RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Conditional marketing approval (China), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Overseas New Drugs Urgently Needed in Clinical Settings (China), Priority Review (Australia), Fast Track (United States)

Structure/Sequence

Sequence Code 10038962H

Source: *****

Sequence Code 10038967L

Source: *****

Clinical Trials associated with Burosumab-TWZA

NCT06202027

/ RecruitingNot Applicable

Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients

The objective of this post-marketing surveillance (PMS) study is to assess the safety and effectiveness of CRYSVITA injection 10, 20, and 30mg, equivalent to in routine clinical settings

Start Date11 Aug 2023

Sponsor / Collaborator

Kyowa Kirin Korea Co., Ltd.

NCT05509595

/ CompletedPhase 2IIT

A Phase 2 Study of Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia

Background:
Fibrous dysplasia (FD) is a disorder that affects bone growth. Affected bone tissue is weakened, and people with FD are prone to deformities, fractures, and other problems. People with FD may also have low blood phosphate levels. This can make bones even weaker. Better treatments are needed.
Objective:
To test a study drug (burosumab) in people with FD who have low blood phosphate levels.
Eligibility:
People aged 1 year or older who have FD and low blood phosphate levels.
Design:
Participants will visit the NIH 3 times in 48 weeks. Each visit will last 5 to 7 days.
Participants will self-inject burosumab under the skin in their belly, upper arm, or thigh. They (or a caregiver) will do this at home 1 or 2 times a month. They will be trained in person on how to inject the drug. Home injections will be guided via telehealth.
During NIH visits, participants will have a physical exam with blood and urine tests. They will have x-rays of different parts of their body. They will have a radioactive tracer injected into their vein; then they will have a bone scan. They will have tests to assess their strength, walking, and movement. They will complete questionnaires about their pain, mobility, and fatigue levels.
Adult participants may have bone biopsies. These will be done under anesthesia with sedation. Small samples of FD-affected bone will be removed for study.
Between NIH visits, participants will go to a local laboratory for blood and urine tests.
Child participants will have an additional follow-up visit 2 weeks after the final NIH visit.

Start Date07 Dec 2022

Sponsor / Collaborator

National Institute of Dental & Craniofacial Research

NCT05357573

/ CompletedPhase 4

An Open-Label, Multi Center, Single-Cohort, Post-Marketing Phase 4 Study to Evaluate the Efficacy, Pharmacodynamics, and Safety of the Anti-FGF23 Antibody, KRN23, in Adult Chinese Patients With Tumor-Induced Osteomalacia (TIO)

The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in adult Chinese patients with TIO

Start Date07 Sep 2022

Sponsor / Collaborator

Kyowa Kirin Co., Ltd.

100 Clinical Results associated with Burosumab-TWZA

100 Translational Medicine associated with Burosumab-TWZA

100 Patents (Medical) associated with Burosumab-TWZA

279

Literatures (Medical) associated with Burosumab-TWZA

28 Feb 2026·JBMR Plus

Postprandial serum phosphorus and calcium concentrations in adults and children with X-linked hypophosphatemia during burosumab treatment

Article

Author: Jan de Beur, Suzanne M ; Heerssen, Heather M ; Munns, Craig F ; Imel, Erik A ; Portale, Anthony A ; Peng, Eric ; Vincent, Marc

Abstract:

