Core Insights - Century Lithium Corp. has reported positive results from its ongoing test work on the recovery of rare earth elements (REEs) from primary lithium leach solutions generated from its Angel Island lithium project in Nevada [1] - Initial testing indicates that high REE recoveries can be achieved without impacting lithium recovery in the extraction process [1] Company Summary - Century Lithium Corp. is focused on the extraction of lithium and rare earth elements, highlighting its 100%-owned Angel Island lithium project in Nevada [1] - The company is actively engaged in test work to enhance the recovery processes for both lithium and REEs [1]

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the most promising stocks with huge upside potential. On November 24, Barclays analyst Gena Wang lowered the firm’s price target on Ultragenyx (RARE) to $50 from $81 and maintained an Overweight rating on the shares. The firm lowered its price target following analysis of the setrusumab COSMIC study, but still maintains an over 70% chance of a positive result. Earlier the same month, Ultragenyx Pharmaceutical reported that it made a total revenue of $ ...

Core Insights - Ultragenyx Pharmaceutical reported a third-quarter 2025 loss of $1.81 per share, which was wider than the Zacks Consensus Estimate of a loss of $1.23, and compared to a loss of $1.40 per share in the same quarter last year [1][6] - Total revenues for the quarter were $159.9 million, reflecting a 15% year-over-year increase, but fell short of the Zacks Consensus Estimate of $168 million [1][6] Revenue Breakdown - Crysvita generated total revenues of $111.9 million, up 14% year over year, with contributions of $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe [4] - Mepsevii product revenues declined by 27% year over year to $7 million, while Dojolvi revenues increased by 14% to $24.3 million due to new patient demand [5] - Evkeeza recorded sales of $16.7 million, up 57% as the drug continues to be launched in territories outside the United States [5][6] Operating Expenses - Operating expenses for the quarter were $330.8 million, a 22% increase year over year, driven by higher investments in late-stage pipeline programs and marketing costs [8] - Research and development expenses were $216.2 million (up 27%), selling, general and administrative expenses were $86.6 million (up 8%), and cost of sales was $28 million (up 33%) [8] Financial Guidance - The company expects total revenues in 2025 to be between $640 million and $670 million, representing growth of approximately 14-20% compared to 2024 [10] - Crysvita revenues are projected to be in the range of $460-$480 million (up 12-17% year over year), while Dojolvi revenues are expected between $90 million and $100 million (up 2-14% year over year) [10] Pipeline Updates - Ultragenyx faced a setback with the FDA issuing a complete response letter for its biologics license application for UX111, requesting more information related to manufacturing [12] - The company plans to resubmit the BLA for UX111 early in 2026 [13] - GTX-102 received Breakthrough Therapy designation from the FDA for treating Angelman syndrome, with data expected in the second half of 2026 [14] - The company has begun the rolling submission of a BLA for its investigational gene therapy DTX401, expected to be completed in Q4 2025 [15]

Core Insights - Ultragenyx reported revenue of $159.93 million for the quarter ended September 2025, reflecting a year-over-year increase of 14.7% [1] - The company's EPS was -$1.81, a decline from -$1.40 in the same quarter last year, and fell short of the consensus estimate of -$1.23 by 47.15% [1][3] - The revenue fell short of the Zacks Consensus Estimate of $167.55 million, resulting in a surprise of -4.55% [1] Revenue Breakdown - Dojolvi generated $24.28 million in product sales, below the average estimate of $25.96 million, marking a year-over-year increase of 13.6% [4] - Evkeeza achieved $16.72 million in product sales, exceeding the estimated $13.24 million, representing a significant year-over-year growth of 56.9% [4] - Mepsevii reported $7 million in product sales, which was below the average estimate of $8.82 million, indicating a year-over-year decline of 27.2% [4] - Total Crysvita revenue was $111.94 million, falling short of the average estimate of $119.46 million [4] - Overall product sales reached $94.99 million, surpassing the average estimate of $89.79 million, with a year-over-year increase of 23% [4] - Royalty revenue was $64.94 million, below the estimated $78.87 million, but showed a year-over-year increase of 4.3% [4] Stock Performance - Ultragenyx shares have returned +10.9% over the past month, outperforming the Zacks S&P 500 composite's +2.1% change [3] - The stock currently holds a Zacks Rank 3 (Hold), suggesting it may perform in line with the broader market in the near term [3]

