CTI BioPharma Corp.
Q1 2015 Earnings Call Transcript

Published: 7 May 2015

[Operator:]

Good day, and welcome to the CTI BioPharma First Quarter 2015 Financial Results Conference Call. Today's conference is being recorded. At this time, I would like to turn the conference over to Monique Greer. Please go ahead, ma'am.

[Monique M. Greer:]

Thanks, Melissa, and welcome, everyone, to our First Quarter 2015 Financial Results Conference Call. This press release reporting our financial results can be found on the home page and in the Investors Section of our corporate website at ctibiopharma.com. Following formal remarks by management, the conference call will be open for questions. Joining me today are Jim Bianco, President and Chief Executive Officer; Matt Plunkett, Executive Vice President of Corporate Development; and Lou Bianco, Executive Vice President of Finance. Before beginning, please note that during the course of the call, we will be making forward-looking statements based on current expectations. Such forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that may cause actual results to differ materially from those anticipated by the forward-looking statements. Additional information concerning these risks and uncertainties is contained in the press release reporting our financial results for the first quarter 2015 in the Risk Factors section of our Annual Report on Form 10-K for the year ended December 31, 2014, and in the company's other periodic reports and filings with the Securities and Exchange Commission. I will now turn the call over to Jim.

[James A. Bianco:]

