CTI BioPharma's (CTIC) CEO Jim Bianco on Q2 2014 Results - Earnings Call Transcript

Aug. 4.14 | About: CTI BioPharma (CTIC)

CTI BioPharma Corporation (NASDAQ:CTIC)

Q2 2014 Earnings Conference Call

August 4, 2014 4:30 p.m. ET

Executives

Monique Greer - SVP of Corporate Communications & IR

Jim Bianco - President & CEO

Matt Plunkett - EVP of Corporate Development

Lou Bianco - EVP of Finance

Jack Singer - EVP of Global Medical Affairs & Translational Medicine

Analysts

Bert Hazlett - Ladenburg

Kim Lee - Janney Capital

Debjit Chattopadhyay - ROTH Capital Partners

Ren Benjamin - H.C. Wainwright

Operator

Good day, ladies and gentlemen, and welcome to the CTI BioPharma Second Quarter 2014 Financial Results Conference Call. Today's conference is being recorded.

And now, I'll turn the call over to Ms. Monique Greer. Please go ahead, Ms. Greer.

Monique Greer

Thanks, Kelsey, and welcome everyone to our second quarter 2014 financial results conference call. The press release reporting our financial results can be found on our homepage in the Investor Section of our corporate Web site at ctibiopharma.com. Following formal remarks by management, the conference call will be open for question.

Joining me today are Jim Bianco, President and Chief Executive Officer; Matt Plunkett, Executive Vice President of Corporate Development; Lou Bianco, Executive Vice President of Finance; and Jack Singer, Executive Vice President of Medical Affairs and Translational Medicine.

Before we begin, please note that during the course of the call we'll 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 these anticipated by the forward-looking statements.

Additional information concerning these risks and uncertainties are contained in the Risk Factors section of our Quarterly Report on Form 10-Q for the quarter ended June 30, 2014 and in the company's other periodic reports and filings with the Securities and Exchange Commission.

I'll now turn the call over to Jim.

Jim Bianco

Thank you, M, and good afternoon everyone. As most of you know, CTI is a biopharmaceutical company with a marketed oncology product and an exciting late-stage pipeline. Our focus is to become a leader in the development and commercialization of new treatment for blood-related cancers.

At the end of May, we announced the renaming of the company from Cell Therapeutics to CTI BioPharma. And as I mentioned before, we made this change at a defining moment in our company's history as we believe it better reflects our vision and expertise. From the company's inception, we feel our expertise in blood-related cancers has enabled us to identify new more effected target cancer therapies to potentially improve side-effect profiles that address unmet medical needs.

Today the advancements we've made with respect to PIXUVRI and Pacritinib are really a testament to this. PIXUVRI is, as you may recall, a first-in-class aza-anthracenediones with a unique structural and physiochemical properties. Our recent preclinical study show that unlike the class in anthracycline-related drugs PIXUVRI is unique in its mechanism for inducing tumor cell death being a potent DNA Alkylator and inducing mitotic instability, supporting the thesis that it is the first approved drug in a new class of anti-cancer agents.

Based on the efficacy and safety profile and its pivotal trial and the clear unmet medical need and aggressive B-cell non-Hodgkin lymphoma, PIXUVRI was the first therapy approved in the EU for the treatment of patients with this blood cancer. Prior to PIXUVRI, patients with aggressive B-cell NHL who had failed two or more prior lines of therapy had no approved treatment options, specifically for this stage of disease.

Earlier this year, NICE published its final guidance recommended for the prescription of PIXUVRI in England and Wales for patients with aggressive B-cell NHL who failed two or three prior lines of therapy. NICE's determination of PIXUVRI is cost-effective in the treatment of patients with multiply relapsed aggressive lymphoma as had an impact across the EU, where many companies look to the rigorous process conducted by NICE as an endorsement for cost effectiveness, and therefore reimbursement in their own country.

Since receiving reimbursement, we started to see stronger demand for PIXUVRI from physicians who have experience with the drug, and others who may have been exposed to recent Advisory Boards or other Medical Affairs Educational Outreach programs.