X-linked hypophosphatemia (XLH) is caused by PHEX gene variants that result in increased circulating levels of fibroblast growth factor 23 (FGF23). FGF23 in turn decreases renal reabsorption of phosphate and suppresses renal production of 1,25(OH)2D, leading to rickets and growth impairment in children and osteomalacia in children and adults. Burosumab is a fully human FGF23-blocking monoclonal antibody approved for treating XLH. Limited data are available on the impact of phosphorus-containing meals or supplements or diurnal variation on serum phosphorus levels, with increases observed in some, but not all studies. It is recommended that serum phosphorus be measured in the morning fasted state when monitoring treatment in patients with XLH. The present substudy of the pivotal pediatric and adult phase 3 clinical trials of burosumab examined the impact of meal consumption and timing around meals on serum phosphorus and calcium levels in children and adults with XLH during burosumab treatment. Thirty-nine participants (pediatric, n = 13; adult, n = 26) were included. The mean (SD) duration of burosumab treatment prior to the substudy was 15.4 (6.6) mo for pediatric and 24.2 (3.7) mo for adult participants. Serum phosphorus and calcium levels were measured before and after breakfast in children, and before and after both breakfast and lunch in adults. In both age groups, there was no clinically meaningful difference in mean levels of serum phosphorus measured at 1 and 2 h after meals compared to fasted levels, and serum calcium levels remained within the normal range for all pediatric participants and most adults, although interpatient variation was observed. These results suggest that, when fasting is not possible, nonfasting serum phosphorus levels may be a suitable alternative in patients with XLH receiving a stable dose of burosumab.

25 Feb 2026·Endocrine Connections

Burosumab for adults with tumor-induced osteomalacia in China

Article

Author: Yung, Chinwei ; Lu, Chunyan ; Kuriki, Masaaki ; Gu, Jiemei ; Sugimoto, Maiko ; Jiajue, Ruizhi ; Zhang, Zhenlin ; Xia, Weibo

A better understanding of the benefit of burosumab for treating tumor-induced osteomalacia (TIO) in Chinese patients is needed. The objective of this open-label, multicenter, single-cohort, post-marketing Phase 4 study was to assess the efficacy, pharmacokinetics, pharmacodynamics, and safety of burosumab after repeated administration in Chinese adults with TIO. Burosumab was administered subcutaneously every 4 weeks for up to 48 weeks at an initial 0.3 mg/kg dose. We investigated the change from baseline in mean serum phosphorus level over time, and changes in serum and urinary phosphorus levels, bone turnover biomarkers, patient-reported outcomes, and other parameters after repeated burosumab administration. Nine patients were treated. Serum phosphorus levels increased and remained above baseline and lower limit of normal at the end of the dosing cycles (averaged over Weeks 20 to 48) (mean [standard deviation] change, 1.5 [0.86] mg/dL). Tubular reabsorption of phosphate and the renal tubular maximum reabsorption rate of phosphate to glomerular filtration rate level ratio increased and remained above baseline values. Alkaline phosphatase (ALP), bone-specific ALP, carboxy-terminal cross-linked telopeptide of type I collagen, and procollagen type 1 N propeptide levels increased, reaching maximums at Weeks 16 or 24. The distance walked in 6 minutes nearly doubled from baseline by Week 48. Patients reported improvements in pain and quality of life over time. There were no serious adverse events and only five treatment-related adverse events (all mild in severity) in three patients. In Chinese patients with TIO, continued treatment with burosumab can provide sustained clinical benefit and a favorable safety profile.

20 Feb 2026·JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Challenges in Estimating Renal Function in X-linked Hypophosphatemia Because of Formula Overestimation and FGF23 Effects

Article

Author: Chapurlat, Roland ; De Mul, Aurélie ; Derain-Dubourg, Laurence ; Lemoine, Sandrine ; Legrand, Mélanie Alice ; Perge, Kevin ; Bacchetta, Justine ; Vignot, Emmanuelle

Abstract:

Introduction:

In X-linked hypophosphatemia (XLH), phosphate and vitamin D analogs increase the risk of nephrocalcinosis and renal impairment. Kidney function assessment is challenging, as creatinine-based estimated glomerular filtration rate (eGFR) may overestimate GFR because of reduced muscle mass. Cystatin C alternatively may be used. Moreover, fibroblast growth factor 23 (FGF23) has been suggested to potentially impact GFR, leading to hyperfiltration and complicating kidney function assessment.

