Cell Therapeutics Management Discusses Q4 2012 Results - Earnings Call Transcript

| About: CTI BioPharma (CTIC)
This article is now exclusive for PRO subscribers.

Cell Therapeutics (NASDAQ:CTIC) Q4 2012 Earnings Call February 28, 2013 4:30 PM ET


Monique M. Greer - Senior Vice President of Corporate Communications and Investor Relations

James A. Bianco - Principal Founder, Chief Executive Officer, President and Executive Director

Steven E. Benner - Chief Medical Officer and Executive Vice President

Matthew J. Plunkett - Executive Vice President of Corporate Development


Robert Cummins Hazlett - Roth Capital Partners, LLC, Research Division

Reni J. Benjamin - Burrill & Company, Research Division


Good afternoon, ladies and gentlemen. Thank you for standing by. Welcome to the Cell Therapeutics Fourth Quarter Full Year 2012 Financial Results Conference Call. [Operator Instructions] This conference is being recorded today, February 28, 2013.

I would now like to turn the conference over to our host, Monique Greer, Senior Vice President of Corporate Communications and Investor Relations for Cell Therapeutics. Please go ahead.

Monique M. Greer

Thanks, Liz. Good afternoon, everyone, and thank you for joining us today for our fourth quarter and full year 2012 results conference call. Following formal remarks by management, the conference call will be open for questions. With me today are Jim Bianco, President and Chief Executive Officer; and Steve Benner, Chief Medical Officer. Matt Plunkett, Executive Vice President of Corporate Development; and Lou Bianco, Executive Vice President of Finance will be available during our question-and-answer period.

A press release was issued after market closed today, a copy of which can be found on the Homepage and in the Investor section of our website at celltherapeutics.com. I also want to point out that going forward, we plan on reporting our quarterly results after market close versus our prior practice of reporting premarket.

The agenda for the call is as follows: Jim will begin with a brief overview of the fourth quarter and full year, particularly regarding the launch and commercialization of Pixuvri in the European Union, Steve will follow with an update on our development pipeline, and then Jim will close and open the call for questions.

Before we begin, 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 continued in the Risk Factor section of our 2012 Annual Report on Form 10-K for the year ended December 31, 2012, which will be on file this afternoon 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?

James A. Bianco

Thanks, Monique. Good afternoon, everyone. I'd like to start out by highlighting how 2012 was a successful year for the company in many ways. The approval of our lead product, Pixuvri, by the European Commission as the first therapy for treatment of patients with multiply relapsed or refractory aggressive B-cell NHL was a transformative event for CTI, representing our third drug approval in the company's history and our second European approval. This event marked our reemergence as a commercial hematology-oncology company. And more importantly, for patients with aggressive B-cell NHL, Pixuvri provides hope and offers new treatment standards.

In May 2012, we expanded our late stage pipeline of product candidates, the acquisition of pacritinib, an oral, once-daily JAK2/FLT3 inhibitor that's demonstrated a meaningfully clinically benefit and good tolerability in myelofibrosis patients in our Phase II clinical studies. Importantly, without treatment emergent myelosuppression, seen with other agents in this class, Steve will discuss this exciting program later on in the call.

So before providing an update on the Pixuvri commercial activities, let me highlight our financial results. For the year ended December 31, 2012, CTI reported a net loss of $115.3 million or $1.98 per share, including the $29.1 million for the acquired in-process research and development expense related to the acquisition of pacritinib from S*Bio. That's compared to a net loss of $121.1 million or $3.53 per share for the same period in 2011. Net loss for the fourth quarter ended December 31, 2012, decreased by 10% to $18.9 million or $0.20 per share compared to a net loss of $20.9 million or $0.47 per share for the same period in 2011, taking into consideration one-time settlement income that we received in 2011.

Total net operating expenses for the year were $101.5 million, again, which also includes the $29.1 million related to the pacritinib acquisition.

