Sanj Patel - President and CEO
Carsten Boess - CFO
Anthony Quinn - Chief Medical Officer and Head of R&D
Mark Goldberg - Head of Global Medical and Regulatory Affairs
Imran Babar - Cowen and Company
Anupam Rama - JPMorgan
David Friedman - Morgan Stanley
Andrew Goldsmith - Canaccord Genuity
Joseph Schwartz - Leerink Swann
David Nierengarten - Wedbush Securities
Synageva BioPharma Corp. (GEVA) Q4 2012 Earnings Call March 11, 2013 4:30 PM ET
Good day, ladies and gentlemen, and welcome Synageva BioPharma fourth quarter and full year results 2012 earnings conference call. At this time all participants are in listen-only mode. (Operator Instructions) As a reminder this conference call maybe recorded. I would now like to turn the conference over to Mr. Matt Osborne, Director of Investor Relations. Sir, you may begin.
Thank you, operator. Welcome to Synageva's year-end 2012 conference call. During the call we plan to discuss financial, clinical, and operational results for Synageva. Before we begin, I would like to remind you of the safe harbor provisions, that certain statements will be made today which are forward-looking within the meaning of the securities laws. Owing to the uncertainties of forward-looking statements, our actual results may differ materially from anything projected in these forward-looking statements. We can give no assurance as to their accuracy and we assume no obligation to update them. For further information on risks and uncertainties, please read our documents on file 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.
Thanks, Matt. Good afternoon, everyone, and thanks for joining us on this call. With me in the room are Carsten Boess, our Chief Financial Officer; Dr. Anthony Quinn, our Chief Medical Officer and Head of R&D; and Mark Goldberg, our Head of Global Medical and Regulatory Affairs. Tony Quinn will provide an overview of our lead development programs, sebelipase alfa and SBC-103 for MPS IIIB. Dr. Mark Goldberg will comment on our global efforts to disease awareness for LAL Deficiency, and Carsten Boess is going to provide an overview of the fourth quarter and full year financial results as well as provide the 2013 financial guidance.
Before they do that, I would like to review our achievements during 2012 and outline our goals for 2013. During 2012, the company made significant progress towards our goal of becoming a leading global organization focused on discovering, developing and commercializing therapeutics for patients suffering from very rare diseases. Progress with sebelipase alfa throughout 2012 culminates in the initiation of the ARISE study. This is a global Phase 3 trial in children and adults with late onset LAL Deficiency.
Meeting this milestone was a result of a tremendous effort on the part of the entire company, particularly, the clinical, medical, and regulatory affairs teams, and also our manufacturing and commercial groups. I am happy with the seamless cross-function integration of these teams and we are now focused on setting up even more U.S. and international sites that continue to enroll more patients into this study. We are also making progress in the Phase 2/3 study in infants with the early onset from of LAL Deficiency, a form of the disease that’s typically fatal within the first six months of life.
The first of the infants treated in the trial who started treatment at around 3.5 months of age, is now past his second birthday, is thriving and running around like any other toddler. We are witnessing this remarkable progress first hand. Not only it only reminds me of our responsibility but also reinforces our determination, drive and desire to make sure we find and treat as many other infants as quickly as possible. It's quite frankly the reason why we are here and Tony will tell you more about that in a moment and describe the company's making in this area.
Now, let's talk about manufacturing. This is another key area of focus for us. As you know, our lead program, sebelipase alfa, is produced by a recombinant DNA technology using Synageva's proprietary manufacturing platform. The system has a number of advantages which includes the production of consistent product that is scalable, capital efficient, and has an ability to rapidly expand production without significant delay or cost. During 2012, we began building a redundant supply chain. This separate facility will be responsible for manufacturing bulk protein and will be located in Massachusetts.