Methods:

We conducted a single-center retrospective study on adults with XLH to compare eGFR formulas (CKD-EPIcreat, CKD-EPIcyst) with true measured GFR (mGFR). Bias, precision, and accuracy were calculated. In a subgroup, mGFR changes after burosumab initiation were assessed. Finally, we analyzed the FGF23/GFR relationship in a pediatric cohort in whom FGF23 was also hypersecreted (fibrous dysplasia) to support our hypothesis that FGF23 leads to hyperfiltration.

Results:

We included 20 patients (17 females, median age 35 years). None had nephrocalcinosis or hypercalciuria, but 8 had secondary hyperparathyroidism. Median mGFR was 90 (85-107) mL/min/1.73 m², significantly lower than CKD-EPIcreat (125 [114-121]) and CKD-EPIcyst (103 [118]) (respectively P < .0001 and P = .01). Bias was significantly greater with CKD-EPIcreat (−22 [−29 to −13]) than CKD-EPIcyst (−13 [−19 to −4]), with lower accuracy (25% vs 93%). In 5 patients with XLH, mGFR decreased from 98 to 84 mL/min/1.73 m² after burosumab introduction. FGF23 moderately correlated with eGFR in 14 fibrous dysplasia patients (R = 0.62, P = .02).

Conclusion:

eGFR overestimates GFR in XLH, increasing CKD misclassification risk. Annual cystatin C measurement is recommended if CKD is suspected. FGF23 impact on GFR should also be considered in burosumab-treated patients.