In July, we announced that the Dutch Healthcare Authority and the Health Insurance Board of the Netherlands approved funding for PIXUVRI as an add-on drug for patients who need a third and fourth-line treatment options of patients with aggressive B-cell NHL. The inclusion on the Dutch list of reimbursed drugs makes PIXUVRI the first registered and reimbursed medicine for the treatment of aggressive B-cell NHL in the Netherlands.

As interest in this new therapy grows, we continue to expand the availability of PIXUVRI to healthcare providers, who treat patients with this late-stage disease.

We recently received approval from the Israeli Ministry of Health for PIXUVRI as monotherapy for treatment of adult patients with multiply relapsed aggressive lymphoma. Neopharm will be distributing and marketing PIXUVRI once it is included in the Israeli National Health Basket of drugs.

So as of today, PIXUVRI is available in 11 countries. That's Austria, Denmark, Finland, France, Germany, Israel, Italy, Netherlands, Norway, Sweden and the U.K. And we have achieved reimbursement decisions in England, Wales, France, Germany, Italy and the Netherlands.

As we ramp up our medical education programs and field activity in various markets, one of our stated business priorities is to generate sufficient sales to achieve a net positive contribution margin for PIXUVRI by the fourth quarter of this year. And if we're able to achieve that goal, then we believe that PIXUVRI commercial effort would be profitable going forward in 2015 and beyond.

Now that we have secured reimbursement and we're seeing renewed interest in potential partners for PIXUVRI in markets where CTI does not have nor plans to have a commercial presence. As such, one of our corporate goals for 2014 is to secure partner to help to expand the product reach and commercial potential of PIXUVRI in territories outside the U.S. And we've made good progress towards this objective, and we hope to be in a position to update you later this year.

So in summary, we continue to see greater adoption and more consistent use by PIXUVRI by healthcare providers as they gain experience with the drug. We're encouraged by the increase in demand, particularly in the U.K. Since our launch there in April, we believe PIXUVRI has the potential to become the second salvage treatment of choice for patients with aggressive B-cell non-Hodgkin's lymphoma.

As we previous described, in connection with the approval of PIXUVRI and an agreement with the European Commission, we're conducting a post-marketing commitment trial known as the PIX-R or PIX306 Study. We continue to bring on-sites in the EU to supplement enrollment to help us reach our goal of completing enrollment in 2015. I know this trial is intended to support the conversion of our EU conditional approval to full approval.

Over the next year, we also intent to initiate a significant number of what we termed "signal-finding clinical trials" across a variety of non-Hodgkin's lymphoma patient populations, and more broadly in hematology either by ourselves or in conjunction with leading investigators in Europe. We believe that these signal-finding studies may expand the opportunity for PIXUVRI, and look forward to sharing those results with you in the future.

Now I'd like to spend a few minutes on our lead investigational program, Pacritinib. Pacritinib, as you recall is an oral tyrosine kinase inhibitor that targets two important activating mutations, JAK2 and FLT3. The activating mutations of JAK2 are implicated in certain blood-related cancers such as the myeloproliferative neoplasms or MPNs, leukemias and certain solid tumors.

FLT3 is a gene commonly found mutated in patients with acute myeloid leukemia or AML. Pacritinib not only inhibits the internal tandem duplication or ITD, but also the A35Y point mutation commonly associated with resistance to Type 2 FLT3 inhibitors.

Based on Pacritinib's efficacy and tolerability profile demonstrated to-date, the current pivotal trial program includes two randomized Phase III trials known as PERSIST-1 and PERSIST-2 for patients with intermediate one, two or high-risk myelofibrosis.

We're obviously happy to report that we recently completed enrollment in the PERSIST-1 pivotal Phase III trial, which enrolled patients with myelofibrosis without limitation on their starting platelet counts. This achievement triggered a $20 million milestone payment from Baxter, which was received this month. This trial is comparing the efficacy and safety of Pacritinib with that of best available therapy, which is any physician selected treatment other than JAK2 inhibitors.

