This protocol has two parts - the Main Study which is 42 days in length and the Treatment Extension which allows the patients who complete the Main Study to continue receiving treatment with uridine triacetate. The purpose of this study is to replace oral administration of uridine with oral administration of uridine triacetate in patients with hereditary orotic aciduria who have received (or would reasonably be expected to receive) clinical benefit from treatment with exogenous uridine. It is also to document the continued clinical benefit of exogenous uridine when patients are switched from oral administration of uridine to oral administration of uridine triacetate.

开始日期2014-04-01

申办/合作机构

Wellstat Therapeutics Corp.

NCT00044785

/ Terminated临床1期

A Phase I Study of Escalating Doses of CPT-11 and 5fluorouracil (5FU) Plus PN401 With a Fixed Dose of Leucovorin in Patients With Solid Tumor Malignancies.

CPT-11 and 5Fluorouracil (5FU) combined with leucovorin has become the standard of care for colorectal cancer. PN401 permits treatment with higher than normal doses of 5FU, which could increase its therapeutic potential. It is hypothesized that adding PN401 to the CPT-11, 5FU, leucovorin regimen will reduce toxicity and will allow higher doses of 5FU to be well tolerated and therefore potentially increase effectiveness.

开始日期2002-08-01

申办/合作机构

Wellstat Therapeutics Corp.

NCT00024427

/ Completed临床3期

An Open Label, Randomized, Controlled, Phase III, Multi-center, Clinical Trial Of PN401 With High Dose 5-Fluorouracil (5FU) Versus Gemcitabine For Treatment Of Patients With Advanced Pancreatic Cancer

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Giving the drugs in different combinations may kill more tumor cells. Chemoprotective drugs such as triacetyluridine may protect normal cells from the side effects of chemotherapy. It is not yet known which chemotherapy regimen is more effective in treating pancreatic cancer.
PURPOSE: Randomized phase III trial to compare the effectiveness of fluorouracil plus triacetyluridine with that of gemcitabine in treating patients who have locally advanced or metastatic pancreatic cancer that cannot be treated with surgery.

开始日期2001-02-01

申办/合作机构

Wellstat Therapeutics Corp.

100 项与尿苷三乙酸酯相关的临床结果

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100 项与尿苷三乙酸酯相关的转化医学

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100 项与尿苷三乙酸酯相关的专利（医药）

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309

项与尿苷三乙酸酯相关的文献（医药）

2025-12-01·CANCER CHEMOTHERAPY AND PHARMACOLOGY

Dihydropyrimidine enzyme activity and its effect on chemotherapy toxicity: importance of genetic testing

Article

作者: Villano, John L ; Pandey, Deepali ; Roberts, Danielle ; Williams, Abby ; Shamaei Zadeh, Alexia

PURPOSE:

Patients with partial or complete DPD deficiency have decreased capacity to degrade fluorouracil and are at risk of developing toxicity, which can be even life-threatening.

CASE:

A 43-year-old man with moderately differentiated rectal adenocarcinoma on capecitabine presented to the emergency department with complaints of nausea, vomiting, diarrhea, weakness, and lower abdominal pain for several days. Laboratory findings include grade 4 neutropenia (ANC 10) and thrombocytopenia (platelets 36,000). Capecitabine is used as a component of first-line adjuvant therapy by approximately 2 million patients worldwide each year. Capecitabine is metabolized to fluorouracil via the enzyme dihydropyrimidine dehydrogenase (DPD). With worsening pancytopenia and diarrhea, genetic testing for DPD deficiency was sent. Prompt treatment with uridine triacetate was initiated for presumed DPD deficiency. Unfortunately, he passed away from an infectious complication and was later confirmed to have a heterozygous DPYD*2A mutation.

DISCUSSION:

Our case demonstrates uneven testing guidelines for DPD prior to initiating 5-FU chemotherapy, appropriateness of treating with uridine triacetate, and analysis of next-generation sequencing (NGS) on tumor samples and co-incidentally obtaining germline DPD deficiency status. Our case also highlights the severe clinical impact of having DPD deficiency even with early uridine triacetate therapy.

CONCLUSION:

It is our recommendation to perform DPD deficiency in curative intent cancer treatment and this information can increasingly be obtained in standard tumor NGS profiling, a growing norm in medical oncology.

