Portola Pharmaceuticals' CEO Presents at Investor Briefing During J.P. Morgan Healthcare Conference (Transcript)

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Portola Pharmaceuticals Inc. (NASDAQ:PTLA) Investor Briefing During J.P. Morgan Healthcare Conference Call January 13, 2014 10:00 AM ET


William Lis – Chief Executive Officer

John T. Curnutte – Executive Vice President, Research and Development


Good day, everyone and welcome to the Portola Pharmaceuticals conference and webcast. This call is being recorded and all remote participants will be in listen-only mode for the duration of the event. I would now like to turn the call over to Mr. Bill Lis, Chief Executive Officer of Portola Pharmaceuticals. Please go ahead.

William Lis

Good morning, everybody and thanks for joining us, that we’re calling our off Broadway JP Morgan presentation. For those of you on the phone, we’re just up the street from the conference. I have a little housekeeping. Callers can find a copy of the presentation on our website in the IR section under events. So again, thanks for coming. 2013 was a pretty good year for Portola. We are off to a pretty good start in 2014, so I’m pleased to present an update of where we are. We’ll be having forward-looking statements today.

So we continue to build a growth Company, we expect to build an enduring Company. We discover, develop and we plan to commercialize our compounds. These are life saving medicines, we’re focused in the areas of thrombus or blood clot, and now on blood cancers, we target lucrative large markets both in the hospital specialty markets. In 2014, 2015 and 2016 we expect to report out either a proof of concept or pivotal clinical trial data.

I think we now have proven as a Management team that we can execute. Our pipeline is unique, it is robust. We think that each of the wholly owned – three wholly owned programs we have, have two opportunities. One is significantly advance the fields in which they are in and two, grab significant market share that each have unique value propositions.

Betrixaban our oral Factor Xa inhibitor, the strategy is a biomarker based patient inclusion into our Phase III clinical trial that we think has the high probability of success, we are enrolling currently into Phase III clinical trial, andexanet alfa first in class and one of a kind recombinant Factor Xa inhibitor antidote for which we own a 100% rights, even within the collaboration that we have with our partners, we expect to start pivotal Phase III studies in the first half of 2013 and report that initial data in 2013 as well, its on an accelerated pathway with breakthrough designation by the FDA. This morning we just announced yet another collaborations with BMS/Pfizer that extends the previous collaboration, I’ll talk a little bit about that.

Cerdulatinib is an exciting up in coming program, we think it’s a next waver innovation for Portola, it is a dual and single pill D cell target inhibitor, it’s a kinase inhibitor and phytokine through the JAK pathway. It acts on a similar pathway to ibrutinib or the PI3 kinase inhibitor; it has an additional mechanism of action. We believe there is – now that we’re understanding the molecular basis, some of the blood cancers in some of the mutations, that there is a pathway forward that is genetically specific.

We are in a dose escalation study in cancer patients at this point, and we expect to report out data in the first half of or in the middle of 2014 and we continue to have our collaboration with Biogen on very Syk selective inhibitors in allergic asthma. I mentioned there was a number of achievement in 2013 and that we are off to a good start in 2014. We have several value-creating milestones ahead of us in the near term; I’ll speak to them a little bit later as well.

So let’s talk about the market that drives two of our products that’s the anticoagulation market. Already there’s a blockbuster in this market. Xarelto is emerging as the second blockbuster. We expect Eliquis will be somewhat farther behind but will reach that status, as well. This is a market that will exceed $10 billion, some have estimated up to $20 billion. We think that provides two opportunities for Portola.

One, our oral Factor Xa inhibitor, this is a big enough market, multiple agents to succeed and have a robust revenue including betrixaban and as the market and adoption for Factor Xa inhibitor increases, so does the need for our Factor Xa inhibitor antidote. With respect to betrixaban, remember there are eight indications that are being pursued, eight indications that are driving this market; we’re pursuing a specific indication where none of the other agents are approved thus far, so we expect to be first into that specific indication, it is a large hospital based indication and we will speak to it.

Let’s talk about betrixaban. Again, we expect to be the first to market and what we consider the largest area of unmet medical need. The real problem in this market currently is that efficiency has been shown, but not risk safety and not without excess bleeding. We think we have the agent and the strategy to provide the efficacy that’s been seen previously, but also without excessive bleeding and we’ll speak about it and again we’re enrolling in Phase III and I’m going to give specific update as to where we are.

