A targeted drug for the treatment of inflammation in cystic fibrosis lung disease

DESCRIPTION provided by applicant Problem to be Solved Cystic fibrosis CF is a global health issue that affects over people worldwide including Americans Lung disease is the primary cause of morbidity and mortality in CF patients and is characterized by intense inflammatory responses in the airways The Product and Long Term Goal Nanometics LLC dba PHD Biosciences PHD is developing a targeted small molecule enzyme inhibitor as a therapeutic for inflammation in CF lung disease It is anticipated that this new approach will be useful to mitigate inflammation in the airways of patients The Technological Innovation The PHD approach is unique to all other CF therapeutics utilizing a targeted enzyme inhibitor with high specificity and picomolar affinity to block recycling of an endogenous metabolite Metabolite accumulation leads to robust and sustained anti inflammatory activity Phase I Hypotheses This Phase I SBIR will demonstrate the feasibility of the PHD enzyme inhibitor as an oral therapeutic in Scnn b Tg mouse models of CF lung disease The hypotheses tested during Phase I SBIR experiments will be a single oral dose will ameliorate inflammation for h and once daily oral doses of the therapeutic are non toxic and will promote a continued anti inflammatory state Specific Aims Specific Aim Demonstrate the Pharmacological and Concentration Dependent Effects of Oral Doses of MTDIA in the Scnn b Tg Mouse Phase II Phase II SBIR studies will further establish the mechanism of action and toxicity profile Commercial Application The PHD approach is a unique approach to anti inflammatory therapy that will provide an extended benefit to patients as a single agent or part of a combinatorial approach The current CF drug market is predicted to reach $ billion by PUBLIC HEALTH RELEVANCE Cystic fibrosis CF is a genetic disorder that affects approximately Americans and people worldwide This project seeks to develop a new long lasting anti inflammatory approach to treat cystic fibrosis lung disease