Umeclidinium Bromide/Vilanterol Trifenatate

Single-dose, oral zoliflodacin achieved 90.9% microbiological cure rate, demonstrating statistical non-inferiority compared to current global standard of care Pivotal Phase 3 data to be presented by the Global Antibiotic Research & Development Partnership (GARDP) in a Scientific Sessions Oral Presentation First collaborative study between industry and a non-profit to address a World Health Organization high priority pathogen and U.S. Centers for Disease Control urgent threat WALTHAM, Mass.--(BUSINESS WIRE)-- Innoviva Specialty Therapeutics, Inc., a subsidiary of Innoviva, Inc. (NASDAQ: INVA), today announced that positive results from the Phase 3 oral zoliflodacin trial will be highlighted in an oral presentation given by the Global Antibiotic Research & Development Partnership (GARDP) at the European Society of Clinical Microbiology and Infectious Disease Global Congress (ESCMID Global 2024) taking place April 27-30, 2024, in Barcelona, Spain. Zoliflodacin is a first-in-class spiropyrimidinetrione, single dose, oral antibiotic that is being developed in partnership with GARDP for the treatment of uncomplicated gonorrhea. “As a single dose, oral antibiotic, zoliflodacin, if approved, could have a profound effect on how physicians across the globe approach the treatment of gonorrhea infections, potentially improving patient access and compliance while helping to reduce the spread of antibiotic-resistant strains of gonorrhea,” said David Altarac, M.D., Chief Medical Officer of Innoviva Specialty Therapeutics. “The presentation of these data for this innovative investigational therapy is an important step in advancing our clinical pipeline strategy, as we now turn our focus on regulatory filing requirements in the U.S.” “Presenting these findings to the scientific community for the first time is a significant milestone in the journey of this important antibiotic in the fight against Neisseria gonorrhoeae, a World Health Organization priority pathogen,” said Dr. Alison Luckey, Senior Medical Lead for GARDP’s Sexually Transmitted Infections programme. “These positive findings not only represent a step forward in the treatment of gonorrhoea if approved, but also demonstrate the pivotal role that this public-private partnership between GARDP and Innoviva Specialty Therapeutics has in addressing the public health failure at the heart of the global antimicrobial resistance (AMR) crisis.” In November 2023, the two organizations announced that the Phase 3 zoliflodacin trial met its primary endpoint, demonstrating statistical non-inferiority of microbiological cure at the urogenital site when compared to treatment with intramuscular injection of ceftriaxone and oral azithromycin, currently the only remaining global standard of care regimen for the treatment of uncomplicated gonorrhea. In addition to the Phase 3 topline data, GARDP will also be presenting three additional posters highlighting details of zoliflodacin’s safety pro additional microbiological data from the Phase 3 trial, as well as data from a drug-drug interaction pharmacokinetic trial. GARDP and Innoviva Specialty Therapeutics plan to submit the Phase 3 zoliflodacin data for future publication in a medical journal. Beyond the zoliflodacin posters, Innoviva Specialty Therapeutics will also be presenting three additional posters at the meeting that feature new data for XERAVA® and XACDURO®. Results from the Phase 3 Zoliflodacin Trial The Phase 3 non-inferiority trial analyzed a total of 930 patients with uncomplicated gonorrhea, including women, adolescents and people living with HIV, making it the largest clinical trial ever conducted for a new treatment against gonorrhea infection, with 16 trial sites in regions with a high prevalence of gonorrhea across five countries, including Belgium, the Netherlands, South Africa, Thailand, and the U.S. The trial compared a single oral 3g