News (Medical) associated with Burosumab-TWZA

12 Feb 2026

·www.biospace.com

Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update

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 (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 reported its financial results for the quarter and year ended December 31, 2025. “The year ahead marks an important turning point for the company, as we approach two potential product launches and a pivotal data readout that, together, could significantly accelerate our commercial revenue trajectory,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “We are implementing a strategic restructuring plan to reduce our operating expenses and ensure our resources are squarely aligned with our highest-impact opportunities, while leading the future of rare disease with multiple first ever treatments.” Fourth Quarter 2025 Selected Financial Data Tables and Financial Results Revenues (dollars in millions), (unaudited) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Crysvita Product sales - Latin America and Türkiye $ 40 $ 22 $ 177 $ 135 Royalty revenue - U.S. and Canada 97 87 275 249 Royalty revenue - Europe 8 7 29 26 Total Crysvita Revenue 145 116 481 410 Dojolvi 32 31 96 88 Evkeeza 17 10 59 32 Mepsevii 13 8 37 30 Total revenues $ 207 $ 165 $ 673 $ 560 Revenues Ultragenyx reported $207 million in total revenue for the fourth quarter 2025, which represents 25% growth compared to the same period in 2024. Crysvita revenue in the fourth quarter 2025 was $145 million, which includes product sales of $40 million from Latin America and Türkiye. Dojolvi revenue in the fourth quarter 2025 was $32 million. Evkeeza revenue in the fourth quarter 2025 was $17 million. Total revenue for the year ended December 31, 2025 was $673 million, which represents 20% growth compared to the prior year. Full year 2025 Crysvita revenue was $481 million, which represents 17% growth compared to the prior year. This includes product sales of $177 million from Latin America and Türkiye, which represents 31% growth compared to the prior year. Dojolvi revenue in 2025 was $96 million, which represents 9% growth compared to the prior year. Evkeeza revenue in 2025 was $59 million, which represents 84% growth compared to the prior year, as demand continues to build following launches in the company’s territories outside of the United States. Selected Financial Data (dollars in millions, except per share amounts), (unaudited) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Total revenues $ 207 $ 165 $ 673 $ 560 Operating expenses: Cost of sales 29 17 109 77 Research and development 203 188 750 698 Selling, general and administrative 89 82 349 321 Total operating expenses 321 287 1,208 1,096 Net loss $ (129 ) $ (133 ) $ (575 ) $ (569 ) Net loss per share, basic and diluted $ (1.29 ) $ (1.39 ) $ (5.83 ) $ (6.29 ) Operating Expenses Total operating expenses for the fourth quarter 2025 were $321 million. Total operating expenses for the year ended December 31, 2025 were $1.2 billion, including $153 million of non-cash stock-based compensation. Net Loss Net loss for the fourth quarter 2025 was $129 million, or $1.29 per share basic and diluted, compared with a net loss for the fourth quarter 2024 of $133 million, or $1.39 per share basic and diluted. Net loss for the year ended December 31, 2025 was $575 million, or $5.83 per share basic and diluted, compared with a net loss the prior year of $569 million, or $6.29 per share, basic and diluted. Cash Balance and Net Cash Used in Operations Cash, cash equivalents, and marketable securities were $737 million as of December 31, 2025. For the three months ended December 31, 2025, net cash used in operations was $100 million and for the year ended December 31, 2025 was $466 million. Financial Guidance and Strategic Restructuring Plan 2026 revenue guidance Total revenues, excluding potential revenue from new product launches, in the range of $730 million to $760 million, an increase of 8% to 13% compared to 2025 Crysvita revenue in the range of $500 million to $520 million, reflecting growing underlying global demand partially offset by expected timing of ordering patterns in Brazil Dojolvi revenue in the range of $100 million to $110 million Strategic restructuring plan and path to profitability in 2027 Ultragenyx has initiated a strategic restructuring plan designed to reduce its headcount and expenses and focus resources on its largest value drivers. The significant reduction and partial reinvestment of expenses, and the planned growth in revenue from current and new product launches, are designed to keep the company on its path to profitability in 2027. Today, in connection with the restructuring plan, the company announced a 10% workforce reduction, impacting approximately 130 employees. Based on the progression of the business and the reductions from the restructuring plan: In 2026, combined R&D and SG&A expenses are expected to be flat to down low-single digits versus 2025. This includes the impact of spend reductions and approximately $50 million for severance, manufacturing, and other non-recurring restructuring charges. In 2027, R&D expenses are expected to decrease from 2025 levels by 38%, or approximately $280 million, driven by the completion of multiple phase 3 studies and reduction of early-stage research efforts. SG&A expenses are expected to increase in support of new product launches and existing approved products. On a combined basis, 2027 R&D and SG&A expenses are expected to decrease at least 15% versus 2025. 