Good day, ladies and gentlemen, and welcome to the Third Quarter 2017 Financial Results and Corporate Update Conference Call.[Operator Instructions] As a reminder, this conference is being recorded. I would now like to introduce your host for today's conference, Danielle Keatley. You may begin.

Good afternoon, and welcome to the Ultragenyx Pharmaceutical Financial Results and Corporate Update Conference Call for the Third Quarter of 2017. We have issued a press release detailing our financial results, which you can find on our website at ultragenyx.com. I am Danielle Keatley, Director of Investor Relations. With me today are Emil Kakkis, Chief Executive Officer and President; and Shalini Sharp, Chief Financial Officer. I would like to remind investors that this call will include forward-looking statements within the meaning of the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995 including, but not limited to, the types of statements identified as forward-looking in our quarterly report on Form 10-Q filed on July 27, 2017; our quarterly report on Form 10-Q for the quarter ended September 30, 2017, that will be filed soon; and our subsequent periodic reports filed with the SEC, which will all be available on our website in the Investors section. These forward-looking statements represent our views only as of the date of this call and involve substantial risks and uncertainties, including many that are beyond our control. Please note that our actual results could differ materially from those projected in any forward-looking statement. For a further description of the risks and uncertainties that could cause actual results to differ materially from those expressed in the forward-looking statements as well as risks relating to our business, see our periodic reports filed with the SEC. I'll now turn the call over to Emil.

Good afternoon, everyone, and thank you for joining us. I'll start by discussing our recent progress and milestones. And Shalini will then give an overview of our third quarter results. We've continued to make regulatory progress during the quarter with two of our clinical programs: burosumab for XLH and vestronidase alfa or rhGUS for MPS 7. For burosumab the U.S. FDA granted priority review status to our Biologics License Application or BLA, and set a PDUFA date of April 17, 2018. As part of the review for process, we recently received the Day 74 filing communication from the FDA, indicating that the review is proceeding according to their internal review timelines and stating that the agency is not currently planning on holding an advisory committee meeting. We're encouraged by the review process to date and the feedback we received from the FDA is in line with our expectations. As a reminder, the FDA accepted our BLA for paediatric and the adult indications and granted priority review. The accepted application is based on currently available data from our multiple ongoing studies in both children and adults. For the paediatric indication, the find includes the 64-week data from the Phase II study in 5 to 12-year olds and 24-week data from the Phase II study in the 1 to 4-year olds. At ASBMR in September, we also presented exciting 40-week data from the Phase II study in 1 to 4-year olds, which showed significant improvement in both rickets and bowing. These young patients had more severe rickets at baseline than the older children that we previously studied and every patient showed substantial healing of rickets at week 40. These younger patients also showed a significant improvement in bowing, which was readily visible on x-rays of some patients at only 40 weeks. The…

Thank you, Emil, and good afternoon, everyone. We issued a press release earlier today that included a financial update, which I will briefly summarize. Total net loss for third quarter of 2017 was $79.2 million or $1.87 per share, basic and diluted, compared with $64.9 million or $1.64 per share, basic and diluted, for the third quarter of 2016. This reflected cash use and operations of $172 million for the first nine months of 2017, compared with $113.3 million for the same time period in 2016. We continue to expect cash use in operations to increase for the remainder of the year. Net loss for the first nine months of 2017 included approximately $44.8 million in non-cash charges, including stock-based compensation of $48.5 million, amortization of premiums on purchased investments, depreciation amortization and other noncash charges, offset by a noncash intercompany foreign exchange re-measurement gain of $8.4 million. We expect stock compensation expenses to continue to increase over time. In the third quarter we recognized revenue of a $198,000 in named patient sales for vestronidase alfa. Our total operating expenses were $83.9 million for the third quarter of 2017. Research and development costs were $60.4 million during this period with our Phase III programs accounting for the greatest proportion of R&D costs. Costs for our multiple pre-clinical translational research programs are also increasing as programs advance towards the clinic. During 2018, we expect operating expenses to increase due to the potential launch activities for both burosumab and vestronidase alfa. We ended the third quarter with $396 million in cash, cash equivalents and investments on the balance. With two U.S. product launches potentially on the horizon, I wanted to take a few minutes to discuss revenue expectations around and vestronidase alfa and burosumab. As you know, the PDUFA date for vestronidase…

