Savient Pharmaceuticals, Inc Q2 2010 Earnings Call Transcript

 |  About: Savient Pharmaceuticals, Inc. (SVNTQ)
by: SA Transcripts

Savient Pharmaceuticals, Inc. (SVNT) Q2 2010 Earnings Call August 4, 2010 9:00 AM ET


Welcome to the Savient Pharmaceuticals Second Quarter 2010 Financial Results conference call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will be given at that time. As a reminder, this conference may be recorded. I would now like to introduce your host for today, Mr. Philip Yachmetz, Senior Vice President and General Counsel for Savient Pharmaceuticals. Sir, please go ahead.

Philip Yachmetz

Good morning and welcome to Savient Pharmaceuticals second quarter 2010 financial results conference call. Last evening we issued a press release providing financial results and highlights for the second quarter of 2010. This press release is available on our website at

Before today's call I would like to read our Safe Harbor statement. Comments made during this conference call will contain forward-looking statements that involve risks and uncertainties regarding the operations and future results of Savient Pharmaceuticals.

In particular, we need to stress that when we discuss information regarding the FDA's review of our resubmitted KRYSTEXXA or Pegloticase Biologics License Application, or BLA, and whether the BLA resubmission combined with the submissions made or planned by our third party API contract manufacturer fully addresses the deficiencies and observations raised and provides the additional materials requested in the complete response letter that we received from the FDA on July 31st, 2009.

The status of remediation efforts by our third party contractor of the 43 deficiencies previously cited by the FDA, any data concerning the efficacy and safety of KRYSTEXXA, our preparations for regulatory submissions for KRYSTEXXA outside the United States, our work with secondary supply sources and the potential for FDA marketing approval for KRYSTEXXA as well as other related matters, no inference of the overall success with respect to these matters can be implied, as they are subject to a number of risks and uncertainties.

We encourage you to review the company's filings with the Securities and Exchange Commission including, without limitation, our quarterly report on Form 10-Q for the second quarter of 2010, which will be filed in the next couple of days and the press release issued by the company on August 3, 2010, which identify important factors that may cause actual results or events to differ materially from those described in the forward-looking statements.

Furthermore, the content of this conference call contains time sensitive information that is accurate only as of the date of this live broadcast, August 4, 2010. We undertake no obligation to revise or update our statements to reflect events or circumstances that occur after the date of this conference call.

Joining me on the call this morning is Paul Hamelin, our President; and David Gionco, Group Vice President and Chief Financial Officer.

Before I turn the call over to Paul, I would like to take a moment to remind listeners that we will continue to have no further comments at this time with regards to the announcement made last quarter regarding the strategic direction of the company. Savient's Board of Directors have determined after a careful and thorough evaluation of potential strategic alternatives for Savient, and with input from its financial advisors that, assuming the KRYSTEXXA BLA is approved by the FDA, a sale of Savient post approval would be the best way to realize the full global commercial potential of KRYSTEXXA and would be the optimal outcome for the company's shareholders and other stakeholders.

As Savient pursues this strategy as well as works its way through the next two weeks of the FDA review process for KRYSTEXXA BLA, we must emphasize that it would be very detrimental to the company's efforts to have any public disclosure or discussion of the status or progress of the process, a likelihood or timing of the sale transaction or the status of the FDA review of the KRYSTEXXA BLA, until such time if there is an announcement to be made for general consumption.

As a result, while Savient and its Board of Directors wish to make its announcement last quarter in order to help our investors understand our strategic plan and provide periodic updates on the FDA review process during these regularly scheduled Earnings Calls, we do not plan to make any further announcements or engage in any discussions regarding our strategic efforts and direction or the status of the FDA review, until and unless a definitive agreement providing for the sale of Savient is executed or we have received formal communication from the FDA with respect to its decision on the prospects of BLA.

I would also like to remind the listeners that this also means that we will not be attending or appearing at various investor conferences in the coming months and we will limit our discussions with analysts and investors to a strictly limited time following this conference call and following any future scheduled and announced conference calls.

At this time, I would like to turn the call over to Paul Hamelin.