Core Insights - Ultragenyx reported a quarterly loss of $1.81 per share, which was worse than the Zacks Consensus Estimate of a loss of $1.23, marking an earnings surprise of -47.15% [1] - The company generated revenues of $159.93 million for the quarter ended September 2025, missing the Zacks Consensus Estimate by 4.55% [2] - The stock has underperformed, losing about 20.4% since the beginning of the year compared to the S&P 500's gain of 16.5% [3] Financial Performance - The loss per share increased from $1.4 a year ago to $1.81 this quarter [1] - Year-over-year revenue growth was observed, with revenues increasing from $139.49 million to $159.93 million [2] - The current consensus EPS estimate for the upcoming quarter is -$1.17 on revenues of $182.02 million, and for the current fiscal year, it is -$5.26 on revenues of $657.62 million [7] Market Outlook - The Zacks Rank for Ultragenyx is currently 3 (Hold), indicating expected performance in line with the market [6] - The Medical - Biomedical and Genetics industry is ranked in the top 39% of over 250 Zacks industries, suggesting a favorable outlook for stocks within this sector [8] - The trend of earnings estimate revisions is mixed, which could influence future stock performance [6]

Product Portfolio - Ultragenyx Pharmaceutical Inc. has a diverse portfolio of approved therapies and product candidates targeting serious rare and ultra-rare genetic diseases [112]. - Crysvita is the only approved treatment for X-Linked Hypophosphatemia (XLH), with approximately 48,000 patients affected in the developed world [115]. - Mepsevii is approved for Mucopolysaccharidosis VII (MPS VII), affecting an estimated 200 patients in the developed world [118]. - Dojolvi is approved for Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), with approximately 8,000 to 14,000 patients in the developed world [119]. - Evkeeza is approved for Homozygous Familial Hypercholesterolemia (HoFH), with around 3,000 to 5,000 patients in the developed world outside the U.S. [120]. - UX143 for Osteogenesis Imperfecta (OI) is in Phase 3, with final analysis expected around the end of 2025 [127]. - GTX-102 for Angelman Syndrome has enrolled 129 patients in the Phase 3 Aspire study, with data expected in the second half of 2026 [128]. - DTX401 for Glycogen Storage Disease Type Ia (GSDIa) achieved its primary endpoint in the Phase 3 GlucoGene study, showing significant reduction in daily cornstarch intake [131]. - UX111 for MPS IIIA received a Complete Response Letter from the FDA, with plans to resubmit the BLA in early 2026 [130]. Financial Performance - Total revenues increased by 15% to $159.9 million for the three months ended September 30, 2025, compared to $139.5 million in the same period of 2024 [139]. - Product sales increased by 23% to $94.993 million for the three months ended September 30, 2025, compared to $77.251 million in the same period of 2024 [141]. - Crysvita product sales increased by 32% to $47.003 million for the three months ended September 30, 2025, compared to $35.604 million in the same period of 2024 [141]. - The company incurred net losses of $180.4 million for the three months ended September 30, 2025, compared to $133.5 million for the same period in 2024 [137]. - Cash used in operating activities for the nine months ended September 30, 2025, was $366.2 million, reflecting a net loss of $446.4 million [166]. - Cash provided by financing activities for the nine months ended September 30, 2025, was $83.8 million, a decrease from $396.1 million in the same period of 2024 [171]. Research and Development - Research and development expenses rose by 27% to $216.212 million for the three months ended September 30, 2025, compared to $170.109 million in the same period of 2024 [148]. - Total research and development expenses increased by $36.6 million (7%) for the nine months ended September 30, 2025, compared to the same period in 2024, primarily due to increases in gene therapy and biologic programs [149]. - The company expects annual research and development expenses to moderate as product candidates advance through clinical development [150]. Cash and Liquidity - As of September 30, 2025, the company had $447.3 million in available cash, cash equivalents, and marketable debt securities [139]. - Cash, cash equivalents, and marketable debt securities totaled $447.3 million as of September 30, 2025, down from $745.0 million as of December 31, 2024 [178]. - The company expects to satisfy future cash needs through existing capital balances and revenue from commercial products [174]. Future Outlook - The company anticipates continued annual losses in the near term due to ongoing product development and regulatory approvals [172]. - Future funding requirements will depend on various factors, including clinical study costs and regulatory interactions [173]. - A hypothetical 10% change in foreign exchange rates would not have had a material impact on the company's financial statements for the nine months ended September 30, 2025 [179]. Obligations and Expenses - Manufacturing and service contract obligations related to clinical stage pipeline amounted to approximately $125.4 million, with $92.9 million due within one year [176]. - As of September 30, 2025, future minimum lease payments under non-cancellable leases were approximately $40.3 million, with $15.1 million due within one year [175]. - Selling, general and administrative expenses rose by $21.9 million (9%) for the nine months ended September 30, 2025, driven by higher employee compensation and marketing expenses [153]. - Non-cash interest expense on liabilities for sales of future royalties decreased by $5.0 million (10%) for the nine months ended September 30, 2025, due to reduced royalty obligation balances [157]. - The provision for income taxes increased by $1.5 million (94%) for the nine months ended September 30, 2025, primarily due to increased foreign activities [160].