Let’s talk about betrixaban. Many of you have seen this slide before, but we will continue to show it probably forever, because it matters, drugs properties matter, we know in the area of thrombosis the drug properties matter. We have three unique properties that distinguish this agent as potential best-in-class agent, it is the longest and really only once daily half-life of all of the Factor Xa inhibitors, that 20 hours versus 10 hours has the low renal clearance and is not metabolized by CYP3A4, why are these properties important? Because in the area of thrombosis its all about net clinical benefit and these property just allow us to have a little bit broader therapeutic window for which the does of this agent and when you talking about frail patients, which is where we are studying it and first indication that is very, very important.

Acute medically I’ll, what is it? Again, these are patients that are hospitalized of diseases such as heart failure, stroke, rheumatic diseases, severe pulmonary infection or pulmonary disease. We estimate – it is estimated that in G7 countries that each year about 15,000 patients that are hospitalized with these diseases survive their underlying diseases and die of a pulmonary embolism. Again, we think that’s tragic, it marks a significant unmet medical need and we are going to try to solve for it where others have not been able to.

There is a standard care in the hospital well established very effective and safe therapy enoxaparin. It’s an injectable agent, only for the hospitalization, the problem is the majority of event greater than 50% of these pulmonary embolism happen outside of the hospital after the patient is discharged, this is problem that we’re solving for. The key is, we have a – that we believe an ideal agent for this patient population, but we also understand the patients now. We understand much better today than was previously, who the patients are that will benefit from these therapies.

That sets up the design of our Phase III clinical trial about 7000 patients that are randomized to either the gold standard enoxaparin or betrixaban compared head-to-head during the hospitalization period and then for about 75%, 80% of the duration of the trial we’re comparing betrixaban against placebo and again we can compare against placebo because there is no approved therapy.

So where are we? we have about 90 greater than 90% power to show a 35% relative risk reductions. So we’re well powered in the clinical trial, it’s a 35 day endpoint an established and validated endpoint for single study approval to composite of asymptomatic blood clots on ultrasound, symptomatic blood clots or blood clots that cause death over the course of 35 days.

So where we are on the clinical trial? here are the metrics, we’re now as of the end of the year greater 25% enrolled of the – about 425 plus clinical trial sites that we plan to have active we now 356, we do now expect a delay from what we had initial projected for completion of enrollment that’s due to this very enriched patient population and more so that we just didn’t get to the 400 sites by the end of the year that we thought we would. The good things as we should have over 400 sites by the time we do our fourth quarter earnings in February and at that time we’re going to update everybody on specific is what it means for the timelines for data and the NDA.

The really good news is we are getting the right patient, we can now say with confidence that when we look at the pooled blinded aggregate event rate that we are on target. And remember what we said that when we announced this clinical trail that this clinical trial will have higher event rates that what was seen before. We now can see that even though we’re a little slower than where we wanted to be, but we are certainly on the right track and that gives us a lot of confidence eventually to succeed in this clinical trial.

Who are these patients? Again, I said it’s biomarker-based. The patients with a positive D-dimer are age greater than 75. If you look at the previous data, this is from the Magellan trial with Xarelto. We know that in these two sub population very large sub groups of the clinical trial called Magellan that the event rates were almost twice that of the overall event rates and the magnitude of benefit as far as reduction of blood clot was almost twice that or are significant greater.

So we know now we are on the right track and we can have confidence that we are going to show benefit as far as preventing blood clots as has previously been shown. The question is, can you do it safely without excessive bleeding and these are the reasons again why we will believe we will succeed, about dosing again in patient selection. I put this slide up because this is the – these are the book ends for well validated markers.

But what you use for choosing doses? These are bookmarks or what I call book ends that represent thrombin generation is defined by INRs which are used – have been used for years to define not only what’s an active anti-clotting agent but what’s a safe dose for an anti-clotting agent and the key is, I don’t know – I don’t have a – can I have a pointer . Okay, I’m on slide 13 for those that are on the phone.

Okay it’s a real simple, anticoagulants thin the blood. You try to think the blood enough to prevent clots but not too much cause bleeding. This is the lower end of INR, you would want your dose to have a thrombin generation inhibition above this to prevent the clots, but not up into this area which causes bleeding which is been validated.

So when we chose the dose for betrixaban 80 milligrams twice, because we want to get the blood levels up quickly in a hospital setting, and then once daily thereafter. We can stay within that range. I talked to you about the properties; we can also stay in that range better than the other agents across patient populations, especially those with severe renal disease. That’s distinctly different than xarelto, which in this indication it’s given once a day, at a shorter half-life, cleared to a much greater extent through the kidney and this is what you see with its PD profile.