2025-03-01·BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Review of the fluoropyrimidine antidote uridine triacetate

Review

作者: Thompson, Jack T. ; Dargan, Paul I. ; Wood, David M.

Abstract:

In 2015, the United States Food and Drug Administration (FDA) approved uridine triacetate to treat overdose and severe toxicity of the fluoropyrimidine chemotherapy agents 5‐fluorouracil (5‐FU) and its oral prodrug capecitabine. Uridine triacetate is as an oral prodrug of uridine that competes with cytotoxic fluoropyrimidine metabolites for incorporation into nucleotides. Two million people worldwide start fluoropyrimidine chemotherapy each year, with 20‐30% developing severe or life‐threatening adverse effects, often attributable to a genetic predisposition such as dihydropyrimidine dehydrogenase deficiency. Whilst genetic prescreening is recommended prior to starting fluoropyrimidine agents, this only prevents 20‐30% of early‐onset life‐threatening toxicity and so does not obviate the need for an antidote. Initial in‐human studies established that uridine triacetate more than doubles the maximum tolerated weekly 5‐FU bolus dose. A lack of clinical equipoise meant a placebo‐controlled phase III trial was not ethical and so the phase III trials used historical controls. These found that uridine triacetate improved survival in those with fluoropyrimidine overdose and severe toxicity from 16% to 94%, with 34% able to resume chemotherapy within 30 days. Five case reports of delayed fluoropyrimidine toxicity demonstrate improvement following uridine triacetate treatment 120‐504 h after last fluoropyrimidine administration, suggesting efficacy beyond the FDA licencing indications. Mechanistically uridine triacetate would be expected to be effective for overdose and severe toxicity of tegafur (a 5‐FU prodrug), but there are no published case reports describing this. Uridine triacetate is available internationally through an expanded access scheme and has been available in the UK since 2019 on a named patient basis.

2025-03-01·Clinical Drug Investigation

Economic Model of Uridine Triacetate Versus Supportive Care for the Treatment of Patients with Life-Threatening Early-Onset Severe Toxicity

Article

作者: Williams, Setareh A ; Ward, Suzanne ; Griffin, Amanda ; Garcia, Jorge J ; Barnard, Michelle ; Beers, Alice ; Bourque, Megan ; Reid, Paige ; Miragliotta, Salvatore ; Trepanier, Chantal

BACKGROUND:

Early-onset severe toxicity following the administration of 5-fluorouracil (5-FU) or capecitabine occurs in approximately 10-30% of patients receiving fluoropyrimidine therapy in the USA and is fatal to at least 0.5% of patients treated. Supportive care measures used to manage symptoms of toxicity are associated with extended hospital length of stay, high cost of care, and poor survival. Uridine triacetate is indicated as an emergency treatment for patients who exhibit early-onset, severe or life-threatening toxicity, and has been shown to significantly improve clinical outcomes. Despite its life-saving capability to reverse early-onset severe toxicity, uridine triacetate may be underutilized.

PURPOSE:

This study aims to evaluate the economic impact of uridine triacetate as a rescue therapy for adult patients from the US hospital payer perspective for early-onset severe toxicity, who are expected to die without treatment.

METHODS:

A decision tree model was developed to compare inpatient survival, hospital length of stay, and inpatient healthcare resource utilization for patients treated with and without uridine triacetate. Costs associated with hospitalization, including supportive care measures and monitoring were evaluated, considering medications and procedures commonly used to manage various severe toxicities experienced (e.g., gastrointestinal, hematological, etc.). The model compared the hypothetical current practice, in which approximately half of patients expected to die from early-onset severe toxicity receive uridine triacetate in addition to supportive care, with the proposed future practice in which all eligible patients receive uridine triacetate during their hospital stay. Hypothetical practical scenarios for US institutions were also considered.

RESULTS:

For each adult patient hospitalized for early-onset severe or life-threatening toxicity who would be expected to die without treatment, adoption of uridine triacetate as a rescue treatment was associated with clinical benefits, including increased inpatient survival (48.5%) and a 7.3-day reduction in total hospital length of stay per patient. Treatment of each additional patient with uridine triacetate was associated with an incremental cost of US$25,247 per patient. Seventy percent of the drug cost was offset by reduction in inpatient healthcare resources utilization. This cost offset is likely underestimated as it does not include additional savings from potential reimbursements associated with changes in hospital length of stay, readmissions and discounting. Hypothetical scenarios demonstrated that model outputs were most sensitive to changes in length of stay and hospitalization costs.