Thank you, M. Good afternoon, everyone. Thank you for joining the call today. Let me begin by stating how pleased we are with the momentum that's building amongst the key opinion leaders in the myelofibrosis community, regarding the PERSIST-1 Phase III results for pacritinib, which is leading up to one of the most important oncology congresses of the year. As you know, in early March, we announced positive top line results and that PERSIST-1 randomized Phase III trial met its primary endpoint and the intent to treat population. And with data in hand, Dr. Ruben Mesa, a co-principal investigator for Mayo Clinic, along with Claire -- Dr. Claire Harrison on the -- co-investigator on the PERSIST-1 trial, submitted a late-breaking abstract to the American Society of Clinical Oncology, or ASCO meeting. And recently, Dr. Mesa was informed that not only was his abstract accepted for presentation, but is also going to be featured as a part of ASCO's press briefing on pacritinib amongst the oncology community and the public at large. To put this in perspective, Dr. Mesa's presentation of pacritinib Phase III results is 1 of 4 hematology abstracts selected for the press briefing on Saturday. This is out of over 5,000 abstracts that were submitted to the meeting. So unlike other myeloproliferative neoplasms, namely PV or ET, myelofibrosis is the most aggressive of the MPNs, with a prevalence of about 18,000 patients in the U.S. And unlike other blood cancers, myelofibrosis is generally a chronic disease and, like their patients, physicians struggle to find effective therapies and are always looking for new treatment options, particularly for patients with low platelets who struggle with cytopenias, namely thrombocytopenia and anemia, as a result of their disease. Our goal for the PERSIST program was to evaluate pacritinib in 2 randomized Phase III trials in patients with myelofibrosis. That would represent a true mix of patients that healthcare providers see every day in their practice, not just better risk patients with normal blood counts, but rather the 40% of patients that present a particular challenge to managing their symptoms and their disease with current therapies. While PERSIST-1 focused on a cross-section of patients irrespective of their baseline blood count, it did exclude patients with prior JAK2 inhibitor therapy, which is how the PERSIST-2 trial complements the patient population in PERSIST-1. PERSIST-2, you may recall, is targeting patients with platelet counts below 100,000, which was, as you also know, the population that was excluded from the COMFORT Phase III studies. But in this trial it will include patients with prior JAK2 treatment and allows ruxolitinib to be used as the best available therapy. If the physician chooses to do so and the patient, obviously, is randomized to the BAT arm, the dose of ruxolitinib in the BAT arm is per the product label, which is a maximum of 20 milligrams per day. And we believe PERSIST-2 has the potential to demonstrate that pacritinib is superior to ruxolitinib in this patient population, as well as addressing the significant differences in treatment-emerging anemia and thrombocytopenia between these 2 agents, data which is now validated from the PERSIST-1 studies, especially in patients with low platelets. And just keep in mind, platelet counts -- normal platelets counts range from 150,000 to 400,000. So we believe in the potential pacritinib to provide potential relief, not only to the patients with fairly stable disease, but also for this 40% of patients with myelofibrosis who have mild life-threatening disease-related thrombocytopenia. Recall the latter definition includes platelet counts below 25,000. Just to help characterize, again, the patient population in the PERSIST-1 trial, 44% of our patients who received pacritinib had platelets counts below 150,000, with a median platelet count of 168,500 and a lower end of the range at 7,000 platelets per microliter. There are other important risk factors and risk differences factor -- differences between the PERSIST-1 and prior Phase III trials like the COMFORT studies. Notably shorter time from diagnosis, the percentage of patients who were JAK2 negative, or who had primary myelofibrosis, in addition to a high proportion of patients with circulating blast counts greater than 5%, patients who had baseline low hemoglobins below 10 milligram -- 10 grams per deciliter or had severe fibrosis on the grading -- on their marrow biopsies. Each of these are considered important risk factors, both for disease progression and for survival. Doctors -- Dr. Mesa's presentation of the full data set at ASCO will provide further insight into the results from the PERSIST-1 trial, from which it will become evident why ASCO selected these results for their press program. What encourages us most by the results of PERSIST-1 is the significant percentage of patients who were able to receive full therapeutic doses of pacritinib over long periods of time, irrespective of their baseline platelet count or the baseline red blood cell count, while in addition to having therapeutic benefit in spleen volume reduction and disease-related symptoms, an improvement in transfusion dependency with regards to red cell transfusions. We are calling pacritinib the next generation multikinase inhibitor with specificity for JAK2 in FLT3, because we believe there is a unique opportunity for pacritinib to address the broader patient population than is currently being served. We can achieve our goal by helping more patients in offering a potentially safer, more tolerable drug. So we're really delighted to build on this positive momentum, as we anticipate completing enrollment in the ongoing PERSIST-2 Phase III trial in the second half of this year, and plan to start a regulatory submission to the FDA and/or EMA on target in late 2015. Our partner Baxter, as you may recall, is responsible for the submission of the MAA. Recall that pacritinib has Fast Track designation by the FDA for the treatment of intermediate and high-risk myelofibrosis, including but not limited to patients with disease-related thrombocytopenia. And this is addressed by the PERSIST-1 patient population. The Fast Track designation also includes patients experiencing treatment-emergent thrombocytopenia on other JAK2 therapy, or patients who are intolerant to or whose symptoms are suboptimally managed on other JAK2 therapy. And this is the patient population we're targeting in our PERSIST-2 study. Looking beyond myelofibrosis, we believe that pacritinib's kinome profile suggests its potential therapeutic utility in a myriad of blood-related cancers, including AML, MDS, CMML and even mantle cell lymphoma and certain solid tumors where JAK-STAT dysfunction is associated with the disease. A separate preclinical study showed that pacritinib overrides thrombo-mediated resistance in FLT3 ITD-positive AML cells, which is believed to be an important mechanism for resistance to FLT3 antagonists in treating AML. And we also have data that is synergistic with commonly used agents in the treatment of AML. The notion of combining an active non-myelosuppressive agent with another drug that may, in fact, be myelosuppressive, is obviously very attractive to many hematologists and oncologists. We're confident that with our [ph] partner Baxter, we will be able to more rapidly advance the development and availability of pacritinib for patients around the world with myeloproliferative neoplasms, AML and other blood-related cancers. So at this point, I'd like to turn the call over to Matt to give you an update on our collaborations with Baxter and Servier. Matt?

