Ryan Martins - VP, Strategy & IR Emil Kakkis - CEO & President Shalini Sharp - CFO Sunil Agarwal - CMO.

Mark - Cowen and Company Brittany - JPMorgan Gena Wang - Jefferies Adam Walsh - Stifel Joseph Schwartz - Leerink Chris Raymond - Raymond James Arlinda Lee - Canaccord Edwin - Wedbush Securities Carol Werther - H.C. Wainwright Mike Guo - SunTrust Robinson Humphrey Yigal Nochomovitz - Citigroup.

Presentation:.

Good day ladies and gentlemen and thank you for standing by. Welcome to the Ultragenyx First Quarter 2016 Financial Results and Corporate Update Call. At this time, all participants are in a listen-only mode, Later we'll conduct a question-and-answer session and instructions will follow at that time.

[Operator Instructions] As a reminder, this call is being recorded. I would now like to turn the call the call over to Ryan Martins. Sir, floor is yours..

Thank you. Good afternoon and welcome to the Ultragenyx Pharmaceutical Financial Results and Corporate Update Conference Call for the first quarter 2016. We have issued a press release detailing our finance results which you can find on our website at Ultragenyx.com. I am Ryan Martins, Vice President of Strategy and Investor Relations.

With me today also are the following members of the Ultragenyx Management Team, Emil Kakkis, Chief Executive Officer and President; Shalini Sharp, Chief Financial Officer and Sunil Agarwal, Chief Medical Officer.

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 plans with respect to our translational research program, the 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; the expectation of increased expenses over future quarters; our belief about adequacy of current cash resources to fund our operations; our intent to file for conditional marketing authorization and the timing of expected decisions regarding approval from regulatory authorities.

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 a 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 Annual Report on Form 10-K for the year ended December 31, 2015, filed with the Securities and Exchange Commission on Feb 26, 2016, our quarterly report on Form 10-Q for the quarter ended March 31, 2016 that will be filed soon and our subsequent periodic reports filed with the Securities and Exchange Commission.

I will now turn the call over to Emil..

Thanks, Ryan and good afternoon, everyone and thank you for joining us this afternoon. I will provide a high-level overview of our recent progress. Shalini will give you an overview of our first quarter financials and Sunil will provide additional details on our clinical development progress.

As we’ve noted at R&D day in December, we are working on expanding our early development program pipeline to get to five to seven programs operating within our Translational Research Group to potentially file an IND every year or every other year.

It is important to engage in separate now to ensure we do not have a development gap in a couple years from now as our current programs are in or are heading to late stage development. We will potentially do a number of different types of deals allowing ourselves to be opportunistic and adapted to the situation.

In general we look for programs that we can grow in value over two-year period and potentially reach a decision about heading the clinic in that timeframe. So I’d start by discussing our recent collaboration with St. Louis University why this is an important part of our long-term strategy to build a diversified company.

In March, we entered into a collaboration with SLU's Center for World Health and Medicine to develop small molecule therapeutics for facioscapulohumeral muscular dystrophy of FSHD. The team at SLU has a tremendous amount of experience as many were from a local Pfizer R&D Center with experience both medicinal chemistry and an early development.

The project lead from SLU was inspired to take action after his daughter was diagnosed with FSHD. We're pleased to be able to work with this talented motivated group and our broader drug development capabilities and rare disease expertise toward finding a meaningful solution for FSHD.

As part of our effort to initiate more early stage programs we also sought to further strengthen our Board in the R&D area and recently appointed Lars Ekman to our Board of Directors. He has deep expertise in building early stage and clinical pipelines and we work -- look forward to working with him.

Turning to our clinical program, we were pleased to have released positive interim data from the Phase 2 program of KRN23 and tumor-induced osteomalacia or TIO last month. Sunil will review this data later on today's call.

All of our clinical programs including three ongoing Phase 3 studies are advancing and we plan to initiate two additional Phase 3 studies this year. We have a number of upcoming anticipated milestones. I'll go over those briefly now.

KRN23 in the pediatric Phase 2 XLH study, we expect the 40-week data in 52 patients and 64-week data in the first 36 patients in the second half of 2016. We plan to file for additional marking authorization around the end of 2016. We plan to initiate a Phase 3 study in pediatric XLH patients in mid 2016.