The primary endpoint of the PERSIST-1 trial is the percentage of patients achieving a 35% or greater reduction in spleen volume from baseline at week-24 as measured by either MRI or CT. And the secondary endpoint is the proportion of patients achieving a 50% or greater reduction in their total symptom score from baseline to 24 weeks as measured by tracking specific symptoms on a form. We anticipate reporting top line results from PERSIST-1 in early 2015.

PERSIST-2 is our ongoing study that's evaluating the safety and efficacy of Pacritinib in patients with low platelet counts, who have moderate to severe thrombocytopenia, meaning patients whose platelet counts are less than or equal to 100,000.

This trial will evaluate Pacritinib compared to best available therapy. But unlike PERSIST-1, in this trial the control arm allows for approved JAK2 inhibitors that are dosed according to the product label for myelofibrosis patients with thrombocytopenia. This trial is expected to enroll approximately up to 300 patients in North America, Europe, Australia and New Zealand.

The PERSIST-2 trial is being conducted under special protocol assessment with the FDA. And we expect that the data from PERSIST-1 and PERSIST-2, if positive, will form the basis for an NDA submission in late 2015 with an MAA submission following shortly after in Europe in 2016.

Pacritinib has a kinome profile unlike any of the other JAK2 inhibitors, providing a rationale to explore its activity across a variety of blood-related cancers. For example, we had encouraging clinical data in lymphoma and preclinical data in FLT3 resistance AML. Pacritinib is currently been evaluated in AML through an ongoing investigator sponsor trial in the U.K. We're excited about the interests we received from investigators in cooperative groups to study Pacritinib in other blood-related cancers in the future.

We're confident that together with our partner Baxter, we'll be able to more rapidly advance development and availability of Pacritinib for patients with myelofibrosis, AML and possibly other cancers around the world.

I wanted to briefly mention to see this stat, which you may recall as in oral selective aminopeptidase inhibitor that is in Phase II development. Unlike normal cells, transformed cells are very dependent on their ability to recycle intracellular proteins, make new proteins and survive. The scientific interest in tumor autophagy has grown over the last decade, following the clinical successes observed with interrupting protein recycling at the proteasome complex.

Attention has now turned to pathways downstream to the proteasome complex that are responsible for removing and recycling essential amino acids off peptides. One novel target for intervention are aminopeptidases, into our family of metalloenzymes that clip and free up amino acids off of peptides and polypeptides, allowing them to be the building blocks for new proteins, essential for tumor cell survival.

At the American Society Hematology meeting last December, researchers reported positive interim results from an investigator-initiated Phase II trial of tosedostat in combination with either cytarabine or decitabine and nearly diagnosed all the patients with AML or high risk Myelodysplastic syndromes patients or so-called MDS. There are several ongoing Phase II cooperative group sponsored trials and IFTs evaluating the activity of Tosedostat in combination with standard agents in AML or MDS and data from the in signal finding trials may help form the appropriate design for a Phase III study.

During the second quarter we announced the U.K.'s National Cancer Research Institute, AML cooperative group initiated a randomized Phase II trial evaluating Tosedostat plus a low dose cytarabine for older patients with AML or MDS.

Now, I'm going to briefly review our financials for the second quarter and the six months period ended just 30th, 2014. Till the second quarter total revenues were $1.3 million that compared to $306,000 for the same period in 2013. Total revenue for the first six months of 2014 was 2.8 million versus the 1.4 million for the same period last year.

The increase in revenue year-over-year is primarily attributable to an increase in net product sales of PIXUVRI. The non-GAAP operating loss, which excludes non-cash share-based compensation expense for the second quarter of 2014 was $21.3 million. The GAAP operating loss for the second quarter was 26.7 compared to 17.9 million for the same period in 2013.

The increase in GAAP operating loss primarily related to the ongoing PERSIST-1, PERSIST-2 Phase III clinical trials and also included $5.4 million of non-cash share-based compensation expense for the second quarter of 2014 compared to 2 million for the same period of 2013.