[Matthew J. Plunkett:]

Thanks, Jim. First of all, let me say that our colleagues at the bioscience division of our partner, Baxter, soon to be called Baxalta, are very optimistic about the opportunity pacritinib may offer to patients and the physicians who treat them. As you might imagine, while pacritinib has not yet been submitted for regulatory approval to the FDA or the EMA, in anticipation of the potential opportunity, the teams from both our companies are focused on developing sales and marketing plans, ramping up commercial and medical affairs teams on a global basis and looking forward to the next indications where pacritinib could best potentially help patients. Our partner tells us that pacritinib is one of the most important products in the Baxalta pipeline. Our other partner, Servier, a private company with $5-billion-plus in revenue, continues to build upon their knowledge of PIXUVRI, and has started promotional activities in some of their European territories, including France, Italy, Ireland, Netherlands and Spain. Servier is expecting reimbursement in most of its European markets by mid-2016. Together we continue to raise awareness of PIXUVRI in the EU. And physician experience with this therapy continues to grow. Our 2 companies will be well-represented at the upcoming European Hematology Association Congress and the International Congress on Malignant Lymphoma, both in June, as we continue to educate physicians through symposia and other medical education activities. We believe that having Servier's commercial and medical teams active in the field, will significantly enhance recognition of the power of PIXUVRI, given their significant resources and the enthusiasm for this drug. Finally, site opening for the European component of the PIX306 confirmatory trial is nearly complete, and patient enrollment has accelerated. We are targeting completion of enrollment for PIX306 by year-end of 2015. Tosedostat is the third promising product candidate that I wanted to briefly mention. Tosedostat is a first-in-class product candidate, an oral inhibitor of aminopeptidases, a family of metalloenzymes that clip and free up amino acids from peptides and polypeptides, allowing them to be the building blocks for new proteins, essential to tumor cell survival. In 2014, we acquired worldwide rights to tosedostat and assisted in the initiation of a Phase II trial evaluating tosedostat plus low-dose cytarabine for older patients with AML or high-risk MDS that is being conducted by the UK's National Cancer Research Institute AML Cooperative Group. Later this year, we expect to report whether tosedostat met the initial efficacy hurdle to enter into the Phase III component of this pick-a-winner trial. Depending on the results, we would plan to consult with the FDA and the EMA regarding the potential registrational strategy that could begin as early as 2016. Clearly, tosedostat fits nicely in our portfolio of novel agents, specifically targeting blood cancers. Now I will turn the call back to Jim to review the financials and upcoming milestones. Jim?

[James A. Bianco:]

Thanks, Matt. So let's look at our financials for the first quarter ended March 31, 2015. Total revenues for the quarter were $2.7 million compared to $1.4 million for the same period last year, of which PIXUVRI net revenues, product revenues made up about $800,000 compared to $1.3 million for the same period in 2014. The decrease in PIXUVRI sales year-over-year was in part due to fluctuations in the foreign currency rate. Additionally, also due to a surge in patient starts that, after reaching agreement on pricing with the German Health Agencies in December of 2013, we did receive a larger wholesale volume from Germany in Q1. So that accounted for the variance that we've seen. The GAAP operating loss for the first quarter 2015 was $27.5 million compared to operating loss of $27.7 million for the same period in 2014. Noncash share-based compensation expense for the first quarter was $4.3 million compared to $7.8 million for the same period last year. Net loss for the quarter was $28.6 million or $0.16 per share compared to a net loss of $29 million or $0.20 per share for the same period in 2014. As of March 31, cash and cash equivalents totaled $44.4 million with total outstanding shares of approximately 180.2 million. So as I stated at the start of our call today, we are really excited about the upcoming full data presentation of pacritinib at ASCO by Dr. Mesa. In addition, we anticipate, again, reiterating the achievement of several objectives that we stated for 2015. So beginning with PIXUVRI, we're focused on completing enrollment in the PIX306 study by the end of this year, initiating a number of additional studies together with our partner Servier, restating our guidance for 2015 sales in the $10 million to $12 million range. Moving on to tosedostat. Matt noted we plan to report an update from the investigator-sponsored Phase II randomized trial of tosedostat in combination -- plus with low-dose cytarabine in older patients with high-risk MDS or AML. And we believe that, and data that's being presented at the EHA meeting from a group in Italy will actually define a registrational path for tosedostat in AML and MDS. And then, of course, for pacritinib, completing enrollment in the second -- in the PERSIST -- second Phase III trial PERSIST-2 in the second half of this year, commencing a regulatory submission to the FDA or EMA as early as late in the fourth quarter of 2015. And then of course, advanced pacritinib development program in a host of other hematologic malignancies in collaboration with Baxalta by year-end 2015. We believe pac has the potential to be the next big advancement in the treatment across the entire spectrum of patients with myelofibrosis. And not only for patients with treatment- or disease-related anemia thrombocytopenia, but also for patients with less-advanced disease and normal blood counts. And we look forward to sharing the additional data from this study, as well as completing our other studies as we bring this through closer to becoming available for patients in need. So at this point, this would end our formal presentation. And we would like the operator to open the call for some questions.

