SAN FRANCISCO--(BUSINESS WIRE)--ActiveSite Pharmaceuticals, Inc., announced today the award of a competitively renewed Phase II SBIR grant from the National Eye Institute, at the National Institutes of Health (Bethesda, Maryland), in continuing support of ActiveSite’s R&D program on novel orally-active plasma kallikrein (PK) inhibitors for treatment of diabetic macular edema (DME), the primary vision-threatening complication of diabetes. This award adds to previous funding the company has received from the NIH and other sources for developing an oral small molecule to treat DME. The grant funds, which could total over $1.3 million, will be used to carry out preclinical safety and toxicology studies with ActiveSite’s lead drug candidate. These studies, once completed successfully, would allow for the compilation and filing of an IND application with the Food and Drug Administration (FDA), for initiating Phase I clinical trials.
DME is caused by diabetes-induced leakage of fluid from the vasculature into the retina, resulting in retinal edema. To combat DME, ActiveSite has targeted the vascular serine protease plasma kallikrein (PK), known to be a pathological mediator of edema in humans. ActiveSite has developed novel potent and selective PK inhibitors, protected by worldwide patent filings, which safely inhibit diabetes-induced leakage of fluid into the retina in clinically relevant rodent models following systemic, non-ocular administration. Based on its projected safety and efficacy profile, a lead drug candidate has been selected to proceed into the safety and toxicology studies required in support of an IND application.
A summary of the research that led to the identification and selection of the lead drug candidate is being presented today by ActiveSite in a paper presentation at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), being held at Fort Lauderdale, FL, from May 6 – May 10, 2012.
“The National Eye Institute’s continuing support of ActiveSite’s research efforts to identify and develop a safe small molecule approach to the treatment of DME has allowed this program to reach this important milestone,” commented Sukanto Sinha, Ph.D., chief executive officer of ActiveSite. “This moves us closer to our goal of bringing this novel, first-in-class, patient-friendly pharmacological approach into the clinic, in order to evaluate its therapeutic potential in DME.”
About Diabetic Macular Edema
DME is a site-threatening microvascular complication of diabetes. It is estimated that, in the U.S. alone, 1.2 – 2.1 million people are affected by DME. The standard-of-care treatment is laser photocoagulation, which reduces risk of future vision loss, but with little improvement in vision. No pharmacological treatments are approved in the U.S., and agents in late-stage clinical development must be delivered intravitreally. An oral therapeutic approach could provide a non-invasive treatment option for this chronic condition with unmet medical need.
About Plasma Kallikrein
Plasma kallikrein is a vascular serine protease which generates bradykinin, excessive production of which leads to edema in humans. Rare individuals with complete deficiency of plasma kallikrein exhibit no clinical abnormalities, suggesting that chronic inhibition of this enzyme would not result in systemic adverse events.
About ActiveSite Pharmaceuticals, Inc.
ActiveSite Pharmaceuticals, Inc., is a privately held, preclinical stage pharmaceutical company, located in the San Francisco Bay Area. The company utilizes proprietary lead discovery technology to discover new inhibitors for therapeutic enzyme targets in diseases with unmet medical need.