For rhGUS and MPS 7 we expect topline data from pivotal Phase 3 study in mid 2016. Moving on UX007, in the second half of the year we expect 78-week data from our Phase 2 study in Long-Chain Fatty Acid Oxidation Disorders.

Based on the interim 24-week data from the Phase 2 study, we plan to initiate a Phase 3 study patients in the Long-Chain Fatty Acid Oxidation Disorders in 2017. Our Phase 2 seizure study in Glut1 Deficiency Syndrome patients continues to enroll patients and data are expected in the second half of 2016.

We plan to initiate a Phase 3 movement disorder study in Glut1 deficiency syndrome patients in the second half of 2016.

For Ace-ER and GNE myopathy, the CHMP opinion on the conditional marketing authorization application in Europe is expected in the second half of 2016 a decision from the European commission that is expected in the first half of 2017. We continue to enroll patients from the pivotal Phase 3 study in GNE myopathy with data anticipated 2017.

That along with the milestone, I'll turn the call over to Shalini now to provide overview of our financial result..

Thank you, Emil. We issued a press release earlier that included a financial update, which I will briefly summarize. Total net loss for the first quarter of 2016 was $52.8 million or $1.35 per share basic and diluted, compared to $21.4 million or $0.63 per share basic and diluted for the first quarter of 2015.

This reflected cash used in operations of $44.9 million in the last quarter compared to $17.7 million in the same period of 2015. We expect modest increases in cash used in operations quarter-over-quarter for the balance of the year.

Net loss for the first quarter 2016 included approximately $12.6 million in non-cash charges with stock-based compensation of $10.2 million, amortizations of premiums on investment securities and depreciation and amortization. In particular, stock-compensation expenses have significantly increased over time and we expect that this will continue.

Our total operating expenses for the first quarter of 2016 were $53.6 million. Research and development costs accounted for $40.4 million or 75% of our operating expenses. Our three Phase 3 programs account for the greatest proportion of R&D cost. As a reminder we share KRN23 development cost 50-50 with our collaborative partners Kyowa Hakko Kirin.

Costs for our multiple preclinical translational research programs, which now includes the FSHD program will continue to increase throughout the year at programs advance toward the clinic.

After excluding the $10 million one-time license fee paid to our tourist in the fourth quarter of 2015, operating expenses continue to increase due to the conduct of multiple late stage clinical studies, manufacturing cost related to clinical supply of multiple programs, increased regulatory activity across the progress, early stage investment in our U.S.

and European commercial infrastructure and patient identification efforts, general and administrative expenses to support these activities and increases in stock-based compensation expenses.

As we have said before our expenses will continue to increase as we invest in advancing multiple product candidate into Phase 3 clinical studies and developing and expanding our pre clinical stage portfolio. We ended the quarter with $487.8 million in cash, cash equivalents and investments on the balance sheet.

We believe that this is sufficient to fund all of our clinical programs through Phase 3 trials and potential launches. We do not have any date outstanding. I will now hand it over to Sunil to provide the development update..

Thank you, Shalini. I will provide an update on our clinical pipeline starting with KRN23. In April, we announced positive interim data from the first eight patients in the Phase 2 study of KRN23 for the treatment of tumor-induced osteomalacia or TIO.

The data indicated that patient treated with KRN23 had improved serum phosphorus levels and other bone metabolism measures. The main baseline serum phosphorus level of these eight patients was 1.7 milligrams per deciliter which is well below the lower of normal for phosphorus which is 2.5 milligram per deciliter.

Six of the eight patients had serum phosphorus level enter the normal range after beginning treatment with KRN23. One of the two patients who did not reach the normal range at the time of the interim data cut, did demonstrate an increase in serum phosphorus and continues to titrate those upwards.

The other patient who did not reached the normal range did not demonstrate any improvement hence serum phosphorus levels and have the highest baseline level of FGF23 of all eight patients. We also saw improvements in bone density in the two patients who completed 24 weeks of treatment.

One of these two patients also showed early evidence of fracture resolution and we look forward to having more bone data in the second half of this year. To date they have not been any serious adverse events in the study. Treatment emergent adverse events are observed in the seven of eight patients.

