PolyMedix, Inc, an emerging biotechnology company developing new therapeutic drug products to treat infectious diseases and acute cardiovascular disorders based on biomimetics, has received clearance to start human clinical trials in the United States with its anticoagulant reversing agent PMX-60056 under the Investigational New Drug application (IND) filed with the US Food and Drug Administration (FDA). PMX-60056 represents an entirely new class of drug, which PolyMedix calls a heptagonist, and is the only reversing agent for heparin and the Low Molecular Weight Heparins (LMWH) in clinical development.
The first phase-I clinical trial will assess the safety of PMX-60056. The protocol for the study involves a dose-escalation study in healthy volunteers in which each subject will receive a single dose of PMX-60056. If the first clinical study is successfully completed, PolyMedix plans to initiate a second clinical trial as a proof of concept study to evaluate both safety and efficacy of PMX-60056. The second clinical trial, also to be conducted in healthy volunteers, will involve first administering heparin to subjects, and then afterwards administering PMX-60056. This study will assess both the safety of using PMX-60056 in heparinized subjects, and will also measure the ability of PMX-60056 to neutralize heparin and normalize blood clotting time, and is thus intended as a clinical proof of concept study. Following these clinical trials, significant additional clinical studies and regulatory submissions will be required to obtain regulatory approval from the FDA and other regulatory bodies before PMX-60056 could be commercially sold.
"The clearance of the IND for PMX-60056 represents a major milestone for PolyMedix," said Nicholas Landekic, CEO of PolyMedix. "This novel heptagonist compound is the first of its kind, and represents a fundamental potential breakthrough for both cardiothoracic surgery and the treatment of bleeding problems with Low Molecular Weight Heparins. PMX-60056 is the only compound in clinical development intended to reverse the activity of LMWH's and heparin. We are proud to be the first and only company to be developing this completely new type of drug to address major clinical needs and market opportunities. We are also very proud of our productivity and efficiency, as this is our second regulatory clearance in the past three months."
Heparin is an intravenous or subcutaneous anticoagulant routinely used after surgery and during cardiothoracic procedures, such as cardiac bypass, to prevent blood clots from forming. Blood clots formed during surgery can be life threatening and cause strokes or heart attacks. After the surgical procedure, the heparin activity must be turned off, which is currently done with protamine, the only agent approved for this use. Protamine has many limitations, including difficulty in adjusting dose, unpredictable efficacy, post-operative bleeding complications, allergic reactions, and other serious potential toxicities.
Low Molecular Weight Heparins (LMWH), such as Lovenox, are a family of drugs which are used for chronic prophylaxis against blood clots in indications such as recurrent deep vein thrombosis (DVT), after hip replacement surgery, in patients that have had heart attacks, and in cancer patients. PolyMedix estimates that as many as 12 million patients annually receive LMWH drugs. Clinical studies have shown that up to 20 per cent of patients receiving LMWH's may experience clinically significant bleeding problems, with 1-4 per cent of patients potentially experiencing serious, life-threatening bleeding. There are currently no approved drugs to reverse LMWH activity.
PolyMedix has designed and is developing PMX-60056, called a heptagonist, as an anticoagulant reversing agent for the reversal of both heparin and the LMWH's. PMX-60056 has been designed to specifically bind to the pentasaccharide region which is found on both heparin and LMWH. The two clinical indications PolyMedix plans to pursue are for a novel reversing agent for LMWH, for which none currently exists, and for a drug that is safer and easier to use than protamine for heparin reversal.
PolyMedix is a publicly traded biotechnology company focused on the development of novel drugs and biomaterials for the treatment of infectious diseases and acute cardiovascular disorders.