Thank you, Shalini. As you've heard, we've made tremendous progress on the regulatory front this quarter with active U.S. and European filings for both vestronidase alfa and burosumab and our potential first approval launch later this month. We are planning to hold an Analyst Day on December 4, in New York, where our management team and experts will provide additional information on our clinical and key pre-critical programs including the Dimension Therapeutics lead programs after the acquisition closes. We'll provide additional detail around our commercial readiness, and launch preparations that are underway as well. We look forward to seeing many of you there. So now let's move to your questions. Operator, can you please provide the instructions for the Q&A portion of the call?

[Operator Instructions] Our first question is from Eric Schmidt from Cowen and Company. Your line is now open.

Good afternoon and thanks for taking my question, congrats on the progress. Maybe for Emil, on the burosumab FDA approval process, it seems quite early to have been informed that no AdCom will be required. Are you getting the sense that the FDA is highly engaged and could complete this review ahead the PDUFA date? And then second question, would just be on the CHMP discussions and whether they're proceeding quite as well, I know that the filing is based there on the Phase II data? Thanks.

Sure, Eric. Thank you for the question. Well I think it's normal for them to determine by the Day 34, whether they're planning AdCom because it takes months to plan an AdCom and execute it, so I don't think it's that unusual, they usually inform you around that time. I think they are readily engaged and working quickly on the application, but we couldn't this point say that would finish that early at this point, so I wouldn't necessarily expect that. On the European review, it's going well. We've -- obviously some of you are tracking time, we've worked through the various steps in the process and passed -- it's a Day 180 point where you are essentially toward the end. And we're still expecting that they will reach the opinion point then by the end of the year is our expectation at this point, but there's still steps to finish in the last put of the year, but so far moving along well with the Europeans as well.

Thank you.

Thank you. And our next question comes from Tazeen Ahmad from Bank of America. Your line is now open.

Hi, good afternoon, thanks for taking my question. Emil, just wanted to get your thoughts on rhGUS. Obviously, that's going to be one of your smaller indications relative to a lot of other things you have in development, but wanted to get a sense from the time, presumably you do get approved, how long will it take for commercialization to actually start and where are you in terms of maybe pre-identifying appropriate patients.

Well, we have been -- on the latter part, identifying patients, we have been doing that really for a couple years. It obviously is a very rare indication. We've still projected around 200, if you found every single person in the world. We've spent more time in the U.S., in some parts of the Europe, not as much time in South America. We continue to find additional patients though and it obviously is a step by step process. We do think there's probably a number patients with hydrops in the first year of life that are not getting diagnosed, and we're working to try to make sure that perinatologists or people handling babies born with hydrops are properly diagnosed. And we have been treating a few patients in compassionate use, who had hydrops under 1 year of age. I would say that's one of the unknowns on the market is we predicted there might be 10 to 20 babies born a year with MPS 7 that have hydrops, so that becomes an important piece of the story in the long run. Our plan on launching is to, of course, get the product out as promptly as possible. I would not expect a long delay in launching the product. We have product ready to go, we just obviously have to do the right things with the final labels and packaging and get that agreement from FDA before you can put it out and distribute it. But we're planning to do what we feel is a rapid launch for the enzyme therapy.

And then maybe for Shalini, what do you think is an appropriate range of pricing that we should be considering here?

Well, we haven't given out a specific dollar amount and we will provide more clarity on that at a later date, but at this point what we have said is that we expect this product to be priced similarly to the other approved ERTs for MPS disorders. Now you should note that this is much more rare actually than the other MPS ERTs but we're still committed to pricing the product in a similar range to the other products.

Thank you.

