IND-enabling development of a biomimetic peptide drug for macular edema

SummaryMacular edemaMEand diabetic macular edemaDMEwhich affects at leastmillion people in theUSare leading causes of blindness in people between the ages ofThe major problem in thesediseases is a leaky vasculaturewhich results in pooling of blood around the retinaThis blood can causeocclusion of vision as well as swelling and increased ocular pressure and detachment of the retinaThe effective therapies approved to date for these conditions are proteins that are injected intravitreally to bind to VEGF to blocks its activityAlthough these drugs are efficacious for somea significant number of ME patients are unable to gain significant improvements to visual acuity using these drugsIn additiondrugs in this space have also proven to be effective for NVAMDa $billion year marketOur peptide drug AXTinhibits signaling of not only VEGF but also of growth factors PDGFHGFand IGFThusit could serve as an anti VEGF monotherapy in patients in whom VEGF is the primary driver of disease but it could also successfully treat patients in whom these other factors contribute to diseaseAXTcould also have advantages over the next generation combination therapy of Fovistaan anti PDGF aptamerwith Eylea or LucentisSpecifically because AXTinhibits PDGF and VEGF simultaneouslyeven as a monotherapy it could be as efficacious as the combination while avoiding the dual injection and the associated complicationsAsclepiX Therapeutics LLC represents the next generation of drug development technologyWe use bioinformatics and systems biology methods to design classes of short biomimetic anti angiogenic and antipermeability peptidesIn additionwe also have technology to prolong the efficacy of our peptide agents through long lasting biodegradable nanoand microparticles as may be neededAlthough he have demonstrated efficacy of our novel lead agentincluding in gold standard head to head models in the mouse and rabbit vsthe leading FDA approved agentswe have not yet conducted the IND enabling GLP toxicity studies needed in two species to enable the filing of an IND for subsequent FDA approvalThis Phase II SBIR Proposal would provide the funding necessary to move this technology from promising pre clinical research to a patient s bedside and the beginning of clinical trialsThis proposal is to optimize the excipients and formulation for our novel biomimetic peptide drugdetermine its local toxicity and biodistribution when administered intravitreallyand determine its potential toxicity when administered at various doses systemicallyWe believe that our Phase II program will lead to the approval of an IND for a novel safe and effective peptide agent for treating macular edemaThis drug has the potential to improve the vision of millions of Americans and may be impactful in multiple ocular diseases Project Narrative There is a large medical need for drugs for the treatment of macular edemaWe have identified a promising peptide therapeutic that has shown remarkable activity in animal models of the human diseaseHere we propose IND enabling safety studies on our drugIf these studies show that our drug is safe in animalsthe next stage will be a Phase I clinical trial to determine safety in people