Robert Anstey - Investor Relations Emil Kakkis - Chief Executive Officer and President Shalini Sharp - Chief Financial Officer Sunil Agarwal - Chief Medical Officer.

Eric Schmidt - Cowen & Company Heather Behanna - Wedbush Securities.

Good day, ladies and gentlemen and welcome to Ultragenyx Pharmaceutical’s Second Quarter 2015 Financial Results. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session with instructions following at that time. [Operator Instructions] And as a reminder, this conference call is being recorded.

And I would now like to turn the conference over to your host, Rob Anstey. Please begin..

Great, thank you. Good afternoon, everyone and welcome to the Ultragenyx Pharmaceutical financial results and corporate update conference call for the second quarter of 2015. We have issued a press release detailing our financial results, which you can be find in our website at www.ultragenyx.com.

With me today are the following members of the Ultragenyx management team, Emil Kakkis, CEO and President; Shalini Sharp, CFO and Sunil Agarwal, CMO. We will keep our prepared remarks brief in order to allow time for the question and answer portion of the call.

But 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 expected release of data from clinical studies, the initiation of additional clinical studies and the designs of same, plans regarding ongoing studies for existing programs and the possibility of being able to file from initial approval and our [indiscernible] were permitted.

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 drug 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 March 31, 2015 filed with the Securities and Exchange Commission on May 12, 2015.

Our quarterly report on Form 10-Q for the quarter ended June 30, 2015 that will be filed today and our subsequent periodic reports filed with the Security and Exchange Commission. I will turn it over to Emil..

Thanks, Rob and good afternoon everyone. Thank you for joining us on the call today. I’ll start things o6f with a quick update since the last call, Shalini will then provide financial update and Sunil will provide overview of our progress on our clinical pipeline.

It’s been an exciting few months at Ultragenyx since the last call, we reported date from our KRN23 program, raised, substantial funding, expanding our senior management team and push forward our late stage programs.

Regarding KRN23, we reached a number of significant milestones since our last earnings calls; first, we reported the first set of data on clinical studies for our KRN23 in pediatric patients with XLH.

We released 16 week data for 36 patients, which showed an increase in serum phosphate in all patients consistent with the data previously generated in adult XLH patients.

Following that in early July, we released interim four week, 40 week data for the first 12 patients showing an improvement in Rickets Bone Disease scores beyond what was achieved with standard of care at baseline, we are encouraged by these results and we look forward to releasing additional 40 week data in 36 patients in Q4 of this year.

Sunil will provide more detail on these clinical results later in the call. We also recently announced the details of our Phase III program KRN23 in adult XLH patients, that two study program including a randomized control study and a small bone biopsy study will kick off later this year.

Additionally, we held discussions with the European Medicines Agency or EMA about the KRN23 program, in those discussions the EMA indicated that we may be able to file for traditional approval for KRN23 based on the 40 week data on 36 patients, if a positive benefit risk is obtained in treating bone disease combined with existing adult data generated to-date.

We now look to forward to following up later this year with both EMA and FDA to discuss the recent Rickets data and determine appropriate next steps in our development program. KRN23 was the highlight of last quarter, but we are on track for a number of key milestones and other programs over the next six months.

So for the remainder of 2015, these milestones include Triheptanoin compassionate use data in instance with FAOD and life trending cardiomyopathy that will be presented at the SSIEM, the Society for Inborn Error Metabolism in Europe.

We will have Triheptanoin data from our ongoing Phase II study in FAOD patients in Q4, with Triheptanoin interim analysis from our ongoing Phase II study in [indiscernible] patients with a focus on the seizure aspect of the disease, these data will be available either late this year or early next year.

We will be filing an MAA for ACR for conditional approval in Europe for GNE Myopathy, in addition KRN23 initial data from the Phase II study of 200 used optimal relation will be released late in 2015 or early 2016.

The KRN23 40 week data including more [indiscernible] in 36 patients from Phase II study in pediatric XLH will get released and initiation of KRN23 Phase III randomized double-blinded placebo control study in adults with XLH will get announced.