Thank you. And our next question comes from Cory Kasimov from JPMorgan. Your line is now open.

Hey guys, this Shawn [ph] on for Cory. Maybe if you could just talk a little bit about your Factor VIII program, that you have in partnership with Bayer, maybe you could just comment a little bit about how this product might be similar or perhaps different from what we've seen from some other companies, say for example, like Spark or BioMarin?

Yes. Well, first of all, we haven't really completed acquisition yet, so I can't really say, it's ours. But when it's ours, obviously, it's going to be way better, right? No. Obviously, it is another Factor VIII program and we will be evaluating after we complete the acquisition. Bayer obviously has been in the Factor VIII space for a very long time. They have tremendous experience and knowledge in Factor VIII and hemophilia. We are not as experienced and knowledgeable in that area and we'll work closely in that partnership going forward on advancing Factor VIII program. Our responsibilities, if the acquisition closes are really primarily on producing the product for the Bayer team, but we're encouraged about the opportunity to work with them and we think that Bayer's strong presence in hemophilia area put us in good position. The Dimension technology around producing the product I think is excellent. And part of why we're acquiring them.

Great. And maybe, just kind of keeping in that vein of hemophilia, so maybe just briefly if you can walk us through kind of the story behind the hem B program that Dimension had and kind of what happened there and if there is any potential read-through whatsoever that you may? Thanks.

Well, obviously we're not experts on the hem B program, it's not our program but what -- we do know and what's in public is that the hem B program used a novel recombinant vector that was different. And they have reported more inflammation to -- in that vector and that may be a factor in what happened for them. I would say that, that vector is not being used for the other programs, is not being used in the Factor VIII nor in the other programs that we would potentially acquire, so we think is that related to the particular vector and that there is no read-through to the other vectors.

Okay, great. Thanks again. Operator Thank you. Our next question is from Joseph Schwartz from Leerink Partners. Your line is now open.

Great. Thanks for taking my questions. So, I know, you said, you'd expect a gradual build over time for burosumab. Can you give us any insight into what you have been able to accomplish in your pre-clinical activities like treatment center and patient mapping to date, and how many of these patients have already been identified, and how you are thinking about commercial field for size and structure?

Well, I can give you some of that, just we have put in the field a 30 patient diagnosis liaison group which is something that Ultragenyx’s special field team that we developed. A field team focused only on disease state and basically finding doctors and identifying patients that they carry and learning about the disease burden. The patient has team – its a 30-person team and we divide it currently, our marketplace in the United States in the 30 territories, and we expect to have 30 UltraCare Liaisons eventually in those places. That's sort of our general sizing that we've proposed. That's based on what we think are the number of targets of around 100 targets per liaison in that space and that's sort of the work we would think about. But right now, our main focus is on to these awareness and identification. And we are doing that work as we do, frankly, for all of our programs, and we haven't released yet information on the numbers of patients and so forth. But we are rapidly looking across the country along a number of specialties using the ICD-9 and ICD-10 codes, primarily, as targeting tools and are finding patients both adults and pediatrics across the country in various clinics. It's really important to get past the key opinion leader centers right, that we have studied and get really out to those secondary places out and around that are seeing patients or treating XLH patients currently.

That's helpful. And then, I guess is a follow-on what -- how does the message differ between the most sophisticated centers and those that are more peripheral out there, be it, you emphasized serum phosphorus or bone pain and stiffness or fractures, or [Indiscernible]. What do you think different segments of the market need to see in order to be convinced that this is the right option for different segments of the market?

Well, I think, one of the first things we're discovering is that people that are far outside the main centers may not even use the word, the acronym, XLH, which is more a modern term, a lot of them use vitamin D resistant rickets or other terms. We're finding through some knowledge gaps around the certain -- the basic science of the disease and naming. And so, part of it is just the explanations around FGF23 is an agent that causes low phosphate and understanding that. The number of doctors are -- that are using phosphate and vitamin D understand the treatment. They may not necessarily have a breadth of knowledge about the burden of the illness across the patients, so we are certainly informing them about the burden of illness in adults and peds. It's the type of problems that are out there and trying to learn from them, in terms of what they see are the biggest issues. The goal is to kind of understand their knowledge of the disease and also give them greater impact in understanding the effect of XLH across the full life of a patient.