CONCLUSION:

Optimal treatment with uridine triacetate for all hospitalized patients in the USA expected to die from early-onset severe toxicity has the potential to improve inpatient survival at a minimal inpatient budget increase. The majority of the drug cost is offset by a reduction in the length of hospital stay and associated costs.

项与尿苷三乙酸酯相关的新闻（医药）

2023-12-11

·GlobeNewswire-Health Care

MaxCyte Announces CEO Transition and Updates Revenue Guidance for 2023

Doug Doerfler, President and CEO, to Retire Effective December 31, 2023; will continue to serve as an advisor to MaxCyte Maher Masoud, EVP, Head of Global Business Development and Chief Counsel, to Succeed Mr. Doerfler as President and CEO, Effective January 1, 2024 MaxCyte is reiterating 2023 expected core business revenue of $28-30 million, and the Company now expects to exceed prior SPL program-related guidance, with revenue of at least $10 million ROCKVILLE, Md., Dec. 11, 2023 (GLOBE NEWSWIRE) -- MaxCyte, Inc., (NASDAQ: MXCT; LSE: MXCT), a leading, cell-engineering focused company providing enabling platform technologies to advance the discovery, development and commercialization of next-generation cell therapeutics and innovative bioprocessing applications, today announced that Maher Masoud has been named President and Chief Executive Officer of MaxCyte, succeeding Doug Doerfler, who will retire as President and Chief Executive Officer, effective January 1, 2024. Mr. Masoud, currently the Company’s EVP, Head of Global Business Development and Chief Counsel, will also serve as a director on MaxCyte’s Board of Directors. Mr. Doerfler will step down from the Board of Directors upon his retirement and will remain an advisor to the Company. Mr. Doerfler founded MaxCyte in 1999 and served as CEO for 24 years, leading the Company from initial technology concept through to the commercialization of its flow electroporation technology. Mr. Doerfler’s team built MaxCyte into a leading cell-engineering company with a significant number of customers and partners, leveraging MaxCyte’s non-viral delivery platform in their pre-clinical and clinical work. During his tenure, Mr. Doerfler steered MaxCyte through numerous key events including initial public offerings on the UK AIM Exchange and U.S. NASDAQ Exchange, the development of the ExPERT instrument portfolio, and supporting the approval of CASGEVY™, the first non-viral cell therapy product approved by the FDA. “Consistent with the Board’s succession planning process, the Board identified Maher Masoud as a leader with a deep understanding of our technology and the cell and gene therapy industry who can continue to build on MaxCyte’s accomplishments. Having worked closely with Maher over the past seven years, we are confident in his abilities to assume the role of President and Chief Executive Officer of MaxCyte," said Richard Douglas, chairman of the Board. "On behalf of the Board, I would also like to acknowledge Doug’s exceptional contributions to, and leadership of, MaxCyte over the past 24 years. Throughout Doug’s tenure, MaxCyte grew to become the partner of choice to leading cell and gene therapy drug developers." "It has been a privilege being part of MaxCyte's exceptionally talented team over the past 24 years as we've worked together to develop our proprietary flow electroporation technology, building MaxCyte into the successful company it is today," said Mr. Doerfler. "Maher has been instrumental in key initiatives at MaxCyte, including building out our roster of Strategic Platform Licenses. He has also played a key role across the operating areas of the Company, including sales, marketing, and business development. I am confident in the Board’s choice and Maher’s ability to execute and drive MaxCyte’s continued success in the years to come." Mr. Masoud brings more than 25 years of experience in the biopharmaceutical industry, including 17 years as an attorney and general counsel, to his new role at MaxCyte. Mr. Masoud has most recently served as EVP, Head of Global Business Development and Chief Counsel at MaxCyte. During his tenure, MaxCyte’s SPL partnership model grew to include 23 partners, as of December 2023. Mr. Masoud started his biopharmaceutical career as a research associate with Glen Research, a Maravai company, before joining Human Genome Sciences as Director and Corporate Counsel, overseeing legal activities for the company’s global clinical trials, until its acquisition by GlaxoSmithKline. Prior to joining MaxCyte, he oversaw the operations of six business subsidiaries at