We continue to build out our global commercial infrastructure. We went public just over 60 months ago and were then primarily a U.S. based organization. Fast forward to today, we have a growing presence around the world and during 2012 we hired regional country managers known to many of us on the team before, and have specific rare disease specific in the U.S., Japan, Poland, Turkey, Germany, UK, France and Latin America. Each of these individuals share the drive, energy and passion that are the essence of all Synageva employees.
We continue to build upon this infrastructure to help not only raise LAL Deficiency awareness and find more patients, but also to support our other pipeline programs as they mature. Our goals for 2013, they are clear, measurable, and once achieved will establish a solid foundation for the organization that we are building. I will review these now.
It's to continue to raise awareness to identify more infants, children and adults with LAL Deficiency. We will progress enrollment in the ARISE Phase III study in children and adults and in the Phase 2/3 study in infants with early onset LAL Deficiency. We will also continue to advance our next pipeline program, SBC-103, the enzyme replacement therapy for MPS IIIB also known as Sanfilippo B. Here we intend to translate our encouraging preclinical data and progress this program into the clinic. We plan to be in clinical trials in the first half of 2014.
We will continue to advance our early stage preclinical programs, SBC-104, 105, 106 and the programs beyond that. All of which are therapeutics aimed at improving the lives of patients with rare and devastating conditions. In 2013, we will also make significant progress towards having a second redundant commercial scale manufacturing facility.
I am now going to turn the call over to Dr. Anthony Quinn, who will review the progress on sebelipase alfa for LAL Deficiency and our next program, SBC-103 for MPS IIIB. Tony, over to you.
Thanks very much, Sanj, and good afternoon everyone. I would like to cover two things in this part of the call. Firstly, I would like to spend a few minutes reviewing the background and the rationale for the design of the Phase 3 ARISE trial, and secondly, I would like to give you a brief update on the progress of SBC-103 on our pipeline program.
The ARISE trial is a global Phase 3 study designed to demonstrate efficacy and safety in 50 patients, both children and adults, with late onset LAL Deficiency. Patients will be randomized to every other week infusion of sebelipase alfa or placebo for the double-blind treatment period of 20-weeks in duration. Data from this double-blind period of 20-weeks will be used to support global submissions for registration. Patients participating in the trial will then enter a longer-term open label extension period with patients on placebo switching to sebelipase alfa.
The biology of LAL Deficiency is straightforward. Deficient enzyme activity leads to buildup of abnormal fat. And it is this build up of abnormal fat that leads to the disease complications including liver cell damage progressing to cirrhosis, and the dyslipidemia leading to accelerated atherosclerosis. As an enzyme replacement, sebelipase alfa addresses the root cause of the disease by replacing the deficient enzyme. This leads to a reduction in the accumulation of the abnormal fat, which in turn reduces the liver damage and improves the dyslipidemia associated with the disease. Based on the preclinical and clinical data we have generated on the effects of sebelipase alfa, insights from discussion with experts in the field and from our discussions with the U.S. European and global regulatory authorities, we have designed ARISE with these liver damage and dyslipidemia endpoints in mind.
The primary endpoint of ARISE is the proportion of patients relative to placebo who achieve normalization of ALP which is a marker of liver damage, and that’s on week 20. In addition, key secondary endpoints will measure the effects of sebelipase alfa on a broad range of abnormalities seen in patients with late onset LAL Deficiency. And this includes reductions in LDL, reduction in [serum] triglycerides, decreases in live fat content and liver volume, and increases in HDL which is a good cholesterol. As a reminder, at six months of the open label Phase 1/2 extension study in adults with LAL Deficiency, sebelipase alfa significantly reduced liver damage with sustained reductions in both ALT and AST.
Additionally, sebelipase alfa also improved patients' abnormal lipid profiles as measured by LDL, triglyceride and HDL. Importantly, these effects took after six months, were sustained at nine months as mostly recently reported at the LBN Conference last month. These improvements in key blood disease markers were accompanied by reductions in liver volume and liver fat fractions assessed by MRI. Sebelipase alfa was generally well tolerated at nine months with mostly mild adverse events that were considered unrelated to the drug. We are pleased to see that we have recently dosed our first patient in the ARISE study, and we are continuing to initiate sites around the world.
