Synageva BioPharma's CEO Discusses Q4 2013 Results - Earnings Call Transcript

Mar. 3.14 | About: Synageva BioPharma (GEVA)

Synageva BioPharma Corporation (NASDAQ:GEVA)

Q4 2013 Earnings Conference Call

March 3, 2014 4:30 PM ET

Executives

Matthew Osborne – Head, IR

Sanj Patel – President and CEO

Anthony Quinn – Chief Medical Officer and Head, Research and Development

Carsten Boess – CFO

Analysts

Navdeep Singh – Goldman Sachs

Chris Raymond – Robert W. Baird & Co.

David Friedman – Morgan Stanley

Geoff Meacham – JPMorgan

Operator

Good day, ladies and gentlemen, and welcome to Synageva BioPharma Fourth Quarter and Full Year Results 2013 Earnings Conference Call. At this time all participants are in listen-only mode. (Operator Instructions). As a reminder this conference call is being recorded.

I would now like to hand the conference over to Mr. Matt Osborne, Head of Investor Relations. Sir, you may begin.

Matthew Osborne

Thank you. Welcome to Synageva’s year-end 2013 conference call. During the call we plan to discuss operational, clinical, and financial results for Synageva.

Before we begin I would like to remind you that certain statements will be made today which are forward-looking within the meaning of the securities laws, including those regarding our product development programs, the expected timeline for achievement of our clinical milestones and our future financial results. Owing to the uncertainties of forward-looking statements our actual results may differ materially from anything projected in these forward-looking statements.

These statements speak only as of today’s date and we assume no obligation to update them. For further information on risks and uncertainties please refer to the risk factor section of our recently filed 10-K for 2013 and other reports and documents we have filed with the SEC.

The format for today’s call will include opening remarks from Synageva’s management team, and then we will open up the call to take your questions. I would now like to turn the call over to Synageva’s President and CEO, Sanj Patel.

Sanj Patel

Thanks, Matt. Good afternoon, everyone and thanks for joining us on this call. Also in the room with me are Carsten Boess, our Chief Financial Officer; Dr. Anthony Quinn, our Chief Medical Officer and Head of R&D; and Dr. Greg Grabowski, our Chief Scientific Officer.

Dr. Quinn is going to provide note overview of our lead development programs, sebelipase alfa for LAL Deficiency and SBC-103 for MPS IIIB. Carsten will provide an overview of the fourth quarter and full year financial results as well the 2014 financial guidance. Before that I will review our achievements in 2013 and outline the goals for 2014 and beyond.

In 2013 we made solid progress towards becoming a fully integrated global organization that’s focused on the development, manufacturing and commercialization of therapies for patients with rare devastation conditions. With sebelipase alfa for LAL Deficiency during the past year we initiated and completed enrolment in the Phase 3 ARISE trial in children and adults with LAL Deficiency.

This is a global trial that includes sites and patients located in key areas of our commercial focus; North America, South America, Europe and Japan. We completed enrolment in this trial at the end of 2013 and plan to release top line results during the third quarter of this year. This will put us on track to complete regulatory submissions in both the U.S. and Europe by the end of the first quarter of next year.

Early this year we also announced that we met the enrolment target in the Phase 2, Phase 3 trial in infants with LAL deficiency, as LAL deficiency is a single disease that presents across a clinical continuum, data of the [mistrial] will also be included in our planned global regulatory submissions. In a moment Tony will describe the initial top line results from the infant study and some of the other compelling data that was presented at last month’s LDN World meeting at San Diego.

Now I want to focus on manufacturing. We have taken a number of steps towards establishing a continuous supply of drug for patients with these chronic conditions that require lifelong therapy. Sebelipase alfa, SBC-103 and most of our other pipeline programs are produced using our proprietary manufacturing platform. This system has a number of advantages including the potential to produce proteins consistently and sustainably. The platform could also produce proteins with favorable glycosylation resulting in better cellular targeting and uptake in key target cells.

We’ve completed construction of an additional protein manufacturing facility in Massachusetts and this complements our existing facilities. In addition we are building a new upstream facility in Georgia as part of our overall corporate strategy and have initiated a process to secure additional ex-U.S manufacturing facilities.