Wellstat, a life science holding company. During his tenure at Human Genome Sciences and Wellstat, Mr. Masoud supported the launch of three FDA approved therapies, Benlysta®, Vistogard® and Xuriden®. Mr. Masoud earned his Juris Doctor degree from Michigan State University College of Law after completing his Bachelor of Science degree in cell and molecular biology genetics from the University of Maryland. "l am honored by the opportunity to lead MaxCyte. While working closely with Doug, the executive leadership team, and the Board over the past seven years, MaxCyte has grown to become the leading non-viral cell engineering company,” said Mr. Masoud. “I firmly believe in the broad and growing commercial opportunity for MaxCyte’s product line and the potential of our partners to bring meaningful product therapies to market. I look forward to leading our stellar team and continuing to build long-term shareholder value.” Updated 2023 Revenue Guidance MaxCyte is reiterating 2023 expected core business revenue of $28-30 million, and the company now expects to exceed prior SPL program-related guidance, with revenue of at least $10 million. Core business revenue consists of sales and leases of instrument and disposables to cell therapy and drug discovery customers and excludes any milestone revenues under SPL programs. About MaxCyte At MaxCyte, we pursue cell engineering excellence to maximize the potential of cells to improve patients’ lives. We have spent more than 20 years honing our expertise by building best-in-class platforms, perfecting the art of the transfection workflow, and venturing beyond today’s processes to innovate tomorrow’s solutions. Our ExPERT™ platform, which is based on our Flow Electroporation® technology, has been designed to support the rapidly expanding cell therapy market and can be utilized across the continuum of the high-growth cell therapy sector, from discovery and development through commercialization of next-generation, cell-based medicines. The ExPERT family of products includes: four instruments, the ATx™, STx™, GTx™ and VLx ™; a portfolio of proprietary related processing assemblies or disposables; and software protocols, all supported by a robust worldwide intellectual property portfolio. By providing our partners with the right technology platform, as well as scientific, technical, and regulatory support, we aim to guide them on their journey to transform human health. Learn more at maxcyte.com and follow us on Twitter and LinkedIn. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including but not limited to, statements regarding the leadership transition and statements regarding expected core business revenue and SPL Program-related revenue for the year ending December 31, 2023. The words "may," “might,” "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," “expect,” "estimate," “seek,” "predict," “future,” "project," “prospect,” "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks associated with the management transition, the timing and outcome of our customers’ ongoing and planned clinical trials; the adequacy of our cash resources and availability of financing on commercially reasonable terms; general market and economic conditions that may impact investor confidence in the biopharmaceutical industry and affect the amount of capital such investors provide to our current and potential partners; and market acceptance and demand for our technology and products. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2022, filed with the Securities and Exchange Commission on March 15, 2023, as well as in discussions of potential risks, uncertainties, and other important factors in our most recent Quarterly report on Form 10-Q and the other filings that we make with the Securities and Exchange Commission from time to time. These documents are available through the Investor Menu, Financials section, under “SEC Filings” on the Investors page of our website at http://investors.maxcyte.com. Any forward-looking statements represent our views only as of the date of this press release and should not be relied upon as representing our views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. MaxCyte Contacts: US IR Adviser Gilmartin Group David Deuchler, CFA +1 415-937-5400 ir@maxcyte.com US Media RelationsSeismicValerie Enes+1 408-497-8568 Nominated Adviser and Joint Corporate Broker Panmure Gordon Emma Earl / Freddy Crossley Corporate Broking Rupert Dearden+44 (0)20 7886 2500 UK IR AdviserICR ConsiliumMary-Jane ElliottChris Welsh +44 (0)203 709 5700maxcyte@consilium-comms.com

