Ultragenyx Pharmaceutical Inc.

Good afternoon, and welcome to the Ultragenyx fourth quarter and full year 2024 financial results conference call. At this time, all participants are in a listen-only mode. At the end of the prepared remarks, you will have an opportunity to ask questions during the Q&A portion of the call. It is now my pleasure to turn over the call to Joshua Higa, Vice President of Investor Relations. Please go ahead.

Thank you. We have issued a press release detailing our financial results, which you can find on our website at ultragenics.com. Joining me on this call are Emil Kakas, Chief Executive Officer and President, Eric Harris, Chief Commercial Officer, Howard Horn, Chief Financial Officer, and Eric Cromdes, Chief Medical Officer. I'd like to remind everyone that during today's call, we will 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. 2024 was a pivotal year for the company as we advanced six late-stage programs in serious genetic conditions, most without any approved therapies, while also expanding access and growing revenue from our four commercial products worldwide. In the middle of the year, we increased our guidance for total revenue and now confirm that we exceeded the upper end of that range for 2024. After filing our first BLA for Sanfilippo gene therapy earlier than planned, we now expect to have a second BLA for GSD1A gene therapy submitted to the FDA in mid-2025. And if both are approved, we would have six commercial products on the market. Combine that progress with the expected Phase III data on UX143 and osteogenesis imperfecta, and expected full enrollment of our Phase III for GHGX102 for Angelman syndrome, we're set for a strong year of value creation, greater than any year in our company's history. Our international growth in 2024 was particularly impressive. We successfully launched FKISA in Europe, Canada, and Japan, while broadening access to our other commercial therapies in Latin America, Canada, and Turkey. We've also been working within these regions to establish clinical trial sites, prepare drug submissions with regulatory authorities, and bring our therapies into more geographies through named patient programs. We're looking forward to another year of strong global revenue growth in 2025, supported by multiple products in launch mode globally. This progress sets us firmly on a path toward full-year gap profitability in 2027. In January, I discussed in depth our priorities for this coming year, so I will use our time today to focus on our UX111 program for Sanfilippo syndrome, which has the potential to be our next approved product in our first commercial gene therapy program. Last week, we presented important new clinical data at the World Symposium in San Diego. that were also included in our BLA submission last December. We filed for accelerated approval based on the substantial and sustained decrease in levels of heparin sulfate and supraspinal fluid, or CSFHS, following treatment with UX111. CSFHS is what I call a disease-caused biomarker because it's directly responsible for disease pathology and progression. It's not just a random measure associated with disease. It is the disease. These new data show that a sustained reduction in CSFHS exposure is statistically associated with significant continued growth in the Bayley-3 cognitive raw score for the subdomains of cognition, receptive communication, and expressive communication when compared to natural history data, which show a climb during this age period. Importantly, we also saw that older children with more advanced disease at the time of treatment were able to retain clinically meaningful functional abilities including communication, ambulation, and self-feeding following treatment with US 111 when the Bayley score is not an effective measure. As we stated in the release, we know from caregivers, clinicians, and others that stabilizing disease so that a child can retain or even slow down the loss of key skills like walking independently, communicating, and self-feeding has profound impact on their quality of life. The earlier we treat, the better is the long-term outcome, and in the long run, newborn screening will identify patients at birth and potentially enable an optimal treatment outcome. Whether treatment from birth or later in life, these gains or retention of function with UX111 treatment are remarkable compared with the progressive loss of function expected in these patients. These results give me confidence that UX111 will be a successful product once approved with the potential to make a meaningful difference for patients with San Filippo Syndrome Type A and their families. Our US-111 program also serves as a strong example of how we are leading and driving changes for the field. Our progress in San Filippo and the progress of other companies in the MPS field is made possible by FDA's willingness to accept CSFHS as a primary biomarker endpoint. Last February, we joined patient advocates, regulators, academics, and industry representatives in a workshop hosted by the Reagan Udall Foundation to discuss qualifying biomarkers in support of rare disease regulatory pathways. This was an opportunity to take decades of work by academic researchers and clinicians in neuropathic MPS diseases and put together a body of information to provide support for leveraging accelerated approval to change the paradigm for drug development in these diseases. Given my long history working on treatment for MTS diseases, including four of the five currently approved enzyme therapies in the U.S., this was an incredible achievement and opens up the possibility of accelerated development for the broader rare disease community, especially those impacted by metabolic diseases of the brain. We're pleased and thankful to see the FDA's focus on the rare disease over the past year with the advancement of first-ever treatments, some of which were at risk of being shelved entirely. We will continue our advocacy and engagement efforts to advance rare disease regulatory policy. With that, I'll turn the call over to our Chief Commercial Officer, Eric Harris, to provide a more detailed update on the progress across our commercial portfolio.