FDA Grants Orphan-Drug Designation for Novel Terlipressin Formulation for the Treatment of Ascites

BOTHELL, Wash. and CHICAGO, Jan. 4, 2013 /PRNewswire/ -- PharmaIN Corporation and LAT Pharma LLC today announced that the US Food and Drug Administration (FDA) has granted their request for orphan-drug designation for terlipressin for the treatment of ascites due to all etiologies except for cancer. Ascites, or fluid accumulation in the abdomen, is a serious complication of liver cirrhosis. The companies' lead new drug candidate PHT101 (or PGC-C12E-Terlipressin) incorporates novel drug delivery technologies that may enable once-daily administration via subcutaneous injection dosing in chronic outpatient populations.

Chronic liver disease/cirrhosis is the 12th leading cause of death due to disease in the US, killing an estimated 27,000 people each year. Approximately 60% of cirrhosis patients eventually develop ascites. Ascites patients face a significantly increased risk of other life-threatening complications, such as spontaneous bacterial peritonitis (SBP) and hepatorenal syndrome (HRS), with a very poor prognosis of 50% survival two years after onset. Current treatments include salt-restricted diet, high doses of diuretics, and frequent paracentesis, the removal of large volumes of ascitic fluid with a needle through the abdominal wall. People with ascites are also often candidates for liver transplantation.

Elijah Bolotin, PhD, President of PharmaIN, said, "Orphan-drug designation by the FDA indicates the importance of this new drug candidate to a desperately ill group of patients. We are excited about the medical potential of PHT101, which synergistically incorporates two of our proprietary technologies designed to enable safer and more effective drug therapy: PGC-Hydrophobic Core nanocarrier-based delivery and unique fatylation that leads to a pro-drug."

"Orphan-drug designation represents a major milestone toward making PHT101 available to patients suffering from ascites due to liver cirrhosis," said John Thottathil, PhD, Chief Scientific Officer of LAT Pharma. "PHT101 would bring a novel, mechanistic approach to ascites therapy as opposed to the current standard of care involving symptom relief. Orphan status may accelerate our clinical development program. It also opens the door to special funding opportunities, such as the Orphan Product Grants Program, and provides important incentives to investors, including seven years of market exclusivity and certain tax credits."

Terlipressin reduces portal vein pressure and increases mean arterial pressure (MAP) in cirrhotic patients with splanchnic vasodilation. Increasing MAP in ascites patients could potentially down-regulate the excessive salt and water retention that leads to ascitic fluid buildup. For more than 20 years unmodified IV-bolus terlipressin has been used in Europe and Asia as rescue therapy for hepatorenal syndrome (HRS) and esophageal variceal bleeding (EVB). Scores of publications have demonstrated the safety and efficacy of IV terlipressin in hospitals, and many have suggested the medical potential of an outpatient version of the drug.

PHT101 is in preclinical testing and is protected by a US patent issued in 2011. International patent applications have been filed. The novel drug delivery technologies are covered by a separate patent estate.

About PharmaIN Corporation

Based in Bothell, WA, PharmaIN is focused on improving the efficacy, safety, and speed to market of injectable peptides/proteins (biologics) for the treatment of cirrhotic liver disease, congestive heart failure (CHF), cancer, inflammation, and other serious medical conditions. The Company has developed proprietary Protected Graft Copolymer (PGC) nanocarrier excipients (15 patent families) and other drug delivery and fatylation technologies. Unique benefits of these technologies include prolonging circulation time in the blood by protecting against enzymatic degradation while preserving drug activity; creating optimal pharmacokinetic/pharmacodynamic profiles to avoid peaks and troughs in drug concentrations; and enabling more effective targeting of cancer, inflammation, and infection sites because of the carrier's uniquely small size (10-20nm). PGC is a highly versatile nanocarrier platform as demonstrated by the improved performance of small peptides through very large proteins, both for subcutaneous and intravenous administration. For more about PharmaIN visit www.pharmain.com.

About LAT Pharma LLC

LAT Pharma was founded in 2006 with the express goal of inventing, developing, and commercializing a lifesaving new therapy for people afflicted with advanced liver disease and its deadly complications. Each member of the Chicago-based management team has decades of pharma/biotech industry experience primarily in the area of new drug development and commercialization. The company is supported by medical advisors who are worldwide leaders in chronic liver disease. LAT Pharma holds an exclusive global license to all unique terlipressin compounds arising from its collaboration with PharmaIN and is responsible for clinical development, marketing, and out-licensing of PHT101. For more about LAT Pharma visit www.lat-pharma.com.

SOURCE LAT Pharma LLC