A long-term, open label, extension study to assess the efficacy and safety of beta-glucuronidase enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII, Sly disease

开始日期2020-01-06

申办/合作机构-

NCT02432144

/ Completed临床3期

A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7

The primary objective of the study is to evaluate the long-term safety of UX003 in subjects with MPS 7.

开始日期2015-11-10

申办/合作机构

Ultragenyx Pharmaceutical, Inc.

NCT02418455

/ Completed临床2期

An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old

The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII participants less than 5 years of age on safety, tolerability, and efficacy as determined by the reduction of urinary glycosaminoglycans (uGAG) excretion.

开始日期2015-07-21

申办/合作机构

Ultragenyx Pharmaceutical, Inc.

100 项与 Vestronidase Alfa-VJBK 相关的临床结果

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100 项与 Vestronidase Alfa-VJBK 相关的转化医学

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100 项与 Vestronidase Alfa-VJBK 相关的专利（医药）

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项与 Vestronidase Alfa-VJBK 相关的文献（医药）

2026-01-01·JOURNAL OF INHERITED METABOLIC DISEASE

Natural‐History Mapping of Lysosomal Storage Disorders ( LSDs ): Gaucher Disease as a Model for Precision Care

Review

作者: Vaishnaw, Maya ; Mistry, Pramod. K. ; Ain, Noor Ul

ABSTRACT:

Natural‐history datasets have become pivotal for drug development and for shaping clinical‐practice guidelines in rare diseases, yet many lysosomal storage disorders would benefit from deep phenotyping and modern analytic methods. Our objective was to integrate the past decade of genomic, cellular, treatment‐outcome, and regulatory advances into a practical framework for capturing and interpreting natural‐history data, using Gaucher disease (GD) as a paradigm. We reviewed more than 300 peer‐reviewed articles (2005–2025), FDA guidance documents, and output from large real‐world registries. Particular attention was paid to long‐read GBA sequencing, biomarkers such as lyso‐Gb1, emerging newborn screening programs, and preliminary observational work that points toward multi‐state disease modeling. Key observations include: (i) Whole‐gene sequencing has expanded genotype–phenotype maps, revealing more than 70 recombinant GBA alleles that confound panel tests; (ii) registry trajectories suggest that formal multi‐state models could capture treatment‐modified courses and silent endpoints—monoclonal gammopathy, malignancy, Parkinson's disease, pulmonary arterial hypertension—better than current summary statistics; (iii) lyso‐Gb1 outperforms legacy biomarkers and now serves as a second‐tier newborn‐screening marker; (iv) Robust natural‐history evidence has already underpinned regulatory approvals across several lysosomal disorders—including olipudase alfa for ASMD, cerliponase alfa for CLN2, vestronidase alfa for MPS VII, and sebelipase alfa for infantile‐onset LAL‐D—demonstrating that well‐curated registries can serve as viable external controls for future LSD submissions. The convergence of deep phenotyping, genotype‐aware analytics, and systematic biomarker capture promises to transform natural‐history registries from descriptive archives into predictive engines. Gaucher disease offers a working template that, when extended across the LSD spectrum, can accelerate precision care and the development of next‐generation therapeutics.Trial Registration:ClinicalTrials.gov identifier: NCT00358943, NCT03291223.

2024-03-01·Molecular genetics and metabolism

Mucopolysaccharidosis type VII (Sly syndrome) - What do we know?