Paul Hamelin

Thank you, Philip, and good morning everyone. Thank you for joining us. Today I'd like to discuss the key accomplishments that we have achieved during the second quarter of 2010, and I think we have had a very productive second quarter. Then I am going to turn the over the call to David Gionco, our Chief Financial Officer, who will review the financial results for the second quarter. We will then open up the call for a brief question and answer session.

As many of you know after acceptance of our resubmission of our BLA for KRYSTEXXA by the FDA in March of this year, we have received PDUFA of September 14. Since receipt of that notification, we have continued to interact with the FDA and in our opinion the dialog with the FDA has remain positive, collaborative and productive as we move into the last five weeks of the regulatory review process.

As we reported in our press release last evening, on July 28 we submitted to the FDA the final three months and six months stability data for the pegloticase API drug substance, validation batches that we manufactured late in October 2009, and also submitted with the final prospects of drug product manufactured from those same validation batches in November.

This was the final data package necessary for the FDA to complete its evaluation and review of our KRYSTEXXA BLA. All the stability data is within our proposed acceptance criteria of assays that the FDA requested as part of our ongoing stability program and we believe the data just submitted is consistent with what we've seen previously with the Phase 3 clinical trials, which has been stable for over three years.

I would like to remind everyone that we submitted this data to the FDA in response to the agency's request made during our September 2009, Type A meeting. At that meeting, the FDA did not want Savient to delay our prospects of resubmission, while we waited for the six months stability data. They recommended we submit early, which we did back in March and then send the stability data not later than 30 days prior to the assigned PDUFA date, which would provide adequate time to review the data.

A senior FDA official at that Type A meeting then asked his colleagues if Savient were to follow this guidance and this submission plan would they all be able to review the submission in time to get the PDUFA date. The answer from the members in attendance was, yes.

We've exceeded the FDA's request by having filed this data 49 days prior to the PDUFA date versus their 30 day request. As a result we believe we provided the FDA with almost three extra weeks of review time on this stability data as part of its evaluation for the BLA, which should be well within its review process timelines to allow for September 14th, PDUFA action date.

Over time, we had many inquiries about the stability data, so please recall from our earlier conference calls, that based upon our conversations with the FDA with the three and six months real time stability that we just submitted on the validation batches and the over 36 months of stability from several other batches that we previously produced, we believe that we're well positioned to receive at least 18 to 24 months of final product dating or shelf life as part of the approval process for the KRYSTEXXA BLA, but of course the always the final determination is in the hands of the FDA.

Over the last few months, we continue to manufacture additional commercial batches of KRYSTEXXA to support a US launch and foreseeable market demand beyond the initial launch period. Combining existing finished KRYSTEXXA drug products already on hand here in the United States, with bulk pegloticase drug substance product currently at Sigma-Tau, awaiting final vial fill-finish, by the time the PDUFA action date arrives or shortly thereafter we will have a total of seven batches of finished KRYSTEXXA product in the United States. This is more than adequate quantities to support a launch in the foreseeable market demand and we will continue to schedule additional batches of KRYSTEXXA to be manufactured in the fourth quarter of this year.

On a going forward basis, our primary API manufacturing partner and fill-finishing organization has adequate capacity to continue to supply bulk and finished product not only to support a US launch, but also launches on a global basis.

In speaking of our primary API manufacturing partner BTG Ltd or Bio-Technology General Limited or BTG as we refer to them, we are very pleased with the results of their remediation actions. BTG has provided documentation including pictures charts, diagrams and analytical data in five substantive reports that have been submitted over the last eight months to the FDA. We believe the remediation steps taken by BTG and its continuing commitments are fully addressing the concerns identified during the FDA's pre-approval inspection in June of 2009.

Let us provide you a little more color on the basis where I believe that we have effectively addressed the FDA's concerns. As you are aware, one of the open items from the FDA, which was specified in the complete response letter we received last July, was the need to remediate the deficiency and observations raised by the FDA in its June 2009 pre-approval inspection. To resolve these open issues, BTG and Savient brought in outside experts to create a plan that we committed to, which included a total quality improvement program and we agreed to provide extensive remediation reports with complete supporting documentation on the progress every other month to the FDA.

