UNITED STATES SECURITIES AND EXCHANGE COMMISSION For the transition period from to . 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. Commission File No. 001-36276 ULTRAGENYX PHARMACEUTICAL INC. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorp ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q1 2023 Earnings Conference Call May 4, 2023 5:00 PM ET Company Participants 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 Conference Call Participa ...

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) (State or other jurisdiction of incor ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q4 2022 Earnings Conference Call February 16, 2023 5:00 PM ET Company Participants Joshua Higa - Head of Investor Relations Emil Kakkis - Founder, President and Chief Executive Officer Erik Harris - Executive Vice President and Chief Commercial Officer Camille Bedrosian - Executive Vice President and Chief Medical Officer Aaron Olson - SVP, Corporate Strategy and Finance Ted Hazinga - Chief Accounting Officer Conference Call Participants Gena Wang - Barclays Chri ...

Financial Performance - For the year ended December 31, 2022, the total revenues increased to $363.3 million, compared to $351.4 million for the same period in 2021, representing a growth of approximately 3%[388] - The net losses for the years ended December 31, 2022 and 2021 were $707.4 million and $454.0 million, respectively, indicating an increase in net losses of approximately 56% year-over-year[387] - For the year ended December 31, 2022, total revenues increased by $11.9 million, or 3%, to $363.3 million compared to $351.4 million in 2021[422] - Interest income increased by $9.1 million to $11.1 million for the year ended December 31, 2022, representing a 474% increase compared to 2021[433] - The provision for income taxes increased by $4.7 million to $5.7 million, a 446% increase due to changes in tax law and significant taxable income from royalty sales[439] Cash and Investments - As of December 31, 2022, the company had $896.7 million in available cash, cash equivalents, and marketable debt securities[389] - Cash used in operating activities was $380.5 million for 2022, reflecting a net loss of $707.4 million[445] - Cash used in investing activities totaled $291.7 million, primarily for property, plant, and equipment purchases of $116.1 million and the acquisition of GeneTx for $75.0 million[447] - Cash provided by financing activities was $501.2 million, mainly from $491.0 million in net proceeds from the sale of future royalties[449] - The company has not experienced a loss of principal on any of its investments as of December 31, 2022[457] Revenue Sources - The increase in total revenues was driven by higher collaboration revenue from Crysvita and an increase in revenue from approved products, partially offset by a decrease in collaboration and license revenue from the Daiichi Sankyo arrangement[388] - Crysvita collaboration revenue in profit-share territory increased by $43.8 million, or 26%, to $215.0 million in 2022, driven by increased demand due to a higher number of patients on therapy[422] - Total product sales rose by $41.9 million, or 54%, to $118.9 million, primarily due to increased demand for Crysvita and the commercial launch of Dojolvi in the U.S.[424] - The increase in Crysvita non-cash collaboration royalty revenue was $3.7 million, or 21%, attributed to launch progress in European countries[425] - The collaboration and license revenue from Daiichi Sankyo decreased by $77.3 million, or 91%, due to the completion of technology transfer[423] Expenses - Research and development expenses increased by $208.6 million, or 42%, to $705.8 million, attributed to higher costs in clinical trials and personnel allocations[429] - Selling, general and administrative expenses decreased by $58.2 million, or 26%, to $220.0 million, mainly due to increased personnel costs and commercialization expenses[431] - Cost of sales increased by $12.3 million, or 77%, to $28.3 million, reflecting higher demand for approved products and amortization of intangible assets[426] Strategic Initiatives - The company’s strategy focuses on time- and cost-efficient drug development to deliver safe and effective therapies to patients urgently[384] - The company is constructing its own GMP gene therapy manufacturing plant to support product candidates[456] - Future milestone payments based on product development success are uncertain and described in the financial statements[455] Risks and Future Outlook - The company anticipates continued annual losses as it develops and seeks regulatory approvals for product candidates[451] - Future funding requirements will depend on various factors, including the success of clinical studies and the ability to raise additional capital[452] - The impact of the COVID-19 pandemic on business operations and results is a significant risk factor for the company[456] - The company faces foreign exchange risk due to transactions in currencies other than U.S. dollars, which may negatively affect revenue and operating income[458] Royalty Agreements - In December 2019, the company entered into a Royalty Purchase Agreement with RPI, receiving $320.0 million for future royalty payments on Crysvita sales, with a capped amount of $608.0 million[406] - In July 2022, the company entered into a Royalty Purchase Agreement with OMERS, receiving $500.0 million for 30% of future royalty payments from Crysvita sales, with a cap of $725.0 million[407] - The effective annual interest rate for the liabilities related to the Royalty Purchase Agreements was approximately 9.3% for RPI and 8.4% for OMERS as of December 31, 2022[409] Tax and Valuation - The company recorded a deferred tax asset of $900.7 million, fully offset by a valuation allowance due to uncertainties in generating future taxable income[420] - A hypothetical 100 basis point change in interest rates would not have had a material impact on the fair market value of the company's cash equivalents and marketable debt securities[457] - The fair value of equity investments decreased by $19.3 million in 2022, a 54% improvement from a $42.1 million decrease in 2021[434][435] Other Financial Information - As of December 31, 2022, total unrecognized stock-based compensation costs were $229.4 million, expected to be recognized over a weighted-average period of 2.28 years[416] - Non-cash interest expense on liabilities for sales of future royalties rose by $13.6 million to $43.0 million, a 46% increase year-over-year[437]

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 | --- | --- | --- | --- | --- | --- | --- | |-------- ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q3 2022 Earnings Conference Call November 2, 2022 5:00 PM ET Company Participants Joshua Higa - Head of Investor Relations Emil Kakkis - Founder, President and Chief Executive Officer Erik Harris - Executive Vice President and Chief Commercial Officer Mardi Dier - Executive Vice President and Chief Financial Officer Camille Bedrosian - Executive Vice President and Chief Medical Officer Conference Call Participants Gena Wang - Barclays Joori Park - SVB Securities ...

Corporate Deck July 2022 Legal Warning 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 expe ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q2 2022 Earnings Conference Call July 28, 2022 5:00 PM ET Company Participants Joshua Higa - Executive Director & Head, Investor Relations Emil Kakkis - Chief Executive Officer & President Erik Harris - Chief Commercial Officer Mardi Dier - Chief Financial Officer Camille Bedrosian - Chief Medical Officer Conference Call Participants Gena Wang - Barclays Joel Beatty - Baird Dae Gon Ha - Stifel Tazeen Ahmad - Bank of America Cory Kasimov - JPMorgan Yaron Werber - ...

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, 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 ju ...