Ultragenyx Pharmaceutical Inc.

Thank you for standing by and welcome to the third quarter 2021 Financial Results Incorporated Meet. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone keypad. If you require any further assistance, please press star 0. Thank you. I would like to hand the conference over to Joshua Higa. Please go ahead.

Good afternoon, and welcome to the Ultragenyx Financial Results and Corporate Update conference call for the third quarter 2021. We have issued a press release detailing our financial results, which you can find on our website at Ultragenyx.com. I am Josh Wahiga, Director of Investor Relations. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Camille Bedrosian, Chief Medical Officer, Eric Harris, Chief Commercial Officer, and Marty Deer, Chief Financial Officer. I'd 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 the 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, see our periodic reports filed with the SEC. I'll now turn the call over to Emil.

Thanks, Josh, and good afternoon, everyone. I'll start off by highlighting our continued execution across Ultragenyx's broad portfolio of clinical and commercial assets. Since our last call, we've made substantive progress on two of our most significant clinical programs focused on larger, rare genetic diseases. The first of these is GTX-102, an antisense oligonucleotide that we are developing in partnership with genetics, biotherapeutics for Angelman syndrome. Over the last few months, we've successfully concluded discussions with three regulatory agencies to get our Phase 1-2 study up and running again. The XUS protocol initially doses four patients with two monthly doses each before our data monitoring committee reviews available safety data. As previously guided, we anticipate providing a preliminary update on the study after this review, which is expected to be around here. A study will then continue to treat those first four patients with two more monthly doses and to enroll an additional eight patients. We plan to provide a more substantial readout from the study after day 128 for all 12 patients, which is expected in mid-2022. Turning to the U.S., we expect to begin dosing patients under the revised protocol later this quarter. The dose in U.S. is lower than XUS, but the patient's are restricted to the younger, smaller, four- to eight-year-old age group. Our experience with the first five patients previously treated suggests that younger, smaller patients can respond to treatment at this dose after drug loading with four repeated low doses over three months. For Wilson's disease, we've also begun enrolling participants in the baseline evaluation phase of our pivotal study, UX. UX701 is an AAV9 gene therapy of a specially designed copper transporter that can restore normal copper metabolism and distribution. The design of our novel Phase 1-2-3 study in Wilson disease is notable because it enables a seamless transition from a traditional dose-finding Phase 1-2 study right into a pivotal study, which will save time. I will note that this is our fourth clinical stage gene therapy program and our first targeting a more prevalent genetic disease. Camille will provide more information on this program and studied later on in her section. Also of note is that the UX701 program for Wilson's disease is our second program to use the Ultragenyx Producer Cell Line, or PCL, gene therapy manufacturing system. Our PCL technology is a novel approach similar to vaccine manufacturing that is designed to yield a more productive and consistent AV production process. The result is that we are manufacturing commercial-grade material at commercial scale. for the first clinical patients with a substantial reduction in cost compared with triple transfection processes. This process allows us to manufacture enough material to treat all the patients randomized to UX701 in the Phase 1-2 portion of the study with a single run, significantly driving down COGS, an important element when thinking about the potential reimbursement challenges in the future for gene therapy products. We are continuing to invest in our PCL system Most recently, it's part of our preclinical AAV program for Duchenne muscular dystrophy. We expect the greater productivity of the PCL system will be especially important for more common and higher-dose indications like Duchenne, where the amount and cost of the product can be an important factor. Moving to the rest of our clinical pipeline, we are advancing four additional programs that further demonstrate the diversity of our portfolio across modalities. All four of these programs have new studies starting over the next few months, and three of these studies will be pivotal. Our gene therapy for DTX401 for GSD1A and DTX301 for OTC deficiency are both moving into Phase III studies based on durable, positive Phase I-II results over multiple years of follow-up. We are also on track to initiate a pivotal Phase II-III study of our newest program, UX143. This monoclonal antibody will be tested in pediatric and adult patients with osteogenesis imperfecta. A larger, rare genetic bone disease is a complement to the capabilities we've developed with CRIS-VITA. Wrapping up with our commercial programs, despite the recent challenges with the COVID Delta variant, our team has continued to be effective at supporting compliance for patients already receiving our therapies, as well as increasing new patient starts across all products. With CRIS-VITA, The successful launch continues, and we are now tracking towards the upper end of our full-year guidance. Adult patients are an increasing portion of patients we are identifying and converting to treatment. In Latin America, CRISPIDA continues to do especially well. Revenue in that region has more than doubled year-to-date in 2021 versus 2020. This growth is backed by increases across the board in patient identification, patients who have a prescription, and are navigating reimbursement process and patients receiving reimbursed therapy. We are nearing the conclusion of the formal reimbursement process in Brazil for access to the product, which should help further growth there. With Duljove, we're continuing to build the momentum of a strong launch. All the key metrics show the teams are able to find patients and quickly get them on reimbursed therapy. We're near 10% of the expected population of LC-FAOD patients now being prescribed Duljove in the first year or so of launch. also growing in Europe, driven by significant increases in named patient requests in France and other countries in the region. Before I turn the call over to Eric, I want to highlight our recently announced collaboration with FDA, NIH, and leading public and private organizations focused on gene therapy development. The Bespoke Gene Therapy Consortium is part of the NIH Accelerating Medicines Partnership Program. It's focused on advancing gene therapies for ultra-rare diseases. At Ultragenyx, we believe we have a responsibility to support the development of treatment for as many rare diseases as possible, including these ultrawares that might not otherwise get treated. We also believe that this joint collaboration will help identify ways to improve the development process and create a further improved regulatory paradigm to improve the efficiency and effectiveness of the development of the next generation of gene therapies for all rare diseases. With that, I'll turn the call now over to Eric.