Corporate Presentation April 29th, 2025 Forward Looking Statements and Disclaimer The presentation contains forward-looking statements. Statements made or presented may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Words such as "believe," "anticipate," "plan," "expect," "intend," "will," "may," "goal," "potential," "should," " ...

Company Overview - Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases [3] - The company has established a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical needs [3] Management and Strategy - The management team of Ultragenyx is experienced in the development and commercialization of rare disease therapeutics [4] - The company's strategy emphasizes time- and cost-efficient drug development to deliver safe and effective therapies to patients urgently [4] Upcoming Events - Howard Horn, the chief financial officer and executive vice president of Ultragenyx, will participate in a fireside chat at Bank of America's 2025 Healthcare Conference on May 13, 2025, at 8:00 a.m. PT [1] - A live and archived webcast of the panel will be available on the company's website [2]

Core Insights - Ultragenyx Pharmaceutical Inc. reported total revenue of $139 million for Q1 2025, reflecting a 28% increase compared to Q1 2024, with Crysvita revenue at $103 million, a 25% increase year-over-year [4][8] - The company reaffirmed its 2025 financial guidance, projecting total revenue between $640 million to $670 million, with Crysvita revenue expected to be between $460 million to $480 million, and Dojolvi revenue between $90 million to $100 million [10][12] - The investigational treatment UX111 for Sanfilippo syndrome is on track for a Biologics License Application (BLA) action date set for August 18, 2025 [14] Financial Performance - Total revenues for Q1 2025 were $139 million, up from $108.8 million in Q1 2024, with significant contributions from Crysvita and Dojolvi [3][4] - Crysvita's product sales in Latin America and Türkiye reached $55 million, marking a 52% increase compared to the previous year [4][5] - Operating expenses totaled $282 million in Q1 2025, with research and development costs at $165.8 million [6][7] Net Loss and Cash Position - The net loss for Q1 2025 was $151 million, or $1.57 per share, an improvement from a net loss of $171 million, or $2.03 per share, in Q1 2024 [8][9] - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $563 million, down from $745 million at the end of 2024 [9][29] Clinical Developments - The company is progressing with multiple clinical trials, including UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome, with key interim analyses expected in mid-2025 [10][11] - The Phase 3 study for DTX401 is anticipated to submit a BLA in mid-2025, following positive results from the GlucoGene study [15][16] Strategic Focus - Ultragenyx aims to manage expenses while prioritizing investments in upcoming commercial launches and advancing multiple Phase 3 programs, which is expected to reduce net cash used in operations in 2025 compared to 2024 [10][12]

NOVATO, Calif., April 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, May 6, 2025, to discuss its financial results and corporate update for the quarter ending March 31, 2025. The live and replayed webcast of the call will be available through the company’s w ...

Core Insights - BridgeBio Pharma reported $36.7 million in net product revenue for Attruby in its first full quarter, with 2,072 unique patient prescriptions written by 756 prescribers as of April 25, 2025 [1][2] - The company has made significant progress in its clinical trials, including the full enrollment of the observational run-in study for hypochondroplasia Phase 2 trial ahead of schedule [1][6] - BridgeBio ended the quarter with $540.6 million in cash and cash equivalents, excluding anticipated regulatory milestone payments of $105 million for ex-U.S. approvals of BEYONTTRA [1][9] Commercial Progress - The first full quarter of Attruby net product revenue was $36.7 million, with 2,072 unique patient prescriptions written by 756 healthcare providers since FDA approval in November 2024 [2][13] - The Chief Commercial Officer highlighted a 42% reduction in all-cause mortality and a 50% decrease in cardiovascular hospitalizations at 30 months for patients treated with Attruby [3] Pipeline Overview - Acoramidis for ATTR-CM is approved in the U.S., EU, Japan, and UK, with the first participant in the ACT-EARLY Phase 3 trial expected to be dosed in 2025 [4] - BBP-418 for LGMD2I/R9 and encaleret for ADH1 have completed enrollment in their Phase 3 studies, with topline results expected in the second half of 2025 [4][6] - Infigratinib for achondroplasia is fully enrolled, with topline results anticipated in early 2026 [4][6] Key Program Updates - Attruby has shown strong clinical efficacy and safety, contributing to its growth in the ATTR-CM treatment paradigm [5][6] - The Phase 2 proof-of-principle study of encaleret in hypoparathyroidism showed 78% of participants achieving normal calcium levels within 5 days, with plans to advance to a registrational study in 2026 [6][7] Financial Updates - Cash and cash equivalents decreased to $540.6 million as of March 31, 2025, primarily due to operating activities and loan repayments [9][10] - Total revenues for the three months ended March 31, 2025, were $116.6 million, down from $211.1 million in the same period the previous year, mainly due to a decrease in license and services revenue [10][11] - The company recorded a net loss attributable to common stockholders of $167.4 million for the quarter, compared to a loss of $35.2 million for the same period in 2024 [20]