Ultragenyx Pharmaceutical Inc.

Good day and thank you for standing by. Welcome to the second quarter 2021 financial results and corporate update conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. And to ask a question during that session, you will need to press star 1 on your telephone. Please note to limit yourself to one question with a follow-up. And if you require any further assistance, please press star zero. I would now like to hand the conference over to your first speaker today, Joshua Higa, Director of Investor Relations. Thank you. Please go ahead.

Good afternoon and welcome to the Ultragenyx Financial Results and Corporate Update Conference Call for the second quarter 2021. We have issued a press release detailing our financial results, which you can find on our website at ultragenyx.com. I am Joshua Higa, Director of Investor Relations. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Camille Bedrosian, Chief Medical Officer, and Eric Harris, Chief Commercial Officer. Marty Deer, our Chief Financial Officer, had an unavoidable flight delay and is not able to join us on today's call. I would like to remind investors that this call will include forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the types of statements identified as forward-looking in our 2020 Annual Report on Form 10-K that was filed on February 12, 2021, our quarterly report on Form 10-Q that will be filed soon, and our subsequent periodic reports filed with the SEC. which will all be available in the investor section on our website. These forward-looking statements represent our views only as of the date of this call and involve substantial risks and uncertainties, including many that are beyond our control. Please note, actual results could differ materially from those projected in any forward-looking statement. For a further description of the risks and uncertainties that could cause actual results to differ materially from those expressed in the forward-looking statements, as well as risks related to our business, Please see our periodic reports filed with the SEC. I'll now turn the call over to Amy.

Thanks, Josh, and good afternoon, everyone. I want to quickly reflect on our progress mid-year and note that we are approaching a period of significant execution on our clinical programs in the second half of this year. For those who are new to our story, we're a diversified global commercial rare disease company with three products approved for four indications. We now have one of the broadest clinical portfolios among rare disease companies in terms of modalities and indications, and one of the most prolific late-stage pipelines of both gene therapy and rare disease. This is a testament to our ability to identify great science, do effective deal transactions, and drive that work forward in the development of our first 11 years as a company. We are now on track to initiate four pivotal clinical trials over the second half of this year, and these include Phase 3 studies for two of our gene therapy programs, DTX401 and DTX301 in GSD1A and OTC, respectively. Two, a seamless Phase 1-2-3 study for gene therapy target UX701 in Wilson disease. Finally, a Phase 2-3 pivotal study for UX143 in osteogenesis imperfecta. Additionally, we will begin our first mRNA therapy, a Phase 1-2 study for UX053 in GSD3. Finally, GTX-102, developed by our partner Genetics, will now be back in the clinic treating angiomal patients again. I'll begin with the angiomal program, and then I'll summarize our clinical and commercial progress before handing it over to the team to provide more details. We previously announced in May and June that Health Canada and the UK's MHRA approved a modified clinical protocol to begin dosing pediatric patients with Angiomal Syndrome in those regions. These approvals followed review by both regulatory bodies of the clinical data on the first five patients in the U.S., as well as abundant non-clinical data. The agreed is moving forward based on both the promising activity seen at the first lower dose of GTX1-2 before any signs of serious adverse event had occurred and the extremely high medical need across the 60,000 patients and their families. We're now working closely with our partners to wrap up the remaining logistical details to get sites enrolling in dosing patients. In the U.S., we continue to make progress in our discussion with the FDA for resuming the study. Those discussions have been productive, and we have received agreement from the agency on a dose and administration plan for naive patients. The agency has requested us to bolster the protocol with some specific additional neurological assessments and documentation to verify the safety of the GTX1 and 2 during steady conduct and to manage the study closely. We'll make further simple adjustments to the protocol based on the feedback in order to get clearance to resume the study in the U.S. At this time, the FDA is asking us to hold back on retreating the prior treated patients. As we have noted before, we plan to provide a preliminary data update from some patients treated with GTX-102, either ex-U.S. or in the U.S. by the end of the year. The amount of data may be limited based on the timing, but we expect to treat the first cohorts of patients. Overall, we are enthusiastic about the process for treating engagement with GTX-102 based on everything we know today. We're looking forward to getting this treatment developed. Shifting to our gene therapy franchise, we've completed all of the known requirements to initiate three pivotal studies in GSD1A with DTX401, ODC deficiency with DTX301, and Wilson disease with UX701. At the ASGCT conference in May, we announced positive longer-term data from the DTX401 phase 1-2 study showing a 100% response rate across all nine patients treated and a durable response extending to two and a half years. Similarly, we shared positive long-term data from the DTX301 Phase 1-2 study that showed a response from all three patients at the Phase 3 dose, and a total of six of nine responders in the first three cohorts of patients enrolled. These patients maintained or improved their response up to three years following treatment. The teams are in study start-up mode for the pivotal studies, and we expect them to get going soon. Our proprietary and commercial-quality HeLa manufacturing platform will be the basis for the next generation of ultra-next-gene therapy products starting with our program for Wilson disease and extending to our preclinical programs for Duchenne, Muscular Dystrophy, and CDKL5 deficiency, a neurodevelopmental disorder. These latter two preclinical programs represent our first non-liver targeted gene therapies and address much larger patient populations. We will provide updates in these programs later this year. Beyond our gene therapy work, we've been on track to initiate two additional clinical programs this year, One is UX143 or citruzumab, our recently licensed antibody for osteogenesis imperfecta or OI. Our partner has already generated promising results in adults with OI showing substantial improvement in bone density. Based on our review of the science, both clinical and non-clinical, we are convinced that the enhancement of bone production via the anti-sclerostin mechanism will improve bone mineral density in a productive manner that will improve bone strength in patients with OI by making new bone right where bone stress is needed, and be superior then to purely antiresorptive actions, for example, bisphosphonates. This should decrease fracturing, so potentially prevent the deformation of spine and bones that comes with repeated fractures early in life, especially in the patients with type 3 or type 4 OI. The clinical study we'll initiate will focus on pediatric and young adult patients that have frequent fractures. We are planning the initiation of a Phase 2-3 study for this population by the end of the year. The other program is UX053, an mRNA program for collection of steroid disease type 3. This first mRNA program for our license with Arcturus Therapeutics is also expected to begin enrolling a Phase 1-2 study by year end. Now turning to our commercial programs, we had another solid quarter with significant revenue increases from Q1 in 2020 with progress over the globe. CRISFIDA particularly continues on a strong growth trajectory even as we enter the fourth year from its initial approval in 2018. This continued uptake is driven by both North America and Latin America. In North America, penetration in the adult exhalation population is a major driver in line with our expanded patient ID and broader physician outreach efforts. In Latin America, our team's efforts in supporting patient reimbursement have helped drive increased volume, and revenue, and patients are staying on Crescita once started and are compliant with their regimens. That is a testament to how much it helps patients with XLH. We also have a number of multigenerational families on Crescita now, and there's nothing better than to get notes from families commenting on how much their lives across generations have changed. Crescita is a paradigm shift in XLH treatment. We're happy to be at the forefront in making this happen. Our strong launch with Crescid has been followed by our successful launch with Dojolvi, which has now been on the market for about a year. Our team established great momentum, identifying patients, securing reimbursement coverage, facilitating new start forms. This strong start shows how much a new option was needed for LC-SAOD and tells us that Dojolvi will also represent a paradigm shift in the management of LC-SAOD. I'll now turn the call over to Eric to elaborate on our commercial progress in the quarter.