Ultragenyx Pharmaceutical Inc.

Good afternoon and welcome to the Ultragenix second quarter 2023 financial results conference call. At this time, all participants are on a listen-only mode. At the end of the prepared remarks, you'll have the opportunity to ask a question during the Q&A portion of the call. It is now my pleasure to turn the call over to Joshua Higa, Vice President of Investor Relations.

Thank you. We have issued a press release detailing our financial results, which you can find on our website at ultragenix.com. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Eric Harris, Chief Commercial Officer, Eric Krambes, Chief Medical Officer, Aaron Olson, Senior Vice President of Corporate Strategy and Finance, and Ted Huizenga, Chief Accounting Officer. I'd like to remind everyone that during today's call, we'll be making forward-looking statements. These statements are subject to certain risks and uncertainties, and our actual results may differ materially. Please refer to the risk factors discussed in our latest SEC filings. I'll now turn the call over to Emil.

Thanks, Josh, and good afternoon, everyone. It has been a year of increasing momentum marked by advances in our lead clinical programs, which we expect to result in multiple important data catalysts over the next few quarters. At the same time, our excellent commercial efforts continue to drive meaningful revenue growth across the portfolio, including Crusvita Revenue in North America with our partner, Kewa Caron, and in Latin America as well. I'll spend a few minutes discussing the Osteogenesis Imperfecta and Angelman Syndrome programs before turning the call over to Eric Harris to talk through our commercial update. Beginning with OI, in June we reported exciting data from the Phase II dose-finding portion of the Pivotal Orbit study showing statistically significant increase in levels of serum P1NP, a sensitive marker of bone formation. The bone production response to these patients was extraordinary. This led to a rapid bone building effect following just three months of treatment with citruzumab, resulting in nearly 10% increase in lumbar bone mineral density. At baseline, these patients had very limited bone mineral density with an average Z-score in the 20-minute cohort of minus 2.12, which means the bone mineral density was two standard deviations below the mean of normal patients for their age. After three months on therapy, the mean Z-score increased by plus 0.65 points, resolving nearly one-third of the deficit from normal in a relatively short period of time. As we've said before, patients are showing meaningful improvements in bone health, and we are highly encouraged with how they're doing. Improved bone health refers to the instance of fractures, bone pain, and relative global health and activity of the patients. In the ongoing phase two, we continue to collect data to compare fracture frequency during the conduct of the study to show the impact of citruzumab and increase the bone marrow density on fracture rates. We expect to share this data at an analyst day in mid-October around the time of ASBMR, the major bone-focused meeting. In July, we announced that we initiated dosing patients in two phase three studies evaluating citruzumab in two different age groups. The Phase III portion of the Pivotal Orbit study is evaluating the effect of cetuzumab compared to placebo on analyzed clinical fracture rate in patients 5 to 25 years old. The newly initiated Phase III COSMET study is an active control study evaluating cetuzumab compared to IV bisphosphonate therapy on analyzed total fracture rate in patients age 2 to 5 years old. Enrollment in both of these studies is going well so far in part because the Phase II data has generated a lot of excitement for the potential of sutruzumab for both the clinical sites and from the patient community. Moving to our Angelman program, in May we announced we received FDA agreement to expand the ongoing global Phase I-II trial of GTX1022 patients with Angelman syndrome in the U.S. The protocol amendment enabled us to harmonize the dosing ranges used between the U.S., and ex-US cohorts of the study. Since then, we've been working to activate US sites that have been on hold for a couple years. We continue actively enrolling the expansion cohorts globally. Enrollment in expansion cohorts is going well, particularly over the last couple months. As of today, we've enrolled more than 20 patients. While we're on track to have eight patients with a full six-month data by the end of the year, We've noted that waiting just a few more months will enable us to report a more substantial update on well more than 20 patients with six months of clinical data, plus safety data, and all enrolled. Though the trial is going well, it seems more prudent to wait for this larger set of expansion core data, which we expect to have in the middle of the first half of 2024, based on enrolled patients to date. We will also be providing the age-run program updated analyst data event in mid-October. This is an opportunity for us to provide more context for the clinical meanfulness of the changes that have been observed in the study. Now, I'll turn the call over to Eric Harris to provide an update on our commercial efforts for the first quarter.