Good day, ladies and gentlemen and welcome to Ultragenyx Pharmaceutical Incorporated Fourth Quarter and Full Year 2014 Financial Results and Corporate Event Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to Rob Anstey. You may begin.

Thank you. Good afternoon, everyone and welcome to the Ultragenyx Pharmaceutical financial results and corporate update conference call for the fourth quarter and year ended 2014. We have issued a press release detailing our financial results which you can find in our website at ultragenyx.com. With me today are the following members of the Ultragenyx management team, Emil Kakkis, Chief Executive Officer and President; Shalini Sharp, Chief Financial Officer; Sunil Agarwal, Chief Medical Officer; and Tom Kassberg, Chief Business Officer. We will start today with an overview and key updates from Emil, followed by highlights of our finance, clinical development, business development and commercial progress. We will keep our prepared comments relatively brief in order to allow plenty of time for the Q&A portion of the call. First, I would like to remind investors that this presentation contains 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, statements regarding the expected release of data from clinical studies, the initiation of additional clinical studies and the designs of same, and the intended effects of our product candidates. These forward-looking statements represent our views only as of the date of this call and involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, the actions of regulatory authorities, the timing of our regulatory filings and other matters that could affect the availability or commercial potential of our product candidates. For further description of the risks and uncertainties that could cause actual results to differ from those expressed in the forward-looking statements as well as risks relating to our business, see our quarterly report on Form 10-Q for the quarter ended September 30, 2014 filed with the Securities and Exchange Commission on November 10, 2014 and our subsequent periodic reports filed with the SEC. With that, I will turn it over to Emil.

Thanks, Rob and welcome to the first Ultragenyx quarterly conference call. We will now be conducting quarterly conference calls regularly going forward. We are also planning an R&D Day in New York later in the year to provide you with a more detailed update on our translational research and development programs. For those of you who are new to Ultragenyx, I found this company in 2010 with a mission of building a next generation biotech company focused on rare and ultra-rare diseases. Our plan is to leverage everything we have learned to-date in this area and apply this experience to develop novel therapeutics based on clear and potent biology. In the last 5 years, our efforts have generated a broad and diversified therapeutic portfolio of rare disease programs and we have also succeeded recruiting an experienced and high-quality team focused on rapidly and efficiently advancing that pipeline, including the exceptional group with me on the call today. This last year 2014 was a transformative year for us in many ways beginning with our initial public offering at the end of January and our first follow-on offering mid-year. We also have driven ahead on multiple programs now in Phase 2 and Phase 3 clinical development. In 2014, a number of clinical regulatory milestones were achieved for all of our programs and Sunil will provide more details on this later in the call. We expect 2015 to deliver another lineup of key milestones across these programs with each expected to either generate Phase 2 data or have advanced into Phase 3 development. In early February 2015, we completed a second follow-on offering of common stock and that provides us the flexibility to strategically invest in company growth and drive effective execution as well as support our efforts to grow value for our…

Thank you, Emil. I trust that you have all had a chance to review the press release we put out earlier today. Total operating expenses for 2014 were $56.8 million. Research and development costs accounted for $46 million or 81% of our operating expenses. OpEx increased significantly from 2013 due to the initiation and conduct of multiple clinical studies, increased manufacturing costs, the addition of new clinical programs to our pipeline, and higher general and administrative costs to support our advancing development portfolio. Total net loss was $59.8 million. Net loss attributable to common stockholders of $64.4 million differs from net loss due to $4.8 million in dividends and other charges related to outstanding preferred stock, which was converted into common stock upon the company’s IPO. The net loss figures also include significant non-cash charges such as $5.4 million in stock compensation expense, $3.3 million for the revaluation of the convertible preferred stock warrant liability, $3.6 million on the amortization of premium on investment securities, and other changes in operating assets and liabilities. We do anticipate our operating expenses to rise in 2015 as we enter late stage clinical trials and prepare for commercialization in the U.S. and other territories. However, due to our recent financing and our commitment to capital efficiency, we expect that our cash balances will be sufficient to fund our current operating plan into 2018, assuming all programs proceed. During this period, we could potentially see pivotal data from all of our clinical stage programs. In terms of the balance sheet, we ended 2014 with $187.5 million in cash, cash equivalents and short-term investments. In February 2015, we raised approximately $175 million in net proceeds from an underwritten public offering of common stock. We have no debt outstanding. I will now hand it over to Sunil to provide a development update.

