Ultragenyx Pharmaceutical Inc.

Good afternoon, and welcome to the Ultragenyx Third Quarter 2024 Financial Results Conference Call. At this time, all participants are in the 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 the call to Joshua Higa, Vice President of Investor Relations.

Thank you. We've 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 Krambes, 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 file. I'll now turn the call over to Emil.

Thanks, Josh, and good afternoon, everyone. This is a great time for Altigenics as we successfully have completed development work across our late-stage programs and now are reaching the submission stage. If we achieve our approval from regulatory authorities, we expect to build further revenue momentum on top of our existing growing commercial portfolio. We continue to see substantial year-over-year revenue growth as we expand geographic access and further reach more patients with our medicines. This growth could further accelerate up to three near-term BLA submissions and approvals for our treatment for both Sanfilippo syndrome type A and GSD1A and potentially for osteogenesis imperfecta. These three late-stage clinical programs could be first-ever approved treatments with a potential to dramatically improve the lives of patients and their families. Doing three submissions within the same year or so is a special opportunity we've earned with our investment and development in these last years, and we understand the importance of these efforts for the patient community. This quarter, we have continued to make progress across our clinical portfolio. I'll touch on a couple points and leave the rest for Eric Rombez, our Chief Medical Officer, to discuss in his prepared remarks. On UX111 for Sanfilippo syndrome, as we announced back in June, we reached agreement with the FDA that cerebral spinal fluid heparan sulfate is a reasonable surrogate endpoint that could be used to support the cellular approval for the treatment of Sanfilippo syndrome. And we already have clinical data to support the efficacy of the product as well. We have since participated in a pre-VLA meeting with the agency where we aligned on the details of the VLA. We are on track to file around the end of this year. We greatly appreciate the FDA's flexibility and thoughtful approach to getting this product submitted and potentially available. Last month, UX143 or citrusumab for osteogenesis imperfecta received breakthrough therapy designation from the FDA as a treatment to reduce the risk of fracture associated with OI types 1, 3, or 4 in patients two years of age or older. We're pleased that the FDA has recognized the potential for UX143 to offer substantial clinical benefit for these patients. Before I hand it over to Eric Harris to talk about the commercial business, I wanted to acknowledge the work our teams have done around the world to bring these important medicines to patients. In Latin America, our team has already generated over $110 million in revenue this year, with a bulk of it coming from ,, but also ,, and . Across Europe and the Middle East, we are successfully navigating the country-by-country reimbursement process for FKESA and METS-AVI. At the same time, we are continuing to address unmet need for CRISPR in Turkey and respond to growing demand for named patient access for Dojolvi. In Japan, we launched FKESA in the second quarter of this year in a recently reached agreement with the PMDA that Dojolvi does qualify for a conditional filing package based on the current clinical data. We expect to file the JNDA in mid-2025. These two products are key components in building our rare disease business in Japan. It could ultimately contribute to the company's overall path to profitability. Finally, in the U.S., we continue to support KKC's efforts commercializing Chris Vita, while also maintaining relationships with the healthcare providers treating patients with XLH and TIO. We clearly have a firm foundation to build from as we think about a future launch of UX143 or cituzumab in OI. We're also calling on prescribers at the Centers of Inborn Errors and Metabolism who treat MEPS-Sevi for MPS-7 and Dozovi for LC-FEOD. These are the same prescribers who could treat patients with UX-111 for MPS-3A, DTX-401 for GSD-1A, and our other gene therapies. The commercial leverage we're gaining in bone and inborn error products will be important contributors to our financial progress in the years ahead. And then I'll turn the call over to our Chief Commercial Officer, Eric Harris, to provide an update on the progress across our commercial portfolio.