Treatment emergent adverse events occurring in two or more patients were primarily -- disorders including -- in muscular skeleton pain these are consistent with the symptoms typically seem patients with TIO.

Two of the eight patients had treatment related adverse events that were possibly or probably related including vitamin D deficiency and rash, both of which were mild. No injections side reactions were observed. Two patients reported symptoms that are suggestive of worsening pre-existing leg syndrome.

Overall the improvements that we have seen in serum phosphorus and other bone mineral metabolism measures in this study are generally consistent with what has been observed in studies of KRN23 in both paediatric and adult patients with XLH.

We're pleased to now have data in two diseases, which indicate potential support for the common mechanism of action of KRN23 which is binding to and inhibiting excess FGF23 activity, the key hormone that leads to phosphate wasting in these patients. Turning to XLH, Dr.

Carpenter presented 40-week interim data from the first 36 patients in the Phase 2 pediatric study in an overall presentation at ENDO in April of this year. In the second of 2016 we expect to have 40 week data from all 52 patients enrolled in the study including the original 36 and an additional 16 with higher baseline bone disease.

In addition we expect to have 64-week data from the first 36 patients in the study. The 64-week data will include additional rickets data, early growth velocity data, other efficacy measures and safety and tolerability information. We intend to file for conditional marketing authorization in the EU around the end of the year for patients with XLH.

We also plan to start a pediatric Phase 3 study in the middle of this year. The primary endpoint of this study will be evaluating rickets using the RGI-C scoring method and will also include a standard of care control arm. This study is expected to be required for potential approval in the U.S.

and could serve as a confirmatory study in the EU if conditional marketing authorization is granted. If conditional marketing authorization is not granted the study could required for approval in EU. We continue to evaluate KRN23 for adults with XLH.

We are currently enrolling two studies, the Phase 3 randomized double blind placebo control study in approximately 120 adult XLH patients, with serum phosphorus as the primary endpoint at week 24 and a brief pain inventory BPI as the key secondary end point and a 48-week open label bone quality study in approximately 10 adult patients to evaluate the effect of KRN23 on the underlying osteomalacia.

Turning to UX007 or Triheptanoin, in the second half of the year, we expect final data from the 78-week Phase 2 open label study in patients with long chain fatty acid oxidation disorder of FAOD.

The primary objective of the 78-week analysis is to evaluate the rates of measure medical events such as rhabdomyolysis, hypoglycemia and cardiac events on UX007 compared to the rates for 18 to 24 months prior to treatment with UX007.

Based on the interim Phase 2 data reported last year, we plan to initiate a Phase 3 study in patients with long-chain FAOD in 2017 for which we continue to optimize the endpoints and overall trial design. As a reminder this trial will be a head to head comparison against MCT.

In addition to the improvements we noted in exercise tolerant testing, we also noted some improvements in quality of life measures in the Phase 2 study and thus we believe having a patient reported outcome end point to compliment a functional end point will be important in the Phase 3 study.

We're in the process of collecting natural history data and qualifying the use of a PRO endpoint before meeting with the regulators. We expect to provide further details on the design of the study after completing discussions with the regulatory authorities.

In the second half of the year we plan to initiate a Phase 3 study in approximately 40 patients with the movement disorder type Glut1 deficiency syndrome. This study is randomized double blind placebo controlled double crossover study that plans to access the impact of UX007 on movement disorder events as recorded by a patient diary.

We continue to have discussions with the FDA regarding the primary end point and collection methodology and we are working on further – the clinical meaningfulness the Glut1 DS movement disorder events prior to finalizing the study design.

In addition our ongoing placebo control Phase 2 study in patients with seizure phenotypes is continue to enroll patients. And we expect data in the second half of this year. If the data are positive the If the data are positive, the seizure and movement disorder studies are intended to support an NDA filing.

We are also on track with rhGUS ACE-3 study in NPS seven patients. Topline data from the pivotal blinded placebo controlled 48-week study are expected in mid-2016. In the EU, the primary endpoint is the reduction in uGAG excretion after 24 weeks of treatment.

Some evidence or trend in improvement in clinical endpoints to support a favorable benefit risk ratio will also be needed. In the U.S., there was no primary endpoint as the FDA has said they will consider the totality of the data appreciating the heterogeneity of the disease.

