BIOORG3.14 LLC — Department of Health and Human Services SBIR Phase I: 200

Description

The overall goal of this program is to develop an oral replacement for injecting insulinThe central premise of this work is that a small molecule called BGor similar moleculescan be taken orally in place of injecting insulin to aid in glycemic controlBGcould completely replace insulin injections or could reduce insulin injections by halfTo begin clinical testing to evaluate this premisea formalregulated toxicological program must be conductedand BGmust be manufactured for human useThe specific aims of this proposal are designed to address these obstaclesPHASE I SPECIFIC AIMMake larger quantitieskgof BGThis aim will produce sufficient amounts of BGto complete all preclinical studies required to begin human testingAimwill also generate analytical knowledgesuch as analytical methods to characterize the drug s stabilityidentitystrengthand puritywhich will enable the manufacturing of BGfor human use and the completion of all preclinical studies required to proceeded to humansPHASE II SPECIFIC AIMSAIMComplete aday repeat dose study in animals with type I diabetesAimwill help defineathe therapeutic oral dose levels of the BGbthe degree to which BGis able to lower blood glucoseandcBGs potential to induce hypoglycemiadiabetic ketoacidosisor other adverse eventsThis information will allow us to power and design initial human trialsAIMDefine the no observed adverse effect LevelNOAELand derive the Maximum Recommended Starting DoseMRSDfor first in human clinical trialsThere is currently insufficient data to determine the dose levels at which toxicity might occur in upon repeat dosing with BGNot knowing these dose levels prevents us from initiating human trialsThis aim will generate the required safety data to enable clinical trialsThis program will lead to the identification of a new class of orally bioavailable molecule that is capable of lowering blood glucose with an activity approaching that of insulinSuch discovery would lead to a novel therapeutic for glycemic controlClinical work represents the only method to determine the extent to which BGor similar moleculeswould make useful and safe oral therapeutic that are capable of reducing the need to inject insulinThis project is designed to reach this clinical stage as early as possible Despite advancements in the management of diabetessuch as oral hypoglycemic agents and insulin therapiesit is estimated that only half ofmillion Americans with diabetes are able to achieve ideal levels of blood glucoseAs a resultindiabetes was estimated to cost the United States $billion$billion in direct medical costs and $billion in reduced productivityThe proposed work is relevant to public health because it will enable the development of a therapeutic to control diabetesreducing individual burdenalong with the acssciated healthcare and societal cost