We continue to build commercial presence around the world. During 2013 we placed regional country managers in key markets including the U.S, Europe, Japan and Latin America. As we plan for the launch of sebelipase alfa we will expand this infrastructure with additional personnel including medical science liaisons throughout the year. Their goal will be continue to raise disease awareness and find even more patients with LAL Deficiency. And longer term they will support the broader portfolio of Synageva programs.

To further build upon the solid achievements Synageva team I’d like to now focus on our key objectives going forward. First we plan to report top line results from the Phase III ARISE trial in children and adults with LAL Deficiency in third quarter of 2014. Secondly we plan to complete the U.S. and European regulatory submissions for sebelipase alfa by the end of the first quarter of 2015. And this includes data from the Phase II/Phase III trial in infants and the Phase III ARISE study in children and adults. Following that during 2015 and beyond we plan to file for approval in Japan and other countries including Brazil, Argentina and Mexico.

Third we will continue to increase disease awareness. We are making tangible progress towards raising global disease awareness and this will remain a key priority for 2014 and beyond. Our teams continue to implement patient identification programs around the world. We are working with various treatment centers, hospitals and academic institutions to take broad patient populations and then apply specific disease criteria to help enrich population for diagnostic testings and to ultimately identify more patients. By the end of this year we plan to have at least 30 of these programs up and running which is double the amount of programs we had last year.

Next we plan to enter SBC-103 to MPS IIIB into the clinic mid this year. MPS IIIB is another devastating, multi-systemic lysosomal storage diseases which has prominent CNS manifestations. The potential therapy that we are developing is the recombinant version of the natural human NAGLU enzyme.

Our goal is provide an effective, practical and safe method of treating these patients. We are focused and driven to build upon the preclinical data that was presented at last month’s LDN World meeting and we believe that we are on track towards doing this with SBC-103.

Beyond SBC-103 our other preclinical pipeline programs continue to advance. All of these programs are protein therapeutics and improving the lives of patients with rate and devastating conditions. We plan to advance two new programs into the clinic by the end of 2016. This means that including SBC-103 our goal is to enter three programs into the clinic over the next three years.

Overseeing all of this is a brilliant team and that team has done a lot of work today, including advance our pipeline programs closer towards the clinic and we look forward to describing these programs in more detail in the future. My personal goal and that of the senior team is to create a generational company. We continue to proactively expand our pipeline opportunities.

We have a solid R&D organization under the direction of Dr. Anthony Quinn. In 2013 we augmented our ability to further expand our opportunity sets with the addition of Dr. Greg Grabowski as Chief Scientific Officer. As many of you on the call know Dr. Greg Grabowski is a leading authority in rare genetic diseases and has been an integral part of bringing several lifesaving treatments to patients. In his role at Synageva Dr. Grabowski is responsible for progressing our current pipeline programs and identifying new rare disease opportunities. I have known Dr. Grabowski for many years now leading up to his decision to join Synageva, I can tell you he is fitting in very well and it’s exciting to have him as part of team.

Since we formed Synageva in 2008, our goal, focus and drive has been to meet the needs of patients with truly devastating conditions and high unmet need. We aim to transform patient lives. Our approach and mantra at Synageva is to be rigorous, thoughtful and truly innovative in everything we do. And this includes our operational expertise in manufacturing, clinical and commercial development and also our strategic plans, including financing, and creating new opportunities for the company. We will continue to do this as we build for the future.

I will now turn the call over to Dr. Quinn who will review the progress on our programs. Tony?

Anthony Quinn

Thanks Sanj and good evening everyone. As Sanj mentioned I’d like to share with you the recent progress with our lead programs. And as we’ve highlighted previously we believe it’s important to first present our data at scientific meetings and last month’s LDN Meeting gave us a great opportunity to file updates on a number of activities.

The Lysomal Disease Network Symposium is an important annual conference for us to share new data with geneticists and key opinion leaders in the area of lysosomal storage diseases. For sebelipase alfa, LAL Deficiency and SBC-103 there were 10 abstracts, three that were selected for oral presentation, including two oral presentations for sebelipase alfa that I’ll now review.

Before I describe the initial data for the ongoing Phase 2/3 studies sebelipase alfa in infants with LAL Deficiency that were presented at the meeting I’d just like to spend a few minutes providing an overview of the natural history of infants with LAL Deficiency in order to put these results into context with the results in sebelipase alfa in this patient population.