Financial Data and Key Metrics Changes - In Q3 2025, Ultragenyx reported total revenue of $160 million, representing a 15% increase compared to Q3 2024 and an 18% increase for the first nine months of 2025 over the same period in 2024 [14][15] - The net loss for the quarter was $180 million, equating to $1.81 per share [15] - As of September 30, 2025, the company had $447 million in cash, cash equivalents, and securities, bolstered by a recent $400 million financing [16] Business Line Data and Key Metrics Changes - Crysvita generated $112 million in revenue during Q3 2025, with contributions of $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe [15] - Dojolvi contributed $24 million, maintaining steady growth, while Evkeeza and Mepsevii contributed $17 million and $7 million, respectively [15] - The company expects total revenue for 2025 to be between $640 million and $670 million, indicating a growth of 14% to 20% over 2024 [16][17] Market Data and Key Metrics Changes - In Latin America, approximately 875 patients are now on Crysvita therapy, with 50 new start forms generated in Q3 2025 [8] - Dojolvi has seen approximately 700 new start forms since its launch in 2020, leading to around 625 patients on reimbursed therapy, with a split of 65% pediatric and 35% adult patients [9] - Evkeeza has approximately 310 patients across 17 countries in the EMEA region, with 120 new patients added since the beginning of the year [10] Company Strategy and Development Direction - The company is focused on maximizing opportunities from its four commercial products and has two BLA submissions in progress for ultra-rare diseases [4] - Ultragenyx aims to maintain financial discipline while preparing for upcoming product launches and pivotal data readouts [14] - The company is positioned for growth with a strong balance sheet and plans to achieve profitability by 2027 [16][48] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming pivotal data readouts for GTX-102 and UX143, which are expected to be transformative for patients with Angelman syndrome and osteogenesis imperfecta [5][23] - The management team highlighted the importance of maintaining cash burn and prioritizing investments as they approach significant clinical milestones [5][16] - The company anticipates continued double-digit revenue growth from existing products and contributions from new launches [48] Other Important Information - The company received $400 million in non-dilutive capital through the sale of a portion of Crysvita royalties, with payments deferred until January 2028 [4][13] - The management team emphasized the unique value of Crysvita and its potential to deliver significant long-term value [14] Q&A Session Summary Question: Data update on Orbit and Cosmic studies - Management confirmed that data from both studies will be reported together, expected in December or January [27] Question: Open label extension from phase II for OI - Management stated that they have not released new quantitative data but are confident in the phase III expectations, aiming for a 40% to 70% reduction in fractures [31][32] Question: Clarification on UX111 and DTX401 submissions - Management explained that the timing of submissions was adjusted due to the need for additional reports, but both filings remain closely aligned [36] Question: Impact of OMERS transaction on financing needs - Management indicated that the $400 million raised will help address investor concerns regarding financing needs in 2026 and support upcoming product launches [42][48] Question: Length of treatment for Setrusumab - Management expressed confidence that Setrusumab will require chronic treatment to maintain bone health, suggesting bisphosphonates may become obsolete [70][73] Question: Loss of exclusivity for Setrusumab - Management highlighted the importance of orphan designation for exclusivity and mentioned ongoing patent protections that extend beyond 2030 [92]

