FORT COLLINS, Colo., May 12 /PRNewswire/ -- PR Pharmaceuticals Inc. (PRP), announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the Company's lead compound, 2-methoxyestradiol (2ME or PulmoLAR(TM)), for the treatment of pulmonary arterial hypertension (PAH).
The FDA granted orphan drug status upon review of the application which includes very encouraging data generated in preclinical experiments and in vitro studies demonstrating the multiple effects of PulmoLAR in addressing many of the pathological processes associated with PAH. In endothelial cell cultures, 2ME can significantly reduce endothelin-1 levels and increase prostacyclin production. Cell culture data have also been generated showing 2ME is a potent inhibitor of vascular smooth muscle and endothelial cell proliferation. These in vitro effects have been translated into significant beneficial effects in various animal models of cardiovascular and renal injury. In animal models of PAH, treatment with PulmoLAR has resulted in markedly reduced vascular remodeling and right ventricular hypertrophy, and reduction of pulmonary hypertension and inflammatory cell infiltration in lung tissues.
"The orphan drug designation for PulmoLAR for PAH is an important step in our development efforts. In addition to recognizing the potential the drug offers for this indication, orphan drug designation offers benefits in terms of regulatory exclusivity, assistance with clinical development and a waiver of PDUFA filing fees," said Dr. Claude Piche, PRP's Vice-President of Clinical Development and Regulatory Affairs.
Stevan P. Tofovic, M.D., Ph.D., Assistant Professor at the University of Pittsburgh School of Medicine and principal investigator on the use of PulmoLAR in animal models of PAH, stated, "PAH is a progressive and fatal disease for which there is no cure. While the newer therapies are helping patients, we believe there may be additional benefit from therapies that target the vascular remodeling that occurs in the small pulmonary arteries of patients. Our work in various animal models of cardiovascular injury, and in models of PAH in particular, indicates treatment with 2ME markedly reduces the damage to the arteries of the lungs and the subsequent right ventricular failure. The net effect is reduced severity of disease resulting in improved survival."
The active ingredient in PulmoLAR is 2-methoxyestradiol (2ME), an endogenous non-estrogenic metabolite of estradiol. 2ME has multiple mechanisms of action, many of which are particularly relevant to PAH. In vitro studies have demonstrated 2ME reduces endothelial cell production of endothelin-1 and increases synthesis of prostacyclin. 2ME is also a potent antiproliferative agent. PulmoLAR is PRP's proprietary sustained release injectable formulation. Animal studies conducted to date suggest a single low-volume injection may provide therapeutic levels of drug for up to one month. PRP plans to file an IND and initiate its Phase I studies in 2005 to support continued development of PulmoLAR.
About PR Pharmaceuticals, Inc.
PR Pharmaceuticals, Inc. (PRP) is a privately held biopharmaceutical company focused on developing, manufacturing and commercializing bioactive compounds in sustained-release formulations. The Company specializes in injectable, biodegradable formulations and has a significant Intellectual Property position in the encapsulation of large molecules such as proteins and peptides as well as encapsulation of classic small molecules into biodegradable microparticles. PRP is applying compelling and patented technology to create a diverse range of candidate pharmaceutical products to address unmet medical needs.
PulmoLAR(TM) is a trademark of PR Pharmaceuticals, Inc.
PR Pharmaceuticals Inc.