Financial Data and Key Metrics Changes - Total revenue for 2025 was reported at $673 million, representing a 20% growth over 2024 and exceeding the upper end of guidance [11] - Net loss for the year was $575 million, or $5.83 per share [12] - Cash, cash equivalents, and marketable securities as of December 31 were $738 million [12] - Guidance for 2026 total revenue is expected to be between $730 million and $760 million, reflecting an 8%-13% growth over 2025 [12][13] Business Line Data and Key Metrics Changes - Crysvita contributed $481 million in revenue, with a 17% growth over 2024 [11] - Dojolvi generated $96 million, representing a 9% growth over 2024 [11] - Evkeeza saw significant growth, contributing $59 million, which is an 84% increase over 2024 [11] - Mepsevii contributed $37 million, continuing to treat patients in an ultra-rare indication [11] Market Data and Key Metrics Changes - The commercial business delivered a significant 20% year-over-year growth in 2025, with treatments reaching over 35 countries [8] - In Latin America, Crysvita's business is anchored in Brazil and Argentina, with solid reimbursement growth in Mexico and Colombia [16][17] - Dojolvi has seen continuous growth in EMEA, with regulatory wins in Kuwait and the U.K. [18] Company Strategy and Development Direction - The company aims for profitability by 2027, implementing a strategic restructuring plan to reduce operating expenses and align resources with high-impact opportunities [9][10] - The plan includes a 10% workforce reduction, impacting approximately 130 employees [10] - The company is focused on launching three additional treatments over the next two years while continuing to grow its base business of four commercial products [10][24] Management's Comments on Operating Environment and Future Outlook - Management highlighted 2026 as a significant year with potential approvals in MPS IIIA and glycogen storage disease type Ia, along with pivotal data readout in Angelman syndrome [4] - The management expressed confidence in the ability to deliver the next phase of growth required to reach profitability [20] - The company is addressing the FDA's incomplete response letter regarding UX111 and is working on resubmitting the BLA with additional documentation [7][21] Other Important Information - The company has a strong commercial infrastructure and experienced team to execute complex rare disease therapies globally [20] - The strategic restructuring is expected to enable the company to diversify its pipeline beyond gene therapy [54] Q&A Session Summary Question: What was the decision behind using cognition as the primary endpoint for the Angelman study? - The decision was based on the belief that cognition is fundamental and intertwined with communication, which takes more time to develop [27] Question: Can you provide insights on the patient baseline profile for the Angelman study? - The baseline data from the phase II trial is reflective of the phase III program, and the company is comfortable with the comparability [33] Question: How does the company plan to achieve profitability in 2027? - The pathway to profitability includes continued growth from current products and a reduction in combined R&D and SG&A expenses [40][42] Question: What is the timeline for the resubmission of the BLA in response to the IRL? - The resubmission is expected to take a couple of weeks for the FDA to determine if all required documents are included, with a PDUFA date set approximately six months after resubmission [72] Question: What is the expected launch trajectory for GSDIa? - The market for GSDIa is expected to develop steadily, with strong demand anticipated, but not as urgent as MPS IIIA [76]

Ultragenyx Pharmaceutical (NasdaqGS:RARE) Q4 2025 Earnings call February 12, 2026 05:00 PM ET Company ParticipantsEmil Kakkis - CEOEric Crombez - CMOErik Harris - CCOHoward Horn - CFOJoshua Higa - VP of Investor RelationsLydia Edelman - Biotech Equity Research AssociateMaxwell Skor - Vice President, Biotech Equity ResearchPriyanka Nagpal - Vice President - Change ManagementRick Miller - Vice President, Biotech Equity ResearchTara Ahmad - AVP, Testing Specialist IWill Soghikian - Research AssociateYigal Dov ...

Ultragenyx Pharmaceutical (NasdaqGS:RARE) Q4 2025 Earnings call February 12, 2026 05:00 PM ET Speaker10Good afternoon, and welcome to the Ultragenyx's fourth quarter and full year 2025 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 the call to Joshua Higa, Vice President of Investor Relations.Speaker5Thank you. We've ...

NEW YORK, Feb. 12, 2026 (GLOBE NEWSWIRE) -- Levi & Korsinsky, LLP notifies investors in Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") (NASDAQ: RARE) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of Ultragenyx investors who were adversely affected by alleged securities fraud between August 3, 2023 and December 26, 2025. Follow the link below to get more information and be contacted by a member of our team: https://zlk.com/pslra-1/ultrag ...

