NanoCor Therapeutics Announces FDA Acceptance of Investigational New Drug (IND) for Carfostin®

CHAPEL HILL, N.C., April 20, 2016 /PRNewswire/ -- NanoCor Therapeutics, Inc. (NanoCor), a biotechnology company focused on treating cardiovascular disease at the molecular level, today announced that the United States Food and Drug Administration (FDA) has accepted the company's investigational new drug (IND) application for Carfostin®, a cardiac gene therapy in development for the treatment of congestive heart failure (CHF). NanoCor intends to initiate a multi-center, open label, dose-escalation Phase 1 trial in patients with advanced stage III/IV chronic heart failure (CHF) in H2, 2016.   

"The acceptance of our IND is an important milestone for us," said Roger Hajjar, M.D., scientific co-founder of NanoCor and director of the Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York. "CHF is a major cause of morbidity and mortality, and there is a critical need to explore new therapeutic approaches. Gene therapy is a viable option for the treatment of CHF, and our proprietary technology platform allows for targeted and minimally-invasive delivery of the therapeutic to the heart." The intended Phase 1 trial will evaluate four escalating doses of the gene product. A group of three patients will be enrolled in each group for a total of 12 patients tested. 

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 the company's proprietary Biological NanoParticle (BNP®) and Self-Complementary Vector Technologies that enable the transfer of therapeutic genes specifically into heart muscle cells. BNPs are derived from recombinant adeno-associated viruses which are non-pathogenic viruses naturally existing in the human body and are pathogenically safe while having a very low immune response profile. Carfostin is a one-time treatment and will be delivered via the femoral artery into the coronary arteries.

Dr. Hajjar continued, "CHF is characterized by multiple defects in calcium-handling proteins, and modulating the calcium cycling protein PP1 is a promising therapeutic approach. Carfostin inhibits PP1 and has shown to increase cardiac contractility and to halt the progression of CHF in preclinical studies. Importantly, BNPs target specifically the heart while de-targeting the liver and can be given at high doses to ensure a higher myocardial uptake."

About Congestive Heart Failure (CHF)

Heart disease is the leading cause of death worldwide with congestive heart failure (CHF) being the final stage of many heart diseases. CHF is a condition in which the heart is unable to supply sufficient amounts of blood and oxygen to the body. CHF can result from conditions that weaken the heart muscle, cause stiffening of the heart muscles, or increase oxygen demand by the body tissue beyond the heart's capability to deliver.  Approximately five million patients in the U.S. suffer from CHF which is one of the most common reasons patients 65 and older are hospitalized.^[1]

About NanoCor Therapeutics, Inc.

NanoCor Therapeutics, Inc. is a privately held biotechnology company focused on treating cardiovascular disease at the molecular level using proprietary vector platforms and genes. NanoCor intends to provide patients currently suffering from CHF with an effective, safe, and minimally-invasive treatment.

Learn more at nanocorthx.com

References
[1] Go AS, Mozaffarian D, Roger VL, Benjamin EJ, Berry JD, et al. Heart disease and stroke statistics—2013 update: a report from the American Heart Association. Circulation. 2013;127:e6–e245

SOURCE NanoCor Therapeutics, Inc.

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