dose of zoliflodacin to a globally recognized standard of care regimen (500mg ceftriaxone intramuscular [IM] plus 1g oral azithromycin). The primary efficacy endpoint was microbiological response at the urogenital site (cure or failure) at the Test-of-Cure (ToC) visit 6+/-2 days after treatment. Secondary analyses included microbiological cure at rectal or pharyngeal sites and safety. The trial met its primary efficacy endpoint, with zoliflodacin (oral, 3g dose) demonstrating non-inferiority to ceftriaxone (IM, 500mg) plus azithromycin (oral, 1g). In the micro-intent-to-treat (micro-ITT) population (n=744), zoliflodacin achieved a microbiological cure rate of 90.9%, a 5.3% difference compared to ceftriaxone and azithromycin which achieved a 96.2% cure rate (95% CI: 1.4%, 8.7%). Microbiological cure rates at extragenital sites were comparable between treatment arms (secondary endpoints). Oral zoliflodacin 3g was generally well tolerated and emergent adverse events were comparable between treatment arms (46.2% vs 46.4%). No deaths or other serious adverse events were reported. Details for the ESCMID Global 2024 presentations are as follows: Oral presentation Title: Oral zoliflodacin is non-inferior to a combination of ceftriaxone and azithromycin for treatment of uncomplicated urogenital gonorrhoea: results of a large global Phase 3 randomized controlled trial Presenter/Author: Alison Luckey Oral session #: 246 Session: 05. New antibacterial agents, PK/PD & Stewardship – Therapeutic expedition: mining old and new drugs and dosing strategies Date and time: Tuesday, April 30, 2024; 8:30-10:30 CEST Location: Hall H Poster presentations Title: Safety pro oral zoliflodacin for uncomplicated gonorrhoea in a Phase 3 trial Author: Gabrielle Kornman, et al. Poster #: 2724 Session: 5e. Safety, hypersensitivity, and adverse effects of treatment Date and time: Sunday, April 28, 2024; 12:00 CEST Location: Poster Area, Sector A, Row 23, Position 2 Title: Pharmacokinetics of zoliflodacin in healthy participants in the presence of itraconazole suggest no clinically meaningful CYP3A4-mediated drug-drug interactions Author: Alison Luckey, et al. Poster #: 2424 Session: 5b. Pharmacokinetics/pharmacodynamics of antibacterial drugs & therapeutic drug monitoring (incl lab methods, models, in vitro and in vivo studies) Date and time: Sunday, April 28, 2024; 12:00 CEST Location: Poster Area, Sector B, Row 16, Position 26 Title: Antimicrobial susceptibility of baseline Neisseria gonorrhoeae isolates from participants recruited in the global zoliflodacin Phase 3 randomised controlled trial Author: Alison Luckey, et al. Poster #: 2527 Session: 5c. New or repurposed antibacterial agents: clinical studies and randomised trials Date and time: Sunday, April 28, 2024; 12:00 CEST Location: Poster Area, Sector B, Row 18, Position 17 Title: Eravacycline susceptibility against Gram-positive pathogens, collected in Europe during 2022 Author: Stephen Hawser, et al. Poster #: 1281 Session: 3a. Resistance surveillance & epidemiology: MRSA, VRE & other Gram-positives Date and time: Monday, April 29, 2024; 12:00 CEST Location: Poster Area, Sector B, Row 1, Position 8 Title: In vitro activity of eravacycline against Enterobacterales and non-fermenter clinical isolates, including resistant isolates, collected in Europe during 2022 Author: Stephen Hawser, et al. Poster #: 1421 Session: 3b. Resistance surveillance & epidemiology: Gram-negatives Date and time: Monday, April 29, 2024; 12:00 CEST Location: Poster Area, Sector A, Row 4, Position 12 Click here for full XERAVA safety and prescribing information or go to . Title: In vitro activity of sulbactam/durlobactam in combination with cefepime against Gram-negative bacterial isolates from a recent Phase 3 clinical trial Author: Sarah McLeod, et al. Poster #: 1816 Session: 3f. Clinical outcome of resistant infections (retrospective and prospective studies, excl clinical