Regarding expense guidance for 2013, net loss from operations is expected to be approximately $60 million to $65 million, excluding any noncash stock-based compensation expense. The projected 2013 net loss from operations is expected to be comparable to 2012 and primarily relates to expected changes in a positive net product contribution from Pixuvri commercial operations as we intend to operate the commercial business with a net positive P&L; management of our SG&A expenses, including management of sales and marketing expenses to drive Pixuvri sales, as well as medical affairs expenses in the support of educational programs for the hemo community in the EU; and lastly, R&D expenses, including the management of cost for ongoing and planned clinical trials involving pacritinib, the post-approval study of Pixuvri, which is pursuant to the post-marketing commitment that we have with the European Medicines Agency.

Turning to our balance sheet, we ended 2012 with cash and cash equivalents of $50.4 million and no debt, and approximately 109.8 million common shares outstanding.

[indiscernible] Marco Rubio. Okay.

Now moving on to our commercial progress. During the fourth quarter 2012, we began making Pixuvri available to health care providers in the EU and initiated our commercial operations on a country-by-country basis. I'm pleased to report the launch of Pixuvri is underway in 8 countries in the EU, including the Nordic countries, the Netherlands, Austria, Germany and the U.K. As you may know, these are referred to as free market access countries. The drug is reimbursed at our market price, 12 months, mostly from hospital funds, the country sick funds, until final pricing negotiations are completed.

All clinical and pricing dossiers have been submitted. Now we anticipate making Pixuvri available to healthcare providers in France, Italy and Spain once reimbursement and pricing discussions have been completed in the second half of this year.

Although it's early in the launch, we're pleased with the interest and receptivity of Pixuvri by healthcare providers in key lymphoma opinion leaders. We're currently focused on educating physicians on the unmet medical need and building brand awareness for Pixuvri as a third and fourth line treatment option amongst physicians in the countries where Pixuvri is currently available. Patients with aggressive B-cell NHL who failed initial treatment with anthracyclines typically go on to receive second line treatments consisting of intensive non-anthracycline-based salvage therapy, with a small percentage of these going on to stem cell transplantation. The literature shows the majority of these patients will relapse after second line therapy. Prior to Pixuvri's approval, there are limited options for patients, mostly palliative care or clinical trials.

Now in the randomized control pivotal Phase III EXTEND trial, our label population consists of a subgroup of third and fourth line patients with B-cell NHL. In that group, the overall response rate was 48% at the end of treatment compared with 12% in the comparative group. Importantly, 28% of the patients achieved a complete remission or an unconfirmed complete remission compared to 4.1% in the comparator arm. There is a 50% reduction in the risk of death or disease progression, and with mean in the overall survival for that group, 13.9 months compared to 7.8 months as to the physician's choice of chemotherapy.

The toxicities were predictable and manageable, and administered at the proposed dose and schedule. Now we have broader intent to treat results from the EXTEND study, you may remember were published in Lancet oncology in May of last year.

Based on the Pixuvri's efficacy and safety profile and the unmet medical need in aggressive B-cell NHL, the European Commission granted conditional marketing authorization for Pixuvri.

Independent third-party market research amongst 250 lymphoma physicians demonstrate that treating physicians favorably assess the Pixuvri product profile established in the EXTEND trial, noting it will allow them to offer salvage therapy as compared to palliation of their symptoms. And through our medical education programs and field activity with our medical science liaisons and sales force, now we're able to highlight the unmet medical need that Pixuvri can fulfill with sharing the results of the EXTEND study with healthcare providers. Our initial launch experience suggests that the market for Pixuvri is highly promotionally sensitive, and when healthcare providers spend time with our sales force, they are more likely to begin to prescribe Pixuvri.

Physicians are also gaining awareness of Pixuvri 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, investigator-sponsored studies and publication. This educational effort is initiated at the American Society of Hematology meeting last December and will continue throughout 2013.

Another important focus for us this year is achieving favorable reimbursement in the 5 major market countries, namely Germany, United Kingdom, France, Italy and Spain. The cost benefit dossiers for PIX were submitted to each of these countries in November. And while we have not yet received the preliminary assessments from Germany's Institute for Quality and Efficiency in Health Care, or IQWiG, we expect it soon based on their calendar.

