The broad long term goal of this project is to develop an improved treatment for neovascular age-related macular degeneration and for diabetic retinopathy: the two leading causes of blindness in the United States.
The specific aims of this proposal Phase 1 proposal are: to formulate sustained release intravitreal delivery systems for the VEGFR inhibitor sunitinib and to demonstrate efficacy in an accepted VEGFR inhibition primate model. Accomplishment of these aims will permit us to move to SBIR Phase II, where the specific aims will be: 1, to manufacture a lead formulation under sufficient Good Manufacturing Procedures (GMP) to permit Phase 1 clinical studies under an Investigational New Drug (IND) Exemption;andm 2, to carry out pre-clinical animal and other GLP studies in support of the IND. The milestones for the successful completion of Phase II work are the granting of an IND to begin clinical trials. The successful completion of this work could lead to a significant reduction in the need for intravitreal injections in hundreds of thousands of patients. We believe that this work is exceptionally significant. We believe that the innovation in delivery technology, drug selection, and approach will allow us to succeed where many others have failed. Our team of investigators is expert in chemistry, pharmacokinetics, drug development, animal models and ophthalmic drug delivery. We have experience in all aspects of the drug development process, from concept to approval and marketing. The co-PI led the team that developed the first intravitreal drug delivery device in clinical use. Auritec Pharmaceuticals is fully equipped and staffed to produce and test formulations for animal and early human studies. Using the strategy that led to FDA approval of the Vitrasert, we will formulate injectable systems for small molecule VEGFR inhibitors, test in vitro and in vivo release and confirm efficacy in primates. The successful completion of this work will be followed by studies and programs to enable an FDA Investigational New Drug Application. Phase I and Phase II clinical studies will form the basis for a decision to proceed to large scale Phase III trials to prove safety and efficacy.
The broad long-term goal of this project is to develop a sustained release intravitreal injectable formulation for the treatment of age-related macular degeneration and diabetic retinopathy: the two leading causes of blindness in the United States. Current standard of care requires intravitreal injection on a monthly basis indefinitely. If we are successful in reducing the injection period to three months, this will constitute a new standard of care with blockbuster potential.