As Sanj mentioned LAL Deficiency is a single disease. It presents across a clinical continuum through infants, children and adults. The infant form of the disease is very rare and even though the clinical presentation is dramatic the low awareness means the cases are either not diagnosed or are diagnosed late in the course of the disease. As a result of our efforts to raise the disease awareness we are identifying more infants with LAL Deficiency. But we are still seeing some cases with advanced disease who died before we could get him transferred to the clinical trial sites.

Infants have the most rapidly progressive presentation with marked failure to thrive and severe hepatic disease due to the build-up of the abdominal fat in the livers and also in the wall of the gut. There is no effect of therapy and typically these infants die before six months of age. We presented two key data updates in LAL Deficiency in infants at this year’s LDN Meeting.

First in the natural history study we described details of the clinical presentations, progression and outcome in infants. Of the 45 infants enrolled in the natural history study all 45 died. 41 had clinical evidence of failure to thrive and 26 fulfilled the same strict definition of growth failure in the first six months of life that was an inclusion criteria in our Phase 2/3 treatment study.

As anticipated this population demonstrated the lowest survival rates with a median age of death of only three months. Aggressive liver disease also contributed to early mortality with transaminase levels up normal diagnosis and increasing with disease progression. The study also confirmed some published findings that there is rapid development liver fibrosis. In addition seven of the 10 infants who underwent Hematopoietic stem cell transplants died before nine months of age and all were dead before full years of age.

Moving on to the Phase 2/3 three study, from the six infants in the ongoing Phase 2/3 study who continued to receive treatment, five have already met the primary endpoint of survival at twelve months of age and the six continues on treatment beyond six months of age. Two infants died shortly after starting study due to the late stage and rapid progression of the disease and one infant died shortly after starting the study due to a non-study related procedure.

Adverse events with sebelipase alfa were mostly mild to moderate and serious adverse events were mainly related to central line infections or hospitalizations for treatments with antibiotics. We continue to treat these infants and will provide further update as to their progress at the appropriate time.

Moving across the clinical spectrum to LAL Deficiency presenting in children and adults, when we started working on this disease four years ago we recognized that a number of aspects of the disease were not well understood. As we’ve highlighted previously LAL Deficiency is an underappreciated cause of cirrhosis and premature cardiovascular disease with a high potential for either a delayed or a misdiagnosis. A poster at this year’s LDN describes some of the key epidemiology and complications in these patients in an observation study in 48 children and adults.

Most currently identified cases were diagnosed in childhood. Elevations of ALT were common, persistent and typically present from a young age. Increases in LDL cholesterol, the bad cholesterol were common with 88% of recorded values greater than 100 mgs per deciliter and a number of individuals had LDL values in a range indicating substantial dyslipidemia by that I mean more than a 119 mgs per deciliter.

Liver fibrosis was also common and six of 48 patients required a liver transplant including four patients who required a liver transplant before 18 years of age. As the data at the world meeting showed the use of lipid lowering medication on a median age of 11.3 years appeared to have limited effect witnessed in improving markers of disease activity, including the LDL level and the ALT levels.

In terms of sebelipase alfa infused patients we continue to treat adult patients as part of the ongoing Phase 1/2 expansion study. Data from six patients out to 90 weeks presented at the meeting continued to demonstrate sustained reductions in ALT and AST which are biomarkers of liver injury with levels frequently into the normal range out to 90 weeks.

In addition sebelipase alfa maintained improvement in the dyslipidemia associated with LAL Deficiency with decreases in LDL, decreases in triglyceride, increases in HDL from the pre-treatment baseline through to 90 weeks. Sebelipase alfa continue to be well tolerated through 90 weeks with every other week dosing, infusions reactions were uncommon and mainly mild, no anti-drug antibodies have been detected in the patients in the extension study and no drug-related serious adverse events have been reported in this study to date.

The findings from the Phase 1/2 study along with our understanding of the disease allowed us to design the Phase 3 study for sebelipase alfa in children and adults with LAL deficiency. And I’d now like to discuss that.

As Sanj mentioned we plan to provide top line results from the ARISE clinical trial during the third quarter of this year. ARISE is a global Phase 3 study that enrolled 66 patients. Patients are randomized to every other weak infusions with sebelipase alfa of placebo for the double-blind treatment period of 20 weeks. All patients who participate in the trial will receive sebelipase alfa after 20 weeks as part of the long term open label expansion period.