Very helpful. Thank you.

Thank you. Our next question comes from Laura Chico from Raymond James. Your line is now open.

Hi. All my questions have been answered. Thank you so much.

Thank you. Our next question comes from Steven Breazzano from Evercore ISI. Your line is now open.

Hi. Thanks for taking my question. Maybe you could just talk about the threshold for expression you're looking for in the OTC Deficiency program? And maybe where you see is the bar for something that you expect to be clinically meaningful?

Well, obviously we haven't finished our acquisition for the program. The measure that's being proposed in the OTC program is to look at ureagenesis and there is a normal level of ureagenesis and we're looking to see what fraction of normal you achieve. I think, the fraction of normal that would be clinically meaningful has not been well determined, and our goal right now would be is, if we can complete the acquisition, would be just to verify that we're seeing some impact on ureagenesis and to use different dose cohorts to try to optimize the quantity of ureagenesis that we think will bring them closer to being self-sufficient for ureagenesis and not needing diversion therapy or having significant events. So, I can't give you an exact percent right now. I'm not sure it's known perfectly well what the right number is, but the ureagenesis will give you an idea, whether we're restoring the ability to make urea and to what degree. So we'll know that as a percent of normal what we're doing, but I would expect that we're going to have to move through some dose cohorts to get to a level of treatment to be able achieve a significant reduction ureagenesis. But at this point, the acquisition is not done yet and the program is just at its very beginning. Those can take a little time to get through some dose cohorts.

Thank you.

Thank you. And our next question comes from Gena Wang from Barclays. Your line is now open.

This is [Indiscernible] for Gena. Thank you so much for taking question. So, maybe just few to question around the burosumab in adult XLH. Just want to get your expectation about the 48-week follow-up for the adult Phase III trial and what do you expect to see there as well as the bone biopsy data. So what do you also submit all this data to FDA as well and so also what your plan for the EU submission for the adult XLH? Thank you.

Yes, so at this point, we have submitted we think all the data they'll use in the review, which is the 24-week bone healing data, the 7 bone biopsies that we just described which showed a reduction from severe osteomalacia to mild. And that would be the body of the data. We would expect to do an additional safety update, but we're not expecting to file the 40-week adult data. We don't necessarily think, it will change in the view. We believe it will be moving in the same direction that is in continued healing and we'd expect the placebo patient to crossover to drug also to begin healing fractures. So that's our expectation. But the data is submitted now is what will be submitted and the safety data is the only additional piece that would come. We think we were able to get additional data in after the filing, including the 40-week peds data and the additional biopsy data, which we think is supportive of the review. And at this point we don't feel there is any deficiency in the quantity of data necessary to get a good review from the FDA, and I think, they are comfortable with what they have, so 40-week [ph] data eventually, we probably would provide, but our expectation is that we will talk about that data once it's available.

Got it. So maybe just a quick follow-up. So just on the use of -- do you expect, like what's your expectation on the timeline?

Well, we still are on track, assuming there is no further stops, that we will be on track by the end of the year we think to get the opinion. The process there has starts and stops. They give you things, you answer questions, it stops. We give it back to them, then it restarts. So our expectation right now is that we should hit opinion point of the end of the year.

Okay. Thank you so much.

Thank you. Our next question is from Maury Raycroft from Jefferies. Your line is now open.

Hi. Thank you for taking my question. For the trihep Phase III Movement Disorder study, I'm wondering if you can comment on where you are in enrollment and any perspective into the types of patients that you are recruiting based on the SLC2A1 mutations and Glut1 protein levels, and maybe any other baseline characteristics that you can comment on?