[Operator:]

[Operator Instructions] We'll take our first question from Charles Duncan with Piper Jaffray.

[Charles C. Duncan:]

Also on the upcoming ASCO press briefing, that's kind of -- that's pretty notable. I wanted to ask you about the PERSIST-1 data that you might anticipate at ASCO. Is it possible that you might be able to point to some of the activity for patients who've been on the drug longer? Or perhaps when could we see 36- and 48-week data from that study?

[James A. Bianco:]

We would probably use the 48-week data for the ASH presentation. And as we said on the initial call, we continue to see as study -- as our study progresses that the treatment effect size continues to increase over time. So we're not concerned about it. None of the KOLs had facts to the contrary. Everybody felt that the drug was extremely active in an extremely high-risk patient population and may, in fact, be disease-modifying. That needs to be defined, but the reception to the data to date has been unanimously overwhelming. So we're really excited by it and can't wait for that presentation on May 30.

[Charles C. Duncan:]

Yes, I'm looking forward to it as well. Jim, given that differentiated activity. I guess, I wanted to ask you about a potential for filing for a breakthrough therapy designation. It would seem to me that at least in the patient population, who have very low platelet counts, this would be potentially a breakthrough.

[James A. Bianco:]

You know, Matt -- Charles, I agree with you 100% because I was supposed to write the intro for that, which I haven't done yet. You're correct, I think people -- not people, the experts look at the data and recognize that whatever signaling mechanism that's inhibited by a JAK1/JAK2 inhibitor is not being affected with this agent. And that in fact is not just differentiating, but opens up a whole host of patients who could otherwise benefit from the drug. So we agree. We think that, that recognition should deserve breakthrough therapy. But obviously, that's an FDA decision.

[Charles C. Duncan:]

Okay. But my question is would you -- is there a time when it makes sense to you that you might consider requesting that or filing for it?

[James A. Bianco:]

Yesterday. Not that we did it yesterday, but, yes, as soon as we can do it.

[Charles C. Duncan:]

Okay, okay. That's helpful. And then -- go ahead, excuse me.

[James A. Bianco:]

I mean, your point's well-taken, because it will help expedite any regulatory submission review and approval potential.

[Charles C. Duncan:]

Okay. And then my last question is, and I think you addressed this on your -- in your prepared remarks, at least towards the end. But what's your perspective on a potential approve label? Would it be focused on -- or would an indication be focused on a restriction based on some clinical criteria, such as low platelet counts? Or do you believe that it makes sense to file for a broader label and just include the studies as you will have done and let that speak and be in the label?

[James A. Bianco:]

It's probably premature to have a discussion regarding what the label will look like. Clearly, the label will be different with 2 trials than it would be with the 1 trial. They address different questions in the Fast Track designation population. But I think it was encouraging the Fast Track designation that we received back from the FDA did not restrict in the language Fast Track for just patients with low platelets.

[Charles C. Duncan:]

Okay, that's helpful. And final question is, any particular milestones? I know, you mentioned several things that you plan to do with your partner, Baxter. But are there any financial cash flows that we can anticipate over the course of the rest of the year from Baxter?