To support these and other efforts including the three Phase III studies that will be running by the end of 2015, we recently raised additional capital, we brought in $287 million in net proceeds to accelerate commercial launch, preparation for ACR rhGUS and KRN23 to invest in early stage research and potential new programs and support continue in late stage events programs.

To drive our development and commercialization efforts, we recently announced two new members of the executive leadership group Jayson Dallas joined us as Chief Commercial Officer leading our Global Commercial Efforts and will first focus on developing our Global Commercial team and our European, U.S. and Latin America operations.

John Pinion joined us as Chief Quality Operations Officer in this new role, John will lead our Global QA, QC functions as Support Manufacturing clinical development as well as support transitional research effort, develop early pipeline projects from his analytical science and research role.

Both of these positions are critical as we move forward towards the commercial stage, Jayson and John have a deep background in building and leading successful organizations including both most recently managing significant operations at Roche, Genentech, we welcome them both to Ultragenyx.

We also recently announced Dan Welch has been elected Chairmen of the board. Dan’s extends experience as well as Chairman and CEO InterMune and mostly his role as a Executive Partner at Sofinnova the valuable asset going forward.

It has been a busy 2015 for Ultragenyx so far in the rate of pipeline advancements, and in-flows is expected to continue to increase. In the next 12 to 18 months will have approved constant Phase II data or pivotal Phase III data from all six of our clinical programs and even more significantly may be on track for U.S.

or EU filings for as many of the three of those programs in the next 18 months. And I’ll turn the call over to Shalini to walk through the latest financials..

Thanks Emil and good afternoon everyone. We issued a press release earlier that included a financial update, which I will briefly summarize. Our second quarter 2015 results included total operating expenses of $30.1 million. Research and development cost accounted for $23.1 million of 77% of our operating expenses.

Our Phase III programs, ACE-ER and rhGUS accounted for the greatest proportion of R&D cost in the first half of 2015 as well as the largest increases and expenditure relative to the first half of 2014. Expenses also increased for both Triheptanoin programs and both KRN23 programs as compared with the first half of 2014.

As a reminder, we share KRN23 development cost 50-50 with our collaborative partner in Kyowa Hakko Kirin. Costs for our preclinical translational research programs also increased over the prior year period.

OpEx increased significantly from the second quarter in 2014 due to the conduct of multiple late stage clinical studies, manufacturing cost related to clinical supply of multiple programs, investments in our commercial infrastructure and patient identification efforts and general and administrative expenses to - for these activities.

Total net loss for the second quarter of 2015 was $29.8 million or $0.83 per share compared to $13.6 million or $0.45 per share for the second quarter of 2014.

Net loss for the quarter ended June 30, 2015 included approximately $6.6 million or $0.18 per share a non-cash charges such as stock based compensation, amortization of premiums on investment securities and depreciation and amortization.

As we continued to build our global commercial capabilities and advance our clinical stage and preclinical pipeline programs, we anticipate that our expenditures will continue to increase in future quarters.

We ended the second quarter with $326 million in cash and cash equivalents and short term investments on the balance sheet that does not include the $287 million in net proceeds raised from an underwritten public offering of common stock in July of 2015. We do not have any debt outstanding.

I will now hand it over to Sunil to provide the development update..

Great, thank you Shalini. I will now provide an update on our clinical progress over the last several months. As Emil mentioned we’ve recently released data from two separate analysis from our Phase II study of KRN23 in pediatric patients with XLH.

The first - was the 16 week analysis in 36 patients that showed a all patients had increased within serum Phosphorus from baseline after treatment with KRN23. At the end of the 16 weeks, the majority of patients in both the monthly and by weekly dosing groups reached the normal range of serum Phosphorus.

Additionally the number of patients achieving the normal range continued to increase after week 16 as patients continued to have their does high rated as needed. Increases in TMP, GFR and vitamin D are also observed in the 16 week analysis.

Overall, these results met our expectations and are consistent with the data generated to date in adult XLH patients. From a safety perspective, no serious adverse events have been reported and no patients have discontinued. The most common adverse events have been injection side related and all of them are deemed mild in severity.