Well, we haven't put out exactly where it's enrolling. It's enrolling a fairly well and our expectation is that we would finish enrolling by early next year, not before the end of the year. But we're on track and I’m putting more in there so we're comfortable with that. We are doing an important amount of patient diagnosis, patient effort, both U.S. and Europe, with our both MSLs and PDLs, and that effort is yielding new patients and patients that have a significant number of movement events, that is we're not having trouble finding people to qualify with enough events. There is quite a few patients, in fact, that have substantial number of movement disorder events. So that part has gone well. So we're encouraged about the process of how this study is going now, and we continue to drive more patient diagnosis going forward in the long run. I don't know enough about the genotypes in the protein to tell you more detail there. We are, of course, getting diagnostic information on the genotypes, but we don't have any, let's say, genotype, phenotype relationship information to provide on how those patients might be like or different from others. There's a lot of literature on that topic, but it's pretty clear that even small amounts of Glut1 can, perhaps, make patients more or less severe. But it's really -- there's enough variation there that you really need to look at each patient individually and the genotype can't really tell you what they're going to be like.

Got it. And what about -- as far as the age goes, can you comment on whether you are getting more children or more adults in the study?

No, I actually don't know enough of the detail. There is a variety of types of patients, both children and adults. So I don't think we're seeing any particular one or the other.

Got it. Okay. Thank you very much.

Thank you. Our next question comes from Arlinda Lee from Canaccord Genuity. Your line is now open.

Hi, guys. Thanks for taking my questions. Can you maybe provide an update on the patients that were treated? I think there was at least one -- one of the hydrops patients that was treated a while ago. And then there was like a theoretical possibility of potentially treating these patients in utero, and I'm wondering about that as well? Thanks.

We have a couple kids that have been treated, like under six months of age, so from a very young age. There are continuing on treatment in general, the ones in U.S. So I don't really have any specific update, so we probably should get some more information on how those guys are doing, but they are continuing to get treatment. And we feel comfortable on their progress. We can't -- I think the question you are really asking is, if we take a kid that young, can we really change their future in a profound way? And I think it's going to probably take a little more time to know how well we've changed it. What I can say though, Arlinda, is that in all the other MPS programs I've been involved with, treating kids from under -- just a few months of age, has resulted in much better outcomes, skeletal and physical growth and other health-wise. So we think it's important to treat some of these kids from an early age and really change their lives in a way that you can't when you are taking patients who are 15 years old and bodies are very physically harmed. Now the update on in utero, there is a research program that we're collaborating with that is looking at an in utero treatment and we don't have anything to report. That will leave that to the scientists who report their results. But they are doing a program looking at the topic. We're not currently planning a clinical trial.

Okay. And then this program would be eligible for a voucher as well. Is your intention to sell that? And what time line could that happen?

I let Shalini answer that.

Yes. So Arlinda, we do expect to receive a priority review voucher for this product for vestronidase alfa for MPS 7 because, obviously, a rare pediatric disorder. Our intention because most all of our products -- or all of our products are expected to receive a priority review anyway would be to divest the priority review voucher. We should receive it upon approval. So if we do get approved, we should receive the PRV simultaneously with that and then the sale process could be anytime following that time.

Okay, great. And then maybe on burosumab, are there any one-time launch costs that we should think about? And then after the five years, what happens to those sales force or medical liaisons that we’re working on burosumab?

On the issue of one-time costs, I don't think it's anything particular. We do have a big patient diagnosis [ph] contract, patient diagnosis force out now that might not continue in the long run that's helping us make -- map all the patients that we can and do disease burden awareness work. That may not continue indefinitely at that level. Sorry, the second question was?

The sales forces that were -- that you guys were using for the first five years of the collaboration.

What would we do with them?

Yes.

We use them to continue to market the many other products that we'll have on the market. Our expectation is that we will have other products on the market at that point in time and we'll be managing it. We'll work with our partner, Kirin, on the transition to make it smooth. They are able to start putting people in the field two years before the transition over, which will help get set up. And then we'll make the transition afterward. But our expectation is to have other products in the market as well and that might be able to help manage that. But with our partner kind of getting going, we think there will be a way to handle that flow in a way that's intelligent and effective for patients.

