Portola Pharmaceuticals Inc's Anticoagulant Gamechangers

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In a few years, Portola Pharmaceuticals (NASDAQ: PTLA  ) might be selling a leading thrombosis prophylactic, as well as a much needed antidote for the medication. In an effort to bring its antidote to the market quickly, the company may also benefit Bristol-Myers Squibb (NYSE: BMY  ) ,Pfizer (NYSE: PFE  ) , Bayer AG, and Johnson & Johnson. Lets take a look at the company's experimental drugs and see how this scenario could play out.

A simpler anticoagulant
Portola Pharmaceuticals may soon turn the world of blood thinners on its head. Less than a year after its IPO, the company has an oral once-daily inhibitor of Factor Xa, named betrixaban, in a pivotal Phase 3 trial for the prevention of blood clots known as venous thromboembolisms (VBTs).

After various types of surgery, patients are typically given enoxaparin as an injection in a hospital setting. The drug itself is safe, but keeping it at an effective level in the bloodstream is difficult. Due to this difficulty, the therapy is typically stopped when they're sent home. Far too often, patients then develop blood clots that pose serious health risks and require rehospitalization.

Betrixaban is a pill that can be taken once daily to continue prevention of VBTs without extensive monitoring. So far, it appears to maintain effective levels in the bloodstream without going overboard. Payers often penalize hospitals if patients are rehospitalized shortly after their discharge. If approved, betrixaban is likely to be very widely prescribed, saving health care providers a bundle.

Inhibition on... inhibition off
Trying to prevent blood clots can create as many problems as it solves. Patients are often treated with Factor Xa inhibitors, like betrixaban, suffer uncontrolled bleeding episodes or require emergency surgery. In either case, the ability to switch off the inhibition of clotting factors can save a life. Unfortunately, physicians do not have an effective switch, but Portola is developing the first.

Portola's most groundbreaking candidate is andexanet alfa, a recombinant protein designed to reverse the anticoagulant activity of Factor Xa inhibitors. Last May, this therapy became the first to...

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How Sangamo BioSciences, Inc. Is Partnering to Success

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Several years ago, Sangamo BioSciences (NASDAQ:SGMO  ) made a big splash when it began clinical testing for its novel HIV therapy. Results so far have been promising, but this therapy is still in mid-stage development. Luckily, Sangamo's proprietary discovery platform has since attracted attention and licensing deals from the likes of Shire(NASDAQ: SHPG  ) and more recently Biogen Idec(NASDAQ: BIIB  ) . Lets take a closer look at these partnerships and see if they can eventually allow Sangamo to advance its own pipeline.

Curing chronic blood disorders
When it comes to outcome-based medicine, Sangamo has a platform that should have a large market opportunity. The company owns a gene-editing technology based on zinc-finger proteins (ZFPs.) The fingertips can be engineered to bind a specific sequence of DNA. The company insists that it can permanently switch the expression of any gene on or off.

The whole idea sounds a little too good to be true, but innovative powerhouse Biogen has been convinced to the tune of a $20 million upfront payment. The companies recently announced a partnership to develop beta-thalassemia and sickle cell therapies. The basic terms of the deal leave Sangamo responsible for research and development activities until it can be proved to work in humans. Biogen Idec would take the reigns at that point, providing Sangamo with milestone payments up to $300 million and...

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