高管变更细胞疗法并购

2023-07-13

·BioSpace

SERB Pharmaceuticals acquires Vistogard® and Xuriden® from Wellstat Therapeutics

West Conshohocken, PA, July 13, 2023 (GLOBE NEWSWIRE) -- SERB Pharmaceuticals, a global specialty pharmaceutical company, announced today that it has acquired two life-saving medicines, Vistogard® (uridine triacetate) and Xuriden® (uridine triacetate), from Wellstat Therapeutics. Both medicines are commercially available in the US and currently distributed across Europe on a named patient or compassionate use basis. Vistogard® is an emergency treatment for adult and pediatric patients following a fluorouracil (5-FU) or capecitabine overdose regardless of the presence of symptoms, or who exhibit early-onset, severe or life- threatening toxicity affecting the cardiac or central nervous system, and/or early-onset, unusually severe adverse reactions (e.g., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of 5-FU or capecitabine administration.[i] 5-FU and capecitabine are chemotherapy agents commonly used to treat a wide variety of solid tumors. Xuriden® is a treatment for adult and pediatric patients with hereditary orotic aciduria (HOA)[ii], an ultra-rare, orphan genetic disorder associated megaloblastic anemia as well as failure to thrive, developmental delay, epilepsy, susceptibility to infection, and orotic acid crystals in the urine.[iii] Jeremie Urbain, President of SERB Pharmaceuticals, said: “We’re proud to add these two treatments to our expanding portfolio of medicines for complex, life-threatening conditions. Acquiring these medicines is in line with our strategy to focus on emergency care and rare diseases.” The products will be sold in the US by BTG Pharmaceuticals, SERB’s US entity. The products will transition seamlessly from Wellstat to BTG Pharmaceuticals and will continue to be available from the same distributors via the same ordering processes: To order Vistogard®, please call 1-800-914-0071. Vistogard® is distributed through Cardinal Health Specialty Pharmaceutical Distribution, available 24 hours/day, 7 days/week. Vistogard® and Xuriden® are available for dispensing through BioMatrix Specialty Pharmacy. To reach BioMatrix Specialty Pharmacy, please call: 844-374-0604. BTG Pharmaceuticals, a SERB company, will promote Vistogard® and continue promoting Voraxaze® through its oncology field force in the US. The companies did not disclose specific financial terms. Today’s news follows the announcement in May that SERB has acquired the exclusive US rights to bentracimab, which is in development as a ticagrelor reversal agent. About Fluorouracil (5-FU) and capecitabine toxicity 5-FU and capecitabine are both trusted chemotherapies received by thousands of patients, often in combination with other chemotherapy agents or radiation. They have a history of success in treating a range of cancers including breast, colorectal (bowel), gastric (stomach), and pancreatic cancer. According to the National Institute of Health (NIH), about 275,000 patients in the US receive 5-FU every year. Capecitabine, a prodrug that is enzymatically converted to 5-FU within the body, has about 60,000 treatment packages sold per year. Approximately 3% of these patients develop serious toxicities and more than 8,000 toxic reactions and 1,300 deaths every year are attributed to 5-FU and capecitabine..[iv] About HOA Hereditary orotic aciduria (HOA) is a very rare inborn error of pyrimidine metabolism caused by mutations reducing the activity of uridine monophosphate synthase (UMPS), which converts orotic acid to UMP. Uridine deficiency underlies many of the symptoms of HOA. When untreated, patients with HOA may develop a blood (hematologic) disorder called megaloblastic anemia as well as exhibiting failure to thrive, developmental delay, epilepsy, susceptibility to infection and orotic acid crystals in the urine (crystalluria).iii About SERB Pharmaceuticals SERB is a global specialty pharmaceutical company with a growing portfolio of medicines for emergency care and rare diseases. For over 30 years we have made treating these complex and life-threatening conditions possible, supporting clinicians, healthcare systems and governments while offering hope to patients and their families. SERB has the broadest antidote portfolio in the world, including medical countermeasures for chemical, biological, radiological and nuclear (CBRN) risks. As a fully integrated company, we have the experience and capabilities to acquire, develop, and manufacture our medicines to the highest standards, and make them available worldwide through our secure supply chain. SERB operates in the US as BTG Pharmaceuticals. Learn more at . About Vistogard® Indication VISTOGARD® is indicated for the emergency treatment of adult and pediatric patients: following a fluorouracil or capecitabine overdose regardless of the presence of symptoms, or who exhibit early-onset, severe or life- threatening toxicity affecting the cardiac or central nervous system, and/or early onset, unusually severe adverse reactions (e.g., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of fluorouracil or capecitabine administration. Limitations of use VISTOGARD® is not recommended for the non–emergent treatment of adverse reactions associated with fluorouracil or capecitabine because it may diminish the efficacy of these drugs. The safety and efficacy of VISTOGARD initiated more than 96 hours following the end of fluorouracil or capecitabine administration have not been established. Important Safety Information In clinical studies, adverse reactions occurring in >2% of patients receiving VISTOGARD® included vomiting (10%), nausea (5%), and diarrhea (3%). One person receiving uridine triacetate experienced grade 3 nausea and vomiting. VISTOGARD® was discontinued for adverse reactions in 2 (1.4%) patients. See full prescribing information. About Xuriden® Indication XURIDEN® is indicated in adult and pediatric patients for the treatment of hereditary orotic aciduria. Important Safety Information Contraindications – None. Warning and Precautions – None. Adverse reactions – No adverse reactions were reported with XURIDEN in patients with HOA. Pregnancy – There are no available data on XURIDEN use in pregnant women to inform drug-associated risk. Lactation– There are no data on the presence of XURIDEN in human milk, the effect on the breast-fed infant or the effect on milk production. The development and health benefits of breastfeeding should be considered along with the mother’s clinical need for XURIDEN and any potential adverse effects on the breastfed infant from XURIDEN or from the underlying maternal condition. Pediatric – The safety and effectiveness of XURIDEN have been established in pediatric patients. See full prescribing information. References [i] Wellstat Therapeutics Corporation (2017). Vistogard (uridine triacetate) US Package Insert. [ii] Wellstat Therapeutics Corporation (2019). Xuriden (uridine triacetate) US Package Insert. [iii] Al Absi HS, Sacharow S, Al Zein N, Al Shamsi A, Al Teneiji A. Hereditary orotic aciduria (HOA): A novel uridine-5-monophosphate synthase (UMPS) mutation. Mol Genet Metab Rep. 2021 Jan 9;26:100703. doi: 10.1016/j.ymgmr.2020.100703. PMID: 33489760; PMCID: PMC7807243. [iv] Santos C, Morgan BW, Geller RJ. The successful treatment of 5-fluorouracil (5-FU) overdose in a patient with malignancy and HIV/AIDS with uridine triacetate. Am J Emerg Med. 2017 May;35(5):802.e7-802.e8. doi: 10.1016/j.ajem.2016.11.038. Epub 2016 Nov 15. PMID: 27884585. Christopher W Sampson SERB Pharmaceuticals +44 7773 251 178 chris.sampson@serb.com

