Cell Therapeutics' CEO Discusses Q4 2013 Results - Earnings Call Transcript

| About: CTI BioPharma (CTIC)

Cell Therapeutics, Inc (NASDAQ:CTIC)

Q4 2013 Earnings Conference Call

March 04, 2014 16:30 ET


Monique Greer - SVP, Corporate Communications & IR

Jim Bianco - President & CEO

Lou Bianco - EVP, Finance


Kim Lee - Janney Capital

Bert Hazlett - ROTH Capital

Ren Benjamin - H.C. Wainwright


Welcome to the Cell Therapeutics Fourth Quarter and Full Year 2013 Financial Results Conference Call. (Operator Instructions). This call is being recorded today, March 4, 2014. I would now like to turn the conference over to Monique Greer, Senior Vice President of Corporate Communications and Investor Relations. Please go ahead.

Monique Greer

Thanks, Dud. Good afternoon, everyone, and thank you for joining us today for our fourth quarter and full year 2013 results conference call. Following formal remarks by management, the conference call will be open for questions.

With me today is Dr. Jim Bianco, President and Chief Executive Officer; Matt Plunkett, Executive VP of Corporate Development; and Lou Bianco, Executive VP of Finance will be available during our Q&A session later on.

A press release was issued after market close today, a copy of which can be found on our homepage and in the Investors section of our website at celltherapeutics.com.

Before we begin, please note that during the course of this 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 Risk Factors section of our annual report on Form 10-K for the year-ended December 31, 2013, 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.

Jim Bianco

Thank you Monique. Good afternoon everyone. For those of you that may be new to CTI, we're a biopharmaceutical company with one marketed oncology drug and a promising late-stage pipeline. Our focus is to become a leader in the development and commercialization of new treatment for blood-related cancers.

I would like to start out by stating the somewhat obvious that 2013 was a transformational year for the company and most important milestone occurred in the fourth quarter of 2013 when we entered into the worldwide license agreement with Baxter International to develop a commercialized lead product candidate, pacritinib. This was a significant achievement towards our goal of bringing this novel promising agent to patients as quickly as possible, particularly to those who are underserved by the current approved treatment options for myelofibrosis.

The Baxter collaboration is expected to help us accelerate our global development timeline and maximize the commercial opportunities for pacritinib in myelofibrosis and in other indications, as well and we’re certainly off to a late start.

I will give you few words on pacritinib, you may recall this is an oral JAK2 and FLT3 inhibitor that we acquired in 2012 and it's certainly being evaluated in key Phase III clinical trials. Pacritinib inhibits two important activating new patients, the JAK2 and FLT3. As you may know activating mutations of JAK2 are implicated in certain blood related cancers such as the myeloproliferative neoplasms, leukemia and certain solid tumors.

FLT3 is a gene commonly found mutated in patients with acute myeloid leukemia or AML and pacritinib inhibits not only the internal tandem duplication but also the A35Y point mutation which is commonly associated with resistance to Type2 FLT3 inhibitors.

Now based on pacritinib’s efficacy and tolerability profile demonstrated to-date, the current pivotal program for pacritinib is focused on myelofibrosis and just as you know it's a chronic malignant bone marrow disorder and that program includes two randomized Phase III trials that we refer to as PERSIST-1 and PERSIST-2. PERSIST-1 is currently enrolling a broad set of patients with myelofibrosis as without limitation of platelet counts and this trial is comparing the efficacy and safety of pacritinib with that of best available therapy, which is any physician selected treatment other than JAK inhibitors.

And as I mentioned there is on exclusion by platelet count. The primary end point for PERSIST-1 trials is the percentage of patients achieving a 35% of greater reduction in spleen volume from baseline to week-24 as measured by MRI or CT scan and the secondary end point is the percentage of patients achieving a 50% or greater reduction in total symptom score or TSS from base line to 24 weeks as measured by tracking specific symptoms on a form.

At the time of initiation of the trial PERSIST-1 utilized the original MPN FAS total symptoms score instrument to measure total symptom reduction. Now we modified the TSS forum based upon feedback we received and the study endpoints and labeling team at the FDA. And through our participation in the patient reported outcomes, or PRO Consortium at the agency. We believe that will become the standardized tool for the industry. Now in connection with this amendment we can expect the enrollment of PERSIST-1 will be increased from 270 patients to approximately 320 patients and we still expect to report top line data from this trial in the second half of this year and enrollment has been going very well.

