Icatibant Acetate

ZUG, Switzerland, Sept. 05, 2024 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to prevent and treat hereditary angioedema (HAE) attacks, today announced a summary of data being presented at the ongoing 7th Bradykinin Symposium. Details of the presentations are outlined below: Long-Term Safety and Efficacy of Oral Deucrictibant for HAE Prophylaxis, a poster presentation by Marc A. Riedl, M.D., M.S. In the current analysis of the ongoing open-label extension of the CHAPTER-1 Phase 2 study, deucrictibant 40 mg/day was well-tolerated, with no new safety signals observed. The results presented provide evidence of the long-term safety and efficacy of deucrictibant for the prevention of HAE attacks and support further development of deucrictibant as a potential prophylactic therapy for HAE. Results of this analysis provide support that: Continuing deucrictibant treatment sustained the early-onset attack reduction seen in the randomized, placebo-controlled portion of the trial, with a median attack rate of zero for every month for over a year in the open-label part of the studyOn average, less than one attack per year per participant was treated with on-demand medication Treatment of HAE Attacks with Oral Deucrictibant: RAPIDe-2 Extension Results, a poster presentation by Emel Aygören-Pürsün, M.D. In the current analysis of the ongoing RAPIDe-2 Phase 2/3 extension study, deucrictibant immediate release capsule was well-tolerated for all studied doses with no new safety signals observed. Results from the ongoing RAPIDe-2 extension are consistent with the randomized, placebo-controlled RAPIDe-1 Phase 2 study and provide evidence regarding the long-term safety and efficacy of deucrictibant IR capsule for repeat treatment of HAE attacks. Outcome analyses showed: Median time to onset of symptom relief as measured by the Patient Global Impression of Change (PGI-C) was 1.1 hours, with 98.5% of attacks achieving onset of symptom relief by 12 hoursMedian time to reduction in attack severity as measured by the Patient Global Impression of Severity (PGI-S) was 2.6 hours, with 97.7% of attacks achieving reduction in attack severity by 12 hoursMedian time to complete attack resolution as measure by PGI-S was 11.5 hours, with 85.8% of attacks achieving complete attack resolution within 24 hoursOverall, 86.0% of attacks were treated with a single dose of deucrictibant immediate-release capsule Prophylactic Treatment with Deucrictibant Improves HAE Disease Control and HRQoL, an oral presentation by Markus Magerl, M.D. In the randomized, placebo-controlled part of CHAPTER-1, a Phase 2 clinical study of deucrictibant for the prophylactic treatment of HAE attacks, health-related quality of life was evaluated using several measures. In the study, it was demonstrated that deucrictibant treatment led to improvements in disease control versus placebo, with 90% of participants in the deucrictibant-groups demonstrating well-controlled HAE at week 12. Presentation details included: Deucrictibant improved Angioedema Quality of Life Questionnaire (AE-QoL) scores, particularly in “functioning” and “fear/shame” domains compared to placeboDeucrictibant-treated participants reported greater satisfaction than those treated with placebo with regards to effectiveness and the domain of global satisfaction, and a comparable satisfaction for side effects, as measured by Treatment Satisfaction Questionnaire for Medication (TSQM) Deucrictibant vs. Standard of Care in HAE: Propensity Score-Matched Analysis, a poster presentation by Marc A. Riedl, M.D., M.S. A propensity score-matched comparison of clinical outcomes between a subgroup of attacks (N=73) from the RAPIDe-2 study and a subgroup of attacks (N=73) from an observational real-world study treated with standard of care, the outcomes were more favorable for the attacks treated with deucrictibant on PGI-C- and PGI-S-based assessments. Deucrictibant had a shorter (1.07 hours) median time to onset of symptom relief as measured by PGI-C “a little better” compared to standard of care (2.38 hours). Cardiovascular safety of repeated oral administration of the B2-receptor antagonist deucrictibant, a poster presentation by Nieves Crespo, Ph.D. In chronic nonclinical safety studies of deucrictibant in non-human primates, no evident effects on cardiac electrophysiology, morphology and hemodynamic parameters were observed. Deucrictibant has showed no evident effects on cardiac electrophysiology and hemodynamic parameters in clinical studies in humans to date, following prophylactic treatment up to 12 weeks of administration in the randomized, placebo-controlled part of the