Financial Performance - Ultragenyx Pharmaceutical Inc. announced preliminary unaudited total revenue for fiscal year 2025, with specific revenue figures for Crysvita® and Dojolvi® to be disclosed in February 2026[6] - Full year results for fiscal year 2025 will be officially reported in February 2026, providing further insights into financial performance[6] Liquidity Position - The company reported cash and investments at the end of fiscal year 2025, indicating a strong liquidity position[6]

Core Insights - Ultragenyx Pharmaceutical Inc. reported preliminary total revenue for 2025 of $672 million to $674 million, exceeding the top end of guidance and representing approximately 20% growth compared to 2024 [1][3] - The company anticipates significant catalysts in 2026, including two potential gene therapy approvals and pivotal Phase 3 data for GTX-102 in Angelman syndrome [1][2] Financial Update - Preliminary revenue for 2025 is estimated at $672 million to $674 million, with Crysvita revenue projected at $480 million to $482 million, reflecting approximately 17% growth from 2024 [3] - Dojolvi revenue for 2025 is estimated at $95 million to $97 million, indicating approximately 9% growth compared to 2024 [3] - As of December 31, 2025, the company reported cash and investments of approximately $735 million [4] Clinical and Regulatory Catalysts - The company plans to present data from the Phase 3 Orbit and Cosmic studies of UX143 (setrusumab) at the J.P. Morgan Healthcare Conference on January 12, 2026 [7] - The rolling submission for DTX401 (pariglasgene brecaparvovec) for glycogen storage disease type Ia was completed in December 2025, with a PDUFA date anticipated in Q3 2026 [7] - UX111 (rebisufligene etisparvovec) for Sanfilippo syndrome type A is on track for BLA resubmission in early 2026 [7] - GTX-102 (apazunersen) data from the Phase 3 Aspire study is expected in the second half of 2026 [7] - Enrollment for UX701 (rivunatpagene miziparvovec) in the pivotal Cyprus2+ study is complete, with data expected in the first half of 2026 [7] Company Overview - Ultragenyx is focused on developing and commercializing therapies for serious rare and ultra-rare genetic diseases, with a diverse portfolio aimed at addressing high unmet medical needs [9] - The management team has extensive experience in the development and commercialization of rare disease therapeutics, emphasizing efficient drug development [10]

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the 12 Stocks that Will Bounce Back According to Wall Street Analysts. On January 6, H.C. Wainwright reduced its price target on Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) from $80 to $60 but kept its Buy rating on the stock. The reduced price target still represents a significant upside from the current price. This update comes after the company announced that its two pivotal trials for setrusumab in osteogenesis imperfecta (OI), Cosmic and Orbit, ...

NOVATO, Calif., Jan. 07, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, January 12, 2026, at 10:30 AM PT. The live and archived webcast of the presentation will ...

NEW YORK, Jan. 06, 2026 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) (NASDAQ: RARE). Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980. The investigation concerns whether Ultragenyx and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.  [Click here for information about joining the class acti ...

FDA Approvals & Rejections - ARS Pharma's neffy, a needle-free epinephrine nasal spray for emergency treatment of Type I allergic reactions, received approval in China, with commercial availability expected in spring 2026. The product generated $31.3 million in U.S. revenue in Q3 2025 [3][4]. - Vanda Pharmaceuticals' NEREUS, an oral NK-1 receptor antagonist for preventing motion-induced vomiting, received FDA approval, marking the first new treatment for motion sickness in over 40 years. The drug demonstrated a meaningful reduction in vomiting in clinical trials [5][6]. Clinical Trials - Breakthroughs & Setbacks - InflaRx's analyses from a halted Phase 3 trial of Vilobelimab in pyoderma gangrenosum indicated potential efficacy signals with longer treatment duration, prompting plans to discuss alternative endpoints with the FDA [15][17]. - SELLAS reported that survival in its Phase 3 REGAL trial for Galinpepimut-S (GPS) in acute myeloid leukaemia is extending longer than anticipated, potentially increasing the likelihood of a positive outcome [18][19]. - Ultragenyx announced that its Phase 3 studies for Setrusumab in Osteogenesis Imperfecta failed to meet primary endpoints, leading to a decline in investor confidence [20][21]. - Genmab decided to discontinue clinical development of Acasunlimab to focus on higher-priority programs, with no impact expected on its full-year 2025 financial guidance [22][23]. Corporate Actions - FONAR Corporation agreed to be taken private by a CEO-led acquisition group for $19.00 per share, valuing the transaction at a significant premium. The deal is expected to close in Q3 2026, subject to shareholder approval [12][13][14].