For the second quarter total operating expenses were $28 million compared to 18.2m for the same period in 2013. The increase in the OpEx was primarily related to clinical development cost related to the persist one and persist two Phase III studies for Pacritinib, and as previously mentioned the $5.4 million of non-cash share-based compensation expense for the quarter.

The net loss for the first six months ended June 30th, 2014 was 56.4 million. As of June 30th, CTI had cash and cash equivalents of $33.2 million. And as I mentioned previously this month we received a $20 million development milestone payment from Baxter following completions of enrollment in the PERSIST-1 pivotal Phase III trial on Pacritinib.

As per our financial guidance for 2014 we continue to expect our loss from operations to be in the range of approximately $45 million to $50 million excluding any non-cash share-based compensation expense, which is as you know a non-GAAP measure. For the primary factors comprising such guidance, you can refer to our press release reporting our financial results for the second quarter of 2014.

So in summary, it's been a very productive first half of the year for CTI. We are excited about the prospects for helping patients and building value for our shareholders. And just to quickly recap our near-term priorities, these would include reporting top line results in early 2015 from the first of two Phase III trials of Pacritinib, continuing to complete the enrollment, opening up sites and enrolling patients in the second Phase III trial, the so called PERSIST-2 study; evaluating Pacritinib and additional indications outside of myelofibrosis, continuing to grow EU sales of PIXUVRI in relapsed or refractory-aggressive B-cell lymphoma with the goal of having a positive net product margin contribution by the end of this year.

And then lastly, pursuing a global development and commercialization collaboration whereby CTI would seek to retain rights to PIXUVRI in the European countries in which we currently market the drug and in the U.S.

Before opening the call to audience, I want to thank our current shareholders for their support, also our employees for their collective passion and dedication to improving the lines of patients with blood-related cancers.

This concludes the formal presentation, and operator, let's open the call for some questions if there are any?

Question-and-Answer-Session

Operator

(Operator Instructions) Our first question will come from Bert Hazlett with Ladenburg.

Bert Hazlett - Ladenburg

Thank you. Thanks for taking the question, and congratulations on the progress, Jim and everyone. In terms of PIXUVRI initially, you've made some good headway in terms of the ability to gain reimbursement in addition to gaining the approval in a number of countries. How rapidly do you think that translates into revenue growth?

Jim Bianco

Bert, as we've been saying from the beginning, this PIXUVRI is really a second half of the year story for us. I think there is a lot of groundwork that was laid both on the education side and as you mentioned, the reimbursement side, the add-on status in the Netherlands; all of these are really positive catalysts.

I mentioned briefly we were pleased and quite frankly surprised at the uptake rate in the U.K. So I think that's a good biomarker if you will that when the drug is considered cost effective and now reimbursed since it is the only proved therapy, we're seeing the uptake in these. And so we're cautiously optimistic that the second half of the year, Q3 and Q4 will be on the forecast that we had hoped so that we can meet that target essentially breakeven by Q4 for the commercial entity for PIXUVRI.

Bert Hazlett - Ladenburg

Okay, thank you. And then I just had one or two on Pacritinib. In terms of the R&D spend 14 million for the quarter; that was in excess of 12 for the first quarter. How should we think about the R&D spend specifically as it relates to Pacritinib going forward and through the remainder of 2014?

Jim Bianco

Sure. Good question. And I'll have Lou take that one.

Lou Bianco

Yes. We could expect it to continue as we expand the trials for the PERSIST-2 program. And as we do the follow-up also on the PERSIST-1, so I think it would pretty much run along the same; incurring the expenses along the same rate as you saw at the first half of the year.

Bert Hazlett - Ladenburg

Okay. And then I think we've discussed before it, Jim, the potential for Pacritinib in settings beyond myelofibrosis. And in particular you're looking at AML with the molecules. At what point do you move from the ISTs and into formal programs, is that going to be host initial Phase III study in myelofibrosis or initial Phase III results, excuse me, or is that further down the road?