So high enough to prevent clots, but really you have to overdose it in order to get 24 hours of coverage. And in a very frail patient population that will cost you. In addition, what we know now is the exclusion criteria. Patients on high-dose aspirin, dual anti-platelet therapy, a hemoglobin less than 10 grams per deciliter or major surgery within the past three months or patients that probably don’t derive a net clinical benefit over 35 days and we have excluded them.

Those are the reasons why we think we are on a path of success. If we are successful as I have said, we have one of the most compelling value propositions in the business. This is an established market with pricing leverage of enoxaparin. They built a $2 billion market in just this indication with six to 10 days of therapy. They have established pay-for-performance measures. Our new Senior Vice President of Commercial sitting here was a gentleman that launched this indication for Sanofi-Aventis. We hope to replicate that.

What is the value proposition for Portola and for investors; this becomes a $3 billion to $4 billion market based on 35 days of therapy in 14 million patients. For patients the value proposition is prevention and morbidity and mortality associated with this condition and for payers and providers it is compelling, because we expect to be at a price discount on a daily basis to generic Lovenox or branded Lovenox in the hospital setting. And the reduction of VTE.

Within the DRC system right now, you’re penalized if patients are rehospitalized within the first 30 days. We expect to reduce hospitalizations due to VTE and the cost associated with it. What is most compelling is, as a small company we can launch this drug, commercialize it ourselves, we’ve done this before; it’s about a 100 person sales force, maybe 125. If we add the antidote on top of this which we’ll talk about maybe it’s a 125, 150 person sales force for two agents, which we think is compelling.

So let’s talk about the antidote. Again, one of a kind, remarkable molecule, breakthrough designation, it is different than anything else that’s being proposed in this area because its immediate and its reversal can be either temporary or can be sustained and that is very, very important, just look at the black box warnings for the anticoagulants. If we stop these agents for a too long of period, you know these patients are pro-thrombotic. So, if you want to reverse the agent to stop bleeding, we know you are going to want to do it, but there are some patients where you’re going to want to reinstitute anticoagulation, we think we have the best mouse trap at this point and it furthers along to do that and to do it safely.

Okay. How do we define the market? Right now again, its fairly simple we think by the year 2020 there is going to be 500 hospital visits for patients that are either on anticoagulant one of the Factor Xa inhibitors or low molecular weight heparin require surgery or have a major bleed an active major bleed and we just take the projections from the analysts and then we multiply it by the percentage of major bleeds and requirements per surgery from the Phase III clinical trials. It’s that simple math.

Another way to look at it is what being done today with warfarin since there is no reversal agent for the Factor Xa inhibitors. What’s done with warfarin? we know of the 4 million patients, a few years ago that were treated in the United States there were about 2000 IV doses of Vitamin K that were given it is an antidote, it reverse it, it can kind of – its like a checks and balances as to whether that 500,000 is real. We also know that FFT or plasma is given in conjunction to reverse warfarin as well in the immediate setting, you know that last year there were about 130,000 doses for the indications that we’re speaking of.

So I think you can kind of triangulate and see what is the number of patients that we are going to try a monetize. In the United States it might look something like that’s on the lower end of the screen, if you think about – if you think about the class of Factor Xa inhibitors as largest warfarin, if you think about the class of Factor Xa inhibitors larger than warfarin as most analyst do in combination with low molecular heparin then you start to look at the number 500,000 through the G7.

Here are the points on Slide 17 that is specific to this; this is a precious and specific mechanism of action. As I said its either temporary or sustainable, the key is this is only agent, I mean at this point we believe its really the only real antidote, because its been able to show reversal of validated markers that being the free fraction of the drug in blood or anti-Factor Xa units which is the gold standard to measure Lovenox and these agents.

We have multiple we have data from with multiple agents and we have safety data now on over 80 healthier voluntaries. I think most of you have seen this Slide before, I’ll present it again, this is the molecule and how it’s designed. Again, it’s a recombinant protein to a modifications – significant modifications made to it. One, the inability of it to assemble on prothrombinase and then removal of its catalytic domain, therefore to cannot act as a anticoagulant or a pro-coagulant its why we think we can do it safely.

Next Slide. This is the Phase II data, I’m not going into it in detail, what I’ll say is this the Eliquis data on the left-hand side of the screen on Slide 19 and the Xarelto data on the right-hand side of the screen. We are taking a look on the left-hand margin as an anti-Factor Xa units; again this is the endpoint that the FDA has said we can use to get conditional approval. Reversal of the Factor Xa inhibitors on measuring the anti-Factor Xa units.