Great. Thank you, Shalini and good afternoon everyone. I will take the next few minutes to summarize the most significant updates from late 2014 and early 2015 as well as provide a brief update on our clinical and regulatory milestones for this year. Starting with rhGUS, significant milestones occurred in both Q4 of 2014 and Q1 of 2015. In December, we announced the initiation of a single pivotal Phase 3 study in patients with MPS 7. The placebo-controlled blind-start study is currently enrolling with the goal of 12 patients who will be treated with 4 milligrams per kilogram of rhGUS every other week. When announcing the start of this study, we also announced that we had reached agreement with both the FDA and EMA on the single pivotal study design. This positive outcome is reflective of the collaborative relationship we have with both agencies across all of our programs. In February 2015, we announced that 36-week results on three patients from the Phase 1/2 study of rhGUS that supported our decision to proceed with the Phase 3 study. These data provided us with several important pieces of information. First, it demonstrated that 4 milligrams per kilogram every other week is the appropriate dose for Phase 3, the previously generated data on the 2 milligram per kilogram dose showed approximately 40% average decline in urinary GAG. This decline was even greater on the 4 milligram per kilogram dose with an average decline of approximately 60%. Second, while the study was not powered for evaluating clinical benefit, the data were encouraging. Specifically, for the two patients who had liver size evaluated at baseline both demonstrated significant reductions in their liver size after treatment. And for the one patient who was able to perform pulmonary function tests, there was a trend towards improvement…

Thanks, Sunil. I will provide a quick update on recent business development activities as well as a snapshot of our current and expected commercial planning activities. We recently executed a license agreement with Inserm and a related French institution for intellectual property related to the use of triheptanoin in Huntington’s disease. Most of you are probably aware of Huntington’s disease, but in short, it is a genetic neurodegenerative disorder characterized by movement disorders, behavioral and psychiatric disturbances and dementia. Death typically occurs within a couple of decades of onset. Huntington’s is fairly prevalent as fare as rare diseases go with up to 30,000 symptomatic patients in the U.S. and an even greater number in Europe. Our decision to pursue the license was driven in part by data from a pilot study in 10 patients with Huntington’s conducted by an investigator in France. The results of the study showed an improvement in brain energy metabolism in response to visual stimulation after one month of treatment with triheptanoin. Triheptanoin also appeared to impact the motor score subset of the Unified Huntington’s Disease Rating Scale. These data were recently published in the Journal of Neurology. In conjunction with the license agreement, we are also providing financial support and drug product for a 100-patient randomized controlled investigator study in early stage Huntington’s disease being run at two centers in Europe. The study will compare triheptanoin to placebo for up to a year looking at its impact on brain energy metabolism, UHDRS scores and caudate atrophy, as well as safety and tolerability. Turning to commercial planning, as Emil noted before, we are actively recruiting for a Chief Commercial Officer who will lead the global expansion of all our sales and marketing activities. In the meantime, we have begun to build out the marketing and commercial…

Thank you, Tom. 2014 was a great year for Ultragenyx making our debut on NASDAQ, now driving ahead on four products and six clinical programs. With funds raised, we are well-positioned to be able to drive our development forward leading to data readouts in multiple programs in 2015. We expect to expand our capability this year to help fill the early pipeline and prepare for commercialization. And we are steadfast in our commitment to look for ways to deliver our treatments faster for patients with rare and ultra-rare diseases. Thank you all for attending today and for your interest in Ultragenyx. And with that, we can start the Q&A session. And can the operator provide the instructions please?

Thank you. [Operator Instructions] Our first question comes from the line of Eric Schmidt of Cowen & Company. Your line is now open.

Thanks for the call and thanks for taking my question. Maybe first on KRN23 and XLH, you have got a lot going on with the 16-week data coming out later this spring and the 40-week data towards year end and then starting out the adult trial. I guess the question is when is the right time to sit down and have a discussion with the FDA about a specific approval path either in children or adults or the combination of the two?