2025 total revenue of $673 million,Crysvita® revenue of $481 million and Dojolvi® revenue of $96 million 2026 total revenue from current products expected to be between $730 million to $760 million Initiated a strategic restructuring plan to significantly reduce and focus expenses and headcount, reiterate path to profitability in 2027 2026 catalysts include two potential approvals and expected pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif., Feb. 12, 2026 (G ...

RARE Investor Alert: A Securities Fraud Class Action Lawsuit Has Been Filed Against Ultragenyx Pharmaceutical Inc. (RARE) [Accessibility Statement] Skip NavigationDid you buy RARE common stock between August 3, 2023, and December 26, 2025?Affected Ultragenyx Pharmaceutical Inc. Investor Summary- Investor action: Contact [Kessler Topaz Meltzer & Check, LLP (www.ktmc.com)] for recovery options at no cost to investor- Key Lawsuit Allegations: Material misstatements and/or omissions concerning the company's dru ...

LOS ANGELES, Feb. 12, 2026 (GLOBE NEWSWIRE) -- The Portnoy Law Firm advises Ultragenyx Pharmaceutical, Inc., (“Ultragenyx” or the "Company") (NASDAQ: RARE) investors of a class action on behalf of investors that bought securities between August 3, 2023 and December 26, 2025, inclusive (the “Class Period”). Ultragenyx investors have until April 6, 2026 to file a lead plaintiff motion. Investors are encouraged to contact attorney Lesley F. Portnoy, by phone 844-767-8529 or email: lesley@portnoylaw.com, to dis ...

Corporate Presentation February 2026 Confidential and Proprietary Forward looking statements Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations, estimates, assumptions, and projections regarding our future operating results and financial performance, including our expectations for profitability in 2027, anticipated cost or expense management, including the company's expectations rel ...

Core Viewpoint - A class action lawsuit has been filed against Ultragenyx Pharmaceutical Inc. for securities fraud, alleging that the company misled investors regarding the efficacy of its drug setrusumab in clinical trials [1] Group 1: Lawsuit Details - The lawsuit covers purchasers of Ultragenyx common stock from August 3, 2023, to December 26, 2025, and claims that the company provided misleading information about the expected results of its Phase III Orbit and Cosmic Studies [1] - Defendants allegedly made positive statements about setrusumab's ability to reduce annualized fracture rates in Osteogenesis Imperfecta patients while concealing material adverse facts about the drug's true potential [1] - The lawsuit asserts that these misleading statements led to Ultragenyx securities being purchased at artificially inflated prices, resulting in investor damages when the truth was revealed [1] Group 2: Legal Representation - Investors wishing to join the class action can do so without any out-of-pocket fees through a contingency fee arrangement [1] - The Rosen Law Firm, known for its success in securities class actions, is representing the investors and encourages selecting qualified counsel with a proven track record [1] - The firm has achieved significant settlements in the past, including over $438 million for investors in 2019 alone [1]

Core Viewpoint - A securities fraud class action lawsuit has been filed against Ultragenyx Pharmaceutical Inc. for allegedly making false and misleading statements regarding its drug setrusumab during the class period from August 3, 2023, to December 26, 2025 [2][5]. Summary by Relevant Sections Lawsuit Details - The lawsuit is filed in the United States District Court for the Northern District of California, under the case name Bailey v. Ultragenyx Pharmaceutical Inc., et al, Case No. 3:26-cv-01097 [2]. - Investors have until April 6, 2026, to file for lead plaintiff status [2][5]. Allegations - The complaint alleges that Ultragenyx misrepresented its knowledge about the effects of setrusumab on patients with Osteogenesis Imperfecta and downplayed the risks associated with the Phase III Orbit study [3]. - It is claimed that Ultragenyx's optimism regarding the study results was unfounded, as the Phase II results lacked a placebo control group, raising concerns about the validity of the reported reduction in annualized fracture rate (AFR) [3]. - The lawsuit asserts that the positive statements made by the company regarding its business and prospects were materially misleading [3]. Investor Actions - Investors affected by the alleged fraud are encouraged to contact Kessler Topaz Meltzer & Check, LLP for recovery options at no cost [5][6]. - The lead plaintiff process allows investors to seek representation in the lawsuit, with the deadline set for April 6, 2026 [4][6]. Law Firm Information - Kessler Topaz Meltzer & Check, LLP is a prominent law firm specializing in securities-fraud class actions and has a history of significant recoveries in securities litigation [8].