引进/卖出上市批准

2022-08-05

·药明康德

礼来阿尔茨海默病抗体疗法和BTK抑制剂获优先审评资格，有望加速上市进程

今日，礼来（Eli Lilly and Company）在第2季度财报中宣布，其用于治疗阿尔茨海默病的抗体疗法donanemab的生物制品许可（BLA）申请已经获得美国FDA授予的优先审评资格。FDA将使用加速批准通道对这一BLA进行审评。同时，该公司的布鲁顿氏酪氨酸激酶（BTK）抑制剂pirtobrutinib也获得优先审评资格，用于治疗经治套细胞淋巴瘤患者。这一审评也将使用加速批准通道。 Donanemab是一款靶向β淀粉样蛋白的单克隆抗体，它在2期临床试验中达到主要终点，将早期阿尔茨海默病患者的临床进展速度延缓32%。在生物标志物研究中，donanemab不但导致淀粉样蛋白沉积的快速清除，而且显著降低患者血浆中P-tau217的水平。这是礼来开发的一个在研血液生物标志物，与淀粉样蛋白和tau蛋白病理，以及AD的诊断相关。即使在停止donanemab用药一年后，P-tau217水平仍然显著降低。 ▲Donanemab停药一年后淀粉样蛋白沉积和P-tau217水平的降低仍然得到维持（图片来源：礼来官网） Donanemab目前正在3期临床试验中接受检验，用于治疗出现症状的早期阿尔茨海默病患者，预计主要终点结果将在明年年初获得。 Pirtobrutinib是一款非共价选择性BTK抑制剂。在去年的美国血液学会年会上发布的结果显示，在100名曾经接受过BTK抑制剂治疗的套细胞淋巴瘤患者中，pirtobrutinib达到51%的客观缓解率（ORR），在11名未接受过BTK抑制剂治疗的患者中，ORR为82%。 ▲Pirtobrutinib治疗套细胞淋巴瘤患者的临床试验结果（图片来源：礼来官网）优先审评资格和加速批准通道均为FDA用于加快创新疗法获批速度而设立的资格认定，加速批准通道意味着FDA可以基于替代终点的结果对监管申请进行评估和批准。优先审评资格意味着FDA将在6个月内对监管申请做出回复，比标准审评流程减少4个月。参考资料：[1] Lilly Reports Second-Quarter Financial Results, Highlights Momentum of New Medicines and Pipeline Advancementshttps://investor.lilly.com/news-releases/news-release-details/lilly-reports-second-quarter-financial-results-highlights 内容来源于网络，如有侵权，请联系删除。