Thanks, Eric. It’s a good question. I think we are constantly talking with the agency through a number of steps in the program, but I will let Sunil maybe answer specifically about regulatory process going forward.

Great, thank you, Emil. And Emil is spot on in that. Our discussions have started from the beginning, they continue as we go. And I will mention a couple of things that I hope help. With respect to the adult plans looking forward, we are right now in active discussions with them, with the FDA and EMA on the next study, which will be as we said in the placebo-controlled randomized study, which would start in the second half of this year. So, those discussions are going well with the agencies. With respect to pediatrics, we have already had several discussions with them and including web and approvable type of endpoint could be in pediatrics and they have made it clear that rickets reduction is an approvable endpoint in future trials. And as you know, our week 40 data will come out at the end of this year or early next year and that will be our first evaluation on rickets and our goal is to see a reduction in that pediatric population on rickets. So, bottom line, the discussions are going well and they are ongoing.

Maybe a second question is moving over to SA-ER and HIBM as the indication, what exactly or what specifically type of scientific advice did you get from the EMA to encourage your filing? And maybe you could characterize your confidence in a potential approval based on that filing?

Sure, Eric. I think we had a very good discussion. I think we have got pretty cleared guidance from them. I will let Sunil describe some of the details.

Yes. Again, thank you Emil. And I think it’s always good to start with our data and that’s our Phase 2 study, which was a rigorous Phase 2 randomized double-blind placebo-controlled study. And bottom line, that data showed that patients receiving 6 grams per day of SA-ER had a statistically significant and clinically meaningful improvement in upper extremity composite muscle strength. And these data were supported by PRO data. These data were supported by lower extremity and muscle strength and function and also some other data, including dose response and the fact that when placebo patients were switched over to 6 grams, they saw benefits. So, we started with that. And then after finishing with that, we had discussions with the EMA. And based on the EMA guidelines, based on their discussions with the EMA and looking at the totality of our data, we are confident in our plans and again that includes filing for conditional approval in the second half of this year.

I think to add one of the piece to Eric was that in discussing I think what they did say and we have noted before is that if we were to apply with this set of data that we would have to restrict our label to upper extremity strength stabilization, which I think is – I mean, we are strong indicator of what they are thinking that we have sufficient proof for the upper extremity, but may be not the lower extremity. And they did indicate they wanted to see some lower extremity in a confirmatory study and that was some of the discussion we had with them. So, I think if you take what Sunil has said regarding the data that input on what the label might look like, I think that gave us clear enough guidance and also guidance from the confirmatory study that we feel we have a reasonable path forward, but I would say very clearly, we are not going to handicap probably approval at this point, while things can happen, but we feel like it’s a solid study. If you look at what rare diseases have got approved on in the past, the 45-patient randomized controlled study. So, it’s a fairly – it’s a solid rigorous test in a very rare disease. So, we think there is a reasonable chance and that’s why we felt it was fair after our discussion to move forward.

Okay, thanks for the color, Emil and Sunil. Maybe just a quick one for Shalini, are you willing to give any more specific net operating expense guidance in 2015?

Thanks, Eric. Well, we are not providing annual OpEx guidance at this point, but what I can tell you is that we do have sufficient cash to fund our operations into 2018 with all the current pipeline programs proceeding. And during that period, we could see pivotal data from all of those clinical stage pipeline programs. We do expect OpEx to be higher both in R&D and G&A in 2015 compared with 2014. And this is due to obviously the progression into late-stage clinical trials and regulatory filings, investment in the commercial infrastructure and planned investment in the translational research capability in early stage pipeline and then again I think 2016 would be higher than 2015 for similar reasons.

Thanks and congrats on all the progress.

Thanks.

Thanks, Eric.

Thank you. Our next question comes from the line of Cory Kasimov of JPMorgan. Your line is now open.

Hi, guys. This is actually Brittany on for Cory. Thanks for taking the questions. With trihep data coming later this year, can you just review what are the key endpoints we should be watching out for and what would determine a positive Phase 2 outcome for each indication? Thank you.

Thanks, Brittany. That’s – we have obviously two programs, the FAOD as well as the Glut1 and so there is potential data in both, plus I point out some other ISPs coming and maybe soon we can talk about the two programs for which we are – our sponsor studies are going.