Okay, great. Thanks.

Thank you. Our next question is from Liisa Bayko with JMP Securities. Your line is now open.

Hi, there. This is Amy for Liisa. So, can you just remind us maybe on the named sales -- the named patient sales for the quarter? What's really driving it? And what's your expectation for this revenue stream going forward? Thank you.

I let Shalini handle that.

So, we do have patients in Europe, who are on treatment under named patient programs, which means that the product is not yet approved, but it can be paid for, for these patients, under certain circumstances, which vary from country to country. We have historically had some named patient revenues for vestronidase alfa in Europe previous to this. But we do expect named patient sales to remain negligible, because it's a small number of patients, and because we recognize revenue on a collections basis. We really don't expect significant revenue and the timing of the revenue is very hard to anticipate.

Great. Thank you.

Thank you. Our next question is from Matthew Harrison with Morgan Stanley. Your line is now open.

Great. Good afternoon. Thanks for taking the questions. I have two. So I guess the first one is, can you just comment, we've heard from some of your orphan [ph] peers that they've been struggling in Latin America a little bit. Can you comment on how you see the value of that territory changing or not changing related to XLH? And then I guess the second question I have is just related to the pipeline. You've obviously added a handful of new assets -- or about to add a handful of new assets. How should we think about prioritization of your pipeline and how are you thinking about capital allocation among the different assets right now? Thanks.

Okay. So, in Latin America there's always been -- there's been a history of sort of lumpy distributions and payments, and challenges in the local economies. And so that will always be an issue going forward. It may be with the increase in the number of rare disease products that's become more challenging. We have our Latin American head, Eduardo Thompson, is extremely well-versed in the work down there and has done a lot of sales work. We think we need to handle it very smartly, intelligently. We're going to focus on countries that are able to reimburse and manage the process in an elegant way. We think the products -- its features I think are very strong and important and that being primarily bone-related and the dysmorphology and the disability that results from it is the kind of thing that fixing would result in a substantially improved child's health. So we think it's the kind of treatment that would probably do well in Latin America from the target populations. So, right now we appreciate, though, that there are increasing pressures on Latin American countries and we have to be really smart about how we handle access and commercialization, in a way that appreciates the challenges they have, while helping press for the health of their patients affected with XLH. Now, on new assets -- prioritization, we do, if we complete the acquisition with Dimension, we'll acquire three products that are at the IND stage. Two of them are really products that are -- would be ours to manage for GSD-I and OTC, which are inborn error products and the Factor 8 product is really partnered with Bayer. They have other inborn error products as we do in our early pipeline. And we're going to try to manage our overall flow of products and look carefully at their tractability and ability to execute. Because we had -- our programs were in phase for after because the one program stopped. We actually have kind of a gap in the Phase I, Phase II area. So there are three programs -- kind of fit nicely into that gap. Our other programs in the early pipeline are really another year to two year out. So I actually think the prioritization will be easy to manage. We'll look at the pre-clinical part of the portfolio, but that is more like 10% of the R&D investment. So 90% in the clinic I think fits very well together without having to push one thing or another out. So we haven't said anymore. We'll talk at R&D Day a little about where the pipeline is and how we're managing it, what else is in there and give you maybe a better picture on how that could be progressed ahead. But we think the Dimension acquisition would give us some good early-stage clinical work in an area that we're strong in, in the inborn errors area. So I do think it fits well.

Okay. Its helpful. Thank you.

Thank you. At this time, I am showing no further questions. I would now like to turn the call back over to Danielle Keatley for closing remarks.

Thank you. This concludes our call and a replay will be available shortly. If you have additional questions, please contact us at 844-758-7273 or ir@ultragenyx.com. Thanks for joining us.

Ladies and gentlemen, thank you for your participation in today's conference. This concludes the program. You may now all disconnect.