NAVIGEN, INC. — Department of Health and Human Services SBIR Phase II: R

Description

PROJECT SUMMARYWhile modern HIV combination therapy has transformed HIV into a manageable chronic infection for many patientsHIV AIDS remains a formidable global epidemicDespite the effectiveness of combining drugs from different classesside effects and drug resistance remain serious concerns for these life long therapiesThusthere is an enduring need for novel HIV inhibitors with new mechanisms of action and stronger barriers to resistanceFurthermoreit is recognized that lack of patient compliance is a major factor leading to treatment failureFor this reasonHIV specialists are excited by the prospect of long acting therapiesand a combination of such therapies would provide a revolutionary new treatment option for many HIV patientsNavigen is a small pharmaceutical company targeting infectious diseases through an innovative discovery and design processWe have identified a novel HIV entry inhibitorcholesterol PIEtrimerCPTthat is a protease resistant D peptidepeptide made from D amino acidsthat targets HIV s conserved entry machineryWith highly potent activity against all major HIV subtypesdesigned barriers to resistanceand in vivo animal efficacyour anti HIV D peptide overcomes the current limitations of the entry inhibitor treatment classAdditionallyCPTis ideal for pre exposure prophylaxisPrEPsince it blocks the first stage of the viral lifecycle prior to infection of target cellsNavigen has advanced CPTto late preclinical development via a recently completed Phase II SBIR awardIn this three year SBapplication we will completeGMP manufacturing of drug substanceGMP formulation of drug product including stability testing and aseptic packagingIND enabling animal toxicologyandour IND packageThese studies will exclusively advance a daily dosing formulation that will be used to establish the safety of our D peptide therapeutic in animals and humansHoweverCPTs high potencyprotease resistanceand favorable pharmacokineticPKand physicochemical properties make it an ideal candidate for extended release depot formulationIn parallel with the studies proposed herewe are working with extended release formulation experts to develop a long acting formulation of CPTCPTLAsuitable for once monthlyor less frequentinjectionMany patients prefer long acting injectables to daily oral pillsThereforeCPTLA will likely achieve broad market penetrationAchieving IND status will greatly facilitate the early investment needed to demonstrate human efficacy with CPTandultimatelya licensing agreement with a large pharmaceutical company who can bring CPTLA to market as a potentially transformative new option for the prevention and treatment of HIV PROJECT NARRATIVE Navigen is developing CPTa novel D peptide inhibitor of HIV entry with remarkable potency and breadthThe goal of this proposal is to complete GMP manufacturingformulation and production of drug productINDenabling nonclinical studiesand an IND packageOnce CPTis established as safe and effective in humanswe will transition to using a long acting formulationbeing developed in parallelrepresenting an important new treatment and preventative option for HIV AIDS