SFS-PNS polymer nanospheres technologies. After formulation selection, Aphios will manufacture and characterize the API and final drug product following cGMP guidelines and conduct preclinical studies in preparation of filing an IND with the FDA to conduct human clinical trials."

Aphios is collaborating with researchers at Louisiana State University Health Sciences Center (LSUHSC). The university will conduct in vitro and in vivo efficacy, toxicity, and pharmacokinetic studies in triple transgenic AD mice. APH-0703 is a highly effective alpha-secretase modulator, several orders of magnitude more potent than typical modulators, according to Jonathan Steven Alexander, Ph.D., LSU's principal investigator. Dr. Alexander and study co-investigator Lisa M. Schrott, Ph.D., hope to identify the most effective, mechanism-based cognitive enhancer formulations of APH-0703. Nanoencapsulated APH-0703 reportedly shows even higher activity than the hydrophobic-based APH-0703 formulation in cell assays and is currently being studied in animal models of AD.

"In dose- and time-dependent studies APH-0703 showed a very high level of effectiveness in alpha-secretase assays, which appears to reflect the powerful activation of PKC-delta and epsilon," Dr. Alexander notes. "An even more impressive finding is that an oral formulation of APH-0703 rapidly reversed cognitive deficits in animal models of AD."