So let's now turn to early onset LAL Deficiency, also known as Wolman's disease, where we continue to enroll our Phase 2/3 study. The first infant was originally treated during the expanded access program, is now being treated at part of the Phase 2/3 study. Has been on therapy for just over 20 months and recently celebrated his second birthday. He continues to meet the developed milestones associated with children of this age. We are encouraged that we are making progress with our disease awareness efforts for early onset LAL Deficiency, as evidenced by the more frequent consideration of this disease and the differential diagnosis of infants with early onset growth failure and liver dysfunction.
We also tend to find more infants with the disease and we have enrolled more than one infant in this trial. We are still healing babies for the first time when they have advanced disease and our goal is to continue to work to find these infants before they reach the terminal stage of the disease. I would now like to turn your attention to our next program, SBC-103, an enzyme replacement approach for Mucopolysaccharidosis Type IIIB, which is also known as Sanfilippo B syndrome. This is a devastating condition with marked CNS involvement leading to a rapid slowing of development with behavioral problems, speech loss, increasing dependency and early death.
Accurate measurement of abnormal substrate in the brain has historically been more challenging in this disease and this is most likely a reflection of the historical methods used to measure substrate. Given the importance of being able to measure substrate to assess disease progression and the effects of enzyme replacement in lysosomal storage diseases, Synageva has developed improved methods for quantifying heparan sulfate, disaccharide. This is a substrate that accumulates in these patients due to the (inaudible) enzyme deficiency.
At the LDN meeting last month, we showed firstly that our new method could clearly differentiate between affected and unaffected (inaudible) deficient MPS IIIB mice, and secondly that treatment with SBC-103, a recombinant human (inaudible) using intrathecal and intravenous dosing approaches, reduced dose dependent reductions in their heparin sulfate levels in the brain, liver and kidney. This new data builds on the work presented last year at LDN in which Synageva demonstrated that is has been able for the first time to make recombinant (inaudible) with good mannose-6-phosphate dependent cellular uptake properties. While the MPS IIIB program is still in the preclinical stage of development, we aim to initiate a natural history study in the second half of 2014 to build a better understanding of the disease phenotypes and how the disease progresses over time.
In partner with these efforts, we will be focused on completing preclinical toxicology and pharmacology studies to allow for the initiation of first in-human trials during the first half of 2014. With regards to our other programs, we are making progress with these programs, SBC-104, 105, and 106. They are all protein therapeutics for rare and devastating conditions, we are moving them forward but we are the behind the development path of SBC-103. So with that I will turn the call over to Mark.
Thank you, Tony. Finding patients is a very significant challenge when working on ultra-rare diseases. And identifying more patients with LAL Deficiency remains a key priority for us. During the next few minutes I want to highlights our cross-functional efforts within the medical affairs and commercial affairs teams to raise awareness of LAL Deficiency. Because the clinical manifestations of the disease in children and adults include fatty liver leading to liver fibrosis and often ultimately to cirrhosis, and premature arthrosclerosis potentially leading to cardiovascular and/or cerebrovascular events.
Our target physician audience consists primarily of hepatologists, gastroenterologists, and lipidologists as well as geneticists. Also, due to the early and progressive nature of the disease, the pediatric sub-specialties within each of these physicians groups play a key role. Interestingly, through our outreach we have also found that it is the pathologists with access to patient biopsies who may be able to inform clinicians of the potential diagnosis by indentifying microvesicular steatosis or the small fat droplets associated with LAL Deficiency. Therefore, it is important that we have programs and strategies in place to reach of these distinct physician audiences. But before we discuss these programs and strategies, let me first orient you with respect to where we are now with respect to awareness of LAL Deficiency.