We’ve shown now that we can do either a short brief reversal of greater than 90% with Eliquis, greater than 80% overall and greater than 90% in many patients with Xarelto, in the doses that we’ve studies. So if we need a short-duration of reversal and we’ve now shown that we can do an extended duration of reversal, which is you see by the light blue lines. So the dark blue lines is bolus and then we’ve shown that with an infusion you can extend it for a longer period of time. This is the dose finding studies from Page II, we will take the data from here and choose our Phase III registration doses that again we plan to start in the first half of the year.

Where are we with the FDA and the development plan? Again after receiving breakthrough designation in December, we’ve had additional discussion with the FDA, we know have the agreement and what the registration studies will be. And therefore we are confident we will start on-time in first half of 2014 and we will report some of the first data from the Phase III trail in 2014. Additional data will come in 2015 with BLA fling at the end of 2015.

What were still working out with the FDA is the confirmatory clinical trial, we’ve made significant progress with them, remember this is the patient trial. The registration trails will be done in healthy voluntaries as the Phase II studies will be and then under accelerated approval, the agreement is to start the confirmatory study – outcome study in patients, we’re rapping up negotiations with the FDA on that clinical trial expect to start it sometime at the end of the 2014 or the beginning of 2015 again the agreement is under accelerated approval that it must be started before you file your BLA.

And we are making progress on CMC John can answer questions on that. I’ll finish up with cerdulatinib. We believe there’s a $2 billion to $3 billion opportunity in these genetically define subtypes. Targeting not only B cell signaling but cytokine signaling via JAK we think is potential beneficial for patients that are not addressed by the other agents including Ibrutinib PI3 kinase inhibitors, rituximab. We have an ongoing, as I said, Phase I/II study. Expect data middle of this year.

How do we see the market in monetizing it? We think there is probably close to 50,000 patient that we can monetize either of the ABC subtype of DLBCL with specific NFKB define mutations, we believe there will be chronic lymphocytic leukemia patients that will still fail BTK inhibitors in PI3 kinase inhibitors based on genetic, therefore that’s how we kind of think about monetizing this. What we see is if you take a look at the Gleevec resistant CML market there was only 7000 patient. So we think it gives you some sense of the opportunity here for our agent.

Again, this is the mechanism of action. In a single pill we are taking about inhibiting two survivals single, pathways based on the B cell side where a PI3 kinase inhibitors and BTK inhibitors work through SYK which is upstream and through the JAK pathway and cytokines. We’ve shown already in cell lines that have the NFKB driver mutations that have been defined to drive tumor progression even with the newer agents that we have activity, we’ve shown that.

We’ve also shown this Slide previously that shows with patients on Ibrutinib that become resistant due do a acquired mutation at the active binding site, that we have activity as well this a particular patients that initially in the clinical trials responded well in the left-hand side of the screen you can see that the vehicle control versus Ibrutinib and lack of tumor progression or cell progression, but on the right hand side of the screen you can see that when the patient relapsed and those cells are taken out and Ibrutinib is tested again, the tumor cells become resistant, but cerdulatinib has activity.

So we think these are two areas where we’ve shown pre-clinical genetically based activity and the question is can we show that in the clinic? And that’s we’re undertaking. We’re now in the second dose cohort, we just got approval by the Safety Committee to move to the second dose cohort, we expect potential three at least four dose cohorts through the first half of this year, we will report out that data this is a combination of CLL patients, non-Hodgkin’s lymphoma.

I believe John has a few follicular lymphoma, as well, patients we might expect and if successful, if we get through this without seeing significant toxicity, then we will go to the expansion phase and that’s when we’ll really start taking and testing our hypothesis of these genetically define blood cancers and whether this drug has specific activity where other drug have not that sets up the milestone slide.

So enrollment increasing for the APEX clinical trial, a little slower than we had hoped, but we get those sites up in the first quarter of this year and we think we can get back on track, the futility analysis is going to move out, the enrollment is going – we said middle of 2015 for enrollment that will move out, we just want to give you, we want to give you specifics we want to wait till the year end earnings call, which will be in February. We’ll have real good an idea of where we’re expected to be and we’ll give specific as to what that the timeline changes are talk about months here.

And andexanet, we will report – we’ll start the Phase III in the first half, we will report out some initial Phase III data and by the end of the year we’ll also have additional Phase II proof-of-concept trials going on for indexing that as well. Lovenox we’ll report out this year, we’ll start the – what’s Daiichi’s compound? It has a new Savaysa. We will start those trials as well and then eventually we will – maybe towards the end of the year we’ll hopefully start betrixaban as well.

