INVESTOR ALERT: Pomerantz Law Firm Reminds Investors with Losses on their Investment in Ultragenyx Pharmaceutical Inc. of Class Action Lawsuit and Upcoming Deadlines - RARE [Accessibility Statement] Skip NavigationNEW YORK, Feb. 12, 2026 /PRNewswire/ -- Pomerantz LLP announces that a class action lawsuit has been filed against Ultragenyx Pharmaceutical Inc. ("Ultragenyx" or the "Company") (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at [[email protected]] or 646-581-9980, (or 888.4- ...

分组1 - Ultragenyx reported a quarterly loss of $1.29 per share, which was worse than the Zacks Consensus Estimate of a loss of $1.20, but an improvement from a loss of $1.39 per share a year ago, resulting in an earnings surprise of -7.32% [1] - The company generated revenues of $207 million for the quarter ended December 2025, exceeding the Zacks Consensus Estimate by 2.01%, and showing an increase from $164.88 million in the same quarter last year [2] - Over the last four quarters, Ultragenyx has surpassed consensus revenue estimates two times, but has only exceeded consensus EPS estimates once [2] 分组2 - The stock's immediate price movement will largely depend on management's commentary during the earnings call, with Ultragenyx shares having increased by approximately 3.8% year-to-date, compared to a 1.4% gain in the S&P 500 [3] - The current consensus EPS estimate for the upcoming quarter is -$1.42 on revenues of $166.49 million, and for the current fiscal year, it is -$4.20 on revenues of $777.45 million [7] - The Medical - Biomedical and Genetics industry, to which Ultragenyx belongs, is currently ranked in the top 36% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8]

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]

Financial Data and Key Metrics Changes - For 2025, the company reported total revenue of $673 million, representing a 20% growth over 2024, exceeding the upper end of guidance [11] - The net loss for the year was $575 million, or $5.83 per share, with cash, cash equivalents, and marketable securities totaling $738 million as of December 31 [12] - Guidance for 2026 expects total revenue 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, while Dojolvi generated $96 million, representing a 9% growth [11] - Evkeeza saw significant growth of 84% over 2024, contributing $59 million, and Mepsevii contributed $37 million [11] - The company anticipates Crysvita revenue in 2026 to be between $500 million and $520 million, with Dojolvi expected to generate between $100 million and $110 million [13] Market Data and Key Metrics Changes - The commercial business delivered a significant 20% year-over-year growth in 2025, with treatments now available in over 35 countries [8] - In Latin America, Crysvita's business is anchored in Brazil and Argentina, with solid reimbursement growth in Mexico and Colombia [17] - Dojolvi continues to grow steadily in the U.S. and EMEA, with recent regulatory wins in Kuwait and the U.K. [18] Company Strategy and Development Direction - The company is focused on achieving profitability by 2027 through a strategic restructuring plan that includes a 10% workforce reduction and realignment of resources towards high-impact opportunities [9][10] - The company aims to launch three additional treatments over the next two years while continuing to grow its existing commercial products [10][24] - The strategic restructuring will allow the company to maintain a diversified pipeline, not solely focused on gene therapy [54] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming year, highlighting key inflection points with potential approvals in MPS IIIA and glycogen storage disease type Ia, as well as pivotal data readouts in Angelman syndrome [4][20] - The management acknowledged the challenges posed by the FDA's incomplete response letter regarding UX111 but emphasized their commitment to providing the necessary documentation for resubmission [7][52] - The company remains optimistic about its ability to deliver meaningful growth and reach profitability, supported by a strong commercial infrastructure and ongoing product launches [20][42] Other Important Information - The company has implemented a strategic restructuring plan to reduce operating expenses and align resources with high-impact opportunities, aiming for profitability in 2027 [9][10] - The company expects R&D and SG&A expenses to decrease significantly in 2027, driven by the completion of clinical studies and reduced early-stage research efforts [15] Q&A Session Summary Question: Can you explain the choice of cognition as the primary endpoint for the Angelman study? - The decision was based on the fundamental nature of cognition and its interrelation with communication, with a focus on a broader assessment through the Multidomain Responder Index [27][28] Question: What is the baseline profile of patients in the Angelman study? - The baseline data from the phase 2 trial is reflective of the phase 3 program, and the company is comfortable with the comparability of the cohorts [33] Question: How does the company plan to achieve profitability in 2027? - The pathway to profitability includes continued growth from existing products, cost reductions, and contributions from upcoming launches [38][40] Question: What is the status of the DTX-301 program? - The DTX-301 program is ongoing, with data expected to be released this year [49] Question: Can you provide an update on the UX111 program and the IRL received? - The company is preparing to provide comprehensive documentation to address the IRL issues and is committed to resubmitting the BLA [52][53]