Financial Data and Key Metrics Changes - In Q3 2025, the company reported total revenue of $160 million, representing a 15% increase compared to Q3 2024 and an 18% increase for the first nine months of 2025 over the same period in 2024 [14][15] - The net loss for the quarter was $180 million, equating to $1.81 per share [15] - Cash, cash equivalents, and securities as of September 30, 2025, totaled $447 million, bolstered by a recent $400 million financing [16] Business Line Data and Key Metrics Changes - Crysvita generated $112 million in Q3 2025, with revenue contributions of $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe [15] - DOJOLVI contributed $24 million, maintaining steady growth, while EVKEEZA and MEPSEVII contributed $17 million and $7 million, respectively [15] - The company expects total revenue for 2025 to be between $640 million and $670 million, indicating a growth of 14%-20% over 2024 [16] Market Data and Key Metrics Changes - In Latin America, approximately 875 patients are now on Crysvita therapy, with 50 new start forms generated in Q3 2025 [8] - In the EMEA region, around 310 patients are receiving EVKEEZA across 17 countries, with 120 new patients added since the beginning of the year [10] Company Strategy and Development Direction - The company is focused on maximizing opportunities from its four commercial products and has two BLA submissions in progress for ultra-rare diseases [4] - A recent $400 million financing through the sale of Crysvita royalties is aimed at strengthening the balance sheet and supporting upcoming pivotal data readouts [4][13] - The company plans to maintain financial discipline while preparing for the launch of new products and managing cash burn [5][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming pivotal data readouts for osteogenesis imperfecta and Angelman syndrome, with expectations for significant commercial opportunities [5][23] - The company reaffirmed its path to profitability by 2027, supported by continued double-digit growth from existing products and potential new launches [16][48] Other Important Information - The company is preparing for the rolling BLA submission for DTX401, expected to be completed next month, and anticipates a BLA resubmission for UX111 in early 2026 [18][21] - Management highlighted the importance of patient support programs in influencing treatment decisions among caregivers and patients [62] Q&A Session Summary Question: Data update on Orbit and Cosmic studies - Management confirmed that data from both studies will be reported together, expected in December or January, with variability in timing due to data cleaning processes [26][27] Question: Open label extension from phase II for OI - Management stated that while no new quantitative data is available, they are confident in the phase II results and expect a fracture reduction of 40%-70% in phase III [30][32] Question: Clarification on UX111 and DTX401 submissions - Management explained that the timing of submissions was adjusted due to the need for additional reports, with DTX401 expected to be filed first [35][36] Question: Impact of OMERS transaction on financing needs - Management indicated that the $400 million raised will help address investor concerns regarding financing needs in 2026, particularly related to priority vouchers [42][48] Question: Commercial opportunity for Setrusumab in OI - Management believes the OI population is larger than XLH and expects similar pricing, indicating a significant commercial opportunity [58] Question: Loss of exclusivity for Setrusumab - Management highlighted orphan designation as a protective factor against loss of exclusivity, with additional patents in place to extend protection [90]