Relative to the same point in clinical development of past enzyme replacement therapies for lysosomal storage diseases, awareness of LAL Deficiency is low. And this is likely due to several factors. First, it was not until 2007 when the first study describing the prevalence of this patient population was published. So an understanding of the size of the population until recently was not well understood. And to be quite frank, it's still evolving. Second, while there are clear signs of late onset LAL Deficiency that would be evident to the astute clinician, there are often minimal symptoms associated with the disease until the disease has progressed to the advanced stages. Not dissimilar to the situation with hypertension or arthrosclerosis.
Therefore, in order to make the diagnosis in a timely manner before the disease has progressed to a very advanced stage, it's necessary for the physician to be well aware of the sometimes subtle signs of the disease. Finally, recognizing LAL deficiency is further [constrained] by the fact that it is a rare disease with a phenotype that’s shared by much more common diseases such as dyslipidemia and liver abnormalities often seen in patients with the metabolic syndrome. However, this does not mean that LAL deficiency is a benign disease. Inexperienced physicians may be lulled into a false sense of security because patients with LAL deficiency may often be asymptomatic with relatively modest enlargement of the liver. Modest elevations in the liver transaminases and/or abnormalities in lipid profile.
We were recently made aware of a newly diagnosed patient like this. However, upon liver biopsy the patient was noted to already have microvesicular steatosis but also importantly with bridging fibrosis. And this patient was only 11 years old. Physicians should not be fooled both early and late onset LAL Deficiency are associated with significant morbidity and early mortality. The good thing is now we can tell physicians that a potential new treatment is in late stage clinical development. However, at this time it's imperative for us to focus our efforts in first raising awareness of LAL Deficiency. It's essential that when a pediatric hepatologist or a gastroenterologist sees a non-obese patient with persistent unexplained elevations in liver transaminases or unexplained hepatomegaly that he or she rules our LAL Deficiency.
Similarly, when a lipidologist sees a non-obese patient with an elevated LDL cholesterol and a low HDL cholesterol, LAL Deficiency should be ruled out. And we are raising awareness in a number of ways. First, driving recruitment to the ARISE trial is a very direct way for us to raise awareness, particularly within trial sites, and to work with the trial site investigators to network and understand the referral groups within their institutions to help find patients. The message that an active protocol is underway, helps to motivate these physicians.
Second, we are implementing screening programs at various institutions where enriched segments of patient populations are being screened to rule out LAL deficiency. We are making solid progress with these initiatives and are continuing to expand them globally. Thirdly, attending medical conferences and sharing our data with key thought leaders is also important, as is publishing these data in leading peer-reviewed medical journals. And we have made progress on each of these fronts. Over the course of 2012, we had a presence at approximately 30 scientific meetings around the world, and during 2013 we plan to attend similar number of scientific and medical meetings, starting most recently with the lysosomal disease network world meeting last month at which five LAL Deficiency related studies were presented. Two of which were sponsored by Synageva.
A manuscript the Phase 1/2 data through the first 12-weeks of the extension trial was published in the journal, Hepatology, a leading hepatology journal. Additionally, an in depth review of the world's literature on late onset LAL deficiency or Cholesteryl Ester Storage Disease was accepted for publication in the journal of Hepatology by an independent group. Additional data providing further insights on the prevalence of late onset LAL Deficiency was recently accepted for publication in the journal Hepatology as a result of a collaboration between the groups at Mount Sinai School of Medicine and the Dallas Heart Study and the University of Texas at Southwestern.
During past year we were pleased to see that a physician paper from the European Society of Pediatric Gastroenterology, Hepatology and Nutrition, and practice guidelines from the American Gastroenterological Association, the Association of the Study of Liver Diseases, and the American College of Gastroenterology for the first time included Cholesteryl Ester Storage Disease for late onset LAL Deficiency, as a disease that should be ruled out before diagnosing non-alcohol fatty liver disease.