Data from the double-blind period at 20 weeks would be used to support global submissions for sebelipase alfa registration. The primary endpoint of ARISE is the proportion of patients relative to placebo who achieve normalization of ALTs a marker of liver injury at week 20.

In addition key secondary endpoints will measure the effects of sebelipase alfa on a broad range of epidemiology seen in these patients including reductions in LDL, serum triglyceride liver fat content and liver volume, increases in HDL and improvements in liver pathology. Following release of the top line results from ARISE we will compile this data, the infant survival data along with pre-clinical CMC and other data in order to complete regulatory submissions in the U.S. and in Europe by the end of the first quarter of 2015.

I would now like to turn your attention to our next program SBC-103 and Enzyme replacement therapy for MPS IIIB which is also known as Sanfilippo B syndrome. This is a devastating disease with marked CNS involvement leading to developmental delay, progressive cognitive decline, behavioral problems, loss of independent mobility and added mortality. We’ve been able to successful produce a recombinant version of the natural human NAGLU enzyme with glycan structures that allow cellular off-take.

Our data from last year’s LDN shown firstly methods for quantifying heparan sulfate disaccharides and this allows measurement of the substrate that accumulates in these patients. And secondly we showed last year that treatment with SBC-103 our recombinant human NAGLU using a variety of dosing approaches produced dose dependent reductions in the abnormal substrate in the brain, liver and kidney tissues.

Building upon that data the SBC-103 pre-clinical data presented at this year’s meeting confirms those results. It describes the potential mechanism for central nervous system uptake and it supports further clinical investigation for the SBC-103 for intravenous administration. Specifically we’ve demonstrated in an in-vitro model of the blood brain barrier and in non-human primates that intravenously administered SBC-103 may have properties that allow it to cross the normal blood brain barrier. Additional analysis indicated that the absorbed specific cellular transport was mediated by the mannose-6-phosphate receptor.

With these data over the next several months we’ll be focused on completing the pre-clinical toxicology and pharmacology studies to allow for the initiation of the first in-human trials in mid-2014 and we’ll provide further details of this study as we approach its start. In addition to sebelipase alfa and SBC-103 we are making progress with our other pre-clinical programs all of which are protein therapeutics for patients with rare and devastating conditions. One of the corporate objectives is to advance two new program into the clinic by the end of 2016 and this will be a major focus for myself and the team.

Bringing Greg Grabowski on board provides us with additional skills which complement the current R&D team, and Greg will be focused on progressing our current pipeline programs and leveraging his exceptional knowledge of genetic disease to support identification of new pipeline opportunities.

So with that I’ll turn the call over to Carsten.

Carsten Boess

Thank you, Tony. I will review Synageva’s year end 2013 results and discuss with you our financial guidance for 2014.

For the year-ended December 31, 2013 Synageva reported a net loss of $95 million compared to a net loss of $43 million for the corresponding period for the prior year. Revenue for the full year ended December 31, 2013 of $13 million consisted of $7 million of Fuzeon royalties from Roche, as well as revenues from $6 million from Synageva’s collaboration with its partners.

GAAP operating expenses for the full year 2013 including R&D and SG&A expenses totaled $109 million. This compares to total operating expenses, including R&D and SG&A expenses for the full year 2012 of $58 million. Synageva has cash, cash equivalents and short term investments totaling $409 million on December 31, 2013 compared with $219 million of cash balance at December 31, 2012.

Now turning to our guidance for 2014. For the full year 2014 we expect a GAAP net operating loss guidance of between of $190 million and $205 million. The GAAP net operating loss is primarily due to investments necessary to support the following: Firstly, global clinical development program for sebelipase alfa; further preclinical and clinical development of SBC-103; continued expansion of the global commercial and medical infrastructure; additional manufacturing capabilities as well as continued investments in the advancement in Synageva’s pipeline programs as Sanj and Tony discussed.

The point is we’re investing in all of these activities progressing in parallel. I’d like to give you a sense of the components of the 2014 operating expenses and where that increased investment from 2013 is going. Since we expect a future royalty revenue to be in the mid-single digit millions of dollars in 2014 our net loss guidance is a closer approximation to our planned total operating expenses. And of the total operating expenses for 2014 we expect approximately 70% of the investment is going towards R&D that includes completion of the Phase III sebelipase alfa clinical trial and other ongoing studies, regulatory submissions, dropped supply for lead and pipeline programs and advancement of the pipeline programs.