trials of new drugs) Date and time: Monday, April 29, 2024; 12:00 CEST Location: Poster Area, Sector B, Row 12, Position 3 Click here for full XACDURO safety and prescribing information or go to . The oral presentation and posters will be available on the “Events & Presentations” page of the Investors Relations section of Innoviva’s website following their presentation at ESCMID Global 2024. About Oral Zoliflodacin Zoliflodacin is a potential first-in-class, orally administered, single dose antibiotic with a novel mechanism of action that is currently in development for the treatment of uncomplicated gonorrhea. In a Phase 3 clinical trial, zoliflodacin met the primary efficacy endpoint by demonstrating non-inferiority compared to a globally recognized standard of care regimen (500mg ceftriaxone intramuscular [IM] plus 1g oral azithromycin). Zoliflodacin was found to be generally well tolerated with the overall rate of adverse events comparable between the two arms, and the majority of adverse events were mild to moderate. In vitro studies have shown that it is active against multidrug-resistant strains of Neisseria gonorrhoeae, including those resistant to ceftriaxone, and azithromycin, with no cross-resistance with other antibiotics. About Gonorrhea Gonorrhea is widely prevalent worldwide, with the World Health Organization estimating 82 million new cases worldwide in 20201, making it the second most prevalent sexually transmitted bacterial infection worldwide after Chlamydia trachomatis. In the U.S., gonorrhea is the second most prevalent sexually transmitted bacterial infection, with an estimated 1.6 million new infections each year.2 The bacterium Neisseria gonorrhoeae has gradually developed resistance to many classes of antibiotics used to treat these infections and as a result, ceftriaxone, given as a single intramuscular injection, has become the last available recommended treatment for gonorrhea globally. About GARDP The Global Antibiotic Research & Development Partnership (GARDP) is a not-for-profit organization that develops new antibiotic treatments for drug-resistant bacterial infections that pose the greatest threat to human health, and makes them accessible to the people who need them. It puts public health needs at the centre of antibiotic drug development to address the immediate crisis of antimicrobial resistance (AMR). Its work is funded by the governments of Australia, Germany, Japan, Monaco, the Netherlands, the Public Health Agency of Canada, South Africa, Switzerland, the United Kingdom, the Canton of Geneva, the European Union (via the Health Emergency Preparedness and Response Authority), as well as the RIGHT Foundation, Wellcome and other private foundations. GARDP was created by the World Health Organization and the Drugs for Neglected Diseases initiative (DNDi) in 2016 and legally registered as the GARDP Foundation in Geneva, Switzerland in 2018. . About Innoviva Specialty Therapeutics, Inc. Innoviva Specialty Therapeutics, a subsidiary of Innoviva, Inc., is focused on delivering innovative therapies in critical care and infectious disease. Innoviva Specialty Therapeutics’ products, through its affiliate, La Jolla Pharmaceutical Company, include GIAPREZA® (angiotensin II), approved to increase blood pressure in adults with septic or other distributive shock, and XERAVA® (eravacycline) for the treatment of complicated intra-abdominal infections in adults. Innoviva Specialty Therapeutics’ products, through its affiliate, Entasis Therapeutics Inc., include XACDURO® (sulbactam for injection; durlobactam for injection), co-packaged for intravenous use approved for the treatment of adults with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by susceptible strains of Acinetobacter baumannii-calcoaceticus complex (Acinetobacter). Our Phase 3 development pipeline includes zoliflodacin, a novel treatment for uncomplicated gonorrhea in adults. For more