PERSIST-2 as you know we recently commenced that study and this trial will evaluate the safety and efficacy of pacritinib in patients with low platelet counts who have moderate to severe thrombocytopenia, meaning the platelet counts that are equal to or less than 100,000 per microliter.

This trial will evaluate the pacritinib as compared to best available therapy but unlike PERSIST-1 this will include approved JAK2 inhibitors that are dosed according to the product label for myelofibrosis in patients with thrombocytopenia and this trial is expected to enroll upto 300 patients in North America, Europe, Australia and New Zealand over the next 12 to 14 months and there is a significant level of interest in site participation for this trial as well.

The PERSIST-2 trial is being conducted under a special protocol assessment which is an agreement which we reached with the FDA on the plan design endpoints and analysis approach for the trial and support a potential NDA submission and we expect the data from PERSIST-1 and PERSIST-2 positive will form the basis for both an NDA submission at the end of this year and the MAA submission early next year.

And I want to underscore the importance of the pacritinib program to CTI. In addition to myelofibrosis we have encouraging clinical data in lymphoma and preclinical data in FLT3 resistant AML. Pacritinib is currently being evaluated in AML through an ongoing investigator sponsored trial underway in the UK. We’re excited about the interest that we have received from investigators and cooperative groups to study pacritinib in other blood cancers in the future in addition to solid tumors.

So in summary we’re confident that our collaboration in fact will allow us to more rapidly advance the development for pacritinib and hopefully make the drug available to patients in need around the world.

Another compound that has drawn greater attention as a result of data presented at ASH last December is our oral selected aminopeptidase inhibitor known as tosedostat. Unlike normal cells transform cells are very dependent on the ability to recycle intracellular proteins to make new proteins and survive. Scientific interest in tumor orthology [ph] has grown over the last decade following the clinical success observed with interrupting protein recycle at the proteasome complex. Attention has now turn to pathways downstream to the proteasomal complex that are responsible for removing and recycling essential amino acids off of peptides. One novel target for intervention are the aminopeptidases, a family of metalloenzymes that clip and free up amino acids off of peptides and polypeptides allowing them to be building blocks for new proteins essential for tumor cell survival.

At the recent American Society Hematology meetings we reported positive interim results and have investigated and initiated Phase II trial of tosedostat in combination with cytarabine or decitabine in newly diagnosed older patients with AML or high risk MDS. Now the patients in this trial were not considered candidates for standard intensive therapy.

The presentation reported on the results of 26 patients got an median age of 69 years were enrolled in the first dose cohort which showed a 54% complete response or incomplete response or CRI with approximately 12 month median survival and this was noted by investigators to be very encouraging given the published data with either decitabine or cytarabine alone in this patient population. The tosedostat combination therapy was well tolerated and was predominantly administered in outpatient therapy. There are several ongoing Phase II cooperative group sponsored trials and ISPs evaluating the activity of tosedostat in combinations with standard agents and patients with AML or MDS and data from these signifying trials may help inform the appropriate design for a Phase III trial in the future.

I’m going to turn over to PIXUVRI. As you may recall PIXUVRI is a first in class anthracenedione unique structural and physiochemical properties. The last fall the results from a preclinical study suggested that PIXUVRI is unique in it's mechanism for inducing tumor cell death, thereby supporting the thesis that it is the first approved drug in a new class of anticancer agents.

As most of you know, based on PIXUVRI’s efficacy and safety profile and the clear unmet medical need and aggressive B-cell non-Hodgkin's lymphoma, the Europe Commission grant the conditional marketing authorization for PIXUVRI in 2012 as the first therapy for the treatment of patients with multiply relapsed or refractory aggressive B-cell NHL. Prior to PIX, patients with aggressive B-cell non-Hodgkin's lymphoma who had failed 2 or 3 prior lines of therapy and no approved treatment options specifically for that stage of disease.