Financial Data and Key Metrics Changes - For 2025, the company reported total revenue of $673 million, representing a 20% growth over 2024, exceeding the upper end of guidance [10] - The net loss for the year was $575 million, or $5.83 per share, with cash, cash equivalents, and marketable securities totaling $738 million as of December 31 [11] Business Line Data and Key Metrics Changes - Crysvita contributed $481 million, with a 17% growth over 2024, exceeding guidance [10] - Dojolvi generated $96 million, reflecting a 9% growth over 2024 [10] - Evkeeza saw significant growth, contributing $59 million, representing an 84% increase over 2024 [10] - Mepsevii contributed $37 million, continuing to treat patients in an ultra-rare indication [10] 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 [6] - The company anticipates total revenue in 2026 to be between $730 million and $760 million, representing an 8%-13% growth over 2025 [11][12] Company Strategy and Development Direction - The company is focused on achieving profitability by 2027 through a strategic restructuring plan that includes a 10% workforce reduction and realignment of resources towards high-impact opportunities [7][9] - The company aims to launch three additional treatments over the next two years while continuing to grow its existing commercial products [9][22] Management's Comments on Operating Environment and Future Outlook - Management highlighted 2026 as a significant year with potential approvals in key programs and pivotal data readouts [3] - The company is addressing the FDA's incomplete response letter regarding UX111 and is working to provide additional documentation for resubmission [5][20] - Management expressed confidence in the ability to deliver the next phase of growth required to reach profitability [19] Other Important Information - The company is implementing a strategic restructuring plan to reduce operating expenses while advancing its product pipeline [7][9] - The company expects combined R&D and SG&A expenses to be flat to down low single digits in 2026 compared to 2025 [12] Q&A Session Summary Question: Regarding the primary endpoint for the phase 3 Angelman study - The decision to use cognition over expressive communication was based on its fundamental importance and the complexities of communication development [24][25] Question: Can you provide insights on the patient baseline profile for the phase 3 study? - The baseline data from the phase 3 program is comparable to the expansion trial, providing confidence in the study's design [29][30] Question: How does the company plan to achieve profitability in 2027? - The pathway to profitability includes continued growth from current products and cost reductions, with expectations for combined R&D and SG&A expenses to decrease significantly by 2027 [35][36] Question: Can you clarify the timeline for the IRL resubmission? - The resubmission timeline is similar to the original submission, with a PDUFA date expected approximately six months after resubmission [68][69] Question: What is the expected launch trajectory for DTX-401? - The launch is expected to develop steadily, with strong demand anticipated, but not an immediate steep uptake [72]

Core Viewpoint - A class action securities lawsuit has been filed against Ultragenyx Pharmaceutical Inc. for alleged securities fraud affecting investors between August 3, 2023, and December 26, 2025 [1] Group 1: Lawsuit Details - The lawsuit claims that Ultragenyx made misleading statements regarding the efficacy of setrusumab, particularly that while it increases bone density, it does not correlate with a decrease in annualized fracture rates [2] - On December 29, 2025, Ultragenyx announced that its Phase III Orbit and Cosmic Studies did not achieve statistical significance in reducing annualized clinical fracture rates compared to placebo or bisphosphonates [2] - Following this announcement, Ultragenyx's stock price plummeted from $34.19 per share on December 26, 2025, to $19.72 per share on December 29, 2025, marking a decline of approximately 42.32% in one day [2] Group 2: Next Steps for Investors - Investors who suffered losses during the specified timeframe have until April 6, 2026, to request appointment as lead plaintiff, although participation in any recovery does not require serving as a lead plaintiff [3] - Class members may be entitled to compensation without any out-of-pocket costs or fees [3] Group 3: Firm Background - Levi & Korsinsky has a history of securing hundreds of millions of dollars for shareholders and has been recognized as one of the top securities litigation firms in the United States for seven consecutive years [4]