Review

作者: López-Valdez, Jaime ; Marsden, Deborah ; Grant, Christina L ; Ezgü, Fatih

Mucopolysaccharidosis type VII (MPS VII) is an ultra-rare, life-threatening, progressive disease caused by genetic mutations that affect lysosomal storage/function. MPS VII has an estimated prevalence of <1:1,000,000 and accounts for <3% of all MPS diagnoses. Given the rarity of MPS VII, comprehensive information on the disease is limited and we present a review of the current understanding. In MPS VII, intracellular glycosaminoglycans accumulate due to a deficiency in the lysosomal enzyme that is responsible for their degradation, β-glucuronidase, which is encoded by the GUSB gene. MPS VII has a heterogeneous presentation. Features can manifest across multiple systems and can vary in severity, age of onset and progression. The single most distinguishing clinical feature of MPS VII is non-immune hydrops fetalis (NIHF), which presents during pregnancy. MPS VII usually presents within one month of life and become more prominent at 3 to 4 years of age; key features are skeletal deformities, hepatosplenomegaly, coarse facies, and cognitive impairment, although phenotypic variation is a hallmark. Current treatments include hematopoietic stem cell transplantation and enzyme replacement therapy with vestronidase alfa. Care should be individualized for each patient. Development of consensus guidelines for MPS VII management and treatment is needed, as consolidation of expert knowledge and experience (for example, through the MPS VII Disease Monitoring Program) may provide a significant positive impact to patients.

2022-05-01·Molecular genetics and metabolism2区 · 生物学

Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII

2区 · 生物学

Article

作者: David Viskochil ; Betsy Malkus ; Lin Zhang ; Antonio Gonzalez-Meneses Lopez ; Pranoot Tanpaiboon ; Esmeralda Martins ; Deborah Marsden ; Heather A. Lau ; Julie Taylor ; Agnieszka Jurecka

Mucopolysaccharidosis (MPS) VII is an ultra-rare, autosomal-recessive, metabolic disease caused by a deficiency of β-glucuronidase, a lysosomal enzyme that hydrolyzes glycosaminoglycans (GAGs), including dermatan sulfate (DS), chondroitin sulfate, and heparan sulfate (HS). β-glucuronidase deficiency leads to progressive accumulation of undegraded GAGs in lysosomes of affected tissues, which may cause hydrops fetalis, short stature, hepatosplenomegaly, and cognitive impairment. An open-label, multicenter, phase II study was conducted in 8 pediatric subjects <5 years of age with MPS VII. Subjects received the recombinant human β-glucuronidase vestronidase alfa 4 mg/kg by intravenous infusion every other week for 48 weeks (treatment period). Those who completed the 48-week treatment were offered to continue treatment with vestronidase alfa 4 mg/kg for up to 240 weeks or until withdrawal of consent, discontinuation, or study termination (continuation period). The level of GAG excreted in urine (uGAG) above normal has been shown to correlate with disease severity and clinical outcomes in MPS diseases. Therefore, the primary efficacy endpoint of this study was to determine the mean percentage change in uGAG DS excretion from baseline to week 48. Statistically significant reductions in uGAG DS from baseline were observed at each visit (p < 0.0001), with a least square mean (standard error) percentage change of -60% (6.6) at week 4 (first post-baseline assessment) and -61% (6.41) at week 48 (final assessment during treatment period). Secondary efficacy endpoints included change from baseline to week 48 in growth and hepatosplenomegaly. Positive trends were observed toward increased standing height Z-score (mean [standard deviation] at baseline, -2.630 [1.17], n = 8; at week 48, -2.045 [0.27], n = 7) and growth velocity (mean [SD] Z-score at baseline, -2.59 [1.49], n = 4; at week 48, -0.39 [2.10], n = 4; p = 0.27). Hepatomegaly was resolved in 3 of 3 subjects assessed by ultrasound and in 5 of 6 subjects assessed by physical examination; splenomegaly was resolved in 1 of 3 subjects assessed by ultrasound and in 2 of 2 subjects assessed by physical examination. There were no new safety signals identified during this study. Mild-to-moderate infusion-associated reactions occurred in 4 (50%) subjects. In conclusion, long-term vestronidase alfa treatment demonstrated a rapid and sustained reduction in uGAGs, maintained growth, and improved hepatosplenomegaly in pediatric subjects with MPS VII <5 years of age. Trial registration: NCT02418455.

