A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED,PARALLEL STUDY TO ASSESS THE EFFICACY,SAFETY, TOLERABILITY, PK, AND BIOMARKER EFFECTS OF PTC857 IN ADULT SUBJECTS WITH AMYOTROPHIC LATERAL SCLEROSIS (CARDINALS)

开始日期2023-10-12

申办/合作机构-

JPRN-jRCT2011230040 / Recruiting临床2期IIT

A PHASE 2, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED,PARALLEL STUDY TO ASSESS THE EFFICACY,SAFETY, TOLERABILITY, PK, AND BIOMARKER EFFECTS OFPTC857 IN ADULT SUBJECTS WITH AMYOTROPHIC LATERALSCLEROSIS (CARDINALS)

开始日期2023-09-29

申办/合作机构-

NL-OMON51552 / CompletedN/A

A Phase 1 Study to Investigate the Absorption, Metabolism, and Excretion of PTC857 Following Oral Administration of a Single Dose of 14C-PTC857 at Steady-State Conditions in Healthy Male Volunteers. - AME study with 14C-PTC857.

开始日期2022-08-18

申办/合作机构

PTC Therapeutics, Inc.

100 项与 Utreloxastat 相关的临床结果

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100 项与 Utreloxastat 相关的转化医学

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100 项与 Utreloxastat 相关的专利（医药）

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项与 Utreloxastat 相关的文献（医药）

2023-02-01·Clinical Pharmacology in Drug Development

First‐in‐Human Studies of Pharmacokinetics and Safety of Utreloxastat (PTC857), a Novel 15‐Lipooxygenase Inhibitor for the Treatment of Amyotrophic Lateral Sclerosis

Article

作者: Klein, Matthew ; Kong, Ronald ; Kaushik, Diksha ; Trimmer, Jeffrey ; Dali, Mandar ; Ma, Jiyuan ; Gao, Lan ; Giannousis, Peter ; Tansy, Aaron ; Johnston, Mayzie ; Golden, Lee ; Thoolen, Martin ; Latham, Joey

AbstractUtreloxastat (PTC857) is a 15‐lipoxygenase inhibitor being developed to treat amyotrophic lateral sclerosis. This first‐in‐human study investigated the safety and pharmacokinetics of utreloxastat in healthy volunteers (N = 82) in a double‐blind, placebo‐controlled trial. The effects of a single ascending dose (100–1000 mg), multiple ascending doses (150–500 mg), and food (500 mg) on the pharmacokinetics and safety of utreloxastat were evaluated. Following single doses, the time to maximum plasma concentration (Cmax) was observed ≈4 hours after dosing and the terminal half‐life ranged from 20 to 25.3 hours. The Cmax and area under the concentration‐time curve (AUC) increased slightly over dose proportionally. Following multiple doses (once daily/twice daily), the apparent clearance reduced and terminal half‐life was ≥33 hours. There was no apparent difference of exposure following morning or evening doses. Varying diets increased the Cmax and AUCs of utreloxastat but did not alter time to Cmax. There were no gender‐based differences in exposure. Utreloxastat showed no marked safety signal following single doses up to 1000 mg and multiple doses over 14 days of 500 mg once daily or 250 mg twice daily. The results support further development of utreloxastat for the treatment of patients with amyotrophic lateral sclerosis at a 250‐mg twice‐daily dose administered with food.

项与 Utreloxastat 相关的新闻（医药）

2024-11-07

·PRNewswire

PTC Therapeutics Provides Corporate Update and Reports Third Quarter 2024 Financial Results