Phase 2 CHAPTER-1 clinical study and up to one year of mean duration of treatment in the ongoing open-label extension (OLE) part. Prophylaxis of Hereditary Angioedema Attacks with Oral Deucrictibant: CHAPTER-1 Results, a poster presentation by Emel Aygören-Pürsün, M.D. The CHAPTER-1 study demonstrated that deucrictibant may significantly reduce the occurrence of HAE attacks, and clinically meaningful reduction in occurrence of moderate and severe HAE attacks, as well as HAE attacks treated with on-demand medication, was observed. CHAPTER-1 results provide evidence of the efficacy and safety of deucrictibant for the prevention of HAE attacks and support its further development as a potential prophylactic therapy for HAE. Clinical Trials Conformity with AURORA COS: a systematic literature review, a poster presentation by Remy S. Petersen, M.D., Conforming to a core outcome set (COS) across various study designs, such as the COS recommended for HAE clinical studies by the Panel of Experts participating in the AURORA Project, may homogenize the use of specific outcomes for clinical studies and support future indirect comparisons among interventions. The design of the RAPIDe-3 Phase 3 study of deucrictibant immediate-release capsule for the on-demand treatment of HAE attacks fully conforms with the AURORA COS based on its prespecified endpoints. Bradykinin Challenge Model in Humanized Bradykinin B2 receptor Transgenic Rat, an oral presentation by Jolanta Skarbaliene, Ph.D. The bradykinin (BK) challenge model is a tool to assess pharmacokinetic and pharmacodynamic activity of bradykinin B2 receptor antagonists. A BK challenge model was successfully developed in humanized bradykinin B2 receptor transgenic rats that are pharmacologically responsive to bradykinin B2 receptor antagonists. The BK challenge model in humanized bradykinin B2 receptor transgenic rats can offer a valuable, easy to manage, and cost-effective tool for efficacy studies compared to those involving non-human primates. Deucrictibant inhibits carrageenan-induced edema in bradykinin B2 receptor transgenic rat, a poster presentation by Anne Lesage, Ph.D. A humanized bradykinin B2 receptor transgenic rat model was used to address the challenge of deucrictibant species selectivity in experimental models. Oral deucrictibant inhibited carrageenan-induced paw edema in humanized bradykinin B2 receptor transgenic rats. The bradykinin challenge model translates across rat, monkey and human, a poster presentation by Juan Bravo, Ph.D. The pharmacokinetics (PK) and pharmacodynamics (PD) of icatibant were analyzed from BK challenge studies in humanized bradykinin B2 receptor transgenic rats, non-human primates, and healthy volunteers. Analyses across species showed similar responses, demonstrating that the BK challenge model in transgenic rats and non-human primates may be predictive of PK/PD outcomes in humans. A novel kinin biomarker assay for characterization of bradykinin-mediated disorders, a poster presentation by Evangelia Pardali, Ph.D. BK is involved in various physiological and pathological processes, including angioedema (AE). Differentiating BK-mediated from histamine-mediated AE and assessing the role of BK in the pathogenesis of other conditions by measuring kinin peptides remains a challenge due to their proteolytic instability and limitations of current analytical assays. A kinin biomarker assay was established and qualified, which could become a key tool for identifying, studying, and managing BK-mediated diseases. A HMWK capillary immunoblotting assay to characterize bradykinin-mediated disorders, a poster presentation by Evangelia Pardali, Ph.D. Activation of the plasma kallikrein-kinin system (KKS) can result in cleavage of high molecular weight kininogen (HMWK) and production of vasodilatory kinins, such as BK. A HMWK immunoblotting assay was established and qualified to reliably measure KKS biomarkers in human plasma and could become a key tool for identifying, studying, and managing BK-mediated diseases. The presentation slides and posters are available on the Investors section of the Pharvaris website at: https://ir.pharvaris.com/news-events/events-presentations. About DeucrictibantDeucrictibant is a novel, potent, oral small-molecule bradykinin B2 receptor antagonist. By inhibiting bradykinin signaling through the bradykinin B2 receptor, deucrictibant has the potential to prevent the occurrence of HAE attacks and to treat the manifestations of attacks if they occur. Based on its chemical properties, Pharvaris is developing two formulations of deucrictibant for oral administration: an extended-release tablet to enable sustained