And again, I told you additional Phase III data, the BLA filing, the critical as we said before CMC we are making progress we have the dual track where we’re taking two processes forward, so we can launch the drug and then maximize the cost of goods overtime with a second process, we’re working with the FDA on this as well. And then as I said cerdulatinib Phase I proof-of-activity middle of the year, potential proof-of-concept towards the end of the year, early next year and then onto the next stage.

And so that’s the story of Portola, we think its pretty compelling and we thank you for your time. Are we taking questions Marty? We are taking question. Yes, Jeff.

Question-and-Answer Session

Unidentified Analyst

Thanks for the update. Could you clarify the andexanet Phase III? What’s the Phase III data that you’ll get at the end of 2014? And then what’s the additional Phase III that’s coming?

William Lis

Yes, it’s a very big question. Remember what we are going to try to de here, just like Phase II we are going to try to study bolus only, we are going to try to study bolus plus infusion. So, what I think you will see is a series of studies that are done with – and then what did we do before two? We stage the molecules, I think I can’t give you specifics but my guess is what you will see is at least one of the agents by the end of the year and perhaps some bolus information by the end of the year with one of the agents, perhaps a little bit more, we will see how the trials goes. Sufficed to say, we’ll be able to report out some of the data. It will be consistent with what we did in Phase II to start to see it come out.

In addition we said, we have the two processes going on. And what we want to do is we want to start shifting to the commercial process. The FDA has said we can use currently with our clinical process to start a trial, eventually we are going to have to produce some data from the commercial process as well before we file with BLA, I think that’s what you will see in the first half. John is that correct?

John T. Curnutte

Yes, sounds right.

William Lis

Am I pointing this correctly, I should probably let you answer the question.

John T. Curnutte

Yes. First half of 2015 we’ll be working with commercial material.

William Lis

Yes, okay. Does that answer your question?

Unidentified Analyst

Sort of, so you weren’t filed with the clinical material, so already filed with commercial material?

William Lis

Correct and that’s what is stage gating, when you see the BLA filing that’s really what is stage gating for the second half of 2015. and make sure we have not only the commercial process in place, but we’ve studied some patients with that commercial process in the Phase III clinical trial.

John T. Curnutte

Yes. I might add Jeff that certainly will use the clinical scale material in the BLA. The question is, does it really represent the actual in for the Phase III and that really hinges on a degree of comparability between the clinical scale and the commercial scale, either way we are in very good shape, because its very similar, we have a even larger data pool that we can combine with the commercial material, if they are more desperate and then the – all of the work with the clinical scale adds to the safety database. But it’s still very readily doable because we’re in healthy volunteers, to do all of work that’s necessary once the commercial material is available for the BLA.

William Lis

Yes it’s really our way of using…

John T. Curnutte

Either way…

William Lis

The program. And we’re really trying to derisk the program here, because as we said, where is the risk starting to fall to. And we’ve been very upfront starting to fall at CMC, we are confident John can talk about where we’re building our confidence and how we are making progress. We hope to have a data set that includes what clinical process, the new commercial process that John said a bigger data set. If we are some reason not and we still have a filable process with this commercial and then we have to start. And that’s why when we talked about that Phase IV confirmatory study, that’s why we want to give ourselves some wiggle room there, too. I think we want to make sure we start certainly with the confirmatory, now we have a process that we agreed to with the agencies of commercialization.

Unidentified Analyst

Sorry Bill. So there’s a possibility that the clinical data that accumulates this year is the dry run for the clinical data that comes with the commercial process next year?

William Lis

Well we hope not, we hope it’s not a driver, I mean hope its part of the BLA as far as the similarity of the things, but it could be. Yes it’s like kind of a risk mitigation.

Unidentified Analyst

And you commented in your presentation about, for betrixaban, you are going to lower the price of generic Lovenox.

William Lis


Unidentified Analyst

Where do you aim to be with andexanet?

William Lis

We have such a wide range, you know we look at – so on the low end -- and I don’t mean to be trite about it, but on the low end you take a look at biologics or what the Kcentra has been priced at, you can say $4,000, $5,000, $6,000 that’s the low-end. On the high-end we look at this antidote, Voraxaze, for methotrexate its $65,000. so b know we’re somewhere in there, right we know we are somewhere in there, I just can’t say today if we are in the middle going this way or down here. I think it will depend on the clinical data where we start to see the market and the real need, is it broader or is it close here and what the value proposition that comes out of our clinical data, I think it’s really important.

