Portola Pharmaceutical’s Andexxa – The Next Big Hit


Portola (NYSE: PTLA) has developed AndexXa, a drug that has shown to reverse the effects of a factor Xa inhibitor.


Factor Xa inhibitors are a relatively new class of blood thinners; Eliquis and Xarelto are such products. Doctors used to prescribe warfarin as the primary blood thinner of choice in the past, but it had issues as well. Patients would have to get frequent blood tests to adjust the dosage and would also have to stay away from foods that were high in vitamin K – a sad day for leafy, green vegetables. With the introduction of factor Xa inhibitors, doctors could then make choices where the couldn’t before. But of course, factor Xa inhibitors have their own issues as well.

One major issue that arises is when a patient stumbles or receives some physical trauma that causes internal bleeding. Currently, there is no known remedy that reverses the effects of factor Xa inhibitors. This makes treating such patients particularly difficult. In the United States alone, there are roughly 100,000 annual cases of patients who were treated with factor Xa inhibitors who experience some sort of bleeding episode.


Enter AndexXa.

AndexXa works by binding directly to the factor Xa and disabling the ability for other drugs to inhibit it. Studies have shown that 37 out of 47 patients achieved and sustained proper hemostasis – stopping of bleeding – while on AndexXa. The news release on this study can be found directly on Portola’s website here.

This is huge news in emergency departments where patients can bleed very quickly internally. They end up requiring multiple transfusions and can end up experiencing strokes, seizures, and death due to intracranial hemorrhages – brain bleeding.

Though there are some risks to stopping the blood thinners, as patients can then have blood clots, the sharp risks of a patient dying while bleeding trumps the risk of thromboses.

On the Topic of the CRL

In August 2016, Portola received a CRL – a rejection – from the FDA that was related to manufacturing issues. Due to this unfortunate news, Portola’s share price plummeted as they were delayed in their drug development and also required additional funding to continue the research.

Then, Pfizer and Bristol-Myers Squibb, undoubtedly the big powerhouses in the pharmaceuticals market, granted a $50m loan to Portola.

I believe that this loan reveals the confidence these two titans have in Portola’s research and future outlook.


According to Portola’s second quarter earnings:

  • Portola had roughly $124m in cash
  • $300m in assets
  • $70m burn rate during that quarter.


With the resubmission of the NDA for AndexXa on the horizon, as well as the recent approval for betrixaban -a factor Xa inhibitor- I see a huge upside in this company.

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Disclosure: I am long PTLA.

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