2026 Clinical and Regulatory Catalysts DTX401 (pariglasgene brecaparvovec) AAV8 gene therapy for glycogen storage disease type Ia (GSDIa): Biologics License Application (BLA) rolling submission completed in December 2025, with an anticipated Prescription Drug User Fee Act (PDUFA) action date in the third quarter of 2026. UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome type A (MPS IIIA): The BLA was resubmitted in January 2026 and included substantial longer-term data, that were recently presented at the 2026 WORLD Symposium , on multiple measures of neurologic benefit to support an intermediate clinical endpoint for accelerated approval supported further by CSF heparan sulfate and other biomarker data, as agreed with the FDA during the last clinical review. Earlier today the company received an Incomplete Response Letter (IRL) regarding its resubmitted BLA. The IRL requests additional supportive documentation related to its CRL CMC responses, which the company will provide in a resubmission. GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): Data from the fully enrolled, pivotal, Phase 3 Aspire study in patients with a genetically confirmed diagnosis of UBE3A deletion is expected in the second half of 2026. Enrollment in the Phase 2/3 Aurora study is also underway in other genotypes and ages, with the first patient dosed in October 2025. UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for Wilson disease: Enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data from this stage are expected in 2026. Conference Call and Webcast Information Ultragenyx will host a conference call today, Thursday, February 12, 2026, at 2 p.m. PT/5 p.m. ET to discuss the fourth quarter and full year 2025 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at . The replay of the call will be available for three months. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: . Forward-Looking Statements and Use of Digital Media Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, including the company’s expectations for profitability in 2027, anticipated cost or expense reductions, including the company’s expectations related to benefits and savings from the strategic restructuring plan, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, the components and timing of regulatory submissions, the company’s ability to provide the requested documentation and address the comments in the CRL for UX111 to the satisfaction of the FDA, the timing of FDA review of the company’s BLA submissions, the timing and outcome of any FDA inspections related to UX111 or other clinical product candidates, the timing of future regulatory interactions related to the company’s clinical product candidates are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercialization of Crysvita in certain major markets, including the U.S. and Canada, and for our commercial supply of Crysvita in those markets, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, , smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, our ability to successfully manage the expansion of our company, delays or unexpected costs and other adverse effects related to the strategic restructuring plan, competition from other therapies or products, regulatory scrutiny of the company’s products and product candidates, the company’s limited experience as a company in operating its own manufacturing facility, market acceptance of our products, uncertainty related to insurance coverage and reimbursement, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidate. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 5, 2025, and its subsequent periodic reports filed with the SEC. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website ( ) and LinkedIn website ( ). Ultragenyx Pharmaceutical Inc. Selected Statement of Operations Financial Data (in millions, except per share amounts) (unaudited) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Statement of Operations Data: Revenues: Product sales $ 102 $ 72 $ 369 $ 285 Royalty revenue 105 $ 93 304 275 Total revenues 207 165 673 560 Operating expenses: Cost of sales 29 17 109 77 Research and development 203 188 750 698 Selling, general and administrative 89 82 349 321 Total operating expenses 321 287 1,208 1,096 Loss from operations (114 ) (122 ) (535 ) (536 ) Non-cash interest expense on liabilities for sales of future royalties (19 ) (15 ) (62 ) (63 ) Other income, net 5 4 26 32 Loss before income taxes (128 ) (133 ) (571 ) (567 ) Provision for income taxes (1 ) — (4 ) (2 ) Net loss $ (129 ) $ (133 ) $ (575 ) $ (569 ) Net loss per share, basic and diluted $ (1.29 ) $ (1.39 ) $ (5.83 ) $ (6.29 ) Shares used in computing net loss per share, basic and diluted 99.9 95.7 98.6 90.5 Ultragenyx Pharmaceutical Inc. Selected Activity included in Operating Expenses (in millions) (unaudited) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Non-cash stock based compensation $ 38 $ 40 $ 153 $ 158 GTX-102 clinical milestone — $ 30 — $ 30 Ultragenyx Pharmaceutical Inc. Selected Balance Sheet Financial Data (in millions) (unaudited) December 31, December 31, 2025 2024 Balance Sheet Data: Cash, cash equivalents, and marketable securities $ 737 $ 745 Working capital 567 473 Total assets 1,532 1,503 Total stockholders' equity (deficit) (80 ) 255 Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com