In November, again in December, again in February, again in April and again in June, we provided the FDA with these detailed reports and supporting documentation demonstrating the remediation of these manufacturing issues and an update on the total quality improvement plan.

As many of you will recall, in February of this year, after the November and December reports had been submitted, we received an unsolicited letter from the FDA stating that the corrective actions implemented and documented in the November and December reports by BTG and continuing additional commitment made by PEG, in those reports, appeared to address the FDA's concerns. The FDA also stated in its February letter that it will verify these corrective actions and additional commitment during the FDA's next GMP inspection of the BTG results.

More recently late last week, we received an email communication from the FDA's review team project manager responding to our enquiry, seeking clarification as to whether the FDA intends to re-inspect BTG's facility prior to the September 14th PDUFA action date. The e-mail states that the FDA re-inspection of BTG is not planned in support of the KRYSTEXXA BLA prior to the PDUFA action date.

While our principal objective as a company, is to obtain regulatory approval of KRYSTEXXA here in the United States, we have continued to make significant progress in other strategic areas that we believe will strengthen the value and expand the market opportunity of this asset, such as the filing in Europe.

The European regulations are of course different than here in the United States, and the European health authorities require a pre-agreed- upon Pediatric Investigational Plan or PIP, prior to accepting a Marketing Authorization Application or an MAA for their review. This is a major gating event in the filing for submission of an MAA in Europe. We have filed a Pediatric Investigational Plan to the EU authorities several weeks ago.

We have received comments on the proposed plan from the EMA committee. From our review and analysis of these comments we anticipate that we will provide our final response to the committee shortly and we believe we will obtain a positive ruling with respect to a Pediatric Investigation Plan. This PIP approval when received will enable us to advance our plan for a centralized European filing late this year.

We continue to believe that KRYSTEXXA represents the first ever approach disease modification for patients who suffer with chronic gout that is refractory to conventional therapy.

As part of the US approval preparedness plan, we have initiated key strategic initiatives for presenting scientific data in support of a potential product launch in the United States.

In doing so, we have actually submitted a total of seven abstracts that include data from our open label extension Program to the American College of Rheumatology for acceptance at their annual meeting in November.

Additionally, we've already submitted three manuscripts to leading scientific peer reviewed publications and within a few weeks we will submit up to three more manuscripts to peer review journals.

If published, we believe these new data will be well received by the physician population and the chronic gout patients' refractory to conventional therapy who currently have no other treatment options available to them. We continue to believe that if approved there will be rapid expansion of this market as patients and physicians seek out new life altering therapies.

As we are now approaching the final five weeks towards our September 14th, PDUFA action date, we as a company continue to remain diligent in our efforts of working with the FDA through the final stages of our regulatory review process to sustain our commercial launch readiness plan and to advance activities associated with a centralized filing in the EU. We believe these core activities will lead to sustainable results that will enhance the value of KRYSTEXXA and will increase value to our shareholders, patient and other stakeholders as we move towards a previously announced strategic objective for the company.

David, at this time I would like to turn the call over to you to give our colleagues here your financial update.

David Gionco

Thanks, Paul. Let's review the financial results for the second quarter and six months ended June 30, 2010 that we reported in last night's press release. Since I will only be discussing highlights from our financial results I refer you to our quarterly report on Form 10-Q when filed, for more specifics and details.

We ended the second quarter of 2010 with $89 million in cash and short-term investments, a decrease of $6 million for the quarter. During the quarter, an investor exercised his warrants and purchased [406000] shares of our common stock where we received cash proceeds from this exercise of $4.2 million. These warrants were issued in connection with our April 2009 registered direct offering.

The net loss for the second quarter of 2010 was $5 million or $0.07 per share, compared with a net loss of $54.8 million or $0.92 per share for the second quarter, a year ago. Significantly offsetting our net loss for the quarter is a non-cash gain of $6.2 million or $0.10 per share due to evaluation adjustment on our warrant liability.