absorption and efficacy for prophylactic treatment, and an immediate-release capsule to enable rapid onset of activity for on-demand treatment. About PharvarisPharvaris is a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to prevent and treat HAE attacks. By directly pursuing this clinically proven therapeutic target with novel small molecules, the Pharvaris team aspires to offer people with all types of bradykinin-mediated angioedema effective, well-tolerated, and easy-to-administer alternatives to treat attacks, both prophylactically and on-demand . With positive data in both Phase 2 prophylaxis and on-demand studies in HAE, Pharvaris is encouraged to further develop deucrictibant. Pharvaris is currently enrolling a pivotal Phase 3 study for the on-demand treatment of HAE attacks and plans to initiate a pivotal Phase 3 study of deucrictibant for the prevention of HAE attacks in the coming months. For more information, visit https://pharvaris.com/. Forward-Looking StatementsThis press release contains certain forward-looking statements that involve substantial risks and uncertainties. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements relating to our future plans, studies and trials, and any statements containing the words “believe,” “anticipate,” “expect,” “estimate,” “may,” “could,” “should,” “would,” “will,” “intend” and similar expressions. These forward-looking statements are based on management’s current expectations, are neither promises nor guarantees, and involve known and unknown risks, uncertainties and other important factors that may cause Pharvaris’ actual results, performance or achievements to be materially different from its expectations expressed or implied by the forward-looking statements. Such risks include but are not limited to the following: uncertainty in the outcome of our interactions with regulatory authorities, including the FDA; the expected timing, progress, or success of our clinical development programs, especially for deucrictibant immediate-release capsules and deucrictibant extended-release tablets, which are in late-stage global clinical trials; our ability to replicate the efficacy and safety demonstrated in the RAPIDe-1, RAPIDe-2, and CHAPTER-1 Phase 2 studies in ongoing and future nonclinical studies and clinical trials; risks arising from epidemic diseases, such as the COVID-19 pandemic, which may adversely impact our business, nonclinical studies, and clinical trials; the outcome and timing of regulatory approvals; the value of our ordinary shares; the timing, costs and other limitations involved in obtaining regulatory approval for our product candidates, or any other product candidate that we may develop in the future; our ability to establish commercial capabilities or enter into agreements with third parties to market, sell, and distribute our product candidates; our ability to compete in the pharmaceutical industry, including with respect to existing therapies, emerging potentially competitive therapies and with competitive generic products; our ability to market, commercialize and achieve market acceptance for our product candidates; our ability to raise capital when needed and on acceptable terms; regulatory developments in the United States, the European Union and other jurisdictions; our ability to protect our intellectual property and know-how and operate our business without infringing the intellectual property rights or regulatory exclusivity of others; our ability to manage negative consequences from changes in applicable laws and regulations, including tax laws, our ability to successfully remediate the material weaknesses in our internal control over financial reporting and to maintain an effective system of internal control over financial reporting; changes and uncertainty in general market, political and economic conditions, including as a result of inflation and the current conflict between Russia and Ukraine and the Hamas attack against Israel and the ensuing war; and the other factors described under the headings “Cautionary Statement Regarding Forward-Looking Statements” and “Item 3. Key Information—D. Risk Factors” in our Annual Report on Form 20-F and other periodic filings with the U.S. Securities and Exchange Commission. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. While Pharvaris may elect to update such forward-looking statements at some point in the future, Pharvaris disclaims any obligation to do so, even if subsequent events cause its views to change. These forward-looking statements should not be relied upon as representing Pharvaris’ views as of any date subsequent to the date of this press release.