No significant changes in serum calcium, urinary calcium or parathyroid hormone were observed. A no patients had serum Phosphorus levels above the upper limit of normal. The second set of data included the first interim look of KRN23 impact on the underlying bone disease in these patients.

The analysis included X-ray data from the first 12 patients evaluating their degree of rickets, via the fracture rickets severity score. The mean rickets score decreased by 58% going from 1.4 at baselines to 0.6 at week 40.

Eight out of 11 patients with rickets at baseline demonstrated an improvement of which three patients no longer exhibited radiographic evidence of rickets at weak 40. Of the 12 patients, six received by weekly dosing and six received monthly dosing.

One patient in the biweekly dosing group did not present with the radiographic evidence of rickets at baseline, and was excluded from the analysis.

All five out of five patients in the biweekly dosing group demonstrated improvement in rickets, the mean base line rickets score was 1.5 and changed to 0.3 at week 40 representing an 80% reduction in the biweekly dosing group. Of the six patients in the monthly dosing group, three out of six demonstrated improvement in rickets.

Two patients did not show a change, and one patient worsen by 0.5 points. The mean baseline score was 1.3 and changed to 0.8 at week 40 representing a 38% reduction in rickets score in the monthly dosing group.

Overall, these interim data are encouraging as they are the first indication that KRN23 may improve rickets beyond what can be achieved with standard of care. No series adverse events were reported and no patients have discontinued. Mild injection [indiscernible] were the most common adverse events.

Pediatric Phase II data in 36 patients at 40 weeks are expected in Q4 this year. We are also expanding the pediatric trail to a total of approximately 50 patients and expect 40 week data on all 50 patients by mid 2016. The additional 40 week data will include rickets as well as safety and pharmaco dynamic for the dynamic results.

Prior to having the 12 patient 40 week analysis, and in meeting with the EMA, the European Agency indicated that a conditional approval filing may be possible based on positive 36 patient, 40 week pediatric data and data from the completed Phase I, II and ongoing Phase II extension studies in adult XLH patients.

Confirmatory studies would be required to maintain and convert any such conditional approval to full approval. Based on FDA and EMA feedback, we plan to initiate a Phase III program in adult XLH patients by end of year, which will include two studies.

The first of the pivotal Phase III randomized double-blind placebo control study of KRN23 versus placebo with approximately 120 patients. Serum prosperous is the primary endpoint at week 24 and the brief pain inventory will be a key secondary end point all in week 24.

The second study will be a smaller open label study in 10 patients evaluating osteomalacia, pre-impose treatment with KRN23 via bone biopsy. We are encouraged about the recent progress with the KRN23 program; we plan to follow-up with both the EMA and FDA to gain clarity on the next steps in the development program.

Moving on to triheptanoin, several data points are expected to be released by the end of 2015. First will be compassionate use data and in infant FAOD patients with severe cardiomyopathy.

At the SSIDM conference in September, five case reports will be presented many with life threatening disease despite treatment with the standard of care medium chain triglyceride oil. Data from our 29 patients, Phase II study of triheptanoin in a broader and older FAOD population will be released later this year.

The study is a learning study, and as measuring a wide range of muscular skeletal, liver, and cardiac endpoints with a goal of determining, the best signals of activity to further evaluate in a potential pivotal trail.

In interim analysis from our ongoing Phase II, study of triheptanoin and Glut1 Deficiency Syndrome is also planned around year end 2015 or early 2016. The primary focus of the study is seizure manifestations of the disease.

Based on the positive investigator responsive trial results in the movement disorder manifestations of the disease presented at AAM earlier this year we are in the planning stages of a second company sponsor studying Glut1. The plant study design is a randomized double-blind placebo controlled crossover study.

We plan to discuss this study design with the FDA by year end. The final two brief updates are for our most two advanced programs. First for rhGUS, our pivotal Phase III study is fully enrolled with 12 patients as of June 2015. The top line data are expected in mid of 2016.

In addition to this Phase III study, a few weeks ago we initiated a Phase II study in patients under five years old with MPS 7. The study will include patients with the severe infantile presentation called non-immune hydrops fetalis. Interim data from this study are expected in late 2016.

We are also now treating two infant MPS patients under named patient programs one in France and one in Turkey.