– Strong revenue performance, supporting increase in full-year revenue guidance to $750-800 million – – Regulatory filings for sepiapterin, Translarna™ and AADC gene therapy under review by FDA – – On track to achieve remaining 2024 clinical and regulatory milestones, including NDA submission for vatiquinone – WARREN, N.J., Nov. 7, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ending September 30, 2024. "I am proud of our team's strong execution," said Matthew Klein, M.D., Chief Executive Officer. "We continue to achieve excellent revenue performance allowing us to raise full-year revenue guidance. In addition, we have submitted three approval applications to FDA so far this year, all of which have been accepted for review, and plan a fourth submission for vatiquinone for Friedreich ataxia in December. We are on our way to accomplishing all of our 2024 objectives and are in a strong financial position to execute on our long-term strategic plans." Key Corporate Updates: Third quarter 2024 total revenue of $197 million Third quarter 2024 revenue for the DMD franchise was $124 million Translarna™ (ataluren) net product revenue was $72 million, driven by continued access for patients in existing geographies, continued geographic expansion, high compliance and adherence, and dose adjustments. Emflaza® (deflazacort) net product revenue was $52 million, driven by new patient starts, continued brand loyalty, high compliance and adherence, and dose adjustments. Key Clinical and Regulatory Milestones: PTC submitted an NDA to the FDA for sepiapterin for the treatment of PKU. The file was accepted for review, and the regulatory action date is set for July 29, 2025. PTC resubmitted the NDA for Translarna for the treatment of nmDMD; the file was accepted, and a regulatory action date was not provided. The BLA for PTC's gene therapy for AADC deficiency remains on schedule for a regulatory action date of November 13, 2024. PTC received Fast Track Designation for the PTC518 Huntington's disease program. PTC plans to submit an NDA for vatiquinone for the treatment of Friedreich ataxia in December 2024. PTC expects to share topline data for the CardinALS trial of utreloxastat for the treatment of ALS in the fourth quarter of 2024. Third Quarter 2024 Financial Highlights: Total revenues were $196.8 million for the third quarter of 2024, compared to $196.6 million for the third quarter of 2023. Total revenue includes net product revenue across the commercial portfolio of $135.4 million for the third quarter of 2024, compared to $144.0 million for the third quarter of 2023. Total revenue also includes royalty and manufacturing revenue of $61.4 million in the third quarter of 2024, compared to $52.5 million for the third quarter of 2023. Translarna net product revenues were $72.3 million for the third quarter of 2024, compared to $69.0 million for the third quarter of 2023. Emflaza net product revenues were $51.9 million for the third quarter of 2024, compared to $67.4 million for the third quarter of 2023. Roche reported Evrysdi® 2024 year-to-date sales of approximately CHF 1.2 billion, resulting in royalty revenue of $61.4 million to PTC for the third quarter of 2024, compared to $50.2 million for the third quarter of 2023. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $161.4 million for the third quarter of 2024, compared to $164.2 million for the third quarter of 2023. The decrease in research and development expenses reflects strategic portfolio prioritization, as PTC continues to focus its resources on its differentiated, high-potential R&D programs. Included in third quarter 2024 R&D expense is $50.0 million in regulatory success-based milestones related to the Censa acquisition. Non-GAAP R&D expenses were $152.0 million for the third quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $150.2 million for the third quarter of 2023, excluding $14.