Core Insights - Ultragenyx Pharmaceutical's shares increased by 15.5% following the completion of the rolling submission of a biologics license application (BLA) to the FDA for its AAV gene therapy, DTX401, aimed at treating glycogen storage disease type Ia (GSDIa) [1][7] Company Overview - GSDIa is a rare, life-threatening metabolic disorder affecting approximately 6,000 individuals in commercially accessible regions, with no approved pharmacologic treatments available [2] - If approved, DTX401 will be the first therapy targeting the underlying cause of GSDIa [2] Clinical Data - The BLA submission is supported by robust clinical data, including results from the phase III GlucoGene study, which demonstrated significant reductions in daily cornstarch intake and improved blood glucose control while maintaining low hypoglycemia rates [3][9] - After 96 weeks of treatment with DTX401, patients showed greater reductions in daily cornstarch intake and enhanced euglycemia [8] Market Performance - Over the past six months, Ultragenyx's shares have declined by 37.2%, contrasting with a 25.2% rise in the industry [4] Development Timeline - The rolling submission of the BLA for DTX401 began in August 2025, with the completion of the chemistry, manufacturing, and controls module marking a significant milestone [5][9]

Company Overview - Ultragenyx shares increased by 15.5% to $22.78, following a significant trading volume, despite a prior 40.9% loss over the last four weeks [1] - The stock's rally is attributed to optimism regarding the completion of the rolling submission of a biologics license application (BLA) to the FDA for its AAV gene therapy, DTX401, aimed at treating glycogen storage disease type Ia (GSDIa) [2] Financial Performance - The company is projected to report a quarterly loss of $1.32 per share, reflecting a year-over-year increase of 5%, with expected revenues of $186.9 million, marking a 13.4% rise from the previous year [3] - The consensus EPS estimate for Ultragenyx has remained stable over the past 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [4] Industry Context - Ultragenyx is categorized within the Zacks Medical - Biomedical and Genetics industry, where another company, Insmed, experienced a 1.3% decline to $174.09 and has seen a -16.6% return over the past month [5] - Insmed's consensus EPS estimate has increased by 4.4% over the past month to -$1.33, which is a 0.8% decrease from the previous year's report [6]

Upgrades - Argus upgraded BHP Group (BHP) to Buy from Hold with a price target of $68, citing firming prices for iron ore, copper, and coal as the global economy improves [1] Downgrades - Jefferies downgraded Mereo BioPharma (MREO) to Hold from Buy with a price target of $0.50, down from $7, due to Setrusumab missing primary endpoints in trials [2] - RBC Capital downgraded DigitalBridge (DBRG) to Sector Perform from Outperform with a price target of $16, down from $23, following SoftBank's acquisition announcement at $16 per share [3] Initiations - Stephens initiated coverage of Commvault (CVLT) with an Overweight rating and a $162 price target, highlighting its evolution in data protection and transition to Software-as-a-Service [4] - Stephens initiated coverage of Rubrik (RBRK) with an Overweight rating and a $105 price target, noting its attractive growth outlook in data protection and data security [4] - Stephens initiated coverage of Varonis (VRNS) with an Equal Weight rating and a $40 price target, emphasizing its growth potential in data security as a Software-as-a-Service platform [4] - Freedom Capital initiated coverage of Ero Copper (ERO) with a Buy rating and a $32 price target, citing its high-grade, low-cost asset portfolio in Brazil [4] - H.C. Wainwright initiated coverage of Terra Innovatum (NKLR) with a Buy rating and a $25 price target, believing its use of off-the-shelf components will reduce regulatory barriers [4]

Core Viewpoint - Ultragenyx Pharmaceutical Inc. experienced a significant rebound of 15.52% to close at $22.78 after a steep decline of 46% due to disappointing clinical trial results for its brittle bone disease treatment [1][3]. Group 1: Clinical Trial Results - Two phase 3 clinical trials for setrusumab failed to achieve statistical significance in reducing the annual clinical fracture rate compared to placebo and bisphosphonates [2]. - Despite the primary endpoint failures, both studies achieved secondary endpoints showing improvements in bone mineral density (BMD) against comparators [3]. Group 2: Company Response and Future Plans - The President and CEO of Ultragenyx expressed surprise and disappointment at the trial results, highlighting the lack of approved treatment options for patients with Osteogenesis Imperfecta (OI) [4]. - The company plans to conduct additional analyses on the data from both studies, focusing on other bone health and clinical endpoints beyond fractures to determine next steps for the program [5]. - Ultragenyx continues to build commercial revenue from four approved products and is preparing for a potentially transformational year with two near-term gene therapy launches and a pivotal Phase 3 readout in Angelman syndrome [5].