Finally, once awareness or the level of suspicion is raised, the dialogue with physicians quickly progresses to how do I test for this disease. And for that there is now a simple to use, robust dried blood spot enzyme assay that offers improved sample stability, ease of transport and quicker time to results than was previously available. This assay is now available at academic and specialty laboratories around the world and we are very pleased to report that LabCorp has validated this assay as well and is the central lab for diagnosing patients as part the ARISE Phase 3 trial and has made this assay commercially available throughout the world.
So we are excited about the progress the teams have made over the past year but we recognize the challenges and are prepared for and eager to embrace the opportunities ahead. And with that I will turn things over to Carsten.
Thank you, Mark. I will review Synageva's year-end 2012 results and discuss with you our financial guidance for 2013. For the year-ended December 31, 2012, Synageva reported a net loss of $43 million compared to a net loss of $25 million for the full year 2011. Revenue from the full year ended December 31, 2012 of $15 million consists of $7 million of Fuzeon royalties from Roche, as well as revenue of $8 million from Synageva's collaboration with Mitsubishi.
GAAP operating expenses for the full year 2012 including R&D and G&A expenses totaled $15 million. This compares to total operating expenses excluding R&D and G&A expenses for the full year of 2011 of $27 million. Synageva had cash, cash equivalents and short-term investments totaling $219 million on December 31, 2012, compared with a $60 million cash balance at December 31, 2011. The cash balance at December 31, 2012 does not include proceeds from the recent follow-on offering completed in January 2013 that resulted in $111 million in net proceeds to the company.
Now turning to our guidance for 2013. For the full year 2013, we expect the GAAP net operating loss guidance of between $87 million and $97 million. The GAAP net operating loss is primarily due to investments necessary to support the global clinical development program for sebelipase alfa, further preclinical development of SBC-103, expansion of the global commercial infrastructure, robust manufacturing capabilities as well as advancement of pipeline programs as Sanj, Tony and Mark discussed.
I would like to give you a sense of the components of the 2013 operating expenses and where that increased investment is going. Of the increase in operating expenses from 2012 to 2013, more than one-third is going towards SBC-103 and the earlier stage pipeline. The rest is going towards the sebelipase alfa ARISE trial building a redundant supply chain as well as our commercial and medical global infrastructure. In addition, we are beginning to invest a proportion of our capital into additional opportunities beyond our first mover pipeline programs to leverage our proprietary production platform to develop improved biologic therapies for unmet medical needs.
With that, let me turn the call back over to Sanj.
Thanks, Carsten. In closing, I am going to reiterate our key objectives for 2013. First is to find more patients and continue to enroll the ARISE study and the Phase 2/3 study in infants with LAL Deficiency. And to continue the development of SBC-103 for MPS IIIB and be in clinical trials in the first half of 2014. And also, to continue to build out our global commercial infrastructure to support not only our lead program but also our pipeline programs. So with that operator, if you could now open up the call to questions.
(Operator Instructions) Your first question comes from Imran Babar from Cowen.
Imran Babar - Cowen and Company
The first question, I'm just wondering, is there any update on the patient who had a grade 2 allergic type reaction that you mentioned previously?
Hey, Imran, this is Sanj. I will ask Tony Quinn to answer that question for you.
Yes, so let me just add reminding people of the background. So this is the first allergic type reaction in this study and I want to just remind people we have given more than 200 infusions to date. And there were no antidrug antibodies detected in the subject but he is having some additional testing done at the moment. And depending on the results of that additional testing, the physician will reassess and make a decision about putting him back on treatment. And as people know these type of allergic infusion reactions are actually pretty common with these classes of therapies and they usually can be managed through.
Imran Babar - Cowen and Company
My next question is just to your comment on the dried blood spot assay and the (inaudible) of adoption at different liver clinics. Are the physicians using it and I'm just kind of curious if you can comment on the adoption of it?
