NanoCor Announces New Data on Congestive Heart Failure Gene Therapy

Up to 25 percent improvement in large animal model

CHAPEL HILL, N.C. September 26, 2017 –(BUSINESS WIRE)–NanoCor Therapeutics, Inc. (NanoCor), a biotechnology company focused on developing gene therapies for cardiovascular disease, today announced publication of a large animal study supporting the efficacy of its lead candidate therapy, Carfostin®. In the study, heart function improved by up to 25 percent using a single treatment of the gene therapy in a large pre-clinical animal (porcine) model. The results of the study, Protein Phosphatase Inhibitor Gene Therapy in a Swine Model of Nonischemic Heart Failure, were published online in the Journal of the American College of Cardiology on September 25th. The NanoCor team plans to study the same gene therapy in a human trial starting next year. The study was conducted at the Icahn School of Medicine at Mount Sinai in New York City.

“In this study of large animals with heart failure, the investigators have shown that the treatment NanoCor will soon be testing in humans appears promising,” said Sheila Mikhail, President and CEO of NanoCor. “There are currently no gene therapy treatment options approved for late stage congestive heart failure. We are excited to move Carfostin forward and make a new treatment option available.”

Congestive heart failure is characterized by defects in calcium-handling proteins. Modulating the calcium cycling protein phosphatase 1 (PP1) is a promising therapeutic approach. Carfostin is a gene therapy that delivers the constitutively active form of PP1 inhibitor (I-1) protein directly into damaged heart cells. Carfostin was created using NanoCor’s proprietary adeno associated viral (AAV) vector, 2i8, and Self-Complementary Vector technologies that enable the transfer of therapeutic genes specifically into heart muscle cells. NanoCor ltc exclusively licensed its 2i8 vector from Asklepsios Biopharmaceutical, Inc. This AAV vector has unique targeting capability tropism for heart tissue and detargets other tissues where delivery of the therapeutics is less desirable, such as the liver. In the study, the team at Mount Sinai treated 13 pigs with severe heart failure induced by mitral regurgitation: six with Carfostin and seven with placebo (saline) solution. The research demonstrated in the animal model that the gene therapy was safe and significantly reversed heart failure by 25 percent in the left ventricle and by 20 percent in the left atrium. The treatment group also showed a ten percent reduction in heart enlargement, a common symptom of heart disease.

“These large animal results suggest that gene therapy may be a viable treatment option for congestive heart failure,” said the study’s senior author, Roger Hajjar, MD, Chief Medical/Scientific Officer of NanoCor and Director of the Cardiovascular Research Center and Arthur and Janet C. Ross Professor of Medicine at the Icahn School of Medicine at Mount Sinai. “We were able to deliver a very targeted therapy in a single dose. Despite advances in medical care for chronic cardiovascular disorders, the five-year mortality rate of heart failure patients remains unacceptably high. Novel therapeutic approaches such as gene therapy and cell therapy hold the promise of complementing, if not replacing, existing therapies for heart failure.”

In 2016, the United States Food and Drug Administration (FDA) accepted NanoCor’s investigational new drug (IND) application for Carfostin® for the treatment of congestive heart failure. NanoCor intends to initiate a multi-center, open-label, dose-escalation Phase 1 trial in patients with advanced stage III/IV chronic heart failure. The intended Phase 1 trial will evaluate four escalating doses of the gene product.

About Carfostin®
Carfostin delivers the constitutively active form of protein phosphatase 1 inhibitor (I-1) protein directly into damaged heart cells and targets type 1 protein phosphatase (PP1), a critical negative regulator of calcium cycling and contractility. Carfostin is based on NanoCor’s proprietary adeno associated virus (AAV) vectors and Self-Complementary Vector technologies that enable the transfer of therapeutic genes specifically into heart muscle cells. Carfostin is a one-time treatment and will be delivered via the femoral artery into the coronary arteries.

About Congestive Heart Failure
Congestive heart failure is a condition in which the heart is unable to supply sufficient amounts of blood and oxygen to the body. It is the final stage of multiple heart diseases. Approximately five million patients in the U.S. suffer from congestive heart failure, which is one of the most common reasons patients 65 and older are hospitalized.

About NanoCor Therapeutics, Inc.
NanoCor Therapeutics, Inc. (Chapel Hill, NC) is a biotechnology company focused on the development of novel proprietary molecular cardiovascular therapeutics. NanoCor’s lead therapeutic is Carfostin®, an intracellular protein therapeutic for chronic heart failure. The technology incorporated in Carfostin was developed in the laboratories of Dr. Roger J. Hajjar, Dr. Evangelia Kranias, and Dr. R. Jude Samulski. NanoCor is a spinout of Asklepios BioPharmaceutical, Inc. (AskBio), which previously spun out Chatham Therapeutics, LLC, sold April 2014 to Baxter, now Shire and Bamboo Therapeutics, Inc, sold August 2016 to Pfizer,