On a non-generally accepted accounting principles basis and excluding the $6.2 million evaluation adjustment on our warrant liability, our net loss for the second quarter of 2010 was $11.2 million or $0.17 per share compared with a non-GAAP net loss for the second quarter of 2009 of $18.9 million or $0.32 per share. The net loss for the first six months of 2010 was $13.3 million or $0.20 per share compared with a net loss of $76.8 million or $1.35 per share for the first six months of 2009.

Looking more closely at the details, total revenues for the second quarter of 2010 were $1 million, an increase of $300,000 from the same period in 2009. Revenues for the first six months of 2010 were $2.1 million, also an increase of $300,000 from the same period in the prior year. Our revenues consist of product sales of Oxandrin, our branded drug that promotes weight gain following involuntary weight loss and Oxandrolone, our authorized generic equivalent of Oxandrin.

Research and development expenses were $7.2 million in the second quarter of 2010, down from $11.6 million in the second quarter of 2009, a decrease of $4.4 million. The lower expenses were primarily due to higher prior year cost of $2 million for consulting services primarily associated with our preparation for the June 2009 FDA Advisory Committee meeting for KRYSTEXXA, coupled with $1.1 million in higher outside laboratory testing services also in the prior year supporting our open label extension clinical study for KRYSTEXXA.

Year-to-date R&D expenses were $13.6 million or $10.8 million lower than 2009. The reasons for this decrease are mostly consistent with the reasons just discussed for the quarter. In addition, the lower year-to-date R&D expenses resulted from a decrease in current year compensation expenses as a result of lower headcount and from our reduction in force initiative in 2009.

Selling, general and administrative expenses were $4.6 million in the second quarter of 2010, a decrease of approximately $2.8 million from the second quarter of 2009. The decrease was primarily due to higher prior year marketing expenses of $2 million, related to preparation for the possible commercial launch of KRYSTEXXA in the prior year.

Year-to-date selling, general and administrative expenses were $9.5 million, $7.4 million lower than the 2009. The reasons for the decreased expenses are mostly consistent with what was just discussed for the quarter. Additionally, compensation expenses were lower year-over-year due to the reduction in force initiative implemented in September 2009.

To summarize, we believe we are financially well positioned to sustain operations. We have substantially lowered our operating costs during the first six months of 2010 resulting in a reduction in our cash burn and will continue to remain financially prudent in regards to cost containment as we monitor our liquidity and cash position on a go forward basis.

Our financial resources will allow us to sustain our operation through the regulatory review process for KRYSTEXXA and to support the ongoing project and initiatives of our strategic plan.

This concludes the financial portion of the conference call. I would like to now turn the call back over to Paul.

Paul Hamelin

Thank you, David. In summary, one, operationally we've continued to manage operations and expenditures in a fiscally sound manner to ensure our ability to sustain and advance all of our key initiatives.

Second, on the KRYSTEXXA BLA front, in our opinion our dialogue with the FDA has remained positive, collaborative and productive in our submission of the PEG modification KRYSTEXXA six month stability data last week.

Third, we believe that we've addressed each issues set forth by the FDA in a complete response letter from last July.

Fourth, we also believe that the remediation steps taken by our primary API manufacturer BTG and its continuing commitments have addressed the FDA's concerns identified during its inspection last June.

Through the coming weeks, we look forward to continuing our interactions with the FDA and to our upcoming PDUFA action date just five week away.

Amy, can I turn the call over to you to open up the lines for Q&A?

Question-and-Answer Session


(Operator Instructions) Our first question comes from Salveen Richter of Collins Stewart.

Laura Ekas - Collins Stewart

Hi, good morning. It's Laura on behalf of Salveen. I just wanted to ask you, I know there have been some changes at FDA with regard to the rheumatology division and I was wondering if you have any concern that's going to affect the timing of the KRYSTEXXA approval?

Paul Hamelin

Good morning. Thanks for the question. Yes, we have actually talked about in the past that the arthritis division has merged if you will with the pulmonary division. It's still remains under the leadership of [Dr. Kirk Rosebow]. He heads up both of these divisions now and Dr. Rosebow was at our advisory committee, and then in addition to that several people at the review divisions still remain responsible for this overall application and PDUFA date. So I don't think any of the changes that have gone on will substantially impact the PDUFA timeline as we go forward.