[James A. Bianco:]

Yes, so we -- I think we gave a little guidance at the beginning of the year that we anticipate earning the $20 million milestone for the PERSIST study, $12 million for the MAA submission, and we have another $10 million that we anticipate from TRISENOX sales. So we're in that range. I think, we said somewhere between $45 million and $55 million in terms of the revenue cycle. When it comes in is, obviously, the fluctuation on the P&L side of it. But we're pretty confident that it -- that he will earn out those milestones this year.

[Operator:]

We'll take our next question from Bert Hazlett with Ladenburg.

[Robert Cummins Hazlett:]

Has the data for PERSIST-1 had any effect on enrollment rates for PERSIST-2, and/or the timing of that study? Has the discussion had any material effect one way or another there?

[James A. Bianco:]

It's probably early to tell, Bert, but we're pretty -- I could tell you, amongst the investigator group, who've all received the top line data, and amongst the steering committee that's seeing essentially the whole presentation, there's a lot of excitement. So the answer is enrollment has picked up substantially. In fact, it's now exceeding what we saw at our PERSIST-1 study on a monthly basis. The misstep that the CRO had was really getting all the sites open in time. One of them got acquired and then the other got acquired, and now the one that is the best one for us has done a great job in essentially playing catch-up. So we're pretty confident that this isn't an end of the year event, given the enrollment rate right now and how much, how far into enrollment we are. But it's not going to be kind of a June-July time frame, but it won't be that much further after that.

[Robert Cummins Hazlett:]

Okay. And in terms of the discussions at ASCO, obviously, as much as you might be able to answer this question ahead of that. But there's obviously going to be a focus on the JAK2 activity and the FLT3 activity to molecule. How much discussion do you expect about the activity at other kinase and, let's call it, the broader kinome profile of the pacritinib molecule?

[James A. Bianco:]

At ASCO probably not much, right? But at ASH, in the hematologic space, probably a lot more, because, again, there's preclinical work that's being done in CLL and CMML, mechanistically in terms of looking at signaling for EPO and TPO. So all of those pieces of data starting to come together should be available for at least abstract, if not presentations, at the end of the year in terms of the ASH meeting. Jack, is there anything [indiscernible]?

[Jack W. Singer:]

No. There'll be some publications starting to appear in the second half of the year that really are exploiting the other aspects of the kinome, particularly the CSF1 receptor and IRAK1, which are both very interesting targets. And the preclinical data is quite exciting on those. We can't go into it more. They'll probably be submitted and certainly will show up as abstracts at ASH.

[Robert Cummins Hazlett:]

Okay. And so just a quick follow-up on that one and then I have one more. But so additional data presentations for pacritinib beyond myelofibrosis, we should be looking to publications and to ASH for a focus there?

[James A. Bianco:]

Absolutely.

[Jack W. Singer:]

Absolutely.

[Robert Cummins Hazlett:]

Okay. And then, Jim, in your opening remarks and in the press release, we talk about -- you discuss the proportion of patients in PERSIST-1 that have low platelets and the unmet need in those patients and the concern of physicians with those patients and the potential utilization of pacritinib there. How do your discussions with physicians inform you with regard to patients that are being driven to that level, to low platelets, as a result of other treatments? How do we think about that patient group? Not those at baseline, but those that are being driven due to other treatments, other JAK2 inhibitors potentially?

[James A. Bianco:]

Yes. So I mean, it's going to -- at least my experience having just been to one ad board in Europe. So it differs geographically. The European arena, the patients that are selected for ruxolitinib, typically have normal amount of polices [ph]. They're typically in pretty good shape in terms of other comorbidities, et cetera. They don't really have profiling on high-risk disease. And in their hands, they tend to see patients stay on therapy for 12 to 18 months before they start to get into the issues with the treatment-related, the emerging anemia or thrombocytopenia, or their disease starts to progress, and they can no longer offer them the therapeutic doses of rux. The IMS data in the U.S. would suggest that half of the patients in the U.S. are being treated on subtherapeutic doses, meaning 20 milligrams or less per day. And that's pretty much -- that's everybody, that's not just the disease-related thrombocytopenia, that is to manage the duration of therapy, even if it's just for mild symptom control, or mild spleen-size reduction. So the experience, I think, in the U.S. clearly is different than what it is outside of the U.S., and we heard that loud and clear. And amongst those physicians, everybody unanimously had the same opinion that this is not only a much -- would be a much welcomed choice for myelofibrosis patients, but they were really astounded by the degree at which you were able to treat from the lowest of low platelet patients to what they've seen in their COMFORT studies. And almost everybody at this meeting was an investigator or a PI on the COMFORT trials. So I think -- and Jack's had some experience with that recently with non-COMFORT investigators. And unanimously, people are excited about the profile.