Approximately 30% is going towards SG&A, primary to support the global commercial infrastructure required to help continue to raise disease awareness, support the planned launch of sebelipase and in the future, our pipeline programs.

In addition we are already planning internationally for commercial and technical operations to create a true, global supply chain. We believe these investments across the functional areas including clinical, research and development, commercial and manufacturing will help drive the business in a scalable yet efficient way.

With that let me turn the call back to the operator to open it up for questions.

Question-and-Answer Session

Operator

Thank you. (Operator Instructions). And our first question comes from Navdeep Singh from Goldman Sachs. Your line is open, please go ahead.

Navdeep Singh – Goldman Sachs

Hey, good afternoon guys and thanks for taking my questions and congrats on the progress in 2013. My first question is on manufacturing. So you have mentioned that your Massachusetts facility is now up and running, I am just wondering how many manufacturing facilities will you need to accommodate the projected demand for sebelipase and in optimal production how many patients can each facility accommodate?

Sanj Patel

Thanks, Navdeep this is Sanj Patel. So I don’t think we have a number. I can tell you our existing and primary facility is already – have the capacity to supply the worldwide supply of the LAL Deficiency patients that we project. The reason why we developed an additional facility one would say for redundancy purposes but also to ensure that we have more than one – obviously more than one supply system.

We may well increase that, we are trying to expand, have a new facility in Georgia as well in addition and also in the future ex-U.S manufacturing facilities. But those are primarily because we are trying to have multi products out there globally. But as I said the primary facility that we’ve had over the last couple of years, three or four years can supply the worldwide supply. We are developing additional ones for our redundancy as well as planning for additional products in the future.

Navdeep Singh – Goldman Sachs

Okay. That’s helpful. And then a follow-up on the Sanfilippo program. At World we saw data come out of SBC-103, also competitive data for BioMarin’s BMN-205. So I was wondering if you could just discuss the competitive position between the two products I understand that BioMarin going ahead with an intra-cerebral injection and I was wondering if the BioMarin product was, I guess more appropriate for the most severe patients or your product might be more appropriate, for I guess like everyone any color on that would be helpful?

Sanj Patel

Yeah so Navdeep I’ll start and then Tony can jump in. So first of all we are not going to comment on other peoples programs. I think it’s very difficult to make direct comparisons, as assays can vary from laboratory to laboratory. What I would say is that we are focused on producing the recombinant version of the natural human NAGLU enzyme. And as Tony mentioned we believe we are getting sufficient amounts of the drug uptake into the brains. Our goal is to provide a very practical safe and effective treatments and we are going to focus on that really primarily on our own goals and own missions for these patients but Tony I am not sure if you wanted to add anything to that.

Anthony Quinn

I don’t have anything to add to what Sanj has said.

Navdeep Singh – Goldman Sachs

Okay, guys. Thanks a lot.

Sanj Patel

Thanks, Navdeep.

Operator

Thank you. Our next question comes from Chris Raymond from Robert Baird. Your line is open, please go ahead.

Chris Raymond – Robert W. Baird & Co.

Hey, thanks. Maybe just a couple of questions on 103. So I just wanted to clarify, I thought I heard you guys say at world that the plan was to go forward when you start that first in human trial in both IV and intra-thecal administration, is that true, can you just verify that and talk about how that trial design might shake out?

Anthony Quinn

Yeah, I will specify, this Tony Quinn the Chief Medical Officer and the Head of Research. – question the key thing is as that we build into our programs is optionality, is that like many diseases and some people we know – and we a lot about the disease but there are some things that we don’t know. If we boxed ourselves in by purely looking at an approach where we were directly injecting into the brain, or we boxed ourselves in by doing an approach where we just inject intravenously then we will be limiting our optionality.

And as I said we presented very compelling data that about the intravenous dosing and that it is able to cross into the brain in normal animals and in a model of the blood brain barrier and we will be moving forward and initially with intravenous dosing.

Chris Raymond – Robert W. Baird & Co.

Okay. So just IV, so then maybe just one more question, follow-up on sebelipase alfa, so you obviously had some impressive infant data but there is going to some sizable amount of time here between now and when you ultimately file and get approval – for approval. Have you had discussions with either FDA or EMA about a sort of a standard access or compassionate use program for specifically in infants between now and then?

