SBIR Phase II: A platform technology that significantly improves drug delivery

The broader impact/commercial potential of this Small Business Innovation Research (SBIR) Phase II project is the development of a drug delivery platform that can improve a drug?s properties such that the drug requires less frequent dosing, and can now be administered by the patient at home. This technology is truly enabling for peptides, a short-lived but potent class of compounds that in their current state are not useful as a treatment for chronic diseases. This technology has significant commercial potential in that it can be broadly applied to numerous emerging and existing drugs in the $140B global biotherapeutics market. Use of our technology will have a significant societal impact by providing more efficient medicines delivered in a more patient-friendly way that could reduce healthcare cost and increase patient compliance. This will significantly impact the health, recovery and quality of life for patients with chronic diseases. Development of this technology will further scientific understanding of drug delivery. This technology will advance the field in that it has significant advantages over the current state-of-the-art, including the ability to allow for less frequent dosing, and by increasing the amount of drug available to the body. These beneficial effects are obtained with one simple modification that does not interfere with the function of the drug. The proposed project is to develop a platform technology that improves the delivery of therapeutics. Peptides and many other types of drugs require frequent dosing because they do not last long in the body and/or require delivery via a needle into the vein because they are not available to the body when given as an injection into the skin. We are developing a way to modify peptides so that they can last longer in the body. As an example, current treatments for a cardiopulmonary rare disease only serve to alleviate symptoms. In this project, a novel peptide that has the potential to be a breakthrough therapeutic for this rare disease will be modified to improve the delivery profile. Current data suggests that the new therapy will be multifaceted and would treat the underlying disease. The objective of this study is to advance the platform technology by exploring other types of modifications, then test these modifications in vitro and in vivo to compare which modifications confer the best delivery profile. A lead compound will be chosen to explore efficacy in animal models, and to examine the immunogenicity and toxicity in rodents to generate critical early safety data. The outcome of SBIR Phase II will be a long-acting peptide therapeutic with strong in vivo preclinical efficacy data for the treatment of a rare disease that has an acceptable safety profile and is ready for further preclinical development.