优先审批

100 项与尿苷三乙酸酯相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D09985	尿苷三乙酸酯	A16AX13	DB09144

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
药物中毒	美国	BTG International, Inc.	2015-12-11
遗传性乳清酸尿症	美国	BTG International, Inc.	2015-09-04

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
不能切除性胰腺癌	临床3期	美国	Wellstat Therapeutics Corp.	2001-02-01
不能切除性胰腺癌	临床3期	加拿大	Wellstat Therapeutics Corp.	2001-02-01
躁郁症	临床2期	美国	Repligen Corp.	2006-03-01
遗传性线粒体呼吸链疾病	临床1期	-	Repligen Corp.	2003-05-08
结直肠癌	临床1期	美国	Wellstat Therapeutics Corp.	2002-08-01
5α-氟尿嘧啶毒性	临床前	美国	Wellstat Therapeutics Corp.	2010-05-20

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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


NCT02110147 (CTgov)	临床3期	遗传性乳清酸尿症	4	uridine triacetate (Uridine Triacetate)	齋襯積鬱蓋壓淵窪糧壓 = 獵網製衊衊鏇廠壓淵餘遞製選範淵鏇齋淵齋憲 (鬱簾衊網範壓淵夢鹽鬱, 鬱築醖夢鏇鹹壓簾襯構 ~ 繭襯繭築簾壓壓齋獵獵) 更多	-	2017-06-28
NCT02110147 (CTgov)	临床3期	遗传性乳清酸尿症	4	uridine triacetate (Single Arm)	憲膚齋衊構鬱築網網憲(蓋鬱醖廠壓醖鏇鬱鬱選) = 鬱廠膚選醖齋願鏇鑰製積鬱鏇築構窪夢鹹繭蓋 (廠醖網齋夢夢壓壓鏇觸, 積顧鬱遞鏇齋衊網壓網 ~ 積艱醖壓憲範鬱網繭憲) 更多	-	2017-06-28
ASCO2016	N/A	药物过量	173	Uridine triacetate	網蓋簾餘糧鬱簾繭構廠(簾鹽窪夢積繭鏇餘繭網) = 鬱壓繭鑰鏇鬱醖繭膚網壓蓋淵顧鹹壓夢顧簾蓋 (夢夢獵壓襯觸鑰鹹網衊 )	积极	2016-05-20
ESMO_GI2013	N/A	dihydropyrimidine dehydrogenase (DPD) deficiency	100	Uridine triacetate	顧衊糧壓鏇選觸獵醖願(襯鏇願夢顧構繭觸糧範) = Mild or no adverse events were attributed to uridine triacetate 壓衊簾顧願糧鬱選醖淵 (淵憲顧鑰糧淵顧鑰齋窪 )	-	2013-07-03
NCT00004233 (Pubmed \| Invest New Drugs)	临床2期	胃腺癌	65	PN401, 5-FU, and leucovorin	淵鏇醖淵壓繭鹹憲築製(顧鹽齋選遞膚築願襯願) = two deaths judged possibly related to treatment; one in a patient who experienced a variety of Grade 2 gastrointestinal toxicities and died at home with an unknown cause of death; and a second patient who also died at home, and for whom treatment-related sepsis could not be ruled out 簾餘獵膚顧鬱艱網願餘 (衊醖遞範積獵鑰製蓋衊 ) 更多	-	2006-11-01