Our next question comes from Andrew Vaino of Roth Capital Partners.

Andrew Vaino - Roth Capital Partners

Just quick questions, you mentioned that you had an adequate number of doses in the United States. Can you comment on specifically how many doses can you just announce it?

Paul Hamelin

Andrew, other than just updating everyone that we've got seven batches that we've prepared for commercial lunch, we never really given quantifies of how much a batch contained. That will be a little bit of giving forward guidance I think if we gave that kind of level of data. So, I appreciate the question but I am going to have to take a pass and giving you an idea how large the batches are.

Andrew Vaino Roth Capital Partners

Okay. Then secondly how see your tech transfer going from the BTG facility in Israel?

Paul Hamelin

Yes, the tech transfer has gone well into Diosynth, which is a secondary API source manufacturer for us. So that tech transfer was substantially complete several weeks, several months actually.

Andrew Vaino Roth Capital Partners

So when do you expect them to [manufacture]?

Paul Hamelin

Yes, we are in the process of doing validation batches as we speak.


Our next question comes from Eric Schmidt of Cowen and Company.

Eric Schmidt - Cowen and Company

Good morning. Thanks for all the updates. Paul, appreciate your assurances on the fact that the FDA won't delay the filing due to the submission of stability data. Do you have any sense that they will also characterize the major amendments, some of your submissions on safety or the REMS program or anything else?

Paul Hamelin

Yeah, Eric, the safety update, the REMS materials, package insert, all of those were part of the original submission back in March of this year. That was actually required for us to send in that information in March directly because it was identified in the complete response as to what we needed this with. So again, all that information went in and March, we've not submitted amendments to those documents to this point in time.

Eric Schmidt - Cowen and Company

Thanks. Then, I was hoping you could outline for us a little bit more about the EMEA pathway here, I understand that the PIP is limiting and you are hopeful to get clearance on that in the short-term here but what else actually do you need to do following the PIP to actually file?

Paul Hamelin

Yes. We tend to think of the filing in three areas here getting ready for Europe. One is the PIP which we've outlined. The second step is actually for us, finishing what we call our 407 report; 407 is the number we assign to the open language extension.

When we file in Europe, the filing actually will be more complete if you will, or more robust than the filing that we originally put in 2008 with the FDA, because we now have the ability in this filing in Europe, to not only provide the Phase 3 study, studies 405, and 406, but the complete 407 open label extension.

So our next point is that, in the EMEA filing we will give all clinical efficacy and safety data, in a resubmission. So that is another important integration step that we are completing in the next few weeks. Then the third component of that, is we do need some additional expert opinions to accompany those filings and so we are working with, experts in Europe to help us in that endeavor.

Eric Schmidt - Cowen and Company

Then did I hear you correctly, the entire filing will go in, you think by year end, or you just to have a plan for the submission by year end.

Paul Hamelin

No, we are attempting to file the application with the totality of the data, by year end.

Eric Schmidt - Cowen and Company

Okay. Then a last question, could you update us on any commercial activities already necessitate that you might be doing, if any?

Paul Hamelin

Well, again in the context of our expressed strategic direction, that we announced in May, and we reiterate, that we think the best action plan for the company is, the sale of the company post approval or post PDUFA. Within that context we think that any potential acquirer wants in, would like to be able to potentially launch this product as rapidly as possible.

So as a result we continue to keep very important commercial activities all going forward, and that is actually what I was trying to refer to in my prepared remarks about the importance of providing more scientific data to ACR of giving upwards of six manuscripts submitted to peer review journals, that's an important of part of launch activity is to continue to have very extensive publication plan.

So we continue commercial activities like that that I think continue to add value to anyone who would be interested in acquiring the company.

Eric Schmidt - Cowen and Company

Other than scientific publications, limited other activities Paul?