We will continue to evaluate named patient treating requests as they come in, the next and final program is Aceneuramic Acid Extended Release or ACR, we are currently enrolling GNE Myopathy patients in the pivotal Phase III study that trial kicked off in May of this year and it is expected to report data in late 2016 or early 2017, the primary end point is a composite of upper extremity muscle strength at week 48, This is the same endpoint used in Phase II which showed a preservation of upper extremity muscle strength over 48 weeks.

Based on the Phase II data, we intend to file for conditional approval in Europe this year, I’m pleased with the milestones our team has reached thus far in 2015, I look forward to updating our investors, the physicians we work with and the patients we serve as we generate more data and advance our pipeline. I will now hand it back to Emil..

Thank you, Sunil. We made substantial progress to our pipeline in 2015 so far and there is more to come in the second half of the year. Our significant cash balance will support [indiscernible] development efforts as we continue or believe to track top notch talent as we extend our critical functions.

Before closing all my comments, I would like to remind listeners that we will be holding a first Ultragenyx Research and Development Day; it will be in New York on December 3 more details will be provided later but we hope to see many of our shareholders there.

Thank you everyone for listening, we can now move onto the Q&A session and can the operator please provide the instructions..

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

Good afternoon congrats on all the progress and thanks for taking my questions.

Maybe just first for Emil or Sunil on the Triheptanoin study in FAOD the company sponsored study because that is the next major company sponsored event and I know you are calling this a learning study as multiple [indiscernible] points but what ideally would you like to see from the trial in order to be convinced that you have said differentiation versus MCT else?.

So I will start with that, maybe Sunil can provide significant comment, the types of patients that enrollment study patients who are on standard of care which usually included MCT oil who were fairly standard of care and we are having significant clinical problems, the largest group of patients have problems with [indiscernible] or muscle disease tolerance, it is essentially muscle problems and many of the patients have impaired ability to walk around or play or go to work and often very sad to avoid worsening their problem.

So that in those types of patients we are hoping to look at some of the impact on their skeletal muscle disease which might translate into either improvements in their walk distance or their exercise output on the [cyclogameter] (Ph) or in perhaps energy efficiency in how well their heart essentially is able to pump out and function during exercise tolerance events.

We will look at [indiscernible] endpoints as well but the study will bring the larger to be able to look at let’s say how many events of - they have so we are going to focus more on the physiological measures in disease, in the hyperglycemic patients or smaller groups that are qualified they are having ongoing and recurring hyperglycemic problems.

Many of them are constantly treated with food or maintain to avoid hypoglycemic, so we are primarily looking at how well they do when they take a faster blood glucose or when they are in fact how many events per day they are having needing supplementation or symptoms suggest hyperglycemic, we will look at their liver tube because usually in these patients the livers can be large or have [indiscernible] and other things related to great count of fatty acids and the inability to produce energy.

So that is the kind of things we look, the cardiomyopathy group in the study - I think there was just one who qualified based on cardiomyopathy issues and so we won’t really learn very much about cardiomyopathy in the study what was happening is that the patients who might qualified with cardiomyopathy were we are actually landing in the emergency room or the ICU and we are getting urgent emerging call for help and we decided not to risk locations wide and would go had let them get treated then in patients capacities program.

There are five of those patients that are in the FSIM release is coming out so I have look to more out release to tell you what you see in patients who are un-standard care having cardiomyopathy problems and they are being treated with triheptanoin. So we have to summarize Eric.

The skeletal muscle part is the phenotype is the most common phenotype in the study and so our goal would be to show that their muscles are functioning better and some physiological measure either really the how much energy they use and they can create site examiner at a particular heart rate or how far they walk or how much their energy efficiently looks like and those are the things that our good measures are whether their muscles that are functioning optimally not using energy optimally which are we think part of the problem you see in [indiscernible] patients and why they have so much exercise in parts kind of long answer I don’t know if I left off something..

No, actually I think you covered well Emil, just remain people as well this is the 24 weeks 6 months analysis, but the study doesn’t end there, we do want to continue to follow for a long-term potential benefit or signal generation so the study continues on for another 54 weeks so again 24 weeks by end of this year, but we will continue to follow these patients longer term..