项与 Vestronidase Alfa-VJBK 相关的新闻（医药）

2026-08-04

·ir.ultragenyx.com

Ultragenyx Reports Second Quarter 2026 Financial Results and Corporate Update

Second quarter total revenue of $214 million, Crysvita® revenue of $156 million and Dojolvi® revenue of $27 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path to profitability in 2027 Catalysts in second half of 2026 include two PDUFA dates and pivotal data readout from GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif., Aug. 04, 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 ended June 30, 2026 and reaffirmed its financial guidance for 2026. “In the second quarter we generated the highest quarterly revenue in the history of the company, supporting our full-year revenue guidance, and keeping us on track toward profitability in 2027,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “As we look to the second half of the year, we are entering a transformative period with multiple important catalysts. We are ready to launch two gene therapy products and are preparing for a pivotal Phase 3 GTX-102 data readout in Angelman syndrome. This puts us in position to broaden our patient impact with sustained growth in the years to come.” Second Quarter 2026 Revenue Highlights and 2026 Revenue Guidance Total revenue in the second quarter of 2026 was $214 million. The company reaffirms its full year 2026 total revenue guidance of $730 million to $760 million, which excludes revenue from potential new product launches. Crysvita revenue in the second quarter of 2026 was $156 million, consistent with expected seasonality in the U.S. and Canada and ordering patterns in Latin America. The company reaffirms its full year 2026 Crysvita revenue guidance of $500 million to $520 million. Dojolvi revenue in the second quarter 2026 was $27 million. The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million. Evkeeza® revenue in the second quarter 2026 was $21 million, driven by increased demand from new country launches and early access. Mepsevii® revenue in the second quarter 2026 was $10 million. Milestones and Upcoming Catalysts Dojolvi for the treatment of LC-FAOD: In May 2026, Dojolvi was listed on the National Health Insurance (NHI) drug price list and was launched in Japan following the receipt of manufacturing and marketing approval under the Conditional Approval System for Pharmaceuticals on March 23, 2026. DTX401 (pariglasgene brecaparvovec) AAV8 gene therapy for the treatment of glycogen storage disease type Ia (GSDIa): In February 2026, the U.S. Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) seeking approval of DTX401 as a treatment for GSDIa and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026. UX111 (rebisufligene etisparvovec) AAV9 gene therapy for the treatment of Sanfilippo syndrome type A (MPS IIIA): In April 2026, the FDA accepted for review the resubmitted BLA seeking accelerated approval for UX111 as a treatment for MPS IIIA and assigned a PDUFA action date of September 19, 2026. GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): The Phase 3 Aspire study, in patients with a full maternal UBE3A gene deletion, enrolled 129 patients, randomized 1:1 to GTX-102 or sham. Data from this study are expected in the September or October timeframe. Enrollment in the open-label Phase 2/3 Aurora study, evaluating GTX-102 in other genotypes and ages, began in October 2025 and is expected to complete in the second half of 2026. UX701 (rivunatpagene miziparvovec) AAV9 gene therapy for the treatment of 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 the fourth quarter of 2026. UX016 novel prodrug for sialic acid used as a substrate replacement therapy for the treatment of GNE myopathy: The FDA cleared the Investigational New Drug (IND) application for UX016 and an externally funded Phase 1/2 study is expected to begin in the second half of 2026. DTX301 (avalotcagene ontaparvovec) AAV8 gene therapy for the treatment of Ornithine Transcarbamylase, or OTC, deficiency: The Phase 3 Enh3ance study continues with patients in both treatment and cross-over groups progressing through 64 weeks of follow-up. Data from the second primary endpoint, which evaluates reduction in treatment burden, including use of ammonia scavengers and dietary management, are expected in the first half of 2027. Summary of Second Quarter 2026 Financial Results Selected Financial Data (dollars in millions, except per share amounts), (unaudited) Operating Expenses Total operating expenses for the second quarter 2026 were $289 million, including $34 million of non-cash stock-based compensation. The company reaffirms its full year 2026 and 2027 guidance for combined R&D and SG&A operating expenses: compared to 2025, combined R&D and SG&A expenses in 2026 are expected to be flat to down low-single digits, and combined R&D and SG&A expenses in 2027 are expected to decrease by at least 15%. Net Loss Net loss for the second quarter 2026 was $92 million, or $0.90 per share basic and diluted, compared with a net loss for the second quarter 2025 of $115 million, or $1.17 per share basic and diluted. Cash Balance and Net Cash Used in Operations Cash, cash equivalents, and marketable securities were $436 million as of June 30, 2026. For the three months ended June 30, 2026, net cash used in operations was $97 million. Conference Call and Webcast Information Ultragenyx will host a conference call today, Tuesday, August 4, 2026, at 2 p.m. PT/5 p.m. ET to discuss the second quarter financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at https://ir.ultragenyx.com/events-presentations. 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: www.ultragenyx.com. 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 regarding Ultragenyx’s expectations and projections concerning its future operating results and financial performance, including its 2026 revenue guidance for total revenue, Crysvita and Dojolvi, its anticipated R&D and SG&A expenses in 2026 and 2027, anticipated benefits and savings from its strategic restructuring plan, and the timing and sustainability of profitability; the timing, progress, results and plans for its clinical programs and studies, including the anticipated timing and outcome of the Phase 3 Aspire study of GTX-102, enrollment in the Aurora study, data from the UX701 and DTX301 studies, and initiation of the UX016 study; the FDA’s review of the BLAs for DTX401 and UX111, including the anticipated PDUFA action dates, the potential approval of either product candidate, whether the FDA may require additional information, studies or manufacturing changes, and the timing and outcome of regulatory inspections; Ultragenyx’s manufacturing and commercial readiness and the timing and success of any potential launches of DTX401 and UX111, if approved; and the potential patient impact, commercial opportunity and growth associated with Ultragenyx’s products and 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 May 6, 2026, 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 (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/). Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com