Jim Bianco

So I think between -- obviously this is a joint effort with Baxter and our colleagues, and so the -- I'll let Jack chime in a minute, but in essence, there is an agreed upon plan for the first set of ISTs. As you start to get a signal that the drug was promising, then obviously the company takes the development direction forward in those particular indications. So, Jack, do you want to talk a little bit about some of the translational work that you're currently contemplating with regards to other ISTs for Pacritinib?

Jack Singer

Right. Just a couple of words about Pacritinib as an AML drug and what makes it very interesting. As you know, there have been other drugs that have targeted FLT3 that have not been overly successful in clinical trials. What's unique about Pacritinib is the fact that it targets both JAK2 and FLT3 in one drug. And JAK2 regulation is one of the ways that resistance occurs to other FLT3 inhibitors as well as what Jim had mentioned earlier, that Pacritinib not only targets the ITD mutation, but several of the common resistance mutations. And we'll expect we'll have some data on this presented at ASH and these are from the pre-clinical side of this.

We have an ongoing study in the U.K. right now looking at patients who have relapsed following standard therapy who have FLT mutations looking at mono therapy with Pacritinib. And we expect that this should give us some idea of how efficacious this would be as a single agent by the end of the year.

We're planning on starting a number of frontline studies in not only AML with and without FLT mutations to look at the additive effect of Pacritinib on standard therapy which such drugs has the hypomethylators. We would expect by some time next year we will have a fairly good sense both from a pre-clinical perspective as well as a clinical perspective on how effective Pacritinib is likely to be in AML, and whether it justifies the Phase III program.

Bert Hazlett - Ladenburg

Thank you. That's very clear. Thank you for the color. I appreciate it.

Operator

(Operator Instructions) We'll move on to Kim Lee with Janney Capital.

Kim Lee – Janney Capital

Good afternoon. Thanks for taking the questions. Two questions for you here on PIXUVRI, can you give us a little more detail on why sales went down slightly compared to Q1? So that's the first question.

And then the second question is on Pacritinib, I believe in the first quarter earnings call you've got into PERSIST-1, top line PERSIST-1 data in the second half of this year, and it sounds like now it's going to be early 2015. Can you give us some detail on why the delay? Thanks.

Jim Bianco

Yes, sure. So on the fixed sales; one of our distributors typically buys in every two months. That happened in Q1. That didn't happen in Q2 despite the fact that demand was actually higher in Q2. That was just a timing issue. So has that buying occurred and sales in Q2 would have been higher than it were in Q1. I wouldn't focus so much on that as it's clearly not a trend. We look at actual new patient starts and demand and we continue to be encouraged by what we see, and specifically as these new countries come online like the U.K. and now the Netherlands.

So we are confident that the trend is in the correct direction even if that wasn't reflected by the actual net sales numbers that were recorded for Q2. With regards to Persist-1 our initial target was to complete enrollments in April. That was based on 270 patients and that would have put to have top line data by the end of the year. If you recall, Kim, based on discussions with the FDA, we increased the sample size to approximately 320 patients. So that additional 50 patients from the 270 initial took us the extra two and a half months to complete the enrollment and that pushes it out by the proportionate amount into early 2015.

Kim Lee – Janney Capital

Okay, great. Thanks for the clarity.

Jim Bianco

Sure.

Operator

Debjit Chattopadhyay with ROTH Capital Partners has the next question.

Debjit Chattopadhyay - ROTH Capital Partners

Hey, Jim, thank you for taking the question. I'm just thinking about Pacritinib as it compares to Ruxolitinib. When you try and analyze what should be the expectations for the PERSIST-1 and PERSIST-2 trials in terms of the number of patients who have greater than 35% reduction in spleen volume, Ruxolitinib had 42% and 28% in the two trials. Given that you have a very different patient population being enrolled in this in the PERSIST-1 and two trials, is there a correlation between the level of thrombocytopenia as baseline versus the spleen volume, and accordingly what should we be benchmarking in terms of spleen volume reduction percentages?

Jim Bianco

Yes. That's an interesting question. One would expect that patients who have lower platelets have more severe disease, have the disease longer and their -- so their spleens are up likely going to be larger than patients for example who have such a compensated marrow function. What percentage, at the end of the day we need to have the proportion of patients achieving 35% reduction in spleen volume being greater on Pacritinib than it is on the control.