Yeah, this is Mark Goldberg. Yes, we are -- it is being widely adopted. It's now available in laboratories. The original one is in Scotland but it's available at academic and specialty labs really around the world. And most recently, and what we are very excited about is that LabCorp who is doing the -- is the central lab for our Phase 3 ARISE trial, is also offering this blood test commercially on a global basis. So we feel that we have gone a very long way towards alleviating that bottleneck.
Imran Babar - Cowen and Company
Okay, great, and my last question is, when can we expect an update on the other infants in the early onset trial?
So we will continue to obviously look out and identify these infants and again continue to enroll them. But this is an ongoing clinical trial so that this moment, we are just going to be focused on enrolling them. We at this moment don’t plan to have any updates until we enroll the original target. The original target was around 8 infants or so. At some point if we feel that is enough information that will be presented at an academic meeting, that’s where Synageva tries to do most of its presentations for data. If we feel there is a large enough cohort, we will present at the scientific meeting. But there will not a presentation outside of that.
Thank you. And our next question comes from Geoff Meacham from JPMorgan.
Anupam Rama - JPMorgan
Hi, this is Anupam Rama in for Geoff Meacham. Just a quick question. Can you give us a better sense of where you are in terms of the patient identification and site initiation for the Phase III ARISE trial and post-world have your thoughts changed all about the enrollment timeline's for the trial? Thanks.
This is Sanj again. So I will start and maybe Tony can jump in. So we have just started as we have said, enrolling and dosing in the Phase 3 study. Our expectations haven’t changed, it's always very difficult to enroll patients in the ultra-rare space particularly the enzyme replacement therapies for LSDs. We are not fully setup as far as setting up the clinical trials sites. We have got about 38 sites listed on the ClinicalTrials.gov. We continue to activate sites. We are going very much globally, activating more global sites. But we have said historically that these trials can take anywhere from 18 to 24 months to enroll and that guidance has not changed. I think we are obviously doing things like the dry blood spot and going various disease awareness programs which continues to help us identify more patients. But at the moment we are not going to change from that. But, Tony, anything to add on the enrollment?
Well, one thing, as everybody knows this is a global clinical trial so it's in many different countries. And so a part of the site initiation as they come online is just walking through the processes in the different countries including regulatory authorities and (inaudible).
Thank you. Our next question comes from David Friedman from Morgan Stanley.
David Friedman - Morgan Stanley
I was wondering if you can just spend a minute explaining exactly where you are with SBC-103 and what specific steps need to occur before it enters the clinic with humans? And then, just when it does enter do you know, is your initial dosing work and Phase I work going to be intravenous, intrathecal or both?
This is Anthony Quinn, the Chief Medical Officer and Head of R&D. So basically where we are -- so as the CFO said, what we talked about historically is we have been able to make this enzyme and with the right uptake characteristics which we presented in the LDN meeting in 2012. And then obviously, the key challenge with this disease that there was not a good substrate assay available and as we have discussed previously, it's very important with lysosomal storage disease with enzyme replacement to be able to show that you can reduced substrate. So the data we presented recently shows firstly that we can differentiate between wild type and (inaudible) mice in terms of the substrate. And then in the initial dosing studies that we presented using both intrathecal and intravenous dosing, we have been able to demonstrate that you can reduce substrate levels, both in the liver, kidney and importantly the brain, because that’s obviously one of the mains organs affected.
There is a lot more work that we need to do and we obviously looked at whole brain and obviously we need to understand which part of the brain we are losing substrate from, clearing out the substrate. So it's an issue. So there is more work to be done in the animal model to build a really solid foundation of understanding to support moving into the clinic. And then obviously that the other activities that you need to do in terms of the toxicological study. And so that’s kind of where we are and with that program at the moment I think we have generated some really important data but we have got to fill in those gaps and build our foundation exactly the same way as we did for sebelipase alfa.