And then Emil you mentioned the 5 I guessed, I guess the patient use cardiomyopathy patients what would be the normal prognosis for such patients not treated with trihep..

Well, many of the patients end with cardiomyopathy basically they die from the cardiomyopathy and there are patients treat with standard care will bounce back some but may the patients start having up former of a severe FLD type and then mortality rate quite high and in this case study patients who were in ICU many of them are fractures on bachelor support or heart cardiac support function devices and things.

So they were pretty sick condition and so the data will review what their heart looks like in terms of echocardiographic at, function and how do they do when put on trial..

Thanks, there may be one quick one for Shalini just on the expense looks like it was pretty decent size bump up in Q2 and you eluted to continued growth, we’re going to grow at a similar phase or just grow more gradually off of the Q2 base..

Well, I think we more continue to grow other fairly significant phase Eric, so we do have multiple programs heading into phase III and we are trying to now build up the commercial infrastructure and it really rapid in efficient way so we do expect that to continue increase and then as I noted in my comments there is a significant non-cash component in there as well, so I would pay attention to that too..

Great thank you..

Thanks, Eric.

Thank you. And our next question is from [indiscernible] from J P Morgan. Your line is open..

Hi guys, this is Britney and for Clarie thanks for taking the questions.

So with the majority of you products and natively stated [indiscernible] can you comment on higher thinking above feeding your pipeline are you actively looking to add any additional products and so what type of products would you be looking for and then quickly on KRN23 is there potential for accelerated approval any as well, thank you..

So I will start with the pipeline and then leave Sunil can talk about the regulatory pathway for KRN23 in U.S.

so we are actively obviously when we have 5 or 6 progress in clinic we were in last couple of years working on driving that portfolio forward and that with our job 1 company over the last year and half that we have started and have a done a quite a bit of work on a number of business development opportunities and we are actively building out the pipeline in the transitional research group.

We currently have two programs that are in preclinical and now we have disclosed Glaxo celldose is our UFO for [indiscernible] other one and in general what I would tell you and we are not going normally disclose our stuff that’s preclinical we will disclosing in general when we are headed to an IND what we know we have got something in hand or we are ready to proceed, our plan now with the two is expand to five or six programs and several things we are looking at right now and working on and we would be expect to be doing that in the coming 12 month period.

Some of those may not get disclose. Some maybe disclose based on the deals that they are and well our goal would be to have five or six early stage program to drive ahead and with the goal of trying to get about an IND every year to keep refilling the pipeline and keep that pace up.

We definitely don’t want to have a gap or everything we use to Phase III and suddenly there is like nothing behind it. So we’re aware of wanting to keep that flow going and I think we are seeing a lot of great opportunities.

To answer another question no one has asked yet, but people always ask, well, how are you competing for these assets or so many people out there, the truth is a lot of these is that people haven’t heard of you, haven’t heard of that have great strategy for treatment and we’re focusing on those things for people are not paying attention to that we think are really important opportunities.

And we are willing to look at sins that needs slightly more work and maybe - answer that one critical question on your own laboratory if it’s necessary to get us to a product that we can develop.

So I think there is a quite a bit out there, actually you have not run into the problem, I think we talked about late stage to commercial assets is a lot of competition and while we look at them, I think it’s less likely to be an effective way to go forward.

In terms of what we’re choosing, we look at [indiscernible] things but we can’t build a whole company on things are small [indiscernible] will do one of those here and there, will have at least a slot in our pipeline for that.

But we are getting that are often that have metabolic, basics that we understand the biology book which is even the treatment. And we try to focus on really simple stories and simple mechanisms where possible something low or something high, and we are trying to reverse that.

So we are finding a lot, there will be things at it, into the pipeline over the coming years. So I’ll let Sunil talk about, reg path for KRN23 in U.S..

Yes, sure. So thanks for the question. I think one general comment is we stayed very close to FDA and EMA to run all the part programs of course, KRN23, so it isn’t we talked to them on a infrequent basis we talk to them regularly.