And then we are pretty confident based upon all of the analyses that we did on the Phase II data both by subset, the patients with low platelets as well as by patients with essentially the normal platelet range that the percentage reduction was approximately comparable across the subsets even though patients with low platelets may start out with larger spleens than the respective cohort of patients with normal platelets.

I think it's important to note we had this discussion with the PIs on the study. Just looking at the demographic of PERSIST-1, the consensus was that this patient population is the most reflective of the actual patient community that they see on a day-by-day basis that is not essentially a representation of a clinical trial artifact, but rather a real life everyday practice sample of patients. I'd like to set INT-1s to high risk with the portion of patients that have low platelets being almost overlapping with what is reported in the CR data or the MPN data on the portion of patients who were thrombocytopenic.

So we think that this will answer a very important question that to-date has not been answered in a randomized controlled fashion.

Debjit Chattopadhyay - ROTH Capital Partners

Thanks, Jim, for the clarity. One more question on the PIXUVRI, based on the data at hand right now, what do you think is the typical duration of therapy for patients on PIXUVRI?

Jim Bianco

I'd say that for patients who are receiving it in the third and fourth line setting that it is not dissimilar to what we saw in the clinical study itself.

Debjit Chattopadhyay - ROTH Capital Partners

Thank you so much, Jim.

Operator

We'll now hear from Ren Benjamin with H.C. Wainwright.

Ren Benjamin - H.C. Wainwright

Hi. Good afternoon, guys. And thanks for taking the questions. Just a couple, I guess, one just following up. On PERSIST-2 -- when we look at PERSIST-1 since its best available therapy excluding JAKs it's pretty easy to figure out what's the delta that we should be looking for in PERSIST-2 since the majority of the controlled patients will be on JAK [5s] (ph). Can you just help us understand how we should be thinking about that delta? Does the trial still win if it's comparable? Or does it need to be statistically better? And at least based on your analysis that difference come about due to dosage reductions in the control arm or how should we be looking at the way that trial might win?

Jim Bianco

So the trial wins if it has a proportion of patients who achieve the end point means statistically significant from the best available therapy arm. But, Ren, you have to put this in the context of what our patient inclusion criteria. So for a patient to get on the study they have to have a 100,000 platelets or less. They have to have a big spleen and they have to have a minimum certain toll of symptom score ID, they are pretty symptomatic.

So if you are on Ruxolitinib and you have low platelets, you have a big spleen and you're very symptomatic. That's not a very good therapeutic response for Ruxolitinib. And that is either because they're on a sub therapeutic dose meaning the less than optimal dose that was studied up to 5 mgs or 10 mgs of b.i.d or they are developing thrombocytopenia as a treatment emergent side effect of the drug and therefore they are tapering their dose and their symptoms in spleen responses are rebounding back.

When we did the sample size analyses we took what is in the literature, but what's seen for Ruxolitinib at 5 b.i.d or 10 b.i.d recognizing of course that that was a single arm Phase III dose titration study. So we feel comfortable that the best available therapy arm will not be superior to Pacritinib. Pacritinib will meet it primary endpoint in the trial.

Ren Benjamin - H.C. Wainwright

Got it. Okay. Just as a follow up, potential expansion in the PV, do you have any thoughts on that especially based in the JAK [5s] (ph) potential expansion into that indication.

Jim Bianco

That, we haven't had any formal thoughts; we've had some discussions with some key opinion leaders about what is the real unmet medical need in (indiscernible), and Pacritinib potentially address that. So it's something that is certainly under considerations, but with no hard plans to pursue it at present. But I think as one starts to look at how this drug's profile is different than other JAK2 inhibitors. There is the whole host of other diseases in blood-related cancers that the biology would make much better sense for going after, was that a true unmet medical need in terms of managing those hematologic malignancies. And we will get a better sense of that as Jack mentioned, with the AML data, but more importantly when the kiname profile itself is published and we start to see the types of diseases that the drug should be biologically active in.