Financial StatementPhase 3OligonucleotideGene Therapy

12 Jan 2026

·www.biospace.com

Ultragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference

Preliminary 2025 total revenue of $672 million to $674 million, exceeding top end of guidance Preliminary cash and investments of approximately $735 million as of December 31, 2025 Anticipated 2026 catalysts include two potential approvals and pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif., Jan. 12, 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 reported preliminary unaudited 2025 revenue results, cash and investments at year end 2025, and provided a corporate update. “In 2025, we delivered another year of significant revenue growth, with preliminary revenue exceeding the top end of guidance," said Emil Kakkis, chief executive officer and president of Ultragenyx. "The year ahead will be transformational for our company and the rare disease communities we serve, with two potential gene therapy approvals of first-ever treatments, and a pivotal data readout from our Phase 3 program in Angelman syndrome, setting the stage for our next phase of growth." Financial Update 2025 Preliminary Revenue (unaudited) Total revenue for 2025 is estimated to be $672 million to $674 million, which exceeds the top end of the guidance range, and represents approximately 20% growth versus 2024. Crysvita ® revenue for 2025 is estimated to be $480 million to $482 million, which exceeds the top end of the guidance range, and represents approximately 17% growth versus 2024. Dojolvi ® revenue for 2025 is estimated to be $95 million to $97 million, at the midpoint of the guidance range, and represents approximately 9% growth versus 2024. 2025 Ending Cash Position (unaudited) Cash and investments were approximately $735 million as of December 31, 2025. The 2025 revenues and cash position included in this release are preliminary and are therefore subject to adjustment. The preliminary revenue results are based on management’s initial analysis of operations for the year ended December 31, 2025. The Company expects to issue financial guidance for fiscal year 2026, including details on previously announced planned expense reductions, and full financial results for the fourth quarter and fiscal year 2025, in February 2026. 2026 Clinical and Regulatory Catalysts DTX401 (pariglasgene brecaparvovec) AAV gene therapy for glycogen storage disease type Ia (GSDIa): Biologics License Application (BLA) rolling submission completed in December 2025, with an anticipated PDUFA date in the third quarter of 2026. UX111 (rebisufligene etisparvovec) AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Resubmission of the BLA is on track for early in 2026 and will be followed by an up to 6-month review per FDA regulations. GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): Data from the fully enrolled, pivotal, Phase 3 Aspire study in patients with a genetically confirmed diagnosis of UBE3A deletion is expected in the second half of 2026. The Phase 2/3 Aurora study is also underway in other genotypes and ages, with first patient dosed in October 2025. UX701 (rivunatpagene miziparvovec) AAV gene therapy for Wilson disease: Enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data are expected in the first half of 2026. Data from the Phase 3 Orbit and Cosmic studies of UX143 (setrusumab) in osteogenesis imperfecta, including data on bone mineral density, vertebral fractures, and patient reported outcomes on pain and physical function, will be presented at the J.P. Morgan Healthcare Conference. The company presentation is scheduled for Monday, January 12, 2026 at 10:30 AM PT. The live and archived webcast of the presentation will be accessible from the company’s website at . About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: . Forward-Looking Statements and Use of Digital Media Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, the components and timing of regulatory submissions and the timing of results from ongoing clinical studies are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercialization of Crysvita in certain major markets, including the U.S. and Canada, and for our commercial supply of Crysvita in those markets, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, , smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, our ability to successfully manage the expansion of our company, competition from other therapies or products, regulatory scrutiny of the company’s products and product candidates, the company’s limited experience as a company in operating its own manufacturing facility, market acceptance of our products, uncertainty related to insurance coverage and reimbursement, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidate. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 4, 2025, and its subsequent periodic reports filed with the SEC. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website ( ) and LinkedIn website ( ). Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com