So we’ve had the recent data set now when we do plan to follow-up with both agencies, we’ve mentioned the EMA discussions and with the FDA our goal is to understand the next steps specifically run the pediatric development program, could there be a potential for something like that, yes, but I don’t want to speculate on the chances of that, because we just having had those discussions with the agency yet..

I would reiterate one thing is that FDA previously said that they wanted standard of care controlled study data for the product that’s sort of a question you had us made of us before we think the data compelling that we can try to work to [indiscernible] pathway, but I would point out that they were reiterated the need for control data and so to acquire significant amount on our part too and to look at the body, the data they make head away with them on a more rapid pathway..

That’s right..

Okay. Great. Thanks so much..

Thank you. Your next question is from Heather Behanna of Wedbush Securities. Your line is open..

Hi, guys. Thanks and I apology in advance for the background noise, just a quick question on TRN23, you could talk about are you rolling people over into the other label extension after 40 weeks, can the dosing be adjusted and if so what kind of things would you be monitoring for durability moving forward..

I will let Sunil answer that question..

Yes. On the pediatric program as you know, it’s a Phase II program currently and the answer is yes, we will be rolling people over into an open label extension. But just remind you the primary study is up to 64 weeks and one of the other 10 points, that 64 weeks is looking at growth velocity.

So we look forward eventually to having that data as well and seeing what that looks like but after 64 weeks, we think it’s critically important to understand to your point on long term safety and efficacy of this agent.

So yes, we are rolling over those patients in addition to that, on the adult side, the Phase I, II was completed but those patients were not rolled over to extension originally. We have reopened an extension for women or recapturing those patients.

So we believe in capturing that long term extension data to your point across both adults and pediatrics..

Great. Thank you..

Next question is from [indiscernible] of Jefferies. Your line is open..

Great. Thanks for talking the question.

I just had a couple on KRN23, firstly while for the EMA the potential through the conditional approval which you are going to play for what is that label going to look like, I can include those pediatrics and adult patients or what’s your view on what that might look like, if you are to get the commission approval..

Thanks for your question. This is Emil. We generally wouldn’t predict what’s on the label, but I would let Sunil talk about what we have data to support..

I think there is may be one general comment about on pediatrics and adults and maybe more some on adults.

As we continue to do drug development, we also continue to work on understanding the disease burdens something that is critically important disease especially also in XLH and we have done a lot of work in adults [indiscernible] and what we are finding is that the adult disease can be pretty severe with respect of the symptoms they have, so when we do go back to the EMA, our plan is to talk to them about adults and pediatrics, not just pediatrics, we have adult Phase 1, 2 study, we have now pediatric phase II study, so our plan is to have a discussion of the totality of the disease and then determine with them what makes sense..

I think they said that we do have data on adults, they did say existing data will be findable, but the question is data primarily but we do show bone marker data, I think you could look at the drug, if you look at the holistically at the program raising phosphate improves bone disease and rickets, it means the [indiscernible] are seeing enough phosphate to make bone, which to me is just the signal that essentially the phosphate levels we are achieving are clinically meaningful and they are changing the bone in stations, so we think that would be true for adult to harder to mention adult because their bone turnover is just slower and the timeframe is taking longer, so but we think we would certainly want to seek both but our best case is certainly is in this area..

Great, okay and then just kind of touching on the same point, what kind of things can or what kind of data we get bone biopsy study that you are talking about will it be bone internal growth will it be things like bone density or just give us a sense maybe what that might look like?.

Yes it is a good question, so really as you know in adults the osteomalacia is the issue, is the histology, the lack of architecture of the bone, so the way we would simply describe this bone quality, you will be looking at really the cellular architecture of the bone and osteomalacia by histology is very easy to recognize and it can be very severe in these adult patients, so it is one of those things when you see it, you see it and by the end of this study after approximately year of treatment we will look again, so it is all about bone quality..

Okay.

And just one last question [indiscernible] for the rickets score obviously this design for nutritional rickets and so the scale is pretty big in terms of what you might expect for XLH patients or is there any concerns about what the sensitivity could be in that big scale and relation to XLH genotypes in response to treatment?.

Yes, I will let Sunil answer the question the scale..