Great, thank you Emil. So, let me start first with the Glut1 program. As you know, we have a Phase 2 study ongoing and we expect interim data by the end of this year and that data will include of course safety and tolerability, but on the efficacy side looking at seizure control, I think it’s important to note one thing we have done in that study that’s in some sense a second look at seizure controller efficacy. We are enrolling patients with generalized tonic-clonic seizures, but we made an amendment recently in that study design to also include patients who only have had some seizures. And that decision was based on a recent publication by Dr. Pascal who showed that patients receiving triheptanoin demonstrated reductions in their absence seizures by EEG. So, we will look at both phenotypes. We will of course look at safety and tolerability and other parameters. And again, that interim data will be in the end of this year. With respect to FAOD, that Phase 2 study is ongoing. It enrolled 29 patients. So, it completed enrollment and we expect the 24-week open label data, which is the primary analysis by the end of this year. Now, this study I would categorize is more of a learning study. As you know, this is our first study, prospective study in FAOD and it’s a very heterogeneous disorder. We have multiple endpoints we are evaluating such as exercise tolerance, such as muscle strength and function, such as looking at hypoglycemia events. So, appreciating that it’s heterogeneous, appreciating that’s a learning study we will look for signals in one or more of the endpoints. And if we see that, we will then determine the best path forward.

Okay, thank you.

Thank you. And our next question comes from the line of Salveen Richter of Sun Trust. Your line is now open.

Good afternoon. Thank you for taking the question. This is [indiscernible] for Salveen. So, just drilling back a little bit more on SA-ER and HIBM and then the conversations you have had with the EMA are there any updates in your thoughts about the submission timelines on how those fit in with the ramp up of the pre-commercial activities that you have out there?

Okay, thank you. This is Emil. So, we have said that we are filing in the second half. And part of the reason for that is we have to get various aspects of the manufacturing lined up for filings since we didn’t expect to file in the timeframe and just getting all the other pieces of an NDA a little bit, we haven’t filed NDA at Ultragenyx, we have filed many NDAs before and the team working with it, but it does take some time. So, we haven’t really provided any greater specificity on that timeline, but we are saying second half. What we talked about in the named patient sales is the fact that there are lot of patients in Europe that don’t walk at all that have upper extremity issues, where we feel that we couldn’t participate in our Phase 3, which will require them to walk. And therefore there is an opportunity maybe to treat some of those patients in between the filing and the conditional – for conditional approval, there is an opportunity to treat some of those patients with named patient sales. They wouldn’t qualify for the Phase 3 study and therefore we think that would be appropriate for named patient sales. And so those activities will be going on in parallel and we will start pursuing named patient sales in the second half and the study start we would expect to have begun already.

Right, thank you.

Thank you. Our next question comes from the line of Chris Raymond of Robert Baird & Company. Your line is now open.

Thanks. The question on KRN23, I know you talked a little bit about the TIO trial on patient numbers etcetera, but just noticing on clin trials that it looks like it’s a study that you have registered as a 48-week study if I am not correct. So, can you maybe talk about how you look at this treatment? Is this something that you think would be a chronic sort of longer duration treatment or what is your sort of sense of how that will play out?

Yes, thanks Chris. I think look TIO patients generally the ones we are talking about treating with KRN23 are ones that have un-resectable tumors. Therefore, they are chronic because you can’t get rid of their source to FGF23. So, we expect their source to continue produce the hormone and therefore our need to block, it will continue, unless, they for example, the source can certainly be found and excised and that might change the situation for a patient. So, we are looking at it as a chronic program. And our choice of it had to do with the fact that we felt it looked like very severe XLH and so maybe to talk about the biology of TIO just briefly and why we think that’s important.

Right. And I think – thank you, Emil and I think that is important, because it is about following the biology and these patients if you can remove the tumor, their symptoms resolve very quickly, but as I have said before in about half of these patients the tumor can either not be found or if it can be found, it’s so disseminated or in a location that’s so sensitive it cannot be removed. And those are the patients, those 50% are the ones we will be targeting and in those patients, they have significant excess FGF23 production and their symptoms are extremely severe in many cases. So, as Emil mentioned, it would be chronic treatment. As I mentioned earlier, we are initiating our studies and we would expect results from that study, including radiographic data by the end of this year or early next year.

