IRVINE, Calif. & KIEL, Germany -- (BUSINESS WIRE) -- Proteo, Inc. (OTCQB: PTEO) and its wholly-owned subsidiary Proteo Biotech AG announced today: The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Elafin for the treatment of pulmonary arterial hypertension.
Orphan Drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs to treat a rare disease or condition affecting fewer than 200,000 persons in the United States. The designation provides the drug developer with a seven year term of market exclusivity upon final FDA approval. Moreover, this designation offers tax credits on certain development costs in the US and a waiver of the new drug application (NDA) user fee and opens the door to special funding opportunities, such as the US Orphan Products Grants Program.
Pulmonary arterial hypertension (PAH) is a life-threatening disease in which the pressure in a patient's pulmonary arteries becomes dangerously high. If untreated, patients have a 40% five-year survival. While the advent of new therapies has likely improved survival to approximately 60%, there remains no specific cure for the disease. Despite the treatment progress during the last two decades there is still an unmet medical need for additional treatments.
Proteo’s Elafin blocks the activity of enzymes that are involved in pulmonary arterial hypertension. This makes Elafin a highly promising compound for the treatment of the disease with a new mode of action. In preclinical studies at Stanford University, the treatment with Elafin attenuated fully developed PAH in an animal model with a pronounced and significant improvement of the vascular pathology, parameters of pulmonary hemodynamics, and right ventricular function. “In humans, the obliteration of distal pulmonary arteries leads to a severe increase in pulmonary artery pressure and subsequently to right ventricular dysfunction. Reversal of this obliteration is a key goal in the treatment of PAH. We therefore propose that Elafin treatment could be a promising option for PAH patients.” said Marlene Rabinovitch, Director of Research, Vera Moulton Wall Center for Pulmonary Vascular at Stanford University School of Medicine.
Birge Bargmann, CEO of Proteo: “We are very pleased that Marlene Rabinovitch, a leading specialist in the field of pulmonary arterial hypertension, and her team have supported this application. Elafin has now obtained orphan drug status for the treatment of PAH in the US and in the European Union which is an important milestone on the way towards the clinical development of Elafin in both regions“.
Further information on the clinical development program for Elafin
Proteo’s pharmaceutical Elafin is a copy of a naturally occurring human anti-inflammatory substance. It is a natural antagonist of the tissue destroying enzymes (proteases) that participate in the inflammatory mechanism of many diseases. Elafin’s ability to block the enzymes that cause these undesirable effects makes it a promising drug for the treatment of e.g. inflammatory lung diseases and severe reperfusion injury. The excellent tolerability of intravenously administered recombinant Elafin has already been demonstrated convincingly in a Phase I clinical trial. The outcome of a Phase II clinical trial on the treatment of postoperative inflammatory reactions in esophagus carcinoma show that intravenously administered Elafin has a very clear positive effect on the period of recovery: 63 percent of the Elafin treated patients required only one day of intensive care. All patients in the placebo group needed several days of postoperative intensive medical care. In addition, Proteo’s licensing and development partner, Minapharm Pharmaceuticals SAE, has initiated a Phase II clinical trial on the use of Elafin for kidney transplantation patients. This trial is concerned with the prevention of acute organ rejection and chronic graft injury (allograft nephropathy). A further clinical trial - EMPIRE (Elafin Myocardial Protection from Ischaemia Reperfusion Injury), a placebo-controlled, double-blinded Phase-II study with 80 patients - has been started in the third quarter of 2011. The study is being performed under the supervision of the cardiologist Dr. Peter Henriksen at NHS Lothian’s Edinburgh Heart Centre in association with The University of Edinburgh, one of the leading European universities in the area of cardiovascular research.
The company researches, develops and markets compounds for biological and medical research as well as for use as pharmaceuticals. The main focus is on anti-inflammatory drugs, in particular on the human protease inhibitor Elafin. Proteo intends to out-license selected indications and to establish international strategic alliances in order to open up new fields of application and for marketing. (www.proteo.de).
Certain statements in this news release may contain forward-looking information within the meaning of Rule 175 under the Securities Act of 1933 and Rule 3b-6 under the Securities Exchange Act of 1934, and are subject to the safe harbor created by those rules. All statements, other than statements of fact included in this release, including, without limitation, statements regarding potential future plans and objectives of the company, are forward-looking statements that involve risks and uncertainties. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those anticipated in such statements. Technical complications that may arise could prevent the prompt implementation of any strategically significant plan(s) outlined above. The company cautions that these forward looking statements and risks and uncertainties involved are further qualified by other factors including, but not limited to those set forth in the company's Form 10-K filing and other filings with the United States Securities and Exchange Commission. The company undertakes no obligation to publicly update or revise any statements in this release, whether as a result of new information, future events or otherwise.