Anthony Quinn

So as we confirmed we’ve completed the targeted recruitment in the clinical trial. We recognize that in the infants and this disease it’s a medical emergency, it’s a very rapid progressive disease, it’s a very rare disease and obviously we will be working through with all the stakeholders about how to manage those infants moving forward and as we proceed to a regulatory filing.

Sanj Patel

So and basically Chris the answer is that while is the enrollment target completed we do have the optionality whereby that if there is an infant that is early enough and we believe could be a good candidate we do have the optionality to be able to include that. We’re also looking at other compassionate use types of programs, we’ve done that in the past. As you know the very first infant initially started off on a compassionate use basis. So we’ve got that optionality as well.

Chris Raymond – Robert W. Baird & Co.

Thank you.

Operator

Thank you. Our next question comes from David Friedman from Morgan Stanley. Your line is open, please go ahead.

David Friedman – Morgan Stanley

Hi. Thanks for taking my question. Just around some of the manufacturing, do you expect that the addition of extra facilities will change your sort of outlook on cost of goods or is it a capacity redundancy sort of part of the equation? Thanks.

Sanj Patel

Thanks, David. It really is a capacity and redundancy and also again building towards multi-product facilities going forward. So I think we’ve started toward that, of course we have this focused on sebelipase alfa and also producing the SBC-103. As I said we’ve got a real mission internally to accelerate the opportunities in front of us and that means being ready for additional products initially in the clinic and then subsequent ally commercially.

David Friedman – Morgan Stanley

Just one quick follow-up. Overtime do you expect that each facility will make all drugs or are you going to have some do one drug, some do another drug, what would be the thought there?

Sanj Patel

So each facility has the ability to have more than one drug. But obviously I think you are right. We’re absolutely very careful as to how we overlay that. So I don’t think we will ever have a same scenario whereby you have all of them in one facility I think it would be very carefully thought about and lot of it depends on the trials and where the patients are in terms of that. So I think we will have to look at that very carefully going forward from now.

David Friedman – Morgan Stanley

Thanks a lot.

Operator

Thank you. Our next question comes from Ian Somaiya from Nomura Securities. Your line is open, please go ahead.

Unidentified Analyst

Hi. Good afternoon. Thank you, it’s Matthew on for Ian. Just a couple of quick questions if I could; first, as we think a little bit further and you’ve talked about some other markets that you’ll be getting ready to file in, I mean Japan and Latin America. Is there anything, any color that you can give us around potentially differences in epidemiology in these territories or may be thought about it different way the level of disease awareness, are there any patients that have already been identified in any of these territories, any color on that would be great, thanks.

Sanj Patel

So let me start and then Tony will jump in. So you are obviously referring to sebelipase alfa in LAL deficiency.

Unidentified Analyst

Yeah.

Sanj Patel

So this is a pan-ethnic disease and they aren’t unlike Gaucher, there isn’t really an ethnic propensity in certain areas. That said there are also in countries, continents that are very important and certainly Latin America we already finding is becoming a very important path of the story, likewise we’ve got a presence in Japan, Turkey and some other countries. So a lot of that comes from physician interest, previous experience of the LSD’s, therapies and then just patient communities.

So that tend to be why there is somewhat, sometimes a different distribution in terms of how the patients are but it’s really more terms of physician interest and the work that been done to identify patients. But Tony you want to jump in?

Anthony Quinn

Yeah I mean the only think to add it is having the global footprint and having the infrastructure in the countries is very important. I’ll give you one example, so we initially based on the common mutation and it was thought that LAL deficiency might be uncommon in Japan but we are having presence in Japan and been able to look at Japanese literature more closely, we now know that the mutations that underlie the disease in Japan are different from the west, that’s been true for other lysosomal storage diseases and I think that just shows you the value of having that global infrastructure, so that you can drill down into the detail in the countries.

Unidentified Analyst

Okay, thank you and if I get a squeeze in one second question, you’ve mentioned a couple times as well potential ex-U.S. manufacturing. And I was just wondering if you could provide a little color on timing and in fact should we assume or think that these are going to be potentially in places with favorable tax treatment as some other biotechnology companies have been doing recently?