Moving to ACR, we continue to enroll patients in the randomized double-blind placebo controlled, 48-week pivotal Phase 3 study of ACR in approximately 80 patients with GNE myopathy. Data from this Phase 3 study are expected in 2017.

We are also expecting a CHMP opinion on our Conditional Marketing Authorization Application for ACR in the treatment of adults with GNE myopathy by the end of 2016 and a decision from the European Commission in the first half of 2017.

Lastly, we continue to advance our Translational Research Programs who presented data from the recombinant human protective protein, Cathepsin A for rh-PPCA program at the World Symposium in March.

As Emil mentioned, there was a lot of ongoing activity with our preclinical pipeline and I look forward to sharing updates with you across all the programs as they progress this year. With that, I will now turn the call back to Emil..

Thank you, Sunil. As you heard, we continue to move our preclinical and clinical programs forward and are building out our teams to support our growing business across all functions. With the five program expected in 2016 and other ongoing clinical studies, we look forward to keeping you updated on our progress throughout the year.

With that, I'll end my comments and we can move to your question. And the operator, please provide the instructions for the Q&A portion of the call..

Yes. At this time ladies and gentlemen, we'd like to open the floor to your questions. [Operator Instructions] And it looks like our first question in queue will come from the line of [Mark] with Cowen. Please go ahead. Your line is now open..

Mark:.

Emil Kakkis:.

Sunil Agarwal:.

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Mark:.

Thank you. Sir. Our next question will come from the line of Corey Kasimov with JPMorgan. Please go ahead, your questions please..

Brittany:.

Emil Kakkis:.

Sunil Agarwal:.

Brittany:.

Thank you. Our next question will come from the Gena Wang with Jefferies. Please go ahead your line is open..

Gena Wang:.

Emil Kakkis:.

Gena Wang:.

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Gena Wang:.

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Sunil Agarwal:.

Gena Wang:.

Thank you, Ma’am. Our next question in queue comes from the line of Adam Walsh with Stifel. Please go ahead. Your line is open..

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Thank you, sir. Our next question in queue will come from Joseph Schwartz with Leerink. Please go ahead. Your line is open..

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Thank you. Next question in queue will come from Chris Raymond with Raymond James. Please go ahead..

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Thank you. Our next question in queue is Arlinda Lee with Canaccord. Please go ahead your line is now open..

Hi guys, thanks for taking my question. A couple of them, first, we had a chance to speak with Dr. Carpenter recently and he mentioned that there was increasing, I guess awareness of XLH and I was wondering maybe kind of related to that expanding to other phosphate raising diseases.

Could you maybe characterize what your interactions with patients and I guess investigators reaching out to you on KRN23 and like and then I have a question on 007..

Emil Kakkis:.

Arlinda Lee:.

Emil Kakkis:.

Arlinda Lee:.

Thank you, Ma’am. Our next question in queue comes from Heather Behanna with Wedbush Securities. Please go ahead. Your line is now open..

Edwin:.

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Thank you. Our next question comes from Kennen MacKay with Credit Suisse. Please go ahead. Your line is open..

Unidentified Analyst:.

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Unidentified Analyst:.

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Thank you. Our next question in our queue comes from Carol Werther with H.C. Wainwright. Please go ahead. Your line is now open..

Carol Werther:.

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Shalini Sharp:.

Carol Werther:.

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Thank you. Our next question will come from Edward Nash with SunTrust. Please go ahead. Your line is open..

Mike Guo:.

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Mike Guo:.

Thank you. Our next question will come from Yigal Nochomovitz. Please go ahead with Citigroup..

Yigal Nochomovitz:.

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Thank you, sir. And at this time, I’m showing no additional questions in the queue. I would like to turn the program over to Ryan for any additional or closing remarks..

Thanks. This is all the time we have for your questions on today’s call. We concluded the call now, with no additional questions. A replay of the call will be available shortly. If there are any additional questions, please contact us at 844-758-7273 or IR at Ultragenyx.com. Thank you everyone, for joining us..

Thank you, presenters and thank you to all of our participants for joining. This does conclude today's conference. You may now disconnect and have a wonderful day..