And David, this is Sanj, we have said that we planned to be in the clinic with SBC-103 in the first half of 2014. And clearly, and as we did with LAL, we are really focused on putting a very robust preclinical package together. As Tony mentioned, this includes the toxicology studies, the preclinical enabling, the regulatory enabling steps that we are doing for the preclinical work. So that’s all underway right now. But clearly data we showed at just month's LDN was very encouraging and part of the reason for the focus on our program in terms of capital allocation.
David Friedman - Morgan Stanley
Great. And then anything that you can say on delivery in humans or what you might expect, or is there still more to be done to decide on that?
So we are looking at different dosing regimens and different routes of delivery at the moment. You can imagine based on the data, that’s obviously important. And obviously that will be part of the work that we will do to support moving into the clinic.
I think it's important to note, we are not ruling anything out at this point, David. We are very encouraged by the data we have seen but we are not resting on that alone. We will continue to do other studies understanding why we are seeing what we are seeing knowing there is very encouraging and very very interesting. We've obviously got to continue to make sure that we have got a package that will serve us in very good steps for the very long term. It isn’t just about getting into the clinic in the first half of 2014 but having a program that can take us all the way and develop a very strong late stage clinical program as we have done with LAL, have a very strong eventual commercial therapy for patients.
Thank you. Our next question comes from Salveen Richter from Canaccord.
Andrew Goldsmith - Canaccord Genuity
This is Andrew Goldsmith on the line for Salveen. Have you provided a time-frame on how long you think it will take to enroll the 8 infants into the Phase 2/3 trial?
This is Sanj. No, we have not. Obviously this is a very very difficult and in terms of just the progressive nature, exceedingly rare as there is no prevalent population in the early onset and these babies born and then they die. We have presented that for historic data showing that they die on average around 3.5 months of age. So there is no prevalent population. It's going to take a while. Having said that, we are now making massive inroads into identifying these babies earlier. It's important to know though, Andrew, that, I think I have said this before, I don’t see the early onset as being limiting from either a commercial point of view, the build of the commercial focus obviously is in the late onset which is two years, children and adults above, but also from a regulatory point of view. We will ultimately file, as part of our package for the late onset, file all the information we have at that time with the early onset.
Having said that, it is obviously incredibly important that we continue to find and treat more infants as we are doing but it won't be very limiting from a commercial or a regulatory standpoint.
Andrew Goldsmith - Canaccord Genuity
Great, thank you very much. And just briefly, I think I saw in the press release you're going to release one year data in the Phase 1/2 trial. Can we just kind of expect every three months to get new data from that trial?
Again, I told you it's very important to represent these information at scientific meetings and have acceptance at the peer-reviewed journal or meetings. So that’s what we are going to be focused on to the extent that we can have those forums and have that avenue vehicles to present, we will certainly provide the updates for that. And the 12-months data should be around, I think, at least around the mid-point of this year at a scientific conference yet to be determined. But, yes, the idea is to try and get these data via scientific conferences or journals.
(Operator Instructions) Our next question comes from Joseph Schwartz from Leerink Swann.
Joseph Schwartz - Leerink Swann
I was wondering if you could talk a little bit more about where you are on the LAL Deficiency patient identification continuum? It sounds like you are doing a good job raising awareness of the spectrum of the disease characteristics among various physicians. I'm wondering what happens next? Are you able to work closely with many physicians to enter patients into a tracker of some sort that you can use going forward?
Yes. We are very happy with how it's going. It is a dynamic process. It's in flux. And I like to think about it in the following way. One arm of our effort is to identify patients that are already known but perhaps not known to us. And the other major emphasis is on identifying patients who have here before not been diagnosed. And we are finding that we are continuing to make advances in this regard, both through routine testing being done by physicians because they are now more aware of the disease. They have the ability and the availability to test for the disease. And so they are just testing on their own. And in addition, we are starting the screening programs with the pediatric heptologists and lipidologists as well as the adult lipidologists. And those are also just beginning to get going at top speed. But we are starting and continue to find more patients as a result.