财报临床3期基因疗法临床申请

2026-08-03

·海螺研习社

手握突破性基因疗法，却输在车间管控 ——Ultragenyx Pharmaceutical Inc.设施FDA 483解读

从“自主制造的王冠”到“合规风暴的中心”。在罕见病和超罕见病的新药研发领域，基因治疗被视作许多绝症患者“一生一次”的最后希望。我们这次解读的就是一家专注于罕见病领域的明星生物制药公司——Ultragenyx Pharmaceutical Inc.在2025年接受FDA检查的483。通过回顾这间原本被寄予厚望的基因治疗工厂在2025年的经历，希望能为整个行业的生产与质量管理带来一些深刻的启示。 Ultragenyx成立于2010年，一直致力于为全球存在极大未满足医疗需求的罕见病患者提供创新疗法（其获批的传统产品如 Dojolvi 和 Mepsevii 已在全球多地区进行商业化销售）。为了解决其高度依赖外部合同定制生产机构（CDMO），导致产能受限且质量难以实现绝对把控的痛点，Ultragenyx斥巨资在距离大波士顿研发中心不远的Bedford地区，买下了一块10.7英亩的土地，建立起一座占地超11万平方英尺的先进基因治疗制造工厂，并于2023年6月正式落成启用，定位为药品制造商，同时具备药物原液和药物制剂的端到端生产能力。随着 Ultragenyx 的核心在研 AAV9 基因疗法 UX111（用于治疗A型圣菲利普综合征）进入上市冲刺阶段，2025年4月21日至4月25日期间，FDA对该设施进行了批准前检查，最终出具了6条缺陷的483报告，覆盖数据完整性、设施维护、偏差管理、质量监督、无菌工艺、污染控制六大领域，最终导致2025年7月11日，FDA 针对 UX111 的生物制品许可申请（BLA）正式下发了完整回复函（CRL），在函件中明确承认该疗法在临床神经发育结果和生物标志物数据上的显著有效性，但由于化学、制造和控制问题，特别是受到刚刚结束的 Bedford 工厂制造设施检查结果的拖累，FDA拒绝了该药物的上市批准。对于基因治疗公司而言，产品上市的延期往往意味着资金链的极度承压。由于获批时间被迫推迟至2026年，Ultragenyx 的财务状况受到了实质性影响。为了削减开支并在漫长的整改期内存活下来，公司不得不在2026年2月宣布了一项痛苦的重组计划：裁减全球约10%的员工（约130人），以将有限的资源集中于应对 FDA 的整改要求和维持高价值项目。好在，经过大半年的痛定思痛、彻查数据漏洞以及全面提升 Bedford 工厂的质量管理体系，公司完成了对 CMC 和检查缺陷的整改。2026年4月，FDA 终于重新接受了其里程碑式基因疗法的 BLA 申请，并将最终审批日期（PDUFA）定档在2026年8月23日。 Ultragenyx Bedford 工厂的这段经历，告诉每一位从业者，仅仅拥有顶尖的科学技术是远远不够的——复杂而严苛的生产合规与质量控制，往往才是决定一款救命药能否真正走向市场的“胜负手”。设施名称：Ultragenyx Pharmaceutical Inc 地址：170 Middlesex Turnpike, Bedford, MA 01730 USA FEI：3025268614 设施类型：药品制造商检查时间：04/21/2025-04/25/2025 检察官：Massoud Motamed, CSO, Lead Inspector Kula; N. Jha, Biological Reviewer Jacob; L. Bitterman, Biological Reviewer Timothy Kamaldinov, Biological Reviewer 01 通过受控文件管理来确保数据完整性的系统存在缺陷。具体如下： …… 欲知详情，请加入海螺企业VIP 具体操作流程参考：【新VIP会员招募】添加小编vx：gmpclub08，注明：VIP

