Development of a Novel Immunomodulatory Pyridone for the Treatment of Heart Failure with Preserved Ejection Fraction

PROJECT SUMMARY/ABSTRACT
Heart Failure with preserved Ejection Fraction (HFpEF) is one of the major drivers of healthcare
costs in the western world and arguably the largest unmet need in cardiology. Currently, there
are no FDA approved drugs to treat the vast majority of patients with HFpEF.
Recently, the Phase II PIROUETTE trial showed that the immunomodulatory drug pirfenidone,
marketed for the treatment of a rare lung disease, has marked beneficial effects in patients with
HFpEF. Unfortunately, pirfenidone is a suboptimal drug. In fact, because of its poor
pharmacokinetics, patients need to take 2 large pills three times a day and they often develop
mild to moderate side effects that lead to dose reduction or discontinuation of treatment (38% of
treated patients had to discontinue pirfenidone in the PIROUETTE trial). Moreover, from a
commercial point of view, pirfenidone has no intellectual property protection and therefore
pharmaceutical companies have no incentive to develop it for further applications.
i-Cordis has PEGylated pirfenidone to obtain “Pegydone”, a new molecular entity with better
exposure, less toxicity and superior therapeutic effects than pirfenidone. i-Cordis has completed
proof of concept studies in rodent models. In this SBIR Phase II grant i-Cordis requests support
to complete critical pre-clinical studies to de-risk its drug development effort in the eyes of
potential investors.
Following completion of the Phase II grant, the company plans to raise dilutive funding to
complete IND filing, and progress through clinical trials, FDA approval and
commercialization of Pegydone as a prescription drug for the treatment of HFpEF.

Grant Number: 5R44HL145833-03
NIH Institute/Center: NIH
Principal Investigator: Jennifer Baltz