Paul Hamelin

I mean relatively speaking, yes. Limited other activity, mean I give another example I highlighted is, we continue manufacture commercial batches of the product, and we'll continue to do commercial batches in the fourth quarter. All of that is part of launch preparedness and post launch preparedness.


Our next question comes from Joseph Schwartz of Leerink Swann.

Joseph Schwartz - Leerink Swann

Good morning. I was wondering since this is somewhat of a dynamic situation with respect to your strategic plans and then we're counting down to the PDUFA, which is coming up pretty soon.

Will you be announcing a price after approval? How will you handle that pricing decision? Are you going out to payers now?

It seems fairly unique since you contemporary, initially, presumably be in discussions with multiple parties who will be doing the analysis, which could influence the price for the company?

Paul Hamelin

Joe, in general companies can have conversations with payers whether they be private or public without having to express prices. So those activities are important in all companies preparedness for launch. Typically then company's post PDUFA or post approval when they begin to introduce product into the market that's the time or the point in time when a price needs to be disclosed publicly. So we will continue to work through our processes here and we will announce the price at the appropriate time just prior probably to submitting or shipping product as we go forward.

Joseph Schwartz - Leerink Swann

Then with that are you contemplating that you would ship the product or will you not ship product because your goal is to have your acquirer own the product and perhaps then?

Philip Yachmetz

Joe, this is Phil. I think it will be premature for us to speculate it to the exact timing of event after approval, given the announced strategy for the company, but we are mindful of the that the normal operating procedures for a company once they get approval in terms of timing for shipment and pricing announcement and so forth and we will be guided accordingly in the context of other things going on with the company.

Joseph Schwartz - Leerink Swann

Okay. Can you just clarify on what you said about the FDA re-inspection not planned and supportive of KRYSTEXXA BLA part of the action date event. Officially mean that day or not requiring it, or that they are just not planning it and there could be delay or something like that?

Paul Hamelin

Joe, what I would first of all say is again late last week we received an email communication from the FDA project team manager responding to our enquiry where we were seeking clarification as to whether the FDA intends to re-inspect the facility prior to the PDUFA. The email then stated that the FDA re-inspection of BTG is not planned in support of the BLA prior to the PDUFA action date.


Our next question comes from Gene Mack of Soleil Securities.

Gene Mack - Soleil Securities

Thanks for taking the question. Just want to follow-up on the a moment ago you were talking about commercial activity and I think what you said in the past is that regardless of where you stood strategically that you plan to have a fairly significant presence on ACR this year and that you would be planning in that along those lines. Is that still the case?

Paul Hamelin

Yeah, I think Gene, the fact that we've submitted seven abstracts to ACR this year is indicative of the fact that we think ACR is a very timely and important congress for us, I mean that is the largest U.S. Rheumatology meeting that's held every year. We think with a PDUFA for action date on the 14th, should we get approved, that's an important date to get information out to rheumatologists in the launch process. So we continue to plan for activities for ACR and that's clearly demonstrated by the fact we submitted seven different abstracts and we hope to have a very successful ACR.

Gene Mack - Soleil Securities

Okay. Can you give us an idea, I know you said you received the one email from FDA but can you give us an idea of, have you guys had pretty fairly frequent interaction with FDA since its reorganization or how much interaction have you had with FDA since the divisions are reorganized?

Paul Hamelin

Yes. In the past, we never go through tremendous amount of details of our interactions with the FDA, but since we have filed our application back in March, we continue to interact and in our opinion, the dialogue with the FDA has remained very positive, collaborative and productive right up until today. So we hope that continues here in the remaining five week. So it's been a consistent normal, good interactive level over the four and a half months since we filed the application.

Gene Mack - Soleil Securities

Okay. Then finally, can you give us some idea in longer term how would two facilities BTG and Diosynth mind wind up working in terms of manufacturing KRYSTEXXA? Is it your desire to it or is it likely BTG will remain the primary facility or at some point it will switch over to Diosynth they will be primary with some sort of back up there?

Paul Hamelin

Our plans have always been and remain to have our current API manufacture BTG and our current fill finisher Sigma-Tau to be the primary supplier of products around the world, and that's central our FDA application that's in and that will go in as the supplier and producer of both and fill finisher for the European application.