Since that time we initiated our Europe Commercial operations on a country by country basis and as of today PIXUVRI is marketed in 10 Europe countries. Of course reimbursement decisions by governmental authorities of each European country has been the greatest barrier to our ability to generate sales for PIXUVRI in 2013. Recognizing that for the most part only in Germany the patients had meaningful market access during the reimbursement of discussions with the regulatory agencies. Now thankfully the interest and support of PIXUVRI by healthcare providers and key lymphoma opinion leaders has greatly contributed to our ability to successfully navigate the reimbursement process in each country.

We spent the better part of 2013 working with these regulatory agencies in the four largest markets being Germany, France, Italy and the UK to ensure successful outcome for getting PIXUVRI reimbursed. And as you know many pharmaceutical companies have been approved by the Europe Commission only to be turned down when it comes to getting new drug reimbursed. So after a number of months of ups and downs in the third quarter of 2013 PIXUVRI was granted market access in Italy and France and followed by Germany in December when we reached agreement for funding and reimbursement with the statutory health funds and just week the National Institute for Clinical Excellence or NICE determination that PIXUVRI, most published it's final guidance in the Journal Lancet Oncology which recommended a prescription of PIXUVRI in England and Wales for patients with aggressive B-cell NHL who have dealt 2 or 3 prior lines of therapy and NICE’s determination that PIX is cost effective in the treatment of these patients have had an impact across the EU where many EU countries look to the rigorous process conducted by NICE as an endorsement for reimbursement in their own country.

Needless to say these accomplishments were no small path especially for a small biotech company like CTI which makes me even more proud of what our team has been able to accomplish. In parallel to our efforts on securing reimbursements our medical affairs team who is educating and building support across key lymphoma opinion leaders. Now, initially many were skeptical of an outpatient monotherapy treatment for relapsed aggressive non-Hodgkin's lymphoma. Since most physicians resort to in hospital more toxic multi-agent regimens. However as their hands on experience with PIXUVRI in their centers began to grow a sense of excitement emerged as they began sharing their results with each other at society meetings, key lymphoma congresses and educational seminars throughout the EU.

The common theme we have been hearing repeatedly is their excitement with PIXUVRI’s ability to overcome chemo-insensitive disease and put third line patients back in remission and eligible to offer them another chance by potentially lifesaving stem cell transplant. Now for patients that they feel are not transplant eligible it allows them the opportunity to give them something they couldn’t with prior therapies which is the opportunity for a durable remission and not just symptom palliation. And the case studies that they have been reporting have been truly remarkable and a real testament to the potential for PIXUVRI.

Physicians are also gaining awareness of PIX through our promotional brand campaign including advertisements, direct mail and congress activities. Our medical affairs organization continues to engage and build relationships with key hematology and lymphoma opinion leaders through one-on-one meetings, scientific advisory boards, medical symposia, IST and publications. And as we ramp up our medical education programs and field activity one of our stated business priorities this year is to generate sufficient sales to achieve a positive contribution margin for PIXUVRI by the fourth quarter of this year and if we’re able to achieve that goal with PIX and going forward PIX will be profitable in 2015 and beyond.

And now that we have secured reimbursement we’re seeing a renewed interest in potential partnering for PIXUVRI in markets where CTI does not have or plans to have a commercial presence and as such one of our corporate goals for 2014 is to secure a PIXUVRI partner to help expand the product reach and the commercial potential. Now we intentionally did not provide 2013 sales guidance given the uncertainty around securing in the time for reimbursement which for the most part didn’t occur until late in the fourth quarter last year. With that in mind net product sales for PIXUVRI for the fourth quarter and full year were 500,000 or $0.5 million and $2.3 million respectively.

Now that we have likely overcome some of the reimbursement barriers we expect to see greater adoption and more consistent use of PIX by healthcare providers beginning in the second half of this year.

So continuing on with our financials for the quarter and full year ended December 31, 2013 we reported revenue of $32.9 million and $34.7 million respectively compared with no revenue for the same periods in 2012. The increase year-over-year included license and contract revenue for 32.4 million which was primarily attributed to the upfront payment received in November from Baxter for the worldwide license agreement for develop and commercialize pacritinib.