Financial StatementPhase 3Gene Therapy

05 Nov 2025

·endpoints.news

Lilly partners with AI biologics company; Amgen ends gastric cancer study

Plus, news about Rigel Pharmaceuticals, Ultragenyx, Neurocrine Biosciences, TransThera, MoonLake Immunotherapeutics, Kelonia Therapeutics, Johnson & Johnson and Harmony Biosciences: 🖊️ Eli Lilly signs deal with XtalPi subsidiary: Lilly will get access to Ailux’s AI-powered bispecific antibody engineering platform, with rights to nominate an undisclosed number of targets. The deal could be worth up to $345 million across upfront and milestone payments. Ailux is a wholly-owned subsidiary of Chinese biotech XtalPi. — Ayisha Sharma 🔬 Amgen stops trial of stomach cancer drug : Head of R&D Jay Bradner said that the company stopped a trial studying bemarituzumab in combination with chemotherapy and Opdivo for patients with first-line gastric cancer due to “inadequate efficacy.” The decision follows an updated analysis of the drug in a separate but similar trial showing that its overall survival benefit when combined with chemotherapy waned over time. A third study, FORTITUDE-103, is testing bemarituzumab in combination with other anti-cancer therapies and has completed enrollment, Amgen said. — Max Bayer 🛑 Lilly terminates CNS work with Rigel: Lilly notified the biotech early last month that it would end their partnership on a preclinical CNS disease program. That partnership was part of a deal announced in 2021 . But the companies will still partner on ocadusertib, which is an RIPK1 inhibitor in a Phase 2a study in certain rheumatoid arthritis patients with moderate to severe disease. – Reynald Castaneda 💼 Ultragenyx divests rare disease drug royalties to Canadian pension fund: The biotech will receive $400 million from OMERS. This is in return for a further 25% of Ultragenyx’s royalty interest from Kyowa Kirin on US and Canadian sales of Crysvita. Proceeds from the deal should help fund four of Ultragenyx’s upcoming launches. — Ayisha Sharma 🖊️ Neurocrine signs deal with TransThera: In an agreement worth up to $881.5 million, Neurocrine will have the rights to develop and commercialize the Chinese company’s preclinical NLRP3 inhibitors for a variety of diseases outside Greater China. – Reynald Castaneda 🌕 Moonlake sells $75M in shares: The drugmaker will use the proceeds to support continued development of its nanobody candidate, sonelokimab, for inflammatory diseases, among other purposes. The offering is expected to close on Nov. 6. The company also reported Wednesday that its 16-week data from a Phase 2 trial showed that palmoplantar pustular psoriasis patients treated with sonelokimab had an average 64% reduction in the area and severity of their psoriasis. — Ayisha Sharma 🤝 Kelonia Therapeutics’ new collaborator: Kelonia will be working with Johnson & Johnson to develop in vivo CAR-T therapies. The biotech will contribute the technology to engineer immune cells directly in the body and J&J will pick the targets. Financial and scientific details were not disclosed, but the deal makes J&J the latest entrant in the red-hot field. Four other pharma companies — AbbVie, AstraZeneca, Bristol Myers Squibb and Gilead Sciences — have acquired startups working on in vivo cell therapies this year. — Ryan Cross ⏸️ Harmony Biosciences said it is pausing work on ZYN002 for a genetic disorder called 22q11.2 deletion syndrome. The synthetic cannabidiol gel previously failed in a Phase 3 fragile X syndrome study. – Reynald Castaneda

Phase 2Cell TherapyImmunotherapyClinical ResultPhase 3

100 Deals associated with Burosumab-TWZA

External Link

KEGG	Wiki	ATC	Drug Bank
-	Burosumab-TWZA	M05BX05	DB14012

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
FGF23 positive Hypophosphatemia	South Korea	Kyowa Kirin Korea Co., Ltd.	17 Sep 2020
Oncogenic Osteomalacia	United States	Kyowa Kirin, Inc. /Sancuso/	18 Jun 2020
Rickets, Hypophosphatemic	Japan	Kyowa Kirin Co., Ltd.	20 Sep 2019
Osteomalacia	Canada	Kyowa Kirin Co., Ltd.	28 Jan 2019
Familial Hypophosphatemic Rickets	European Union	Kyowa Kirin Holdings BV	19 Feb 2018
Familial Hypophosphatemic Rickets	Iceland	Kyowa Kirin Holdings BV	19 Feb 2018
Familial Hypophosphatemic Rickets	Liechtenstein	Kyowa Kirin Holdings BV	19 Feb 2018
Familial Hypophosphatemic Rickets	Norway	Kyowa Kirin Holdings BV	19 Feb 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Congenital Abnormalities	Phase 3	Germany	Kyowa Kirin GmbH	21 Dec 2020
Dwarfism	Phase 3	Germany	Kyowa Kirin GmbH	21 Dec 2020
Genu Varum	Phase 3	Germany	Kyowa Kirin GmbH	21 Dec 2020
Protoporphyria, Erythropoietic, X-Linked Dominant	Phase 3	Italy	Kyowa Kirin Pharmaceutical Development Ltd.	25 Oct 2018
Chronic Pain	Phase 3	United States	Ultragenyx Pharmaceutical, Inc.	31 Jan 2018
Sebaceous of Jadassohn Nevus	Phase 3	United States	Ultragenyx Pharmaceutical, Inc.	31 Jan 2018
Hypophosphatemia	Phase 1	United States	Ultragenyx Pharmaceutical, Inc.	01 Jul 2019