2026-07-29

·BioSpace

Biotechs bring big launches and pharma rivalries into Q2 earnings period

iStock, ismagilov With the second quarter earnings period underway, BioSpace examines five biotechs with meaningful catalysts that analysts and investors are watching. Biotechs are heading into the second quarter earnings period ready to pressure their Big Pharma peers with new drug launches. More than ever, smaller biotechs are choosing to go at it alone to market their drugs, rather than partner up or sell. This has created a dynamic environment among these upstart companies, with plenty of eyes watching to see if they succeed. From Agios Pharmaceuticals to Ultragenyx, BioSpace takes a look at five biotechs with major catalysts to report on over the next few weeks. ezstandalone.cmd.push(function () { ezstandalone.showAds(129); }); Earnings Q2 earnings arrive as pharma picks up steam, buoyed by dealmaking, policy tailwinds Biopharma is entering its second-quarter earnings season riding high on a wave of massive deals and venture capital flow, plus a clearing of regulatory and policy overhangs. What can industry watchers expect to hear on the upcoming investor calls? July 15, 2026 · 6 min read · Tristan Manalac Read more Agios Pharmaceuticals Q2 stock performance: 35% Earnings date: July 30 After reporting the failure of a Phase 2 study for tebapivat in sickle cell disease, Agios’ management will have to show confidence in the biotech’s remaining pipeline when reporting second quarter earnings. Last week, Agios announced that the next-generation PK activator would be discontinued after failing to differentiate from the company’s own mitapivat or Novo Nordisk’s etavopivat. Analysts hadn’t given tebapivat a lot of credit heading into the readout, but the candidate served as a “narrative enricher for longer-term franchise durability,” according to Truist Securities. The failure of the candidate puts more “concentrated risk” on mitapivat, just as the drug class gets more competition from Novo Nordisk. Mitapivat is already approved as Pyrukynd for pyruvate kinase (PK) deficiency and Aqvesme for alpha- or beta-thalassemia. In the second quarter presentation, Truist will be watching for details on the thalassemia launch and the drug’s potential in sickle cell disease with tebapivat out of the running. The drug is under FDA review via the accelerated pathway for the indication with a decision date set for November 1. Arrowhead Pharmaceuticals FY3Q stock performance: 567% Earnings date: August 4 Arrowhead Pharmaceuticals has already begun its fiscal fourth quarter of 2026 on strong footing, with the announcement last week that two Phase 3 studies of plozasiran in severe hypertriglyceridemia (sHTG) were successful . The results sent the company’s shares up 20% that day to $91.48, while rival Ionis Pharmaceuticals dipped. Analysts had been anticipating the readout in the leadup to fiscal third quarter earnings. Beyond the outstanding results of those sHTG trials, analysts are watching for a second half readout for Arrowhead’s obesity programs , ARO-INHBE and ARO-ALK7. ARO-INHBE has already shown a doubling of the weight loss achieved by Eli Lilly’s tirzepatide in an interim readout from a small group of patients in two Phase 1/2a trials. “ARWR is ahead of peers across both the INHBE+ALK7 programs, w/ multiple paths forward depending on data,” Jefferies wrote in an earnings preview. Arrowhead is also expecting data for the Alzheimer’s disease therapy ARO-MAPT in the third quarter, which could help validate the company’s blood-brain barrier delivery tech. On the commercial side, Arrowhead celebrated its first ever approval late last year, when plozasiran was approved to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). As of the company’s last earnings call, the drug was “trending better” than Ionis’ rival product, according to Jefferies. The firm is expecting sales of about $6 million for the quarter. C-suite Caught In Sarepta ‘Downdraft,’ Arrowhead Becomes RNAi Knight in Shining Armor Sarepta’s troubles had nothing to do with Arrowhead’s assets, and yet both companies have seen their stock prices decline this past month. BioSpace caught up with Arrowhead’s Chris Anzalone to talk about the biotech’s role as an RNAi pipeline savior. August 12, 2025 · 7 min read · Annalee Armstrong Read more BridgeBio Q2 stock performance: 125% Earnings date: TBD BridgeBio is on