Ultragenyx Pharmaceutical Inc.

Good afternoon, ladies and gentlemen, and welcome to the fourth quarter and full year 2020 Financial Results and Corporate Update Conference Call. At this time, all participants are in listen-only mode. Later, we will conduct a question and answer session, and instructions will follow at that time. If anyone should require assistance during the conference, please press star zero. I would like to turn the conference over to your host, Mr. Josh Rojiga. Sir, you may begin.

Good afternoon. and welcome to the Ultragenyx Financial Results and Corporate Update Conference Call for the fourth quarter and full year 2020. 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, and joining me on this call today are Emil Kakas, Chief Executive Officer and President, Camille Bedrosian, Chief Medical Officer, Eric Harris, Chief Commercial Officer, and Marty Deer, our Chief Financial Officer. I would like to remind investors that this call will include forward-looking statements within the meaning of the state's 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 quarterly report on Form 10-Q that was filed on October 27, 2020, our annual report on Form 10-K that will be filed soon, and our subsequent periodic reports filed with the SEC. which will all be available on our website in the Investors section. 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 that actual results could differ materially from those projected in any forward-looking statement. For 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 Emil.

Good afternoon, and thank you, everyone, for joining us today. 2020 was a transformative year for Ultragenyx as we executed on many of our strategic priorities. We now completed the fourth approval from our portfolio we had at IPO, and over the last couple of years, it's now refilled our portfolio with substantial opportunities in six clinical stage programs, four of which are entering pivotal studies. We also have 14 preclinical programs and are advancing our first mRNA program to the clinic this year. The efficient conversion of our pipeline to our next opportunities will allow us to accelerate our value creation and treat more rare disease patients with the first-ever specific treatment. From a commercial perspective, we achieved the upper end of CRISVIDA revenue guidance despite a global pandemic and received two U.S. approvals and launched treatments for two more diseases with no other approved treatments. From our clinical programs, we released important data updates from our gene therapy programs in GST1A and OTC deficiency, as well as the first glimpse of results for the antisense oligonucleotide GTX102 in Angelman syndrome. Those initial Angelman results suggest promising activity in this relatively large rare disease with no approved therapies, but certainly much more to do to realize the potential. On the gene therapy side, we initiated one of the largest ever gene therapy licensing deals by providing non-exclusive rights to Daiichi Sankyo to utilize our manufacturing platform, including our HeLa producer-selling technology. Later in the year, we partnered with Solid Biosciences and Duchenne Muscular Dystrophy to pair their differentiated microdystrophin construct with our AV8 variants and our productive HeLa PCL system. Most recently, we broke ground and began construction on our gene therapy, manufacturing facility in Bedford, Massachusetts, that will initially provide 30 manufacturing runs per year at the 2,000 liter manufacturing scale. Ultranet now has one of the broadest gene therapy franchises, which originated from our acquisition of Dimension Therapeutics in 2017. Our portfolio now includes three in-house pivotal clinical stage programs, one additional clinical program partnered with Bayer, and two publicly disclosed in-house preclinical programs Our robust HeLa PCL manufacturing platforms enable us to make AV vectors at large scale with a highly efficient, robust process that will enable the next generation of larger clinical programs. But that is just the gene therapy side of the business. Our development strategy is to choose the right modality for each disease, and we have a variety of other therapeutic modes in our portfolio. For genetic disease of bone, monoclonal antibodies can be the most effective way for affecting change in their biology. In December, we added a new late-stage monoclonal antibody program via collaboration with Myrio Biopharma. Citruzumab, or UX143, has completed a Phase IIb study in adult patients with multiple types of osteoarthritis imperfecta, or OI, which is one of the largest rare genetic bone diseases and significantly more common than XLH. Camille will provide more detail on this program later. But I will note that OI and the UX143 program are a perfect complement for our bone franchise and the expertise we have built with Chris Vita. Their learning from our Chris Vita development program will be very helpful in the future development of UX143. And there is significant overlap between the physicians who treat OI, XLH, and TIO. Importantly, this collaboration provides us with commercial rights to the product throughout the world, with the exception of Europe, where we will receive a royalty. While 2020 was a year full of unforeseen challenges, Ultragenyx was able to make substantial progress executing our strategic plan across our preclinical, clinical, and commercial programs with great success this past year. I'll hand it over to Eric to provide more detail on our commercial performance for the year.