Emerging Drug Developer: Portola Pharmaceuticals

Cash-hungry Portola aims at a blockbuster market

Portola Pharmaceuticals isn't cheap to operate.

When the South San Francisco-based developer announced it had completed raising $60 million in July, executives were clear that much of that cash would go to the mid-stage development of two key antithrombotic programs: Betrixaban, an oral Factor Xa inhibitor, and PRT060128. Portola researchers had already blueprinted a second Phase II study of betrixaban, looking to recruit 500 patients in a trial that would compare it to warfarin. PRT060128 is being advanced in a planned mid-stage study involving 800 patients to evaluate its chances at becoming a successor to clopidogrel, better known as Plavix.

In both cases, the company believes it is on track to emerge with a best-in-class replacement for standard therapy. At the same time, it is advancing early-stage programs: A factor Xa inhibitor antidote as well as Portola's Syk and JAK inhibitor program for inflammation, cancer and thrombosis.

And the company believes it has potential blockbusters on its hands.

"We have significant Phase II studies planned," says Charles Homcy, MD, the CEO. When they're wrapped, Portola will have data on 1,300 patients and multiple doses in each program.

Betrixaban, Portola's lead development program, is an oral anticoagulant being studied for stroke prevention in patients with atrial fibrillation as well as the secondary prevention of stroke and heart attack. The small molecule therapy also boasts a long half life, making it more appropriate for chronically ill patients and possibly releasing providers from the kind of intense monitoring associated with the use of warfarin. Getting the data to prove that the drug is effective without presenting the same risks as warfarin would open up a multibillion-dollar market.

PRT060128, meanwhile, is an antiplatelet agent being developed for thrombosis in patients with acute coronary syndrome, the prevention of cardiovascular events in patients during and following percutaneous coronary intervention, and the secondary prevention of heart attack and stroke.

These trials won't be a quick turnaround. Portola will need about 17 months to get its Phase II data back, a point that could present the developer with a unique "value-creation opportunity," says Homcy. "The money in the bank we have would not take us to that point and leave $40 to $50 million in the bank. We do have a requirement to raise more money, which can be met. In addition, we have two other programs which are eminently and imminently partnerable."

Homcy never takes his eye off the company's bank balance.

"Having been in biotech 14 years, one always needs to do two things," he says. "One is being cautious and looking at the things outside of our control, watching the money. And the other thing that is important is clinical data. Nothing is more important than good clinical data. What we're focusing on right now is protecting the portfolio by keeping it well funded without diluting equity. If we keep control of those two issues," he adds, then Portola will be able to explore a variety of options like partnering and a possible IPO at some point.

In the meantime, Portola has found the kind of deep pocket backers it needs to fund the work through proof of concept. Portola raised $137 million in its first three rounds of venture capital as well as additional equity financing. The D.E. Shaw group, Adage Capital Management, BBT Capital Management/Apothecary Capital, Janus Capital Group and PAC-Link BioInvestors joined a group of existing investors like Abingworth and Alta Partners to raise the latest $60 million.

They're backing a company with 74 staffers, which includes 12 biologists and co-founder David Phillips, PhD, an expert in Factor Xa. Phillips and Homcy worked together at Cor Therapeutics before it was bought out by Millennium Pharmaceuticals, and continued together for some time on the R&D side of Millennium before launching Portola in 2003.

And Homcy doesn't expect to add many more employees as he steers Portola through the mid-stage straits. That's not in the budget.