Yes sure it is a great question and the score is 0 to 10 and I think without understanding or some of them may not understand the disease or how it is going to suddenly work, one could maybe make the misunderstanding that well if you - in the middle of the bad disease and that’s actually not the case on how the scoring system works.

For example when I’ve seen X-rays, myself of a score of three or even two that is extremely noticeable and looks very problematic for the wrists and knees for these patients.

So when we look at how the scoring system is operating scores of two, three or even one are very noticeable by our expert independent reader and when you talk to the specialist they consider that very meaningful disease.

In addition to that to your point about sensitivity, it does appear very sensitive to ask that changes based on the data we’ve seen, based on talking to the experts and also the person who invented this scoring system, Dr. [Patcher] (Ph).

Okay. Great. Thanks for taking the questions..

Thank you. Our next question is from [indiscernible] of MOV. Your line is open..

Hi, guys. Thanks for taking my question.

Can you maybe provide an update on the name patient sales progress for ACR and then on maybe you talked about how you think about the pipeline going forward, could you maybe comment on the kind of pipeline that we might be seeing from the existing drugs that are in the clinic already and your appetite for maybe adding additional indications and what the timing of that might be? Thanks..

All right. We’ll start with the name patient sales in UX-01, we are planning to pursue name patients treatment for 01 patients where IMA filing is happening in this half of the year, we are working on name patient sales process for UX-O1, we think there is substantial interest on it.

Our expectation will be to be working with investors who want to treat their patients.

Because of the various rules of that, we will be focusing on people that don’t qualify for the phase III study, because that they qualify for phase III they should be and phase III program not in the name patients health and so generally in these type program we will start looking in the key countries in Europe or it’s possible with the best cares you have severe patients they don’t qualify for a study, so that process ongoing we have initiate patients and they name new patient treatment yet in UX001 but as its proceeding take some time to work through.

The second question is are we pursuing other indications for our current pipeline I think right now the pipeline opportunities for the current targets are set, but there are something we are doing investigator studies that could be common part of the story, so we are adding the movement [indiscernible] to the Triheptanoin program as you know and I think we have already discussed that as part of good one indication.

We are supporting investigator study on [indiscernible] diseases as well which is a 100 patient analysis control study so not in significant IST that’s is up and running so that is that another potential indication that come in and if the study shows efficacy and safety and identification so it would be potential in other way for Triheptanoin to move forward there are actually a number of ISTs ongoing or Triheptanoin, some of them can be discovered by going in clinicaltrail.com and we get a lot of request in fact for other possible indications.

Right now we are focused on the two genetic indications that were must comfortable with, but we are supporting some other lower efforts which could potentially become sponsored efforts that they show I mean that there is a reason to invest.

The KRN 23 at TIO and XLH are really the important ones are is [indiscernible] is ongoing dominant types of Hypophosphatemia which are also associated with elevated FGF23.

There are relatively smaller indications that are at this point we are not actively pursuing them don’t think it adds to the story I think the TIO was important to do because there are lot of patients in very severe conditioned and we were getting a lot of capacities request so we thought like that was a disease there we thought that was, there was distinctly different and perhaps in the area where we could show substantial benefit of the treatment is effective.

So with that those are those to for some of the other programs like enzyme Triheptanoin MPS 7and we are on anticipating large number of major markets for MPS 7 enzyme, but if you found one let us know but that’s the small, population, but we think a really important disease states.

So we continued to do some products that are like that, they don’t have large secondary indications.

I think that probably KRN 23 [indiscernible] in terms of volume important one regard to that, I think right now are also our effort is really clean in the pipeline and the next R&Ds and I think we are seeing some really interesting things and I feel pretty good about us still they are very nice early stage by planning help we sell pipes move forward..

Thank you. End of Q&A.

Great well, that there are no additional questions I guess. So that concludes call today. A replay of this call will be available shortly on by webcast and phone. If you have any other questions offline, feel free to contact us at 844-758-7273 or by email ir@ultragenyx.com. Thanks everyone for joining us..

Ladies and gentlemen, thank you for your participation in today’s conference. This concludes the program. You may now disconnect. Have a great day..