Ren Benjamin - H.C. Wainwright

Got it. And just switching gears to see the stat with this pick a winner design that's ongoing right now, could you give us any color as to when enrollments may complete, when we might see some initial data?

Jim Bianco

I think it'll be too earlier this year to say. I mean it is a large -- obviously this is a cooperative group that does the majority of AML patients in the European theatre, so to speak. So we think enrollment will be robust, but with regards to data that is a follow-up period obviously for this patient population.

Jack Singer

Yes. Just to add a little color to that. This trial started to enroll a couple of months ago and it's only picking up steam. The initial analysis at this point is when 60 patients have completed therapy on the Pacritinib on a controlled arm, excuse me, and there is a good chance there by the end of the year given their past grow records. So I'd expect that possibly by EHA, next -- European Hematology, next year we might have -- we'll be able to give an answer. That would be the earliest we'd expect to have data.

Ren Benjamin - H.C. Wainwright

Terrific, thanks for that clarity. And just a couple more questions; one, we notice that the SG&A was slightly lower than the previous quarter, is there any changes in the sales force or any changes regarding people at the firm that we should be aware of?

Unidentified Company Representative

No, I just think we're very cost conscious, and we're just looking at it that way.

Ren Benjamin - H.C. Wainwright

Okay. And then, just one last one on Opaxio; Jim, do you have any update as to when the next interim look may occur? And then, we know that Opaxio is obviously been evaluated in a maintenance therapy at the frontline, but given that Avastin has a PDUFA date in November for the second-line patient population, is there anything with that approval that could potentially change how we should view Opaxio at all?

Jim Bianco

I don't think so. Ren, if you recall the frontline of Avastin data as maintenance in varying cancer similar population to what's in the GOG-0212 did not show a benefit on survival, it prolonged CFS, and it had an adverse event rate of G.I. preparation in bleeding, which was alarming, which is why Avastin in the frontline setting was not recommended even in the editorials that accompany that publication.

In a relapse setting, again, I think maintenance in frontline if you hit survival I think Opaxio will be the drug of choice just on an ethical basis, because again it's the first large maintenance study that does show up benefit on prolonging overall survival that would be a first in that disease since the 80's, with the addition of platinum to Taxane, the platinum-based regimen. So we see them as co-existing.

Ren Benjamin - H.C. Wainwright

Got it. When might we see the next interim? And when do you -- do you have any update as to when the final results might be available?

Jim Bianco

No. As you know, it's event-driven, what we get is every six months or so. We get the GOG composite based on the event rate. We don't think that we're going to have an analysis this quarter or this half. So we haven't heard from the GOG firmly, but our bet is they typically do their analyses around their annual meetings, which one just passed and the second one is next year. So I don't think we'll see data this year.

Ren Benjamin - H.C. Wainwright

Excellent. Thank you very much, and goodbye.

Jim Bianco

Okay, thanks, Ren.

Operator

And we'll move on to Bob (indiscernible) Investor Capital.

Unidentified Analyst

Hi, thank you for taking my call. One of my questions is answered, I've got two others. One is can you help us anyway if you can, how should to model the second half of the PIXUVRI sales? You mentioned it's going to be a second half story, but how should we think about -- is it going to be twice, double, triple levels on what do we have now?

Jim Bianco

Yes. Since we haven't really provided guidance, I feel I'm pretty reluctantly to set an expectation that it would double or triple, etcetera. Internally, we know what our spent is and we know what the revenue run rate would have to be to offset that spend. So, it was neutral to positive on margin contribution perspective. And then obviously there are some other elements we talked about forming a global partnership.

So, all of those things, Bob, are variables that would influence whatever answer I gave you today. So we prefer to stick with the guidance that our goal is to get at the breakeven by Q4, and have it be profitable as a standalone entity in 2015 and beyond.

Unidentified Analyst

The other questions is on (indiscernible), and you're talking about some rationale for lymphoma and FLT3 mutation AML, I think you're talking about in details about why it makes sense FLT3 mutation AML, can you share what you have more for lymphoma?