Financial Data and Key Metrics Changes - In Q3 2025, the company reported total revenue of $160 million, representing a 15% increase compared to Q3 2024 and an 18% increase for the first nine months of 2025 over the same period in 2024 [13][14] - The net loss for the quarter was $180 million, equating to $1.81 per share [15] - As of September 30, 2025, the company had $447 million in cash, cash equivalents, and securities, bolstered by a recent $400 million financing [16] Business Line Data and Key Metrics Changes - Crysvita generated $112 million in revenue during Q3 2025, with contributions of $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe [14] - Dojolvi contributed $24 million, maintaining steady growth, while Evkeeza and Mepsevii contributed $17 million and $7 million, respectively [14] - The company expects total revenue for 2025 to be between $640 million and $670 million, indicating a growth of 14% to 20% over 2024 [16] Market Data and Key Metrics Changes - In Latin America, the company added approximately 50 new patients on reimbursed therapy for Crysvita, bringing the total to around 875 patients [8] - In the EMEA region, Evkeeza has approximately 310 patients receiving treatment across 17 countries, with 120 new patients added since the beginning of the year [10] Company Strategy and Development Direction - The company is focused on maximizing opportunities from its four commercial products and has two BLA submissions in progress for ultra-rare diseases [3] - A recent $400 million financing through the sale of a portion of Crysvita royalties is aimed at strengthening the balance sheet and supporting upcoming pivotal data readouts [3][12] - The company plans to maintain financial discipline while preparing for the launch of new products and managing cash burn [4][12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming pivotal data readouts for GTX-102 and UX143, which are expected to be transformative for patients with Angelman syndrome and osteogenesis imperfecta [4][23] - The company reaffirmed its path to profitability by 2027, supported by expected double-digit growth from existing products and new launches [12][48] Other Important Information - The company is preparing for a rolling BLA submission for DTX401, with expectations to complete this filing in December [23] - Management highlighted the importance of patient support programs in ensuring access to treatments, especially in a competitive landscape for Angelman syndrome therapies [64] Q&A Session Summary Question: Data update on Orbit and Cosmic studies - Management confirmed that data from both studies will be reported together, expected in December or January [26][27] Question: Open label extension from phase II for OI - Management stated that while no new quantitative data is available, they are confident in the phase III expectations, anticipating a fracture reduction of 40% to 70% [30][32] Question: Clarification on UX111 and DTX401 submissions - Management explained that the timing of submissions was adjusted due to the need for additional reports, but both filings remain closely timed [36][38] Question: Impact of OMERS transaction on financing needs - Management indicated that the recent financing strengthens the balance sheet and supports the path to profitability in 2027 [44][48] Question: Length of treatment for Setrusumab - Management expressed confidence that Setrusumab will require chronic treatment to maintain bone health, suggesting bisphosphonates may become obsolete [72][74] Question: Loss of exclusivity for Setrusumab - Management highlighted orphan designation and additional IP protections that extend exclusivity beyond 2030 [98][99]

Revenue Performance - Total revenue for Q3 2025 was $160 million, representing a 15% growth compared to Q3 2024[4] - Crysvita revenue in Q3 2025 was $112 million, including $47 million from Latin America and Türkiye[4] - Dojolvi revenue in Q3 2025 was $24 million[4] - Total revenues for Q3 2025 reached $159.933 million, a 14.7% increase from $139.494 million in Q3 2024[30] - Product sales increased to $94.993 million in Q3 2025, up from $77.251 million in Q3 2024, representing a 22.9% growth[30] - Royalty revenue for Q3 2025 was $64.940 million, compared to $62.243 million in Q3 2024, showing a 4.3% increase[30] Financial Guidance - The company reaffirmed 2025 revenue guidance of $640 million to $670 million, with Crysvita revenue expected between $460 million to $480 million[13] Operating Expenses - Operating expenses for Q3 2025 totaled $331 million, including $37 million in non-cash stock-based compensation[5] - Operating expenses totaled $330.823 million in Q3 2025, a 21.8% increase from $271.481 million in Q3 2024[30] - Research and development expenses rose to $216.212 million in Q3 2025, up from $170.109 million in Q3 2024, reflecting a 27.1% increase[30] Net Loss - Net loss for Q3 2025 was $180 million, or $1.81 per share, compared to a net loss of $134 million, or $1.40 per share in Q3 2024[7] - Net loss for Q3 2025 was $180.413 million, compared to a net loss of $133.516 million in Q3 2024, indicating a 35.1% increase in losses[30] - Net loss per share for Q3 2025 was $1.81, compared to $1.40 in Q3 2024[30] Cash and Assets - Cash, cash equivalents, and marketable debt securities were $447 million as of September 30, 2025[8] - Cash, cash equivalents, and marketable debt securities decreased to $447.315 million as of September 30, 2025, down from $745.029 million at the end of 2024[34] - Total assets as of September 30, 2025, were $1.190 billion, a decrease from $1.503 billion at the end of 2024[34] - Total stockholders' equity fell to $9.159 million as of September 30, 2025, down from $255.297 million at the end of 2024[34] Future Developments - UX143 for osteogenesis imperfecta is expected to have phase 3 study readouts by the end of 2025[12] - The company plans to resubmit the BLA for UX111 early in 2026 following constructive discussions with the FDA[16] Balance Sheet Improvement - The company received $400 million from the sale of a portion of its Crysvita royalty interest, enhancing its balance sheet[9]