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $73.5 million for the third quarter of 2024, compared to $80.9 million for the third quarter of 2023. The decrease in selling, general and administrative expenses reflects lower employee costs as a result of the reduction in workforce in 2023. Non-GAAP SG&A expenses were $63.1 million for the third quarter of 2024, excluding $10.3 million in non-cash, stock-based compensation expense, compared to $67.9 million for the third quarter of 2023, excluding $13.0 million in non-cash, stock-based compensation expense. Change in the fair value of deferred and contingent consideration was a loss of $0.7 million for the third quarter of 2024, compared to a loss of $1.5 million for the third quarter of 2023. Tangible asset impairment and losses (gains) on transactions, net, was a loss of $1.8 million for the third quarter of 2024, compared to $0.0 million for the third quarter of 2023. These results were primarily related to fixed asset impairments. Net loss was $106.7 million for the third quarter of 2024, compared to net loss of $133.0 million for the third quarter of 2023. Cash, cash equivalents, and marketable securities was $1,013.4 million on September 30, 2024, compared to $876.7 million on December 31, 2023. Shares issued and outstanding as of September 30, 2024, were 76,952,124. PTC Updates Full Year 2024 Financial Guidance: PTC anticipates total revenues for full year 2024 to be between $750 million and $800 million. PTC anticipates GAAP R&D and SG&A expenses for full year 2024 to be between $740 million and $835 million, including expected R&D expense milestone payments of up to $65 million. PTC anticipates non-GAAP R&D and SG&A expenses for full year 2024 to be between $660 million and $755 million, including expected R&D expense milestone payments of up to $65 million and excluding estimated non-cash, stock-based compensation expense of $80 million. PTC anticipates up to $90 million of payments for full year 2024 upon achievement of potential regulatory success-based milestones from previous acquisitions, of which up to $65 million will be recorded as R&D operating expenses. As of September 30, 2024, $85 million in milestones have been achieved, of which $65 million has been recorded as R&D operating expenses and $20 million as contingent consideration payable. Non-GAAP Financial Measures: In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms: AADC: Aromatic l-Amino Acid Decarboxylase ALS: Amyotrophic Lateral Sclerosis BLA: Biologics License Application CHF: Confoederatio Helvetica Francs (Swiss francs) DMD: Duchenne Muscular Dystrophy FA: Friedreich Ataxia FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's Disease NDA: New Drug Application nmDMD: Nonsense Mutation Duchenne Muscular Dystrophy PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder: To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at . A replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at and follow us on Facebook, X, and LinkedIn. For More Information: Investors: Ellen Cavaleri +1 (615) 618-6228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Updates Full Year 2024 Financial Guidance", including with respect to (i) 2024 total revenue guidance, (ii) 2024 GAAP and non-GAAP R&D and SG&A expense guidance and (iii) 2024 acquisition related milestone payment guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions, including whether the European Commission adopts the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) for the conditional marketing authorization for Translarna in the EEA, or PTC's ability to identify other potential mechanisms by which it may provide Translarna to nmDMD patients in the EEA; PTC's ability to use the clinical data from its international drug registry study and real-world evidence concerning Translarna's benefits to support a continued marketing authorization for Translarna for the treatment of nmDMD in the EEA; PTC's ability to use the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; expectations with respect to Upstaza, including any regulatory submissions and potential approvals, commercialization, manufacturing capabilities, the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to sepiapterin, including any regulatory submissions and potential approvals, commercialization, the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; the timing of and actual expenses incurred in connection with the discontinuation of PTC's preclinical and early research programs in gene therapy and reductions in workforce, which may be in different periods and may be materially higher than estimated; the savings that may result from the discontinuation of PTC's strategic pipeline prioritization and reductions in workforce, which may be materially less than expected; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna, Emflaza, Upstaza, Evrysdi, Tegsedi, Waylivra or sepiapterin. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 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财报申请上市

