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Treatment of Heart Failure with Preserved Ejection Fraction
Leveraging recent findings in cardio-immunology and Phase II clinical trials, we have developed a first-in-class immunomodulatory molecule to treat heart failure with preserved ejection fraction.
Heart Failure Death Rate per 100,000 (any mention), All Ages, All Races/Ethnicities, Both Genders, 2014-2016. View interactive map
The Unmet Need
The Unmet Need
We are losing the fight against heart failure
We are losing the fight against heart failure
• 6,000,000 Americans have heart failure
• This costs our nation >$30bn/year
• >50% of patients have a form of heart failure, heart failure with preserved ejection fraction (HFpEF), for which we have few treatment options
• Heart failure is a leading cause of hospitalizations and death every year
• Currently there is only 1 FDA approved therapy to treat HFpEF
We need new tools to treat heart failure with preserved ejection fraction
We need new tools to treat heart failure with preserved ejection fraction
A Game Changer
A Game Changer
The Phase II PIROUETTE trial has recently shown that pirfenidone can treat HFpEF
The Phase II PIROUETTE trial has recently shown that pirfenidone can treat HFpEF
Nature Medicine, volume 27, pages1477–1482 (2021)
The Problem
The Problem
Pirfenidone is limited by lack of intellectual property and poor pharmacokinetics resulting in the need for three times a day dosing, CNS penetration, and frequent side effects
Pirfenidone is limited by lack of intellectual property and poor pharmacokinetics resulting in the need for three times a day dosing, CNS penetration, and frequent side effects
The Solution
The Solution
Optimize pirfenidone through conjugation
Optimize pirfenidone through conjugation
We eliminate the known shortcomings of pirfenidone by adding a small PEG chain (<500 kD)
We eliminate the known shortcomings of pirfenidone by adding a small PEG chain (<500 kD)
Tried and true method to extend half-life, and reduce CNS penetration of suitable small molecules
Does not reduce bioavailability after oral administration
Same technology used for Movantik®
The Breakthrough
The Breakthrough
We have identified a PEGylated pirfenidone superior to pirfenidone
We have identified a PEGylated pirfenidone superior to pirfenidone
Better tolerated
Better tolerated
in vivo
More cardioprotective
More cardioprotective
in vivo
Composition of matter IP and multiple potential applications
Composition of matter IP and multiple potential applications
Intellectual property
Intellectual property
Patentability and FTO assessment performed by Global Patent Group, LLC
FTO cleared and path identified for broad composition of matter IP protection
Patent application filed by WashU in July 2018, key claims upheld by international searching authority
Potential applications
Potential applications
Treatment of HFpEF (see PIROUETTE trial)
Prevention of post MI heart failure
Treatment of Muscular dystrophy associated cardiomyopathy (Orphan indication, see Pirfenidone in mdx mice)
Prevention of Post-op Afib (see Pirfenidone studies in dogs)
Prevention/treatment of Immunotherapy associated myocarditis e.g., COVID-related)
Treatment of Idiopathic pulmonary fibrosis (current application of Pirfenidone)
Etc.
Fit for the Task
Fit for the Task
Meet The Team
Meet The Team
Douglas Mann, MD
Founder, Chief Scientific Advisor
Lewin Distinguished Professor of Cardiovascular Disease;Professor of Medicine, Cell Biology, and Physiology, Washington University School of Medicine
Luigi Adamo, MD, PhD
Founder, CEO
Heart Failure Specialist and Director for Cardiac Immunology at Johns Hopkins University.
Jennifer Riggs, PhD
Head of Medicinal Chemistry
21 years experience; lead chemist for 2 successful PEG small molecules at Nektar Therapeutics
Bradley Keller, PhD
VP Preclinical Development
Drug development expert;30+ years in pharma;7+ years in small/startup biopharma companies
Jennifer Baltz, PhD
Chief Operating Officer
Harvard-trained biochemist;Serial entrepreneur;Phase I and II SBIR PI
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