As you know -- or as you may know, in Germany, for example, the federal joint committee, which is called the GBA, is the ultimate decision-maker in German healthcare and consults with IQWiG to assess patient-related benefit. GBA is typically focused on evidence-based medicine, patient outcomes in terms of morbidity, mortality and quality of life.

In Germany, when an agent is the first drug to be approved in an indication, IQWiG is restricted from determining benefit. So it's common to see preliminary assessments from IQWiG among first-to-market agents in a given indication that do not reflect the opinion of the ultimate decider, which is the GBA. GBA is aware of this issue within the IQWiG guidelines, and as a result, has overturned 7 of the last 28 IQWiG rulings.

It's also important to keep in mind that these reimbursement discussions have no impact on the reimbursement of Pixuvri or physician's ability to prescribe this new treatment. We believe PIX will offer those patients suffering from relapsed aggressive B-cell NHL a safe and effective therapy as there are currently no approved alternatives, and we'll continue to work with the healthcare regulatory bodies to ensure a successful outcome as we navigate the reimbursement process.

We're off to a productive start in 2013 and look forward to what's shaping up to be an already busy and exciting year. We have the right people in the right place and the right product portfolio to build a leading hematology company committed to sustainable growth.

So at this time, let me turn the call over to Steve to provide an update on our post-marketing trial for Pixuvri, in addition to providing an update on our pacritinib Phase III program.

Steven E. Benner

Thank you, Jim. Based on Pixuvri's efficacy and safety profile, with a clear unmet medical need progressive B-cell, non-Hodgkin's lymphoma European Commission-granted conditional marketing authorization for Pixuvri. As we have previously described, the connection with the approval and an agreement with the European Commission, we are conducting a post-marketing commitment trial, known as the PIX-R or PIX306 study. As you may recall, this is a randomized trial of pixantrone-rituximab versus gemcitabine-rituximab in patients with aggressive B-cell NHL relapse after receiving CHOP-R therapy or an equivalent regimen and are ineligible for stem cell transplant.

The primary endpoint is overall survival with secondary endpoint progression-free survival, complete remission and overall response rate. We are considering revising the primary endpoint to assess and plan to discuss this with the European regulatory authority this year in the second half.

We know that in the European public assessment report that either progression-free survival or overall survival would be an acceptable endpoint for full approval. We plan to open additional sites in the EU in the second half of this year, which we expect will increase patient enrollment and also increase the visibility of pixantrone among EU thought leaders. The trial is intended to support the conversion of our EU conditional approval to a full approval.

Now I'd like to turn to the development of our program evaluating pacritinib. To those of you may not be familiar with this product candidate, pacritinib is an oral, once-daily, JAK2/FLT3 inhibitor that inhibits 2 important activating mutations, JAK2 and FLT3. JAK pathway is important in many biological processes, including the regulation of immune function and formation and development of blood cells. JAK2 is implicated in a spectrum of blood cancers, such as the myeloproliferative neuroplasms, or MPNs, leukemia and lymphoma. FLT3 is a commonly mutated gene found in patients with acute myeloid leukemia, or AML, and in lymphoma.

JAK2 inhibitors have been shown to be effective in treating myelofibrosis and have an established regulatory approval pathway, which lowers the risk of development. MPNs are a group of diseases in which specific types of blood cells are overproduced in the body and disrupt their normal functioning. It's a chronic malignant bone marrow disorder that triggers an inflammatory response, resulting in fibrosis within the bone marrow and eliminating its ability to produce blood cells. Splenomegaly and organomegaly are consequences of the disease process. This thing can grow to 10x the normal size, causing great discomfort, and at this stage, the disease is termed myelofibrosis.

It's estimated that there are approximately 30,000 people living with myelofibrosis in the United States. Myelofibrosis is typically diagnosed in people between 50 to 80 years of age, but can occur at any age. Myelofibrosis, myelosuppression typically thrombocytopenia, a relative decrease in blood platelets, is both a consequence of the disease and has emerged as a limiting treatment related side effect of JAK1/JAK2 inhibitors. Where a normal platelet count in adults is in the range of 150,000 to 450,000 platelets per microliter of blood, thrombocytopenia refers to platelet counts lower than 150,000. As the myelofibrosis progresses and platelet production decreases, thrombocytopenia develops [indiscernible] identifying a high-risk population in patients with myelofibrosis who have a normal platelet count. The estimated 30,000 people living with myelofibrosis, based on peer review publications, between 25% to 30% of these patients are thrombocytopenic with platelet counts below 150,000.