2024-05-24

·BioSpace

Ferroptosis Targeting Therapy Market Opportunity and Clinical Trials Insight

“Currently More Than 10 Ferroptosis Targeting Drugs Are In Clinical Trials” In the intricate landscape of cancer therapeutics, the spotlight has shifted towards reducing the risk of cancer death, unveiling novel opportunities for precision interventions. In the recent years, the discovery of ferroptosis targeting therapies has emerged as a game changing player for the treatment of various malignancies, such as cancer diseases, autoimmune, inflammatory, eye diseases, kidney ailments, cardiovascular disorders, etc. In dichotomy with known regulated cell death pathways, like necrosis and apoptosis, ferroptosis is an innovative form of cell death which is categorized by iron ion gathering as well as considerable lipid peroxidation mediated membrane damage. Since, the conventional therapies, like radiotherapy, chemotherapy or targeted therapy, have illustrated significant response in the treatment of cancer malignancies; however, the come along with one of the key difficulties i.e. development of drug resistance. To prevent the progression of drug resistance, ferroptosis cancer therapy can be considered as promising approach. Download Research: Furthermore, combination of ferroptosis with radiotherapy, chemotherapy in conjugation with immunotherapy has displayed auspicious results in preclinical studies. Additionally, the data from modern studies suggests that ferroptosis targeting therapies can also be utilized with other contemporary therapy, like nanotechnology, photodynamic therapy, magnetic field, sono dynamic therapy, for the management of various ailments, including neurodegenerative diseases, viral infections, cancer, autoimmune and list goes on. Few reasons which influence the market growth of ferroptosis targeting therapies include rise in research and development, upsurge in preclinical, translational as well as clinical trials, mounting collaborations together with investments, FDA designations, increasing cancer and other diseases incidences and fatalities. For instance, Utreloxastat, also known as PTC857, is a 15 lipoxygenase inhibitor developed by PTC Therapeutics has received EMA Orphan Medicinal Product, in 2022, for the treatment of nuemrus patietns suffering from amyotrophic lateral sclerosis. Moreover, Vatiquinone (PTC-743) has also received FDA and EMA Orphan Drug designation as well as FDA Fast Track designation to treat Friedreich’s ataxia (FA). Coupled with that, scientific progressions in areas like drug delivery systems, nanoparticle formulations, and targeted therapies have facilitated the development of more effective and specific ferroptosis targeting agents, contributing to market expansion. Additionally, increasing research and development investments from pharmaceutical companies, academic institutions, and research organizations have recognized the potential of ferroptosis targeting therapies and are driving innovation in addition to ferroptosis market growth. Apart from that, many grants in order to encourage and initiate surplus studies for ferroptosis targeting therapies have been received to numerous scientists. For instance, professor at University of Würzburg, based in Germany, has received ERC Consolidator Grant worth EUR 2 Million (US$ 2.15 Million) to research the regulation of ferroptosis in November 2023. In spite of promising results witnessed in ferroptosis targeting cell therapies, the global market for this innovative modality faces several significant challenges, such as long term safety and efficacy concerns, translation of preclinical findings into clinical applications, competition from other therapies, complex and multifaceted nature of ferroptosis which must be addressed to drive further growth and widespread adoption. As of May 2024, the preclinical in conjugation with clinical research market of global ferroptosis targeting cell therapy for the management of cancer, autoimmune and neurodegenerative diseases have reached late phase of development; whereas ferroptosis targeting therapies for viral infections, cardiovascular diseases, eye illness and others are in early phase studies. Even though, the clinical trials market has increased, currently the US dominating ferroptosis targeting therapy realm. At last, with the increasing prevalence of cancer along with other diseases, including autoimmune as well as inflammatory diseases, viral, bacterial, diabetes, etc. at an alarming gait urges the need to develop better as well as groundbreaking ferroptosis targeting therapies which could provide cure in addition to anticipation amidst the populace suffering from cancer across the globe. Furthermore, an inclining market trend towards utilizing the realm of ferroptosis targeting therapies has been observed due to involvement of various stakeholders from pharmaceutical as well as biotech companies such as MitoImmune Therapeutics, Kojin, PTC Therapeutics, Sumitomo Pharma and Prothegen. With the current market scenario, the future of the ferroptosis targeting therapies market looks promising and is expected to multiple further as evident from the growing clinical trials, mounting research and development and involvement of various stakeholders in the domain.