track to deliver a strong second quarter report, as Attruby continues to steal market share in the crowded transthyretin amyloid cardiomyopathy (ATTR-CM) space, according to Jefferies. The drug is expected to make sales of $215 million for the quarter thanks to increases in front-line uptake, the firm predicted in a July 19 note to clients. This growth is expected to continue quarter-over-quarter going forward, which will in turn build investor confidence in BridgeBio’s next three planned launches in rare disease, Jefferies said. The company is working toward FDA approvals for BBP-418 in limb girdle muscular dystrophy type 2, infigratinib in alternating hemiplegia of childhood and encaleret in autosomal dominant hypocalcemia type 1. Infigratinib was approved in 2021 for cholangiocarcinoma and is marketed as Truseltiq. Each of these could mean $1 billion opportunities for BridgeBio, Jefferies noted, while Attruby is on track to deliver $3–$4 billion in peak sales in ATTR-CM. The drug faces competition from Pfizer’s Vyndaqel and Vyndamax, Alnylam’s Amvuttra and Ionis/AstraZeneca’s eplotersen in the space, but fears that BridgeBio will lose significant market share are subsiding as the market becomes more mature, according to Jefferies. Ionis Pharmaceuticals Q2 performance: 190% Earnings date: July 29 Ionis is well ahead in the marketplace with Tryngolza, which now has two approved indications in familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG) but is still facing immense pressure from Arrowhead. Analysts are expecting Ionis’ overall revenue to decline by more than half when the company reports earnings this week, as the price of the drug declines significantly to match the entry into a more common sHTG indication. Guggenheim analysts have revenue estimates above consensus at $209 million. This would mark a year-over-year decline of about 54% from last year, when revenue was $452 million . The slightly optimistic take as compared to consensus stems from higher than expected estimates for R&D collaboration revenue, since Recordati paid a $30 million upfront fee for a partnership with Ionis at the end of June. With the price reduction, Guggenheim has lowered its sales expectations to $2.9 billion as compared to $3.2 billion prior. But the analysts are optimistic that the dip for Tryngolza, which is now approved for FCS and sHTG, will be short lived as the sHTG label highlighted the therapy’s ability to reduce the risk of acute pancreatitis. Recent data also showed its ability to normalize hepatic fat. Moving forward, analysts will be focused on three upcoming Phase 3 readouts, including the Lp(a) HORIZON trial of pelacarsen, which is testing the drug for reducing lipoprotein(a). Rare diseases Ionis Paves Its Own Path as Initial Tryngolza Launch Defies Expectations Long an R&D company that partnered off assets, RNAi biotech Ionis Pharmaceuticals shifted in 2025 to bring two medicines to market alone. Analysts are already impressed—and there’s more to come in 2026. February 18, 2026 · 3 min read · Annalee Armstrong Read more Ultragenyx 2Q performance: -1.33% Earnings date: TBD Ultragenyx has been through the wringer this year, witha high pro for a bone disease drug and an FDA rejection for a rare disease gene therapy. Heading into the third quarter, analysts are keenly watching for any details on a Phase 3 readout for apazunersen in Angelman syndrome, which could help turn the ship around. Jefferies anticipates peak worldwide sales of about $1.87 billion for the drug in 2036 if ultimately approved. Ultragenyx executives have had to make some tough decisions following the events of 2025, including implementing an expense reduction plan and layoffs . They also sold off two priority review vouchers that were awarded with the recent approvals of rare disease therapies Mepsevii and Crysvita. The biotech is awaiting two key regulatory decisions. Pariglasgene brecaparvovec, an AAV8 gene therapy, is under review for glycogen storage disease type 1a with an FDA action date of August 23. And rebisufligene etisparvovec, an AAV9 gene therapy, was accepted for priority review by the FDA with a decision date of September 19. Jefferies believes that both launches could be successful because there is unmet need in both indications. As for sales, Jefferies predicts Crysvita to come in between $500 and $520 million while Dojolvi could take home $100–110 million.