Core Insights - Ultragenyx Pharmaceutical Inc. reported total revenue of $673 million for the year ended December 31, 2025, representing a 20% increase compared to the previous year [5][4] - The company has initiated a strategic restructuring plan aimed at reducing operating expenses and headcount, with a goal of achieving profitability by 2027 [9][10] - Key catalysts for 2026 include two potential product approvals and pivotal Phase 3 data from the GTX-102 study for Angelman syndrome [1][11] Financial Performance - In Q4 2025, total revenue was $207 million, a 25% increase from Q4 2024 [4][6] - Crysvita revenue for 2025 was $481 million, up 17% year-over-year, with significant growth in Latin America and Türkiye [5][6] - Dojolvi revenue for 2025 reached $96 million, reflecting a 9% increase compared to the prior year [5][6] Operating Expenses - Total operating expenses for Q4 2025 were $321 million, and for the full year, they amounted to $1.2 billion [7][6] - The company incurred a net loss of $129 million in Q4 2025, slightly improved from a net loss of $133 million in Q4 2024 [8][6] - For the year, the net loss was $575 million, compared to a net loss of $569 million in 2024 [8][6] Strategic Restructuring - The strategic restructuring plan includes a 10% workforce reduction, impacting approximately 130 employees [10] - Revenue guidance for 2026 is projected to be between $730 million and $760 million, indicating an 8% to 13% increase compared to 2025 [10] - The plan aims to align resources with high-impact opportunities and reduce costs to support future growth [9][10] Clinical and Regulatory Developments - The company expects pivotal data from the GTX-102 Phase 3 Aspire study in the second half of 2026 [11] - Anticipated regulatory actions include a PDUFA date for DTX401 in Q3 2026 and ongoing developments for UX111 and UX701 [15][11]

Core Viewpoint - A securities fraud class action lawsuit has been filed against Ultragenyx Pharmaceutical Inc. (RARE) for alleged material misstatements and omissions regarding its drug setrusumab, affecting investors who purchased stock between August 3, 2023, and December 26, 2025 [1] 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., Case No. 3:26-cv-01097 (N.D. Cal.) [1] - Investors have until April 6, 2026, to file for lead plaintiff status [1] Allegations - The complaint alleges that Ultragenyx misrepresented the reliability of information regarding the effects of setrusumab on patients with Osteogenesis Imperfecta, downplaying the risks associated with the Phase III Orbit study [1] - 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) [1] - The lawsuit asserts that the positive statements made by Ultragenyx about its business and prospects were materially misleading and lacked a reasonable basis [1] Investor Actions - Investors are advised to retain counsel or contact Kessler Topaz Meltzer & Check, LLP for a free case evaluation [1] - The process for becoming a lead plaintiff is outlined, emphasizing that the lead plaintiff represents the interests of all class members [1]

Core Viewpoint - A class action lawsuit has been initiated against Ultragenyx Pharmaceutical, Inc. for allegedly misleading investors regarding the efficacy of its drug setrusumab during the Class Period from August 3, 2023, to December 26, 2025 [1]. Group 1: Legal Action - Investors have until April 6, 2026, to file a lead plaintiff motion in the class action lawsuit [1]. - The Portnoy Law Firm is encouraging affected investors to contact them for legal rights discussions and case evaluations [2]. Group 2: Allegations Against Ultragenyx - The complaint claims that Ultragenyx misrepresented the reliability of information regarding setrusumab's effects on Osteogenesis Imperfecta patients and downplayed the risks of failure in the Phase III Orbit study [3]. - It is alleged that Ultragenyx's optimism regarding the Phase III Orbit study was unfounded, as the results were based on Phase II data without a placebo control group, potentially leading to misleading conclusions about the drug's effectiveness [3]. - The lawsuit asserts that the positive statements made by Ultragenyx about its business and prospects were materially misleading and lacked a reasonable basis [3].

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 ...