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

CHAPEL HILL, N.C.,  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 atMount 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."

Source: NanoCor Therapeutics, Inc.