information about Innoviva Specialty Therapeutics, please visit here and follow us on X (formerly Twitter) and LinkedIn. About Innoviva, Inc. Innoviva is a diversified holding company with a core royalties portfolio, a leading critical care and infectious disease platform known as Innoviva Specialty Therapeutics (IST), and a portfolio of strategic investments in healthcare assets. Innoviva’s royalty portfolio includes respiratory assets partnered with Glaxo Group Limited (GSK). Innoviva is entitled to receive royalties from GSK on sales of RELVAR®/BREO® ELLIPTA® and ANORO® ELLIPTA®. Innoviva’s other innovative healthcare assets include infectious disease and critical care assets stemming from acquisitions of Entasis Therapeutics, including XACDURO® (sulbactam for injection; durlobactam for injection), co-packaged for intravenous use approved for the treatment of adults with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by susceptible strains of Acinetobacter baumannii-calcoaceticus complex and the investigational zoliflodacin currently being developed for the treatment of uncomplicated gonorrhea, and La Jolla Pharmaceutical Company, including GIAPREZA® (angiotensin II), approved to increase blood pressure in adults with septic or other distributive shock and XERAVA® (eravacycline) for the treatment of complicated intra-abdominal infections in adults. For more information about Innoviva, please visit here and follow us on LinkedIn. ANORO®, RELVAR®, BREO® and TRELEGY® are trademarks of the GSK group of companies. Forward Looking Statements This press release contains certain “forward-looking” statements as that term is defined in the Private Securities Litigation Reform Act of 1995 regarding, among other things, statements relating to goals, plans, objectives, and future events. Innoviva intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. The words “anticipate”, “expect”, “goal”, “intend”, “objective”, “opportunity”, “plan”, “potential”, “target” and similar expressions are intended to identify such forward-looking statements. Such forward-looking statements involve substantial risks, uncertainties, and assumptions. These statements are based on the current estimates and assumptions of the management of Innoviva as of the date of this press release and are subject to known and unknown risks, uncertainties, changes in circumstances, assumptions and other factors that may cause the actual results of Innoviva to be materially different from those reflected in the forward-looking statements. Important factors that could cause actual results to differ materially from those indicated by such forward-looking statements include, among others, risks related to: expected cost savings; lower than expected future royalty revenue from respiratory products partnered with GSK; the commercialization of RELVAR®/BREO® ELLIPTA®, ANORO® ELLIPTA®, GIAPREZA®, XERAVA® and XACDURO® in the jurisdictions in which these products have been approved; the strategies, plans and objectives of Innoviva (including Innoviva’s growth strategy and corporate development initiatives); the timing, manner, and amount of potential capital returns to shareholders; the status and timing of clinical studies, data analysis and communication of results; the potential benefits and mechanisms of action of product candidates; expectations for product candidates through development and commercialization; the timing of regulatory approval of product candidates; and projections of revenue, expenses and other financial items; the impact of the novel coronavirus (COVID-19); the timing, manner and amount of capital deployment, including potential capital returns to stockholders; and risks related to the Company’s growth strategy. Other risks affecting Innoviva are described under the headings “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” contained in Innoviva’s Annual Report on Form 10-K for the year ended December 