Great. And at the risk of asking you to repeat something, I think I heard at the end of your prepared comments mention of named patient sales, could you maybe give a little bit more color on that in terms of I know, I think I already said not in 2015, but maybe a little bit more color on which specific products you are talking about?

Yes, so this is Emil. We are planning to initiate the process of doing the in-patient sales and personally we have done this before. So, we are initiating that process, but maybe I will Tom fulfill sort of the details on what we think is going to happen on named patient sales timing.

Sure. There are two products primarily we are thinking about for named patient sales and they are in the sequence of rhGUS is the first product that we are sort of proceeding with and trying to identify patients that might be through physician request that come from a capacity user named patient sales basis from territories in Europe whereas named patient sales are possible. And then we work with those physicians to try to facilitate gaining access to the product pre-approval and there are certain pathways available particular countries that this pathway exists. Turkey, France, Italy and Spain are the ones that primarily are supportive of named patient sales opportunity. So, we are working with some identified patients and physicians that have initially expressed interest in gaining access to rhGUS and working through the systems of distribution and dossier submission and support that’s needed to help with that. So, it’s a modest effort. Obviously, MPS 7 is in terms of prevalence, there is small number of patients that we are talking about. And then we are beginning as the second effort with UX001. Sunil and Emil have talked about our filing strategy. So, we are also looking at opportunity there. And I think we have already received some request and interest for access through named patient sales to the product. And so we are working through further identification of patients and physicians and then thinking through various operational aspects of that. And just to clarify you also need to think about pricing, so part of what we are doing this year currently is looking at the type of pricing that we think would be appropriate for named patient sales in Europe.

Great, thanks.

Thank you. And our next questions comes from the line of Adam Walsh of Canaccord Genuity. Your line is now open.

Hi, guys. Thanks a lot for taking my questions. Can you hear me?

Hello, Adam. Yes, we can hear you fine.

Okay, terrific. Thanks. My first question is on – it’s great that you guys are sponsoring an investigator trial in Huntington’s disease. Do you have any indication about the timing in terms of when we might see data from that particular trial?

At this point, we don’t – being investigator sponsored, we are certainly funding the study and supporting Dr. Michelle on that. And she has been very productive investigator and she has done a lot of work. So, I actually think she is someone who can actually, but right now, we are not willing to put out exact timelines, but it will be important to get a handle on when. I think the protocol is done and I think they are just – they are in the setup mode.

And as you mentioned on the call, that’s 100-patient trial?

Yes, so 100-patient randomized-controlled study.

Okay, terrific. And then with everything going on I guess this is more of a strategic question, with everything going on in terms of new expansion into TIO for KRN23, the Huntington’s disease, the possibility of getting early approval for SA-ER, do you feel like you are right-sized from an HR perspective or from a management perspective to handle and possibly even further expand the pipeline?

Well, I think it’s a good point. We have grown a lot actually in the last year, but we are scaling up a lot right now. I would say from a development standpoints come in and hired a number of senior people in the development area. Clin ops and then the clinical affairs and then the other function drug safety, etcetera. So, we have actually really ramped up a lot and have sold out the team on that side. I would say, our work right now is on the commercial side. We are hiring and we have some basic pieces of commercial, but I think we are well short of what would take the launch of product. So, we need to definitely pull that in and that’s why we have been recruiting a Chief Commercial Officer, but we are taking our time. We have been doing that for a few months. And it’s critical to meet those people be someone that can mesh well with a really high performing team, someone who can run their our speed and drive as we do. I think what I also say is the company is made up of very motivated and hard working people. They absolutely drive and get stuff done. So, even though we don’t have as many companies, I think we are actually – I think we are very productive as a company. And part of that has to do with how we put things together and how we delegate people to do the work, but as no question you are right, if we are going to be commercializing in two products and doing all the work we do we need to scale up further and I think it’s particularly in the commercial side. So, there is more to do, but at this point, I would say giving how many people we have hired and how many great people we have hired has grown a really good spot to execute on our plans. So, I feel very comfortable we can do it.