Although we believe pacritinib will benefit all patients with myelofibrosis, the benefit will be clearly demonstrated in patients with low platelet counts. We believe pacritinib may offer an advantage over to other JAK inhibitors with the effective treatment of symptoms, while having less treatment of emergent, but related thrombocytopenia and anemia.

Pacritinib has been studied in 2 Phase II trials in a total of 65 myelofibrosis patients. In these trials, 30% to 74% improvement in 7 of the myelofibrosis assessment form scores was observed relative to baseline. Among the valuable patients, approximately 1/3 achieved a 35% or greater reduction in spleen volume as measured by MRI. We believe these effects are independent of baseline platelet counts.

Pacritinib is different from other JAK2 inhibitors because it appears to be associated with less myelosuppression and does not appear to induce a clinically-relevant degree of treatment-emergent thrombocytopenia. Importantly, in the 2 Phase II trials of pacritinib, comparable reductions of splenomegaly were observed among patients with platelet counts of less than 50,000, 50,000 to 100,000 and greater than 100,000 baseline platelets. The most common adverse events were diarrhea and nausea, which we now know can be proactively managed with the use of agents such as anti-diarrheals or anti-nausea medications.

With early intervention at the first sign of GI symptoms, these side effects are readily manageable and resolved with time. We believe pacritinib addresses an unmet need, particularly for patients living with myelofibrosis who face treatment-emergent thrombocytopenia anemia, a serious consequence of certain therapies and also, the disease. We believe that pacritinib may offer patients an effective therapy with a safety profile that could allow for longer-term management of the disease.

We recently initiated the first of 2 planned Phase III clinical studies in patients with myelofibrosis. PERSIST-1 is a multi-center randomized controlled Phase III trial comparing the efficacy and safety of pacritinib with that of best available therapy in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis and post-essential thrombocytopenia myelofibrosis.

The total of 270 eligible patients are planned to be enrolled and randomized in a 2:1 fashion to receive either pacritinib, 400 milligrams taken orally, once-daily, or the best available therapy. Available therapy includes physician-selected treatment other than JAK inhibitors. There are no entry exclusions for patients based on baseline platelet count. The primary endpoint will be the percentage of patients achieving a 35% reduction or greater in spleen volume, measured by MRI at the week 24 assessment. The trial plans to enroll at approximately 80 clinical sites in Europe, Australia and the United States. We've recently held investigative meetings in Australia and Europe. I can tell you that the sites are enthusiastic about the trial and are identifying patients. We anticipate fully enrolling the trial within 12 to 14 months.

The second Phase III clinical trial, the PERSIST-2 trial, is currently being planned to evaluate pacritinib compared to best available therapy, including JAK2 inhibitors, in patients with myelofibrosis whose platelet counts are less than 100,000. This trial is expected to initiate in the second half of 2013. We expect it will have the same primary endpoint as the PERSIST-1 trial.

Interest in this study among U.S. investigators has been encouraging. Although our primary focus is on blood cancers, we continue to work with our other pipeline candidates targeting both hematologic and solid tumors, including tosedostat, OPAXIO, paclitaxel poliglumex, and bistaliphin [ph] with both cooperative group and investigator-sponsored trials. We plan to report results from several investigator-sponsored trials at upcoming scientific meetings.

I will now turn the call back over to Jim.

James A. Bianco

Thanks, Steve. I want to underscore the importance of pacritinib and the pacritinib program to CTI. In addition to myelofibrosis, we have encouraging clinical data in lymphoma, and preclinical data in FLT3 resistance AML.

From an intellectual property perspective, pacritinib has composition of matter protection through 2026 an important designation both in the United States and in Europe for myelofibrosis. We believe PAC is an attractive asset. As we've previously mentioned, we're in the process of looking for an x-U.S. development partner. We anticipate that such a partnership could provide us with non-diluted capital and external validation for the program.