Jim Bianco

There is a -- if you go on the Web site under our publication scientific meetings etcetera, there is a paper that a Phase I study that was conducted in a variety of aggressive (indiscernible) lymphomas that showed a pretty good signal. Jack, if you want to talk about …

Jack Singer

Yes. This is a study out of Wendy Anderson by Dr. Younas, who has published the journal Clinical Oncology I think in 2012, and it showed interesting activity of mono-therapy with almost no toxicity; very interesting study.

Unidentified Analyst

Okay, thank you.

Operator

And we'll take a follow-up from Bert Hazlett.

Bert Hazlett - Ladenburg

Thank you very much. Just a quick follow-up on Ruxolitinib, and it's potential with regard to additional indications. You have Ruxolitinib with myelofibrosis and PV being considered. Are there -- and we spoke about the FLT3 and the AML opportunities there, but are there additional indications for Ruxolitinib more broadly that Novartis and Insight are pursuing that it make sense that are on the shortlist, maybe for Pacritinib as you move forward beyond AML and beyond the FLT3 activity, but straight in the development scheme for Ruxolitinib?

Jack Singer

Yes. I'd rather comment on the Ruxolitinib development scheme. I think it's important that you let the biology direct where you investigate these agents. JAK1 JAK2 kiname profile is distinctly different than the JAK2 FLT3 and the other kiname targets that we hit, and again, these are in similar low nanomolar concentration.

So, our development path would clearly be distinctly different for the most part from those diseases that are not just JAK2 mutation-directed, like PV or MS, so it wouldn't make sense for us. For example, the JAK1 component is an important action for the JAK1/JAK2 molecule for us to pursue those types of diseases if we don't have JAK1 inhibition activity.

Jim Bianco

I'll just say we're taking a very broad approach to the potential spectrum of activity for Pacritinib in hematologic diseases as well as solid tumors, and with the approach being driven by the potential for some of the pathways that Pacritinib interferes with have a major effects in solid tumors, particularly those with selected mutations. And I think until more information is available, that's really all I'd like to say about at this point.

From a liquid tumor perspective, the unique profile of Pacritinib is that it does not cause either anemia or thrombocytopenia. It allows you to explore it in disease where cytopenia is part of the disease, and it is extremely interesting in NDS, and another hematologic neoplasm which I think it's going to be very specific for chronic myelomonocytic leukemia, again, accompanied by significant cytopenias. And we'll be exploring those in both preclinical as well as clinical studies.

Bert Hazlett - Ladenburg

Thank you for that.

Jim Bianco

I should say neither of those would you choose to use a drug such as Ruxolitinib, which is associated with anemia and thrombocytopenia because you'll be working across preferences, and could not use it in combination with another drug such as hypomethylator or high NAK inhibitor that also cause cytopenias. So I think we've got a lot more flexibility due to the profile of Pacritinib.

Bert Hazlett - Ladenburg

That's great color. And I guess just in terms of the MO for the development, for the consideration in these additional settings, right now that appears to be IST-focused initially, then if you see success or signals more broadly developed, do you see that changing with the potential success in myelofibrosis? Do you see that formally being explored by the partnership more aggressively at some point?

Jim Bianco

Yes, I think that is pretty standard if you'll in the industry when you know you have a successful molecule that's on a road to potential regulatory approval that you'd take clearly a wider cast of not just looking at investigator studies, but actually a company's sponsored trials.

Bert Hazlett - Ladenburg

Terrific. Thank you for the color. I appreciate it.

Jim Bianco

Thanks, Bert.

Operator

We have no further questions. Dr. Bianco, I'll turn the conference back to you for closing or additional remarks.

Jim Bianco

Thanks, everyone for joining us on the call. We appreciate your support. Look forward to seeing many of you at the upcoming investor conferences and meetings this fall. Have a good evening, and enjoy the rest of your summer.

Operator

And again, ladies and gentlemen, that does conclude our conference for today. We thank you all for your participation.

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