Over time after hopefully approval of both in the US and in Europe we will then take the opportunity for submitting and sBLA to then hopefully have Diosynth and an another fill finisher become a secondary supplier or a backup supplier to our global needs and global demand.

I said earlier in my comments or two we really believe that the current supply from BTG and Sigma-Tau were very much adequate to handle global launches and product supplies for the next few years. Again, if we just think from business standpoint it's prudent to have to backup or a secondary supplier that is approved in many markets around the world.


Our next question comes from Kim Lee of Global Hunter Securities.

Kim Lee - Global Hunter Securities

Good morning. Just a couple of quick questions on, will there be any follow up on patients on the extension studies and are you foreseeing any additional amendment to safety to the FDA?

Paul Hamelin

Kim, last year in July of 2009, we thought the drug exposure portion of the open label extension, for all intents and purposes patients were no longer receiving drug. At that point in time from July to the end of December we continued to follow and collect data on these patients for the six months that they were drug free immediately within that study.

That data, the totality of that safety data was submitted to the FDA March, and you will recall at the time in March, we said that the safety data of now the totality of the open label extension was very similar and we took that to the Phase 3 data that we had already submitted and was reviewed by the FDA and advisor committee, and we didn't any additional safety signals, we didn't see any difference in profile and we didn't see any difference as patients remained on drug, for in this case, we had almost 63 patients who've been on drug two to two and half years.

So there was no increase safety risk by being on longer-term therapy. So, all the safety data has been in the FDA for last few months, and it looks very consistent with everything that we reported prior to this point in time.

Kim Lee - Global Hunters Securities

Great. Then have you had any follow-up label discussions with the FDA?

Paul Hamelin

Well, again other than the comments that we have made in general about we had a very good productive dialogue through the course of last few months and the fact that we'd mentioned that we got an email about, that the agency doesn't believe there is a need to re-inspect the facility prior to PDUFA.

Other than those comments, we really don't want to and historically I've not commented on any kinds of dialogues that maybe ongoing or anything like that. I think if you can probably appreciate this close to PDUFA date five weeks away, call me conservative or superstitious but I think I just wanted to be a little bit cautious here in our remaining five weeks as we approach the PDUFA date.


Our next question comes from Katherine Xu of Wedbush Securities.

Katherine Xu - Wedbush Securities Inc

I am sorry, I jumped on a call a bit late, so can I ask a question an other way, have you received any increase or demands from the FDA that you think you wont be able to address by the PDUFA so far?

Paul Hamelin

Katherine, again as you would expect through the course of the review we had questions raised and we've responded to the FDA through those. The last remaining important information that's been requested was the six months stability data and that data went in on July 28.

So we've provided the FDA, not only there are 30 days that they have requested us to review the stability data but we've actually added almost three weeks of additional review time. So we've substantially responded to any and all enquiries over the last few months that the agency has raised and with the last component being an important one, which is the stability data that went in on the 28th of July.


Our next question comes from Carol Werther of Summer Street.

Carol Werther - Summer Street Research Partners

Could you describe in a little bit more detail when you think that pivotal trial results will be published?

Paul Hamelin

Yeah, good morning Carol. As I was indicating, we've submitted three manuscripts already to peer review journals, so we've got three more. Each peer review journal has a different time frame and process with which they review and either accept or decline.

So I think it's very difficult for me to give you any kind of specificities as to when we think these manuscripts will get published. I think the importance is that we are submitting them now. Then just again, depending on the journal, some of those can get published in relatively short time. Relatively short time might be three months, four months, and an intermediate time, six months, eight months or longer time frame of 8 to 12.

We think by submitting six difference manuscripts, what we will do, should they all be accepted is that we will have a steady flow of peer review journals coming up through before the end of 2010 and throughout 2011, which we think is very important commercially to do.

Carol Werther - Summer Street Research Partners

Then with the abstracts filed to the ACR, will we see, what happens to the patients after they come off KRYSTEXXA, in the open label, I assume that gradually there your [efficacy] levels will start to increase again?