Carsten Boess

Yes, hello, thanks a lot for the question. This is Carsten Boess, the CFO. We are building a global infrastructure and it’s encompassing both the manufacturing, the commercial and the medical infrastructure outside the U.S. So you can rest assured that we are taking a true global view also in terms of our tax planning and our tax calculations going forward. We have not set a particular time for it but rest assured we are focused on it.

Unidentified Analyst

Okay, thanks, that’s very helpful, thank you.

Operator

Thank you. Our next question comes from Salveen Richter from Canaccord. Your line is open. Please go ahead.

Unidentified Analyst

Good evening. This is Andrew on the line for Salveen, and my apologies if you answered this, just wanted to clarify, are you ruling out intra-thecal dosing in 103 or you are just going to start with IV and leave that option open?

Anthony Quinn

As I said earlier is that the key thing is to build in the optionality or being able to look at both approaches. So we have our optionality, and but as I said earlier we will be progressing with intravenous dosing.

Unidentified Analyst

Okay, fair enough. And then I wanted to, I realize it’s just a little preliminary but can you give us any thoughts on how you are thinking about pricing here?

Sanj Patel

So obviously we understand the pricing dynamics very well in the space. We have not determined the price yet and there is an awful lot of work that needs to be done before we come to that conclusion. But we are rest assured very focused on continuing to execute. We’ll do that and we clearly have a drug that has already made a big impact. We are going to continue to approach that in the right way but we have not determine that and that’s why we do understand very – what we have here as far as potential therapy.

Unidentified Analyst

Okay, great thanks.

Operator

Thank you. Our next question comes from Geoff Meacham from JPMorgan. Your line is open. Please go ahead.

Geoff Meacham – JPMorgan

Good afternoon guys and thanks for taking the question. Guess, I got one for Tony. So when we look at the ARISE study you guys have learned a lot on LAL over the years. I was just curious in terms of what you guys have found out in terms of the patients that were able to normalize ALT’s without sebelipase? Then what’s just kind of the natural background rate of, I guess you called, recovery or something like that in these patients then obviously is a proxy for what could go right and what could go wrong in the ARISE study?

Anthony Quinn

Okay so Geoff I’ll answer that question. So basically we did present data from our observational study and at the world meeting in Santiago and that’s probably the most useful dataset addressed by question. And the bottom line is as I said earlier is the ALT’s are tend to be persistently elevated from early childhood and once they elevated they stay elevated and clearly that’s very different from the path from that we observe and in CLL1 and CLL4 studies we have shortly after initiating an Enzyme treatment we saw a rapid and sustained reduction in transaminases.

Geoff Meacham – JPMorgan

That’s right and then as a follow up to that the enrolment in the study obviously went well ahead of expectations, Street expectations and went a lot faster. Curious though if there any way to read through what centers worked better than others in terms of awareness or may be diagnoses or things like that as kind of a read through so what could happen or what strategies could be utilized commercially?

Sanj Patel

Yeah, Geoff. It’s Sanj, so I think bottom line is you have to execute and certainly in the areas whereby there was – there were these patients identification programs, speed programs going on, using the dry blood spot, while it was working well, while the physicians were engaged, why they understand the system, you had a better read through, and I think that’s a learning that will definitely go towards the commercial launch.

There is nothing new there, it’s all about execution and making sure you have those communication lines open. So we are continuing on that, we are expanding it and we really building upon that. But those – it’s just really hard grasping and getting out to all the global areas and making sure you focus in the right markets which we have done. You can’t have a blanket approach. There is no magic bullet, you do have to customize for certain countries. But we go choose in place and now it’s a matter of executing on them towards a launch.

Geoff Meacham – JPMorgan

Okay thanks.

Sanj Patel

Thank you.

Operator

Thank you. I would like to hand the conference back over for closing remarks to management.

Sanj Patel

So yeah, thank you very much for all the questions. As I said earlier the bottom line is we’ve got lot more to do. We are solidly focused on executing on LAL and the launch obviously, but most importantly also accelerating our overall opportunity set, the pipelines, opportunities that are in front of us, and also our mission to help patients with these devastating rare conditions. So we are executing on that. So we are excited. We’ll keep going and thank you for your support.

Operator

Ladies and gentlemen, thank you for participating in today’s conference. This concludes our program for today. You may all disconnect and have a wonderful day.

Sanj Patel

Thanks.

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