Joseph Schwartz - Leerink Swann
Okay, great. Thanks. And then how wide is the phenotype that you expect to see in the ARISE trial? Do you have pre-specified stratifications in mind for your analyses? Because it is my understanding that there's a pretty broad spectrum of how the disease, how severe it can be?
So, Tony Quinn again. So basically, obviously as you know we have got children and adults and I mean the disease is fairly similar in terms of from a phenotype perspective and there maybe some differences in terms of some (inaudible). But you know if you look at the badly affected children, their transaminase is up typically in the same range as the adults. Obviously age is an important potential covariant. There are other things like potentially the use of lipid loading therapy. And so we have thought very carefully about the disease. We have used all of the information that we have available and we will be factoring those types of things into the analysis.
Thank you. Our next question comes from David Nierengarten from Wedbush Securities.
David Nierengarten - Wedbush Securities
I've a couple of questions, one that you might not answer and another one that maybe you could shed some details on. So the first question is, I was wondering if you had or if you could tell us about the number of patients you screened to get the first patient enrolled or dosed in the ARISE trial?
David, this is Sanj. So we will not be disclosing that. Suffice to say we continue to screen for patients, find more patients. The disease awareness programs we have just discussed are certainly helping us move the needle. Awful lot more work to be done. But we are focused on getting it done, we will not be discussing that at this point.
David Nierengarten - Wedbush Securities
Yes, can't blame me for trying. And then my other question was a little bit more big picture question. When you look at the SBC protein that you have manufactured, are those or is its ability perhaps to reduce substrate in the brain generalizable to other protein targets that you could look at, say, I don't know, the neuronal form of Gaucher's disease for example?
This is Sanj again. So I will make a few comments and then Tony may jump in. But again, we are very encouraged by the data we have seen with SBC-103 for MPS IIIB and clearly with flow into the uptake. Substrate reduction from the brain in the MPS III animal model. You know there could a number of reasons why that’s happening. Certainly we have always been very pleased with the glycosylation we have from the proteins from our proprietary manufacturing system, the amount of mannose-6-phosphate, mannose-terminal GlcNAc are very important. That could be one reason. There could be some other disease specific ones. We are certainly looking into the application beyond that one program from MPS IIIB. But we can't rely on that completely. We better make sure we stay focused on the 102 program, the sebelipase alfa program, then the Sanfilippo B program now. We will certainly look at leveraging those learnings. We will do it both for the Sanfilippo B program and for other programs. So it's a very important areas of focus. But please buy it, a lot more work to be done. Tony, anything to add on that?
Yes, I would just briefly add. So basically, I will say the data we have shown at the moment, if you take whole brain, you can see there is evidence of substrate reduction with IV dosing. As I said earlier on the call is that our job is also to understand which parts of the brain are we clearing substrate from. These diseases, they are all a bit different and you know you have to be careful extrapolating between one disease and the next. But as Sanj said, obviously it's an intriguing observation that we have made. And we well obviously will be following up to best to understand that that says and then also to understand whether some aspects of it, and I'm sure it will some, not all, may be generalizable to others and related diseases.
Thank you. I am showing no one else in queue at this time. I would like to hand the conference back over to Sanj for any closing remarks.
Yeah, thank you, very much. So I would really just like to thank everybody for getting on the call and obviously support. We are, to reiterate, very much focused on planning more LAL Deficiency patients both for ARISE and beyond. Very much also focused on developing the pipeline, SBC-103 for Sanfilippo B, clearly, and also our other pipeline programs. And really putting together some very solid packages there. Beyond that Carsten highlighted our development of our manufacturing capabilities. We are certainly working towards having these redundant supplies as we launch these products is a very key point for us. But longer term we are also focused on investing and building for the long term. And that’s clearly the mantra that Synageva has aside from the things that we have talked about today. So with that thank you very much and I look forward to speaking to you tall again soon.
Ladies and gentlemen, thank you for participating in today's conference. This concludes the program for today. You may all disconnect and have a wonderful day.
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