For the fourth quarter ended December 31, 2013 we posted a net income of $10.2 million or $0.08 per share compared to a net loss of $19 million or $0.20 per share for the same period in 2012. Net loss for the year ended December 31 was $49.6 million or $0.43 a share compared to a $115 million or $1.98 per share in 2012.

Non-cash share based compensation expense for the year was $9.1 million compared to $7.9 million for the same period in 2012. We exited 2013 with cash and cash equivalents of $71.6 million. With regards to expense guidance for 2014 loss from operations is expected to be approximately 45 million to 50 million excluding any non-cash share based compensation expense which as you know is a non-GAAP measure. For the year ended December 31, loss from operations excluding non-cash share based operation expense was 32.5 million. So the projected 2014 net loss from operations is primarily related to the following. The achievement of a positive net product contribution from PIXUVRI commercial operations by year-end as we intend to operate that business with a net positive P&L in 2015.

License and contract revenue associated with the achievement of the clinical development milestone for pacritinib, specifically related to the completion of enrollment in PERSIST-1 in 2014 and PERSIST-2 in early 2015. Management of SG&A expenses including management of sales and marketing expenses to drive PIXUVRI sales as well as medical affair expense and support of educational programs for the Hemat [ph] community in Italy. And lastly R&D expenses including management of costs for ongoing and planned clinical trials involving pacritinib and our investigative sponsored studies with tosedostat.

So our key business priorities in 2014 on the development front for pacritinib included completing patient accrual in PERSIST-1 Phase III trial of pacritinib and to report the top line results in the second half of this year. Enrolled the majority of the patients in the second Phase III PERSIST-2 study that recently opened at the clinical trial sites and initiate investigator sponsored trials evaluating pacritinib in additional indications outside of myelofibrosis.

With regards to PIXUVRI as mentioned we expect the gross sales in current indication in the EU with a positive product margin contribution by the fourth quarter of 2014. And lastly to pursue a partner for commercializing PIXUVRI in additional markets within EU and another emerging markets outside the EU and the U.S. I can tell you that we’re off to a very productive start in 2014 and we certainly look forward to what is shaping up to be another busy and very exciting year for the company. So before opening the call for questions as always I like to thank our employees for the collective passion, dedication to the patients that we serve.

With that operator can we open the call for questions?

Question-and-Answer Session


(Operator Instructions). Our first question is from the line of Kim Lee with Janney Capital. Please go ahead.

Kim Lee - Janney Capital

My first question is I apologize if I missed this, but did you say that you changed the primary end point for PERSIST-1?

Jim Bianco

No we did not say that we changed the primary endpoint for PERSIST-1. We said we made change to the MF SAS form consistent with the agencies desire to standardize that form going forward so that was an amendment change to the measurement tool which was as you know is the agency moving away from the version 2 that was utilized in the comfort study with some learning [ph] changes that they felt will ultimately when validated would make it a more standardized tool across the industry.

Kim Lee - Janney Capital

And as far as milestone payments from Baxter, should we expect any milestones this year relating to the PERSIST-1 top line data and potential NDA filing and if so how much we expect?

Jim Bianco

As we reported I guess in our press release when we announced the deal we will earn $20 million at the time of the last patient enrollment in PERSIST-1 and $20 million at the time of the last patient enrollment in PERSIST-2. So we certainly feel confident that the first $20 million will be earned this year, in fact that we need to be that in order for our expectation to have data on that trial out by year-end.

Kim Lee - Janney Capital

And as far as R&D and SG&A expenses go that’s quite a big jump up from last quarter. Should we expect fourth quarter operating expenses to be a good base for to startup 2014?

Lou Bianco

It should be consistent and you could expect to see similar growth as the sales also start to pick up.

Jim Bianco

I think the one thing that we do need to mention is that because the way the agreement with Baxter is structured for the reimbursement of 75% of the PERSIST trial expenses going forward that you will see a higher burn in one quarter and then the offset when we earn the milestone in the subsequent quarter but on an average basis our burn forecasted for the year would be in that $45 million to $50 million range.

Kim Lee - Janney Capital

And one last question, I will jump back in the queue there. The investigator initiated trials which should start this year. Can you give us a sense of some of what trial design would look like and how many sites and how many patients? Thanks.

