The Little Pharma That Could - How PolyMedix Was Able to Bring New Drugs to Clinical Trials

A lesson in supply and demand economics: 50% of pharmaceutical products are losing their patent over the next two years and Big Pharma doesn’t have enough products in the pipeline to replace them.

While Big Pharma’s dry pipeline can be attributed to any number of factors—increased FDA regulation, the expense and time it takes to bring a drug to market—a relatively unexamined aspect of the pipeline gap is Big Pharma’s traditional “brute force” method. That is, Big Pharma uses a scattershot approach to drug creation, synthesizing and then testing in laboratory experiments millions upon millions of compounds in the hope that one will work.

This haphazard method doesn’t come cheap: a recent study from Tufts University’s Center For Drug Development reports that it takes an average of $198 million out-of-pocket per compound to reach the FDA’s Investigational New Drug (IND) stage, and $615 million in fully burdened costs. For all the expense, however, this “brute force” approach has not been particularly successful, as evidenced by the empty product pipelines of virtually all of the major pharmaceutical companies across the industry. Mathematicians have estimated that there may be 1070 to 10200 potential drug compounds that could exist– that is, one followed by 70 to 200 zeroes – meaning that even billions of test compounds represent an infinitesimally tiny fraction of all possibilities, further argument against a “brute force” approach.

Based in Radnor, Pennsylvania, PolyMedix, Inc. is an emerging biotechnology company developing new therapeutic drug products to treat infectious diseases and acute cardiovascular disorders based on biomimetics. Rather than mimic the roughshod trial-and-error approach of Big Pharma, PolyMedix uses patented, proprietary computational drug design tools developed from the University of Pennsylvania. As company President and Chief Executive Officer Nicholas Landekic explains, rather than making millions of keys in the hope that one might work, PolyMedix’s computer aided or structure-based drug design allows researchers to take the lock apart, see how it works, then craft proper keys that fit.

As the following chart illustrates, the results of PolyMedix’s more rational approach to drug design are fairly staggering:

                   R&D + Admin empl.    Annual R&D ($ millions)        Annual INDs or equiv
Merck                  31,700                       $4,882                                     9
Pfizer                   86,600                       $8,089                                   25
PolyMedix             14                             $13                                         2

Equally remarkable, PolyMedix’s two leading drug candidates currently undergoing clinical trials—PMX-30063 antibiotic and PMX-60056 heptagonist—are wholly unique, one-of-a-kind products that are neither analogues or “add-ons” to pre-existing blockbuster drugs of the type commonly seen in the pharmaceutical industry.

In mid-August, PolyMedix received regulatory clearance to begin human trials in the United States with its anticoagulant reversing agent PMX-60056 under the Investigational New Drug application (IND) filed with the 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.

Similarly, in late-August, PolyMedix commenced a Phase I clinical study in Canada for PMX-30063, its defensin mimetic antibiotic compound. The first and only defensin mimetic compound to enter human clinical trials for systemic use, PMX-30063 represents an entirely new class of antibiotic drug. Entirely different from other antibiotic compounds currently on the market, PMX-30063 is a synthetic chemical mimic of host defense proteins, one of the oldest and most effective antimicrobial defense systems found in virtually all living creatures. PMX-30063 is the first small molecule mimetic of host defense proteins to enter clinical trials intended to treat systemic infections. The unique mechanism of action of PMX-30063 makes bacterial resistance unlikely to develop, thus addressing one of the greatest needs in medicine.