We believe we have a unique asset in pacritinib and we're encouraged by the strong interest so far. At the end of the day, our intent is to evaluate those partnerships that we believe are in the best interest of the company, our patients and our shareholders.

This is clearly an exciting time in CTI's corporate evolution. We have an opportunity to serve patients, their caregivers, and to ensure rapid and widespread access to new, less toxic therapies that will make a difference.

Additionally, I want to thank our employees for their commitment and tireless efforts. It's their collective passion and dedication to patients that help bring Pixuvri to market.

Now as we look ahead to 2013, allow me to summarize the key takeaways in today's update. So we intend to drive the use and adoption of Pixuvri in relapsed aggressive B-cell NHL in the EU with a positive net margin contribution; we want to secure reimbursement in Germany, U.K., France and Italy in the second half of this year; and complete patient enrollment in the PERSIST-1 Phase III trial of pacritinib, but within the 12- to 14-month period. I want to initiate the second Phase III trial for pacritinib in the so-called PERSIST-2 trial in patients with myelofibrosis in the second half of this year, and remain opportunistically focused on business development activities outside the U.S., including securing non-equity based capital through an x-U.S. partnership for pacritinib, in addition to establishing channel partners for Pixuvri, and then reporting results from investigator-sponsored studies on tosedostat and OPAXIO in hematologic and solid tumors at the scientific meetings.

So with that as a note, let me ask the operator to open the call for questions.

Question-and-Answer Session


[Operator Instructions] And our first question comes from the line of Burt Hazlett with Roth Capital.

Robert Cummins Hazlett - Roth Capital Partners, LLC, Research Division

I have 2 questions actually. First on Pixuvri, you mentioned you were going to talk with the regulatory agencies in Europe about moving from OS to PFS in terms of the endpoint. Can you characterize how much discussion you think that might take? And then the second, can you -- when we talk about your JAK2 inhibitor, there is a discussion generally with investors, there's a discussion generally about the GI AEs. Can you talk about how did those -- the AEs were managed in prior trials and how you're characterizing -- or how you're managing those side effects in the ongoing trials? Because I think there's a little bit of a question as -- in investors' minds as how they've been handled previously and how they will be handled going forward.

James A. Bianco

I'm going to actually ask Steve to address both of those questions with respect to the process for scientific advice, and since he's closest to the data under GI AEs and how they're managed.

Steven E. Benner

Sure. First for Pixuvri, we anticipate talking with the EMA representatives in the second half of this year. We believe those discussions should be straightforward based on their recent published guidelines and past discussions that the company has had with the EMA. Can't give you more than timing than second half of this year.

Robert Cummins Hazlett - Roth Capital Partners, LLC, Research Division

Would you like it to be an iterative process? Will you go back and forth, or is it potentially a one and done?

Steven E. Benner

I think the initial discussions will probably be with the rapporteur, and then we'll follow his guidance in terms of what subsequent discussions we should have with the agency more broadly.

Robert Cummins Hazlett - Roth Capital Partners, LLC, Research Division

And because of the endpoint change, would there be any potential for the data to come any earlier?