孤儿药快速通道免疫疗法

2024-05-02

·药事纵横

盘点值得关注的5种中期ALS 药物

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。在经历了长达数年的空窗期之后，随着2022年 Amylyx 的 Relyvrio 和2023年Biogen 的Ionis' Qalsody 的批准，肌萎缩侧索硬化症（ALS）领域的进展开始加速。但现在，这一进展似乎已经停滞不前。本月早些时候，在III期试验失败后，Amylyx宣布将 Relyvrio从市场上撤出，并密切关注来自Seelos Therapeutics， Sanofi和Denali Therapeutics的临床试验，他们的最近错过了主要终点。“我们从很多试验到突然之间，现在没有那么多，或者它们很小或很早，”马萨诸塞州总医院神经病学系主任、领先的ALS研究人员Merit Cudkowicz表示。在2023年10月撤回细胞疗法NurOwn的生物制品许可申请后，BrainStorm Cell Therapeutics 已经达成协议与FDA合作设计IIIb期试验。与此同时，Cleene预计将于今年启动其研究性金纳米晶体悬浮液CNM-Au8的III期试验。但是，尽管后期ALS管线可能已经枯竭，但仍有超过100名在研候选人处于临床开发的各个阶段。最终，Cudkowicz说，“我们需要更多地关注生物学。这就是阻碍我们前进的原因。它不了解什么是最好的目标。她说，现在有一个很大的推动力，即发现更多的生物标志物，以更好地了解如何对患者进行分层以获得最佳临床结果。小编总结针对ALS病理学各个方面的五种中期疗法。Denali Therapeutics的DNL343幸运的是Denali这家总部位于加利福尼亚州南旧金山的公司在其研究疗法的箭袋中还有另一支箭。Denali目前正在招募DNL343的IIb/III期试验，DNL343是真核起始因子2B（eIF2B）的小分子激活剂。1月，Denali宣布完成该试验的入组是2024年的关键里程碑。DNL343旨在通过干扰应激颗粒形成和TDP-43聚集来抑制细胞综合应激反应并预防或减缓疾病进展，这几乎存在于所有ALS患者中。2023年4月，Denali来自一项Ib期试验数据显示DNL343“在表现出对与综合应激反应相关的生物标志物的强大抑制的剂量下通常耐受性良好。Calico Life Sciences的ABBV-CLS-7262比如DNL343，Calico Life Sciences的药物（与Abbvie合作开发）是一种针对综合应力响应途径的EIF2B激活剂。ABBV-CLS-7262是2014年Abbvie和Google支持的Calico之间达成的一项交易的果实，以开发与年龄相关疾病的新型治疗方法。ABBV-CLS-7262目前正在Healey ALS平台试验中进行评估，Cudkowicz是该试验的首席研究员。II/III期双盲试验包括约300名患者，将持续六个月，随后是治疗延长期。Cudkowicz说，招募现已完成，试验预计将于今年10月结束。NeuroSense Therapeutics的PrimeC本月早些时候，在美国神经病学学会（American Academy of Neurology）的年会上，NeuroSense Therapeutics（神经感知治疗学）公布来自IIb期 PARADIGM 试验PrimeC的数据，一种FDA批准的两种药物环丙沙星和塞来昔布的新配方。PrimeC旨在通过调节microRNA合成、减少神经炎症和影响铁积累来治疗ALS。Cudkowicz是该试验的首席研究员之一，他表示，在功能，呼吸和神经丝生物标志物水平方面，结果“朝着正确的方向发展”。2023年12月，NeuroSense从为期六个月的双盲试验中公布的营收结果表明PrimeC达到了该研究的主要安全性和耐受性终点，并且还达到了次要临床疗效终点。接受研究药物治疗的患者在ALS功能评定量表修订版中看到了“有意义的疾病进展减缓”，与安慰剂相比，数据支持PrimeC的比例为29%。2月，公司报告了更多数据表明该药物对生活质量和无并发症生存期具有临床意义。PTC Therapeutics的Utreloxastat2022年12月PTC Therapeutics报道I期试验，其ALS候选药物utreloxastat（PTC857）的结果表明，该药物“在健康人群中是安全的，耐受性良好，并显示出有希望的药理学特性”。PTC857是一种小分子，可抑制 15-脂氧合酶以减少氧化应激（ALS 疾病进展中广泛认可的因素），旨在防止谷胱甘肽的消耗，谷胱甘肽是一种保护性抗氧化剂，研究表明可能减少在ALS患者的运动皮层中。PTC857目前正在II期CardinALS试验，预计顶线数据将在2024年第四季度公布。Coya Therapeutics 的 COYA 302Coya Therapeutics牵头计划，COYA 302型，是一种调节性T细胞（Treg）靶向疗法，正在开发中，用于ALS，额颞叶痴呆，帕金森病和阿尔茨海默病。目前，它正处于ALS的II期试验中。COYA 302是一种生物联合疗法，具有双重免疫调节作用机制，旨在增强Tregs的抗炎功能，抑制活化的单核细胞和巨噬细胞产生的炎症。它由低剂量白细胞介素-2和CTLA-4 Ig 组成。2023 年2月，总部位于休斯顿的Coya来自一项概念验证研究公布的结果，其中一小群 ALS 患者接受了为期48周的双机制免疫治疗。该研究评估了安全性和耐受性、Treg功能、氧化应激和炎症的血清生物标志物以及通过ALSFRS-R量表测量的临床功能。Coya报告说，该疗法耐受性良好，最常见的不良事件是轻微的注射部位反应。没有停药或死亡。来源：https://www.biospace.com/版权声明：本文转自生物药大时代，如不希望被转载的媒体或个人可与我们联系，我们将立即删除-END-药事纵横投稿须知：稿费已上调，欢迎投稿各位朋友好，觉得本文对您有帮助，请随手点一下下方的在看，以便让你的朋友也能看到哦。

临床2期临床结果细胞疗法临床3期

100 项与 Utreloxastat 相关的药物交易

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