Jim Bianco

I think we’re probably best off we anticipate on several trials in AML, we started one. As a FLT3 positive AML in the relapse setting is single agent and FLT3 positive AML in a different population of patients in front line in combination with standard therapy and those would be essentially the two big buckets if you will. And then outside of AML there is a several solid tumor trials that are currently in being flushed out if you will to start probably in the second half of this year. So can we rather than getting into the trial size, et cetera, some of those when they are published on clinicaltrials.gov you can pick it up there. The trial in the UK Reeve Cardiff [ph] probably on their website will describe what their pick-a-winner design looks like for that trial.


Thank you. Our next question is from the line of Robert Hazlett with ROTH Capital. Please go ahead.

Bert Hazlett - ROTH Capital

Could you describe maybe some of the details about that commercial infrastructure you were planning and establishing in the EU for PIXUVRI. Again I know that the comment is around positive contribution margin and profitability sometimes in 2015 but positive contribution margin around the end of this year but give us the blueprint as it stands now for the commercial infrastructure in the EU and maybe if there is potential for inflection point should things either better or worse than expected.

Jim Bianco

Absolutely. So we currently have 20 quintile employees that we deploy in the Nordics, Germany, Austria, and the UK. Italy and France will come online in the second quarter of this year. By year-end we would have a total comp of about 28 field based quintile employees and in addition to the three CTI employees that we have in the UK which would be market access, medical affairs and regulatory. So, that cost structure and that structure we have found to be very cost effective and obviously without having to have legal entities in the 10 countries that we currently market the product.

With regards to inflection point I think it's critical to note that clearly NICE was a very big shot in the arm for us so to speak since many countries will draft off of the NICE opinion on cost effectiveness. With regards to Italy and France they come on a little slower even though we have market access so we wouldn’t expect to see a contribution from Italy or France in terms of unit sales and so the second of the year likely in third and fourth quarter.

UK, Germany, Austria and the Netherlands have been the primary source of the revenue growth that we have seen coming into the first and second quarter this year and so the commercial team is tracking to forecast. We’re very pleased in terms of what we’re seeing on the trend lines. We think based upon having just come back from Europe and our colleagues on the phone are currently in another part of the world. There is a fair amount of interest in PIXUVRI, I think it is probably an understatement to say that there is a lot of excitement in lymphoma in Europe as they start seeing really dramatic ability to rest to savage patients in the third or fourth line setting and then clipping back essentially cure the therapies.

So that theme is resonating as really a viable commercial potential for the product and because of that we’re seeing companies come up and say, hey Europe is a big territory there is other big emerging markets that they would be interested in moving the product into. Even some have said, hey can we co-promote alongside of you we have existing products in the heme space and there's some synergies. So the long and the short of it is you know we think that this can become a very attractive viable business in 2015 and beyond and so we’re still in kind of the building block stage if you will, if you look at the immune Esbrit launch, it's much bigger market obviously for them but still the first year, first 18 months coming out as an approval is really just building blocks to get the product up to where it needs to be in terms of it's run-rate and totally obviously very different than what you see here in the U.S. which is turnkey for the whole country.

So we’re pleased by the progress, we’re excited by what we’re seeing in the first actually in the last three months in terms of unit growth and by the interest in some very familiar names and potentially good partners to expand the commercial potential for the product with us.

Bert Hazlett - ROTH Capital

Just one another on pacritinib and one on tosedostat. On pacritinib you’ve stated the enrollment goals for PERSIST-2. Again, that looks pretty rapid in terms of the expected enrollment goals. Can you just reiterate why you think that rapid enrollment is achievable for PERSIST-2?

Jim Bianco

Yeah absolutely. So about half the sites or so will be PERSIST-1 sites that will be rotating off of PERSIST-1 as it completes it's enrollment and then start the screening for PERSIST-2 patients. So that saves us about a 4-6 month lead in time have those been new sites with whom A, we didn’t have contracts and B, essentially patient screening and experience with from the CRO perspective site initiations et cetera. So that’s really becomes a turnkey as PERSIST-1 completes in the second quarter, you would have this ability to have 40 to 50 sites in Europe start screening immediately and we have been working on that front getting the work in advance of when PERSIST-1 would complete, again it takes about a 3 month lead time. So we started last year on that effort. And then the U.S. sites as they continue to come online this is a patient population that has been excluded for the most part for most clinical trials and so we know from the bigger centers here like Mayo, et cetera that there is this pent up demand for this trial and so we have heard we have x-number of patients already in clinic that would be eligible beyond your study.

