Core Insights - Ultragenyx is accused of misleading investors regarding the potential success of setrusumab's Phase 3 study in reducing fracture rates [1] Company Summary - The allegations suggest that Ultragenyx may have provided inaccurate information about the efficacy of setrusumab, which could impact investor confidence and stock performance [1]

Core Insights - The upcoming year is a pivotal moment for the company, with two potential product launches and a crucial data readout expected to significantly boost commercial revenue [1] - A strategic restructuring plan is being implemented to lower operating expenses and align resources with high-impact opportunities in the rare disease sector [2] FDA Update - The company received an Incomplete Response Letter (IRL) from the FDA regarding its resubmitted marketing application for UX111 (rebisufligene etisparvovec) gene therapy for Sanfilippo syndrome type A, requesting additional documentation for resubmission [3] Earnings Snapshot - The company reported a fourth-quarter loss of $1.29 per share, missing the consensus estimate of $1.14, while sales increased to $207 million from $165 million year-over-year, surpassing the consensus of $196.97 million [4] - Revenue from Crysvita was $145 million, Dojolvi reached $32 million, and Evkeeza sales were $17 million [4] Strategic Restructuring - A strategic restructuring plan has been initiated, including a 10% workforce reduction affecting approximately 130 employees, aimed at reducing expenses and partially reinvesting to support future growth [5] - The company aims to achieve profitability by 2027 through significant cost discipline and revenue growth from current and new product launches [5] Outlook - The company forecasts fiscal 2026 sales between $730 million and $760 million, below the consensus of $786.93 million, with Crysvita revenue expected to be between $500 million and $520 million and Dojolvi revenue projected at $100 million to $110 million [6] Market Reaction - Following the announcements, Ultragenyx Pharmaceutical shares fell by 10.21% to $21.10 [7]

Core Viewpoint - A class action lawsuit has been filed against Ultragenyx Pharmaceutical Inc. for alleged misleading statements regarding the efficacy of its drug setrusumab, impacting investors who purchased shares between August 3, 2023, and December 26, 2025 [1][5]. Group 1: Lawsuit Details - The lawsuit claims that Ultragenyx made false statements about setrusumab's ability to reduce fracture rates despite increasing bone density, which misled investors [5]. - Investors who purchased shares during the specified period may be eligible for compensation without incurring out-of-pocket costs through a contingency fee arrangement [2]. Group 2: Participation Information - Interested investors can join the class action by visiting the provided link or contacting the law firm directly for more information [3][6]. - A lead plaintiff must be appointed by April 6, 2026, to represent the class in the litigation [1][3]. Group 3: Law Firm Credentials - The Rosen Law Firm has a strong track record in securities class actions, having achieved significant settlements for investors, including over $438 million in 2019 [4]. - The firm has been recognized for its success in securities class action settlements, ranking highly in the industry since 2013 [4].

Core Viewpoint - A class action lawsuit has been filed against Ultragenyx Pharmaceutical Inc. for alleged misleading statements regarding the potential of setrusumab and its study protocols, impacting investors who purchased stock between August 3, 2023, and December 26, 2025 [1][5]. Group 1: Lawsuit Details - The lawsuit claims that Ultragenyx made false and/or misleading statements about setrusumab's ability to increase bone density and its correlation with fracture rates, which were misrepresented to investors [5]. - Investors who purchased common stock during the specified period may be entitled to compensation without any out-of-pocket fees through a contingency fee arrangement [2]. Group 2: Participation Information - Interested investors can join the class action by visiting the provided link or contacting the law firm directly for more information [3][6]. - A lead plaintiff must be appointed by April 6, 2026, to represent the class in the litigation [1][3]. Group 3: Law Firm Credentials - The Rosen Law Firm has a strong track record in securities class actions, having achieved significant settlements, including the largest securities class action settlement against a Chinese company [4]. - The firm has consistently ranked highly in securities class action settlements and has recovered hundreds of millions of dollars for investors [4].

Core Insights - Ultragenyx reported $207 million in revenue for Q4 2025, marking a year-over-year increase of 25.6% [1] - The company's EPS for the same period was -$1.29, an improvement from -$1.39 a year ago, but below the consensus estimate of -$1.20 [1] - The revenue exceeded the Zacks Consensus Estimate of $202.92 million by 2.01%, while the EPS fell short by 7.32% [1] Revenue Breakdown - Dojolvi generated $32 million in revenue, surpassing the average estimate of $30.28 million, with a year-over-year increase of 2.9% [4] - Evkeeza achieved $17 million in revenue, exceeding the estimated $16.37 million, reflecting a significant year-over-year growth of 63.9% [4] - Mepsevii reported $13 million in revenue, above the average estimate of $10.81 million, also showing a year-over-year increase of 63% [4] - Total Crysvita revenue reached $145 million, exceeding the average estimate of $135.25 million [4] - Overall product sales were $102 million, which was below the estimated $114.76 million, but still represented a year-over-year increase of 41.9% [4] - Royalty revenue amounted to $105 million, surpassing the average estimate of $91.57 million, with a year-over-year change of 12.9% [4] Stock Performance - Over the past month, Ultragenyx shares have returned -0.8%, compared to a -0.3% change in the Zacks S&P 500 composite [3] - The stock currently holds a Zacks Rank 3 (Hold), indicating potential performance in line with the broader market in the near term [3]

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]