临床3期上市批准临床2期财报

100 项与 Vestronidase Alfa-VJBK 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11004	Vestronidase Alfa-VJBK	A16AB18	DB12366

研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
粘多糖累积病Ⅶ型	美国	Ultragenyx Pharmaceutical, Inc.	2017-11-15

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02418455 (Pubmed \| Mol Genet Metab)	临床2期	粘多糖累积病Ⅶ型 uGAG DS excretion	8	Vestronidase alfa 4 mg/kg	鬱選蓋製製夢憲糧夢顧(積齋顧襯糧廠築窪窪醖) = 衊憲壓齋鏇壓遞觸鹹壓艱築獵襯廠鹹艱鏇構遞 (鏇簾製襯鹽鏇艱餘膚築 )	积极	2022-05-01
NCT02418455 (CTgov)	临床2期	粘多糖累积病Ⅶ型	8	UX003	觸醖艱選範簾廠廠簾襯(醖構鹹範鹹艱積製鹽糧) = 壓襯顧蓋夢衊鹹顧壓衊憲願淵鬱遞蓋鏇壓鏇範 (遞齋淵觸簾鑰齋餘積齋, 16.915) 更多	-	2019-10-16
NCT02432144 (CTgov)	临床3期	粘多糖累积病Ⅶ型	12	UX003	觸艱鏇構齋艱製範選蓋 = 鏇憲鹽鹹積範簾衊觸觸築鑰憲衊繭鬱願鹽襯餘 (簾壓齋憲觸齋鏇積範觸, 餘選醖鏇廠簾蓋繭選構 ~ 鏇選艱鏇廠糧範範鹽襯) 更多	-	2019-07-30
NCT02230566 (Pubmed \| Mol Genet Metab) 人工标引	临床3期	粘多糖累积病Ⅶ型	12	vestronidase alfa	齋壓艱選構構顧鹽餘壓(積選製積鏇淵遞醖積鬱) = 夢構憲憲遞壓襯範廠遞糧鏇觸範積鑰鬱簾鹹觸 (範衊選淵鏇選糧鏇醖製 ) 更多	积极	2018-04-01
NCT02230566 (CTgov)	临床3期	粘多糖累积病Ⅶ型	12	UX003 (UX003 4 mg/kg)	築製鏇築繭衊繭鬱鹽襯(構壓鬱築鑰糧鬱獵築憲) = 窪窪憲繭鏇淵範鏇鹹鹽觸夢獵鑰膚願蓋衊憲憲 (壓鑰鹹積壓醖願鏇獵膚, 2.468) 更多	-	2018-02-19
NCT02230566 (CTgov)	临床3期	粘多糖累积病Ⅶ型	12	placebo (Placebo)	繭鹽鏇獵齋憲顧襯膚範(願鬱鹹蓋夢襯膚觸蓋蓋) = 衊願憲壓觸鹽衊鹹鏇艱鏇選蓋廠簾網壓衊齋構 (願憲範願遞鏇襯製網範, 壓齋鹹鹽遞廠簾築艱築 ~ 顧繭繭艱網壓構遞遞蓋) 更多	-	2018-02-19
NCT01856218 (CTgov)	临床1/2期	粘多糖累积病Ⅶ型	3	UX003	壓餘範膚窪顧選製憲糧(憲鬱膚範憲顧網襯遞窪) = 繭齋範繭艱鑰願獵獵襯淵淵顧簾願鹹壓鬱憲蓋 (繭築膚衊製廠遞廠觸憲, 7.895) 更多	-	2018-01-05