And that’s really helpful. And then if I might just one quick follow-up to that, is your focus right now primarily on execution with the current portfolio or you are still looking to in-license additional products and develop them further?

Job one is executing the portfolio, because if we simply execute in this program, right, there is so much coming out of the loan. It will be enough to drive value. However, from my own experiences, from everyone’s experiences, here you can’t keep your eye only on that. You do have to keep looking and being active at finding other opportunities. I would say since IPO we have been getting an even higher rate of flow – deal flow and opportunities brought to us, but we are going to be very selective. So, if anything we bring on, has to be something we strongly in. It’s going to add value and be worth expanding. Our focus is primarily on earlier stage assets right now, but we are seeing some clinical stage and even commercial stage assets that we look at, but we will be really careful about our choices, but I think right now, we execute on six programs. I think you would agree that, that is tremendous amount of value creation. But I want to be ready now for just these six with the next stage for the company over the next few 2 to 3-year period and that means getting some new things in now.

Okay, that’s great. Thanks a lot.

Thank you. Our next question comes from the line of Ryan Martins of Jefferies. Your line is now open.

Hi, this is actually Chris on for Ryan. Just had a couple of questions. One follow-up on the endpoints for KRN23 obviously you discussed ricket reduction would be an approvable endpoint for pediatrics, but what in your view would be an approvable endpoint for an adult population?

Yes, that’s part of the discussion Sunil has been talking about. I will let him fulfill some details on that.

Sure and thank you Emil. Yes, Emil is exactly right. This is what we are working with the FDA on now and the EMA on now. So, we don’t have a definitive answer yet, but I will say we are actually having really positive discussion with both agencies. They appreciate the significant unmet medical need, not only in pediatrics, but in adults and they have clearly said that to us in our face-to-face meetings. So, to be determined and we will have more information when we start this study in the second half of this year.

Okay. And then just another question I had on triheptanoin, obviously an academic group is going to be publishing a randomized-controlled trial comparing it to medium change triglyceride oil and most in the abstract it seems like they are having some problems with compliance. And I was just curious what steps you have taken to overcome any of those potential issues?

Yes, it’s important area. We have talked about GI upset with triheptanoin as an oil as one of the potential issues. And we have – there are number of ways to manage those things to help that, which we have in our current program. And I can’t – we are not really in charge of that program. So, we can’t really say what they are doing exactly in all aspects of it. It is a program that wasn’t actually initiated with our product at that point. So, it’s more of what they are doing. It’s more of a physiological study and not much more we can say, but maybe I will let Sunil and he can tell more about what we are doing in our program to manage this.

Right, so thanks Emil. So, you are right, GI tolerability is a potential issue with triheptanoin, but we found with our experience with our investigators that this actually can be managed with how you administer it, for example one of our investigators told us and this actually was Dr. Fannie Michelle [ph] who is doing the Huntington study who is very well-versed in how you dose trihep. She was saying that for example if you give it in someone of a short type of administration, you have significant GI tolerability, but once you found out if you actually slowly give it over a period with a meal, then it is much more tolerated. So, we do think it’s manageable. We have updated all of our programs so that it can be manageable and has been throughout our studies to-date.

Okay, I agree. Congrats on the quarter and thanks for taking my question.

Thank you.

Thank you. And our next question comes from the line of Carol Werther of CRT Capital Group. Your line is now open.

Thanks for taking my question. I know you don’t want to give specific guidance for this year in terms of exactly what your burn is going to be, but can you give us an idea of how many people you are planning to add as you approach commercialization?

Thanks Carol. This is Emil. Well, many, let me ask, I will leave that to Sharp.

Sure, Carol. So, you will see when we file our 10-K that we went from 59 people to 170 people over the course of 2014. And I think you could see a similar trajectory of growth over 2015 if that’s helpful to you.

That’s helpful. Thank you.

Thank you. [Operator Instructions] And I am showing no further questions at this time.

Great, thanks. With no additional questions, this concludes the call and replay of the call will be available shortly. If there are any additional questions, feel free to contact us at 844-758-7273 or by e-mail at ir@ultragenyx.com. Thanks everyone for joining us.

Thanks, everyone.

Ladies and gentlemen, thank you for participating in today’s conference. That does conclude today’s program. You may all disconnect. Have a great day everyone.