31, 2022, and Quarterly Reports on Form 10-Q, which are on the Securities and Exchange Commission (SEC) and available on the SEC’s website at . Past performance is not necessarily indicative of future results. No forward-looking statements can be guaranteed, and actual results may differ materially from such statements. Given these uncertainties, you should not place undue reliance on these forward-looking statements. The information in this press release is provided only as of the date hereof, and Innoviva assumes no obligation to update its forward-looking statements on account of new information, future events or otherwise, except as required by law. References View source version on businesswire.com: Contacts Innoviva Specialty Therapeutics David Patti, Corporate Communications +1 908.421.5971 David.Patti@inva.com Innoviva, Inc. Investor Relations Argot Partners +1 212.600.1902 Innoviva@argotpartners.com Source: Innoviva Specialty Therapeutics View this news release online at:

▲5月30-31日 第八届广州生物医药创新者峰会 扫码立即报名 注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。药物临床试验是新药上市的必经之路，但往往付出和回报不成正比，根据数据统计，临床I、II、III期的成功率分别为63.2%、30.7%和58.1%。据不完全统计2024年1-3月有23项失败临床研究，小编在此进行汇总，并介绍部分失败临床案例以供参考。1AMX0035 III期研究未达主要终点2024年3月8日，Amylyx Pharmaceuticals公布AMX0035治疗肌萎缩性侧索硬化症（ALS）三期临床PHOENIX的顶线数据，研究没有达到主要终点和次要终点。Amylyx将与监管进行沟通，包括自愿撤市的计划。受此消息影响，Amylyx股价暴跌83.5%。AMX0035的成分为苯丁酸钠和牛磺酸二醇，于2022年9月获得FDA批准上市，定价为每年15.8万美元。PHOENIX研究共招募了664例ALS患者，结果并未达到主要终点，第48周时，与安慰剂组比，AMX0035治疗组肌萎缩侧索硬化功能评定量表（ALSFRS-R）评分较基线变化不具有显著统计学差异（p=0.667）。此外，该研究也未达到患者报告的生活质量结果评估、总生存期以及慢肺活量（SVC）衡量的呼吸功能等次要终点。2Pimavanserin III期研究未达主要终点2024年3月11日，Acadia Pharmaceuticals公布Pimavanserin治疗精神分裂阴性症状三期临床ADVANCE-2的最新研究结果未达主要终点。研究的主要终点为NSA-16总分从基线到第26周的变化，治疗组和安慰机组26周NSA-16评分分别为-11.8 vs -11.1%，结果不具有统计学意义。安全性和耐受性与之前的临床试验一致，不良事件发生率较低。受此消息影响，Acadia Pharmaceuticals股价盘后下跌15%。3Keytruda+Lynparza III期研究未达主要终点3月21日，默沙东宣布其PD-1疗法Keytruda联合维持性PARP抑制剂Lynparza（奥拉帕利）一线治疗某些转移性非鳞状非小细胞肺癌（NSCLC）患者的III期KEYLYNK-006试验未达到总生存期（OS）和无进展生存期（PFS）的双重主要终点。除此之外，2023年12月默沙东还终止过一项K药+Lynparza用于转移性鳞状非小细胞肺癌的III期研究。终止原因是独立数据监测委员会（DMC）审查了计划中的中期分析（IA3）数据，发现与Keytruda联合化疗后进行Keytruda加安慰剂治疗相比，接受Keytruda联合化疗后进行Keytruda+Lynparza治疗的患者并没有显示出总生存期（OS）改善。而无进展生存期也没有统计学意义。早在2022年8月份，默沙东还公布了K药两项III期临床试验均未能达到终点。分别是：K药与卫材的仑伐替尼（Lenvima）联合用于新诊断肝癌的三期临床试验结果和K药+Lynparza联合用于先前治疗过的转移性去势抵抗性前列腺癌。4Enspryng III期研究未达预期3月21日，罗氏合作伙伴Chugai制药（中外制药）公布了Enspryng（萨特利珠单抗）治疗全身型重症肌无力（gMG）的III期LUMINESCE研究结果。虽在其主要终点观察到具有统计学意义的数据，但结果没有达到对临床获益程度的预期。Enspryng是一种白介素6(IL-6)受体拮抗剂，2020年8月获得美国FDA批准用于治疗抗水通道蛋白-4(AQP4)抗体阳性的视神经脊髓炎谱系障碍(NMOSD)成人患者。此后，萨特利珠单抗于 2021年5月在国内获批上市，成为国内首个视神经脊髓炎谱系疾病治疗药物。中外制药表示此次研究结果不影响Enspryng在NMOSD患者中的使用。该公司还在开发Enspryng用于其他神经自身免疫性和炎症性疾病。5DNL788 II期研究未达主要终点2月16日，Denali Therapeutics在SEC文件中透露，DNL788（SAR443820）治疗肌萎缩侧索硬化症（ALS）的II期HIMALAYA研究未能达到显著改善修订版ALS功能评定量表（ALSFRS-R）评分的主要终点。受此消息影响，Denali的股价下跌了近8%。2018年10月，赛诺菲和Denali公司就RIPK1抑制剂的全球开发和商业化展开广泛合作。赛诺菲将领导SAR443820/DNL788治疗ALS和MS的1期临床和2期临床开发，并与Denali公司一起进行该药治疗ALS、MS和阿尔茨海默病的3期临床试验。SAR443820/DNL788是由赛诺菲和Denali共同开发的一款有效的、选择性的、具有脑渗透性的RIPK1抑制剂。赛诺菲将继续在多发性硬化症患者中开展SAR443820/DNL788的K2 2期研究；1月8日，Denali在新闻稿中透露，赛诺菲已完成SAR443820/DNL788治疗多发性硬化症（MS）的2期试验，共招募174名参与者。6Yupelri IV期研究未达主要终点2024年1月5日，Theravance Biopharma宣布Yupelri（revfenacin）吸入溶液用于慢性阻塞性肺病（COPD）维持治疗的IV期PIFR-2研究未达主要终点。Yupelri 是美国批准用于慢阻肺维持治疗的唯一每日一次的雾化长效毒蕈碱拮抗剂（LAMA）。这项PIFR-2研究旨在评估，Yupelri较噻托溴铵（Spiriva，粉雾剂）在严重至极为严重COPD患者的肺功能改善情况。研究结果显示，第85天时第一秒用力呼气谷值较基线变化的主要终点指标方面，与对照组相比，Yupelri并未有统计学显著差异。Yupelri的安全性与耐受性与既往研究一致。本次研究的失败也导致Theravance股价下跌10%。总结新药的研发是现实且残酷的，没有主角光环，研究的失败不仅使巨额研究费用打水漂，往往也伴随着股价的大幅下跌。但凡事都有两面性，没有这些负重前行的研发企业，患者也便失去了一线希望，失败也是探索路上的重要部分，为成功奠定基石。2024年第一季度也有很多临床成功的好消息，比如：1月，Vertex公布Nav1.8抑制剂VX-548治疗中至重度急性疼痛三期临床达到主要终点；2月，信达生物宣布IGF-1R抗体治疗甲状腺眼病的三期临床达到主要终点，将向NMPA递交上市申请；强生宣布FcRn抗体Nipocalimab治疗全身性重症肌无力（gMG）三期临床达到主要终点；3月，Acelyrin公布IL-17类抗体Izokibep治疗银屑病关节炎（PsA）的2b/3期临床达到主要终点。参考资料：各公司官网往期文章：全球失败临床研究TOP18（1-2月） 活动推荐  5月 • 第八届大湾区生物医药创新者峰会  关键词：  小分子，XDC，多肽及GLP-1热点话题，80+行业专家（点击下方图片查看详情）▼【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。