And to give you a sense for what I mean about PERSIST-1, we screen between 11 and 14 patients a week on PERSIST-1. We think the enroll for PERSIST-2 should be in that timeframe. We think that 12 to 14 months is achievable.

Bert Hazlett - ROTH Capital

And for tosedostat I have a phone connection difficulty, my apologizes but the additional next steps for tosedostat either an AML or beyond AML if you can describe those things as you see them at this point.

Jim Bianco

So maybe a little too premature to lock in to what we’re saying but with respect to our scientific advisory board which as you may know contains the expert in myeloproliferative neoplasm, specifically CML, Dr. Druker, and leukemia and MDS with Dr. List and company. So the interest with tosd, as we call it, would be in MDS, given that the signal that we have seen in both the OPAL study and in the patients from the Seattle Cancer Care Alliance who were essentially MDS patients I suppose to the high risk AML and there could be in two realms one being patients who are progressing on hypomethylating agents and adding tosedostat in that setting to see if you can resensitize them with a hypomethylating agent continuing and then the other would be more of a traditional frontline where you take the hypomethylating agent plus and minus tosedostat. And both of those are still undergoing what we call the feasibility stage with thought leaders in the MDS community.

There is some interest in putting tosedostat in a solid tumor study like in pancreatic cancer giving the science published about pancreatic cancer being exquisitely sensitive to the need to have protein sources in order for it's survival and I think that’s been relatively well demonstrated and so Dan Von Hoff , who is on our SAB who is an expert in, he is the one who got gemcitabine approved for pancreatic. He is the one who brought the albumin-bound nano particle, paclitaxel to pancreatic, again for its registration and approval. And Dan things that based upon the biology and the science with tosedostat that we should look at it in that tumor as well. So I think that is kind of just an outline, again none of this has been vetted to the point where we can say how many patients with centers and when would they start but that’s certainly the activities for our medical affairs group in the first half of this year.


Your next question is from the line of Ren Benjamin with H.C. Wainwright. Please go ahead.

Ren Benjamin - H.C. Wainwright

Just a couple of questions, starting off with pacritinib. I'm sorry, Jim, did you mention that the number of patients had increased from 270 patients to 320? Or did I miss-hear that somewhere? And what would be the reason for it, if I heard correctly?

Jim Bianco

No that’s correct, again as I mentioned with the MF SAS Version-2 which was the version that was used in the COMFORT studies, the FDA had some suggestions about how to modify, how the question is either asked or including an additional question into the tool and wanted us to make that change to the form. So in changing that when that amendment becomes active, you want to increase the sample size so you’ve an adequate number of patients that have been evaluated with the amended tool and just to give you a sense we’re working in a group with Sanofi and with the PRO Consortium at the Critical Path Institute on essentially validating that tool as what then would become a standard for the industry that meaning the other studies that are looking at myelofibrosis as a validated tool for the sector and so we were pleased that the field group at the FDA was so engaging if you will on trying to take lessons learnt from the COMFORT studies that have gone before us and then essentially to make improvements that they feel will become again essentially could become a standard for the assessment form [ph].

Ren Benjamin - H.C. Wainwright

And I guess just related to this, can you give us a little bit more color as to what the amendment could be, or what additional questions there might be there? And will patients who have already been enrolled and been assessed? Do they get reassessed with this new amended tool? Or do you just increase the sample size so that you can have a new set of patients that can be assessed by this new tool completely?

Jim Bianco

It's the latter, you do use both obviously because you don’t discard the data and so to speak. So the changes that were made were there is small and simple things but it's how you ask a question on the other tool and you can go on clinicaltrials.gov and you can pull up the modified or the version 2 on the MF SAS form and the FDA added one additional question it's really a clarity on the question as opposed to, tiredness one of the categories as well. Tiredness can mean a lot of things to a lot of different people. So it's stuff like that Ren. It was literally only three changes that were not if you looked at them it didn’t change the categories per se but changed the way in which they are worded as a question. And again we have had dialogue obviously with the FDA on this and don’t forget that this is the tool that is in PERSIST-2 in the entire patient population as well.

