Portola Pharmaceuticals Inc (PTLA) Corporate Presentation Transcript

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Andexanet Alfa last year, not only positive Phase 3 data for submission of the conditional approval but additional Phase 2 data with edoxaban and enoxaparin, and now, we see the initiation and agreement with the FDA on the Phase 4 trial was a significant accomplishment.

Cerdulatinib, you will see even more data today of the positive responses we are starting to accumulate as we increase the dose. From a corporate standpoint, we are very successful with an additional follow-on last year and the indefinite commercial manufacturing agreements that we did with both CMC Biologics and Lonza, taking a dual-source approach to not only mitigate risk but allow us to launch on time and then extend the product globally. This is an important slide because it talks about the amount of leverage we have in the thrombosis space with our oral factor Xa inhibitor and andexanet.

On the left hand side of the slide on Slide 6 is the current projections for the novel oral anticoagulant, now expected to achieve $13 billion as a franchise. We see the market in two spheres, from two sides. One, how do we compete in the oral factor Xa inhibitor market with our differentiated oral factor Xa and the betrixaban? We see that opportunity by going into a specific indication, being the first into that market and being standard of care, that is acute medically ill. A $3 billion to $4 billion indication in and of itself and then also the antidote. The antidote is an important component of the anticoagulant market. At this point, we are ahead of the competition and have the potential and have the only and first true antidote onto the market next year.

To give you a better sense of where this market is going, and again, it drives both the opportunity for betrixaban and the antidote, we look on the side of the screen here. Currently, a drug like Xarelto has five indications already.Pradaxa,edoxaban, and apixaban of BMS Pfizer are looking to catch up to that. There are future indications as well. All this evolves into the future, but there are additional indications that our competitors are pursuing as well. Again, we are wellahead by years of our competitors in acute medically ill. At this point, only J&J and Bayer have made an indication that they are going continue to pursue this indication.

Again, what do we talk about when we say acute medically ill? We are really talking about 24 million patients in the G7 that currently have an indication in the hospital or prevention of VTEs, where the medical need is pretty simple. Despite the use of Lovenox and injectable agents this year in those territories, there will be over one million blood clots and 150,000 death, just in this patient population.

We start to accumulate this amount of deaths on an annual basis and then you start to put even now in the G7. That is more death than you see in the combined hepatitis, AIDS, and the combination of all the cancer indications. That is a pretty big human health issue that we are tackling in acute medically ill.

Why do we think we will succeed? The properties of betrixaban are unique. This is not just from a clinical standpoint, from a commercial standpoint if we do get there first with differentiating outcomes. This drug really has the only true once-daily half-life. That is very, very important from an efficacy and safety standpoint. Lowest renal clearance and not metabolized by CYP3A4. It is just a more stable drug when you talk about blood concentration as being an important parameter of defining or determining efficacy and safety. These three characteristics are clearly differentiated from all of the other agents.

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