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05509595 (CTgov)	Phase 2	Fibrous Dysplasia of Bone \| Hypophosphatemia	12	Burosumab	tvlwodmlzy = ndugbkemmw turniuhimb (siluigbkdq, rcydpisnma - desecifqwk) View more	-	02 Feb 2026
ESPE2024	Not Applicable	Familial Hypophosphatemic Rickets	24	Burosumab	hbozyervlz(xqmusagowq) = bvsezroosy kjihduaezb (occlzumcgt, 0.2) View more	Positive	16 Nov 2024
ESPE2024	Not Applicable	Familial Hypophosphatemic Rickets	19	Burosumab (X-linked hypophosphatemic rickets)	kgblkpxgfi(apdplmayws) = fwhobycqzv lgxhjtslfk (scxhfnqubx ) View more	Positive	16 Nov 2024
ENDO2024	Phase 3	Familial Hypophosphatemic Rickets PHEX mutation	-	Burosumab	dzlbmrchmt(mzmtdbfvbm) = nczwmzpuvo orfwmpmuid (nglvclnryb ) View more	-	01 Jun 2024
NCT04146935 (CTgov)	Phase 4	Familial Hypophosphatemic Rickets	10	Burosumab Injection [Crysvita]	gnourgkoan(jurpsubkjz) = whbeuxwwfs hogmoogvsu (xmqkerkivo, eubbelfkdu - ivzlyjrfwr) View more	-	24 Aug 2023
NCT03920072 (BUR02, WCO_IOF_ESCEO2023)	Phase 3	Familial Hypophosphatemic Rickets	35	Burosumab	dtsgcnoidi(fkkjasdjgh) = The most frequent of which were injection-site reactions, which resolved without requiring dose changes fgoszzwhzz (iuwgdtjoaa )	Positive	04 May 2023
IMPROVEMENT IN PATIENT OUTCOMESFOLLOWING INITIATION OF BUROSUMAB INADULTS WITH XLH: A SINGLE CENTRE EXPERIENCE (WCO_IOF_ESCEO2023)	Not Applicable	Familial Hypophosphatemic Rickets FGF23	28	Burosumab	yixkopngaf(nmpkaivdxn) = rrdutgnksa rdhhfffysx (ewovfgpmcd ) View more	Positive	04 May 2023
NCT02304367 (UX023T-CL201, ENDO2022)	Phase 2	Oncogenic Osteomalacia FGF23	14	Burosumab	penqgzgkho(ymprjqyfya) = qizvotlwgn hllkvoiipm (znasprdnvq, 0.5)	Positive	01 Nov 2022
NCT02915705 (phase 3 trial, ESPE2022)	Phase 3	Familial Hypophosphatemic Rickets	62	Burosumab continuation group	cgwhfrmntc(swvisvebhl) = The mean change from baseline exceeded the MID for Pain Interference at weeks 64 and 88 and for Fatigue at week 64 in the burosumab continuation group, and for Pain Interference and Fatigue at week 88 in the crossover group dgjebjlqgt (vnuogjbehh ) View more	Positive	15 Sep 2022
NCT02915705 (phase 3 trial, ESPE2022)	Phase 3	Familial Hypophosphatemic Rickets	62	Crossover group		Positive	15 Sep 2022
NCT02312687 (UX023-CL203, Pubmed) Manual	Phase 2	Familial Hypophosphatemic Rickets	20	Burosumab-TWZA	tgghrrdkpt(idhlxyhxhv) = mztoaelznz qbgnfmwdzi (iyksnbxznl )	Positive	08 Sep 2022

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