Steven E. Benner

We would hope so because if we were able to switch to progression-free survival, it leaves with substantial decrease in sample size from 350 down to the range of 200, 220. That would still make this a very large trial in this disease, in this patient population. So it would still be a very robust data set. And we're going to be in the process, as we said, about getting additional EU sites now that we're also launching the drug commercially in the EU, and hoping that those EU sites will help us boost accruals so that we could complete the study more quickly than under the current design. With regards to the GI adverse events, the most common side effects are diarrhea, the second is nausea. These GI side effects tend to occur within the first month of treatment. So 89% of the patients that have been exposed to pacritinib had experienced some diarrhea within the first month. Because there's an opportunity to assess early, these patients. And in the clinical protocols, the patients will be contacted by their sites and asked, if they're using a formal script, whether or not there's any change in the GI symptoms. And that will allow the sites to give them specific instructions with regards to what additional medications they will take. It will also be given at the time they start the medication, a prescription for IMODIUM or some comparable anti-diarrheal, and with the instructions to start taking that medication in the event that they have any problems. So those patients whom the clinicians are concerned about, if the possible risk of diarrhea might seem to be higher or the responsiveness of the patients to taking medicine might be decreased, those patients could be instructed to take the IMODIUM prophylactically just as they start the therapy. This kind of aggressive management was not done in Phase I, understandably, because it was trying to elucidate the toxicity profile of the drug overall. So it's only as you get to the Phase II experience that you start to see a difference in terms of the presentation of GI side effects. The most recent example we have from publication would be the lymphoma study that is published in the Journal of Clinical Oncology in September in which patients with lymphoma were dosed with pacritinib up to 600 milligrams, well above our 400-milligram per day dose in myelofibrosis. No Grade 3 or 4 GI toxicities were observed. So the GI toxicity is predictable. It's on target due to FLT3 and it's well managed by standard agents that the clinicians and patients are both very familiar with.


Our next question comes from the line of Ren Benjamin with Burrill & Company.

Reni J. Benjamin - Burrill & Company, Research Division

Maybe just starting off with Pixuvri, Jim. Can you talk a little bit about the sales force that you have in place? Is it optimal for Europe? And if not, how to best leverage? Is it just increasing the sales force, how do you best leverage forces out there to maximize the potential of revenues in Europe for 2013? And then just related to the previous question regarding PFS, just help me -- just remind me, I thought OS was decided as a prime [indiscernible] will no longer be used for [indiscernible].

James A. Bianco

I'll toss it over to Steve [indiscernible]. We currently have 19 people in the field. That includes 5 sales people, a country manager and an MSL in Germany, with 2 additional, what we call, key account managers, coming into Germany in the second week of March. We have a field complement that covers the Nordics and the Netherlands. And then we have a field complement in the U.K. And obviously, we wouldn't put people into territories where we don't have big access pricing. With respect to is it enough critical mass, the answer is this product is very promotionally sensitive. Salespeople, unlike in the U.S., is the #4 source of information for physicians in Europe that influences their treatment practices, coming right behind like journal ads and symposia. So they play a very important role. These are very advanced degree individuals, typically Ph.D.'s, Pharm D's or M.D.'s in the case of MSLs. And we have seen that as we have [indiscernible] into the field complement, because as you may recall, what we talked about in the fourth quarter, while we may have hired these folks under German law, for example, they need a 90-day waiting period before they can start and we work that into our timeline. So a lot of these folks have started out of the gate, January 1, and again, with some of the complement coming on in early March. So the answer is we think we have the right [indiscernible] with total by the end of the first quarter in the field. It doesn't include the 3 leadership positions that we have in terms of the General Manager, market access folks, et cetera. So from that perspective, again, when we say it's promotionally sensitive, we see that directly in terms of call frequency and then orders that come in behind that. Just one other note to give you a sense in Germany. In Germany, there's a process whereby the hospitals, when they're interested in the product, they can apply to the payers' systems, have it put on what would essentially be like formulary here in the U.S. Of the 2,000 hospitals in Germany, we targeted only the top 200 where the majority of the lymphoma patients are treated. And of those 200 hospitals, after seeing just a one-sheet product profile for Pixuvri, 146 of them applied to the regulatory agencies for formulary requests. So we're encouraged, we think we have the right complement now. And as sales start to ramp up, and importantly, when we get pricing decisions, then obviously, we'll implement the second half of the year, the additional sales personnel to try to make this a successful launch. Steve...

Reni J. Benjamin - Burrill & Company, Research Division

I guess just related to that, Jim -- sorry, just before we get to Steve, what are -- any thoughts regarding securing a marketing partner in Europe? And I guess this is another way of asking, has Novartis sort of just backed out of their option for taking over Pixuvri in Europe?