[Jack W. Singer:]

If I could just make one addition. If you look at the patients that we're able to -- that are being recruited for the U.S., for the PERSIST-2 trial, most of those will have had prior rux, and they're improving very well. And so there is a substantial proportion of patients out there with fewer than 100,000 platelets, and to be eligible for the trial, they have to have a fairly large spleen and be symptomatic. So I think that there really is an unmet need, which everybody is seeing, particularly for those patients with compromised platoplesis [ph] that this drug will really meet that need.

[Operator:]

We'll take a question from Ren Benjamin with H.C. Wainwright.

[Reni J. Benjamin:]

I guess, Jim, just talking about the enrollment completing by the second half of 2015, so I'm assuming kind of late third quarter or maybe early fourth. You also mentioned that you would be filing with the MAA at the end of this year as well, and I would have thought that -- the filings for both the U.S. and the EU would have been pushed out to sometime in the first half of 2016. Can you just help me understand the discrepancy?

[James A. Bianco:]

Yes. And I think it's pretty clear that Baxter, Baxalta, is pretty impressed as well with the trial data. And I know that they are having or plan to have discussions with the rapid tours [ph] about moving forward on just a single study.

[Reni J. Benjamin:]

Okay. So based on PERSIST-1 they may go ahead and start that filing in the EU.

[James A. Bianco:]

Right. And then for us, we will have all of the components with the exception of the analysis of PERSIST-2, so we can start our rolling submission under the Fast Track guidelines that start to get the other NDA modules review underway. And so that would, obviously, save us some time.

[Reni J. Benjamin:]

Okay. And then, I guess, just -- if you can remind me, in the PERSIST-2 study that's ongoing right now. I guess, 2 things, one, do you have a breakdown or an idea of how many patients in the -- that are coming in, might be treated with rux versus some other best available therapy? Or do you think the majority of the patients are being treated with rux in terms of the controller?

[James A. Bianco:]

No, sir. It's pretty much behaving what we had -- the assumptions that we made coming into the trial. So we, again, we said this before, we know about half the patients have platelet counts of 50,000 or less. So those patients are not typically going to be eligible for ruxolitinib per the label, unless they start on labrux [ph] and fall below 50,000, where they can try to taper, but that's the exception. Think about 30%, 35% of patients are coming out to the study on rux. And then, of course, they get randomized 2 to 1, so obviously, you have twice the chance of getting on pac than you do on BAT. And then for the BAT arm, we know the dose of rux is per the label or lower. So again, as we expected, nobody is going to try to be aggressive with that agent. The median dose is, what, 10 milligrams a day?

[Jack W. Singer:]

5 milligrams to 10 milligrams.

[James A. Bianco:]

Yes, 5 milligrams to 10 milligrams. So again, clearly subtherapeutic from the perspective of the pharmacology for that agent.

[Reni J. Benjamin:]

Okay. And can you just remind us what's the difference in the 24-week response rate that the trial is powered to detect?

[James A. Bianco:]

We never got into the specifics. We can say that it's significantly overpowered. And we just -- the same way we were pretty much on target for the PERSIST-1 study. And Ren, just -- no, I mean for clarity, if you look at the pharmacology section for the basis of approval that the FDA published, which you can get online, you will see that at 20 milligrams a day, no patient, 0, will hit 35% reduction in spleen and 50% reduction in symptoms. And so we just told you that the median dose is 10 milligrams, patients going for 5 milligrams to some getting 20 milligrams. And so we don't think that the treatment arm are going to reach the threshold to be counted as an endpoint. And that was obviously the primary objective of the study. We are doing PK-PD on these patients as well.