So we want to do the work around that I think we still have quite a bit of time before we have to make that final decision. We’ve brought Mark onboard, he is doing some work there, we are not asking him to speak specifically about it, but I think we’ll get there and be able to give some guidance, I mean you know perhaps next year at this time we will start giving guidance on price. In the mean time, it’s a really big broad range and we just – yeah I don’t think more.

Unidentified Analyst

Last question related to that. Can John’s commercial process give you a gross margin that’s drug –like [ph] at the low-end of that pricing range for andexanet?

William Lis

Yes I think we’ve not stated what it will be, our guess is yes, but we haven’t stated what it will be, but we are confident if John’s commercial process will allow us to have a – start to build a profitable drug, but again we said we ant to maximize cost of good, we’ve always said through the second process that will come probably 18 months after we nailed down the initial commercial process. If you remember in our earnings call and Marty was that August? We said we’ll have a commercial launch with a certain cost of goods and about 18 months later we’ll have another cost of goods that will optimize it. I think that’s how we would like to put it today, but as you know we are going to continue to try to be transparent and give updates as they come and we do work. Is that fair, John?

John T. Curnutte

Very fair and its also important to point out that the follow on second generation process is in active work right now so the two first and second generation process development programs being run in parallel which enables us to minimize the length of time we’ll be on the marker with the first generation and to be able to follow with the second generation.

William Lis

So this is where our risk is, but we are confident, I mean we are making really good progress, I think we are feeling pretty good and we are going to get there, its just going to take us some time as we said and hopefully as we’ve said the CMC process will catch up to the accelerated development process. Remember we are going to go from IND to Phase III in 24 months, so we have some catch up work to do. any other questions? Yes.

Unidentified Analyst

Congratulations on having a path forward to the regulatory filing. If I could just confirm, this will be a normal, not inpatients, for the initial filing?

William Lis


Unidentified Analyst

You’ll follow that up later. How big do you expect this trial to be? And do you have to look at each individual anticoagulant to get the claim?

William Lis

Yes. So the second question. We believe we have to study them all, the molecules are similar but they are a little bit different, so we think we have to study them all, how big will the trial be, what we said before is somewhere between – each one is somewhere between 30 and 50 patients, 25 and 50 patients and we give our selves a little bit of a wiggle room for the point that John said – or as Jeff, I think, his comment – are you doing a dry run or are you pulling the full data set in, because you can see the end to statistical significance we don’t need many, all right its that striking. So we’ll be in healthy volunteers it’s been agreed to with the FDA, but one thing I want to just correct you and you said the healthy volunteers for the filing. It will be healthy volunteers for the data, then there will be – we will be required to at least submit some patients all right, we’ll have to start it so there will be some, you don’t know what that number will be but it will be some patients and again that’s defined as in accordance with the FDA accelerated approval. Right, go ahead ask.

Unidentified Analyst

So, once you’ve started in patients you don’t know how many there will be, but they will have to see that data before they’ll give you approval.

William Lis

Yes, they will have to see that data, I mean again it’s not going to be, this is not data where we are going to be showing the efficacy or even the PD extent. I think it’s a fact that you start the trial, I think they are probably looking to see if anything catastrophic happens in patients that is different. The good things is they will let us submit that data all along during the filing process. So you know you file and even during the course of that they will allow us to submit more data. I have to say its – I have to give this agency credit, I mean this has been pretty collaborative, so there is a lot of ways in which it’s going to – the whole goal of collaborative is, what are all of the metrics that you can push on to try to move the drug to the markets in there.

Unidentified Analyst

And what’s the trigger or the gating factor for actually starting it in patients?

William Lis

Yes, so we want to get our commercial process down and then we have to have agreement on the trial and get it built. Again, we hope to do that in the next few months and then be able to start it whether it’s the end of this year or early next year hope to be able to start it, we think that’s a pretty low bar, we should be able to do that.

Unidentified Analyst


William Lis

Yes it should be, it could be, but again we want to – we are looking at the CMC process, because we could start that with some of the other material too and then move into the commercial process, but we’ll just – we have some flexibility here. Is that accurate? I got a thumbs up. Okay any other questions?

Again thanks for coming to our off Broadway presentation here at JP Morgan and we’ll look to see you around through the rest of the year, we appreciate your support.


Ladies and gentlemen this concludes today’s Portola Pharmaceuticals event. You may now disconnect.

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