James A. Bianco

Let me put it in this way. We know that Novartis has, internally, a threshold for a product to have to have at least $500 million in sales before they would consider it a product that they would take to market. So clearly, we've never considered Pixuvri in the EU to be a $500 million marketed product, so it would not be surprising that Novartis would not want to take it forward in the EU. And then obviously, with respect to other partners, as I've mentioned in the script, we have been forming and will continue to form channel partners. In Turkey, we have an existing one, and we'll have one in Israel. There are mid- to large-sized pharma that have interest in taking Pixuvri in rest of world. So into Eastern Europe, China, the Pac Rim, India, South America, et cetera. And so those are the things that can -- I'll turn it over to Matt and give you a little bit more color on that since it's been his primary driver for the first half of the year, that and the PIX deal -- the PAC deal.

Matthew J. Plunkett

Yes. Just to add to Jim's comments, I think we're very gratified at the interest that we've seen in Pixuvri. And one thing to look forward from us later in the year is the opportunity to broaden the availability of the drug to cancer patients in territories beyond Western Europe, where we currently have plans to market the drug.

Steven E. Benner

Coming back to your initial question on progression-free survival, our goal for the PIX306 trial is to have that trial serve as the basis for obtaining full approval in the EU, and we're confident that PFS would be an appropriate endpoint for that body. We currently have no plans to refile pixantrone, and so we have it in the United States until we have additional data. And as you correctly raised, the FDA has not been favorably disposed to accepting progression-free survival as an endpoint. What we'll do is we'll complete that trial, look at the results, and then decide overall where we are as a company and what makes the most sense in terms of future filings for pixantrone in the U.S.

Reni J. Benjamin - Burrill & Company, Research Division

Okay, great. And then just switching gears to -- you mentioned in several scientific meetings where data will be presented. Can you potentially spell out? Would it be at ACR, ASCO, ASH? Do you know -- have these abstracts been submitted already? And will we see any follow-up studies or follow-up data or long-term data from the Phase II trials of pacritinib?

James A. Bianco

Information abstracts have been submitted to a variety of meetings throughout the year. Certainly, the biggest ones, both in the hem space and in the solid tumors space for both pacritinib and Pixuvri, so -- and tosedostat and OPAXIO. So I think you'll see some visibility on that in the future as those meetings come together.

Reni J. Benjamin - Burrill & Company, Research Division

Okay. And any -- will the pacritinib data, will that be further follow-up data from the ongoing Phase II trials, or will it be more preclinical, exploring the drug in indications like AML?

James A. Bianco

Steve, do you want to just briefly -- generally talk about what kind of -- obviously, there's no new data, not any new clinical trial data. But Steve will tell you on the rest.

Steven E. Benner

So we would anticipate the next presentations will take place at the DHAP and then at ASCO, abstracts we had shared with you today, actually. So we obviously don't know yet exactly what would be presented. We've done additional work on describing the pharmacokinetic properties of pacritinib. We have also reviewed the safety profile for the drug as well and summarized that data. So those are the kinds of presentations that you may be seeing in the next few months.


And I am showing no further questions. I'd like to turn the call back to Mr. Jim Bianco for any closing remarks.

James A. Bianco

Absolutely. Clearly, we're focused on delivering on the commitment -- our commitment to patients through the acquisition, development and commercialization of less toxic, more effective ways to treat and cure cancer. We're excited about our prospects for helping patients and driving shareholder value.

We'd like to thank you for your support of our mission, and have a good evening.


Ladies and gentlemen, this concludes the Cell Therapeutics Fourth Quarter Full Year 2012 Financial Results Conference Call. If you would like to listen to a replay of today's conference call, please dial 1 (800) 406-7325 or (303) 590-3030 and enter access code 4603217. We would like to thank you for your participation. You may now disconnect.

Copyright policy: All transcripts on this site are the copyright of Seeking Alpha. However, we view them as an important resource for bloggers and journalists, and are excited to contribute to the democratization of financial information on the Internet. (Until now investors have had to pay thousands of dollars in subscription fees for transcripts.) So our reproduction policy is as follows: You may quote up to 400 words of any transcript on the condition that you attribute the transcript to Seeking Alpha and either link to the original transcript or to www.SeekingAlpha.com. All other use is prohibited.


If you have any additional questions about our online transcripts, please contact us at: transcripts@seekingalpha.com. Thank you!