Unidentified Analyst Thank you. The follow-up is on OI. So are there any specific reasons for preferential suitability of setrusumab on AI versus other anticlotting antibodies like Evenity SHR-1222 or the others? Emil Kakkis No. Well, the only other one is that they are romosozumab, romo, I guess we call it for short. Both target the same class and there are differences though. Remember our program setrusumab is a fully human antibody, which we think is a better choice for a long-term therapeutic because of ...

ultrageny% Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations and projections regarding our future operating results and financial performance, anticipated cost or expense reductions, plans with respect to commercializing our product and product candidates, our translational research program, expectations regarding our manufacturing capabilities, the expected timing of release of ad ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended September 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to . Commission File No. 001-36276 ULTRAGENYX PHARMACEUTICAL INC. (Exact name of registrant as specified in its charter) (State or other jurisdiction of i ...

Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) Q2 2023 Earnings Conference Call August 3, 2023 5:00 PM ET Joshua Higa - Director, IR & Corporate Communications Emil Kakkis - Founder, President, CEO & Director Eric Crombez - Chief Medical Officer & EVP Aaron Olsen - SVP, Corporate Strategy & Finance Erik Harris - EVP & Chief Commercial Officer Operator Thank you. We have issued a press release telling our financial results, which you can find on our website at ultragenyx.com. Joining me on this call are Emil ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to . Commission File No. 001-36276 ULTRAGENYX PHARMACEUTICAL INC. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other ju ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q1 2023 Earnings Conference Call May 4, 2023 5:00 PM ET Joshua Higa - Vice President and Head of Investor Relations Emil Kakkis - Founder, President and Chief Executive Officer Erik Harris - Chief Commercial Officer and Executive Vice President Eric Crombez - Chief Medical Officer and Executive Vice President Aaron Olsen - Senior Vice President of Corporate Strategy and Finance Ted Huizenga - Chief Accounting Officer Gena Wang - Barclays Maury Raycroft - Jefferie ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended March 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to . Commission File No. 001-36276 ULTRAGENYX PHARMACEUTICAL INC. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other j ...

Company Participants Conference Call Participants Operator Joshua Higa 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. Emil Kakkis In 2022, we generated more than $350 million of global revenue in our fifth year as a commercial company. This year, we expect revenue to be in the ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-K ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File No. 001-36276 Ultragenyx Pharmaceutical Inc. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other jurisdiction of (I. ...

400 0 1 Cash used in operations, Gene Therapy Manufacturing Facility (GTMF) Capital Expenses and select non-recurring uses of cash 2 2021: ~$55M GTMF; 2022: ~$90M GTMF, $75M GeneTx Acquisition, $30M Evkeeza License; 2023: ~$20M GTMF 3 Estimated cash, cash equivalents, and available-for-sale investments as of December 31, 2022 (unaudited) 9 Confidential and Proprietary Abnormal bone metabolism leads to increased bone resorption, inadequate bone production | --- | --- | --- | --- | --- | --- | --- | |-------- ...