Ren Benjamin - H.C. Wainwright

And just related, you mentioned an NDA submission at the end of this year, and MAA at the end of next year. But as I understand, you need both PERSIST-1 and PERSIST-2 for a filing. So, for an NDA submission to occur at the end of this year, is it that you'll be submitting it in modules or will the NDA submission also be next year?

Jim Bianco

I’m sorry if I said this year that would be incorrect. What we stated that the PERSIST-1 top line results would be that that study would be analyzed in the second half of this year and that PERSIST-2 would complete it's enrollment in early 2015 with the targeted NDA by the end of 2015 and the MAA in early 2016. The MAA is obviously is controlled by Baxter and so the NDA submission goes first and then they create the common technical document from the NDA components into their MAA if you will.

Ren Benjamin - H.C. Wainwright

Just jumping quickly to Opaxio. You guys have completed enrollment. Can you just give us a sense as to when we might see the next interim analyses?

Jim Bianco

Sure. So I will put it in the context, that the GOG meets twice a year and that’s typically if they were going to do an analysis it would be around one of their meeting date and so we really don’t have feedback about when obviously it wasn’t January, we don’t know if it will be the summer but that’s typically when they meet as a group and look at their trials components. So when they tell us you will be the first to know.

Ren Benjamin - H.C. Wainwright

Another question regarding data presentations. Obviously we have several conferences coming up. Can you give us a sense as to any data updates or presentations that you may have, maybe ACR or ASCO or ASH?

Jim Bianco

I don’t know if we have that the list yet, but again that’s I’m not certain Ren. I mean we can certainly go offline and have a discussion about what is on the drawing board but I’m not sure about what’s been submitted.

Ren Benjamin - H.C. Wainwright

Okay. And then just one last question. PIXUVRI, you mentioned that 2014 should be able to generate sufficient sales so that it’s; I'm paraphrasing, but cash flow positive or at least net neutral from a unit perspective. Any color you can provide us as to maybe how much the expenses are for that particular unit within the Cell Therapeutics franchise?

Jim Bianco

Not yet. We’re seeing some good trends, when those trends become things that we can rely on we will provide that guidance at that time. I think we provided you with the headcount would be and so obviously that is typically the sales force headcount is a good portion it's not 67% of your expenses in the commercial effort and then the rest would be direct in terms of marketing aids, tools and advisory meetings et cetera. So one could probably do back on the envelope on that.


And we do have a follow-up from the line of Robert Hazlett with ROTH Capital. Please go ahead.

Bert Hazlett - ROTH Capital

Just with regard to the form and the additional clarification of the question, that is for PERSIST-1 in terms of the clarifying document and those are incorporated into PERSIST-2, as well? Or, just any more, just a little bit more clarity on which study that was driven by and is it being incorporated already into PERSIST-2.

Jim Bianco

Absolutely. So we have reached an agreement, I don’t want to say agreement, we agreed with the FDA last year and made the amendment to change way before PERSIST-2 if you will PERSIST-2 was already into the SPA discussions and that was already a given that that was going to be the instrument for PERSIST-2 in terms of how the agency wanted to see the program progress. And if you’re familiar with the PRO group, I mean that’s the whole intent there is that you’ve an instrument, that you’ve buyoff, signoff essentially by the FDA and in this case the reported outcomes group with a validated tool and we do plan on validating that tool as part of the clinical trial effort in this year and so that then becomes, then the differences we’re willing to share that with the community I guess the prior tools that were utilized while validated were not made available to the FDA or the public.


At this time I would like to turn the call back over to Dr. Bianco for closing comments.

Jim Bianco

Thanks everyone for joining us on the call. We obviously appreciate your support and we look forward to seeing many of you at the upcoming investor conferences and meetings and have a good evening.


Ladies and gentlemen that does conclude our conference for today. If you'd like to listen to a replay of today's conference please dial 303-590-3030 or 1800-406-7325 and enter the access code, 4670750. I would like to thank you again for your participation. You may now disconnect.

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