Paul Hamelin

Yes, in the, in some of these abstracts, not all, but in some there is updates from the open label extension, and I think will continue to flush out, if you will what happens with these patients.

We know as I stating earlier from a safety standpoint, the drug continues to be very safe in these patients, and just qualitatively from an efficacy standpoint, we know these patients are receiving outstanding benefits because they continued, and wants to remain on therapy, and that is what I was indicating earlier, where we end up with.

So thinking in total now, we had almost 47%, 48% of patients who started on the trial, remained on drug for over 18 months, and we had 27%, no, actually closer to 30%, who have been on drug for between 2 and 2.5 years. So, I think that is a testimony to the safety and the efficacy of the drug.


Our next question comes from Steve Byrne of Merrill Lynch.

Steve Byrne - Merrill Lynch

Hi, thanks Paul. Presumably you have some patients that were not in this open label extension trials, and are interested in returning to KRYSTEXXA therapy, and you would have clinicians that participated in those trials that would also require, no selling pressure to move product, would you consider selling some product to these folks, if you're negotiations for a company sales drug drag out, just to register some quick sales?

Paul Hamelin

Steve, I certainly appreciate and understand your inquiry. We as a company are very mindful of the fact that this is an orphan drug. It is disease modifying in these patients. As I think we've all seen the results, it is life changing. So, we are very conscious of our obligation as a biotech or a biopharmaceutical company for the need to be able to provide patients access to this drug is quickly as we can.

Other than saying that we will attempt to do so and we'll just have to wait and see how advancing time lines plays itself out as we go forward post PDUFA, but we are concerned and we do want to help these needy patients who have approached us. We know there are patients who really want on this therapy as quickly as they can, we will attempt to accommodate that, however we can as we continue through this process.

Steve Byrne - Merrill Lynch

Okay, At UR there was poster on rasburicase for tophaceous gout and I was wondering what your sense is among European rheumatologist about the awareness or interest level in using rasburicase. It has a short half life, but what do you think the awareness level is for this for treating gout and what limits its utilization more broadly?

Paul Hamelin

Steve, just if I understand the question, what limits the use of rasburicase more specifically?

Steve Byrne - Merrill Lynch


Paul Hamelin

Let me start for that aspect. Rasburicase itself first of all indicative for tumor lysis syndrome and it's a very short course of therapy, it is five days. The reason why its five days and in tumor lysis syndrome is because this is the naked enzyme. Let's say it's a raw uricase protein and it is highly androgenic and any time beyond five doses and even in fact even within five dose exposure on five consecutive days for patients you can end up with frank anaphylaxis.

So it is very difficult managed drug. I think what was reported at UR has been actually some information that's been available for about two years, and basically there were some attempts to using it very small numbers of patients in gout to see if it could have some disease modifying benefits but every one.

So now I am going to transition from just that report to almost all rheumatologists in Europe and certainly all rheumatologists here in United States recognized the increased safety risk of using rasburicase on anything other than short-term one-time use in tumor lysis syndrome. That's why there is so much excitement both in Europe and here in the US. We're giving KRYSTEXXA out on the market, because this clearly is a more sustained and safer approach to treating chronic gout in patients who are refractory to conventional therapy.

The enthusiasm here in the United States in the rheumatology community is much higher right now because the prospects of approval are closer or more eminent. So believe me in all the market research that we have conducted in Europe and all the thought leaders that we interact with in Europe they are equally excited and anxious to begin utilizing the products whenever they can.

Steve Byrne - Merrill Lynch

Thank you.

Paul Hamelin

It would also Steve, I just want to comment. I know we get asked about market size. Market size in Europe is actually larger than the market opportunity here in the United States. So we continue to think that in the future, Europe will be a very, very important market for KRYSTEXXA. Any other questions?


I am showing no additional questions sir.

Paul Hamelin

Okay. All right. With that I want to again thank everyone for joining us here this morning and we are going to conclude our second quarter earnings call. Thanks everybody. Have a great day.


Ladies and gentlemen, this does conclude today's conference. Thank you for your participation and have a wonderful day. You may all disconnect.

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