[Operator:]

[Operator Instructions] And we'll take a question from David Lebowitz with Janney Capital.

[David N. Lebowitz:]

Just a kind of follow-up on the prior question, with respect to the PERSIST-2 trial. Since there is the requirement regarding thrombocytopenia and platelet counts, will there be the type of numbers for patients on rux to actually provide a direct statistical comparison between rux and not the remainder of the best available therapy? Or will they be certainly included as one group together?

[James A. Bianco:]

They'll be analyzed separately in terms of that choice of best available therapy. If you recall that we mentioned this on prior calls, there's different patients streams. There's going to be a patient population that has had prior JAK2 therapy and come in and not on JAK2 therapy. There are going to be patients who are on ruxolitinib who've developed thrombocytopenia from the drug. And when they get randomized during the wash-out period, if their platelet counts rebound above 100,000, they are stratified separately. And they can go back on ruxolitinib. Obviously, that's treatment-emergent thrombocytopenia, which is why they would be -- had come to the trial, because they have had symptoms, a large spleen and low platelets, suggesting that, at a tapered dose of ruxolitinib, it's not very therapeutic for them. And then there is the patients who come in with treatment-related thrombocytopenia on low dose rux, who still have a big spleen and a lot of symptoms. And all of these patients get randomized BAT, and then again, as I mentioned, it's the physicians' choice if they want to put them back on rux. And of course, we will analyze the BAT group on an ITT as well as on a subset. And there should be adequate power to have some data that would be able to compare directly on duration of therapy, tolerability, spleen size reduction, symptom control on these doses of ruxolitinib for patients in that sub-100,000 platelet population.

[David N. Lebowitz:]

It's good to know. To jump over to PIXUVRI, given the fluctuation in the recent quarter and the currency behind it and things of that nature, how should we think of sales going forward for the rest of the year?

[James A. Bianco:]

So, David, we're pretty confident on the $10 million to $12 million sales -- total sales for the year, which would be a big step up from -- what did we do last year, Lou, $7 million? A lot of this is, again, on this investment stage that we're currently doing with Servier, so there was a little bit of out-of-the-field activity in the first quarter. If you look at the -- just the FX alone was a 25% hit. But if you actually look at demand, Q1 last year over Q1 this year, the demand numbers are actually up, say, on an encouraging trend from the demand perspective.

[Operator:]

[Operator Instructions] We'll take a question from Bert Hazlett from Ladenburg.

[Robert Cummins Hazlett:]

Jim, I just would like to clarify the filing strategy for both the EU and the U.S. as it stands now. I know Charles asked the question a little bit earlier. But the filing strategy for the EU is for a single study PERSIST-1 to be submitted? Or am I incorrect in that thinking?

[James A. Bianco:]

No. We believe, based on our discussions at a team level with Baxalta, that they are exploring with the health regulatory agency in Europe, the potential to file off of the PERSIST-1 study alone, obviously, with PERSIST-2 coming in for subsequent approval in the future, if it's positive.

[Robert Cummins Hazlett:]

And is that being pursued in the U.S.? Or is it still required to have both PERSIST-1 and PERSIST-2 for the U.S.?

[James A. Bianco:]

We have not had that discussion with the FDA yet. So I really don't have an answer for that.

[Operator:]

That concludes today's question-and-answer session. Dr. Bianco, at this time, I will turn the conference back to you for any additional or closing remarks.

[James A. Bianco:]

Thank you. Thanks, everyone, for joining us on the call. As always, we appreciate your support, and look forward to seeing many of you at the upcoming Investor Conferences in New York and, importantly, at the ASCO scientific meeting. And if you're traveling to Europe at the EHA, which will also have data from the pacritinib study that will be different than the data that's presented at ASCO. But thanks, again, for your interest in CTI.

[Operator:]

This concludes today's conference. And thank you for your participation.