成都圣诺生物科技股份有限公司成立于2001年7月，2021年A股上市（SH：688117）。圣诺生物从创立起就专注于多肽领域，具备多肽原料药和制剂的全流程研发管线和全产业链平台，能够为多肽创新药和仿制药提供从工艺路线设计，到小试、中试、工艺验证和质量研究等药学研究服务，以及药物研发及商业化生产所需原料药、制剂产品的全链条定制生产服务。 1. 深耕多肽领域二十余载，原料药+制剂+CDMO服务协同发展 深耕多肽领域二十余载，圣诺生物是我国多肽药物领域的重要玩家。圣诺生物公司成立于2001年，于2021年在上交所上市，是国内成立较早的专业多肽药物生产企业之一。圣诺生物致力于为国内外医药企业的多肽类创新药研发提供药学研究和定制生产服务，自主研发、生产和销售多肽类仿制药原料药和制剂产品以及多肽药物生产技术转让服务。经过多年的研究与开发，公司在多肽药物研发和生产领域积累了先进的核心技术和丰富的研发生产经验，具有自主多肽合成和修饰核心技术，并积累了大量非专利技术，依托技术的不断创新，持续满足多肽医药市场需求。 业务构成多元，原料药+制剂+CDMO服务协同发展。公司业务聚焦于多肽医药领域，主营产品和服务包括多肽原料药、多肽制剂、多肽创新药CDMO服务、受托加工服务以及多肽药物生产技术转让服务： 多肽创新药CDMO服务：公司的药学研究服务针对客户筛选或指定的多肽分子进行药学研究，并将形成的研究资料和所生产样品交付客户用于进一步研发或注册申报，服务过程中的相关知识产权归客户所有。公司按照技术合同约定收取研发服务费用，相应费用计入主营业务成本。 多肽原料药：公司已拥有19个自主研发的多肽类原料药品种。在国内市场，公司取得14个品种多肽原料药生产批件或激活备案，其中恩夫韦肽、卡贝缩宫素为国内首仿品种；在国外市场，公司比伐芦定、依替巴肽、利拉鲁肽等10个品种获得美国DMF备案，其中9个品种处于激活状态，可被制剂生产企业引用申报；艾替班特为首家提交此品种美国DMF备案的仿制原料药，并被我国卫健委纳入《第一批鼓励仿制药品目录建议清单》，比伐芦定国外客户成为美国第二家获批的仿制药制剂生产商。 多肽制剂产品：公司多肽制剂立足于公司多肽原料药规模化生产能力，终端客户为国内医疗机构。公司多肽制剂产品均使用自产原料药生产，具有质量可靠、稳定性好等特点，已在全国31个省、自治区、直辖市销售。 多肽类产品定制生产服务：多肽类产品定制生产服务包括为客户提供的定制肽生产服务，以及多肽原料药、制剂定制生产服务。定制肽生产服务包括为客户提供新药研发筛选阶段所需多肽化合物、对照品的定制生产，以及按照客户提供的产品结构定制生产的多肽化合物产品。  多肽药物生产技术转让服务：公司多肽药物生产技术转让服务是指公司出于自身资本实力、药品审批风险、市场推广成本等因素考虑，将自主研发并已经向国家药品审批部门提交药品注册申请的多肽仿制药品种（包括原料药和制剂）的整体权益转让，包括但不限于知识产权及开发、商业化、销售和分销、再许可、对外许可和其他使用的权利、所有权和利益。受让方通常会与公司约定继续以公司名义申请生产批件，在获取相应的生产批件后，公司按照受让方指令组织相关产品的生产并收取相应的加工费用，相关产品销售及市场开拓由受让方负责。 2.多肽药物CDMO一站式服务，CDMO业务持续增长，技术优势助力CDMO发展 多肽创新药CDMO服务是圣诺生物的主要业务构成之一，主要提供药学研究服务，针对客户筛选或指定的分子进行药学研究，并将形成的研究资料和所生产样品交付客户用于进一步研发或注册申报，符合中、美、欧申报要求，服务过程中的知识产权归客户所有。 圣诺生物的多肽药物CDMO服务拥有国内完善的多肽药物研究平台，包括独立的原料小试生产车间、独立的原料中试生产车间、独立的制剂中试生产车间和相应独立的研究团队。且公司的研究团队专攻多肽领域多年，积累了宝贵的经验，致力于保质保速地完成产品申报，为创新药物抢占注册先机。公司在多肽药物研发生产领域获得较高的知名度和认可度，已先后完成50多个创新多肽药物的CMC研究，其中1个多肽创新药已获批上市进入商业化阶段，2个多肽创新药已申报生产，另有20余个多肽创新药进入临床试验阶段。 CDMO为公司业绩增长的潜力点。自2018年起，公司CDMO业务占主营业务收入比例不断上升，2023年占比达到24%。营收端，从2017年到2023年，公司CDMO收入从0.50亿元上升至1.03亿元，营收占比自2018年12%上升至2023年的24%。 公司具有CDMO业务多项优势： 多肽合成和修饰核心技术优势：公司自主研发并掌握了长链肽偶联技术、单硫环肽规模化生产技术等八项多肽合成和修饰类自主核心技术。 质量稳定、收率高、规模化生产能力的业务优势：相比较于小分子CDMO，多肽创新药药学研究技术在生产过程中更加容易产生杂质过高、稳定性不达标等问题，更加考验服务企业的工艺研发和质量研究能力。 从初始物料到原料药、制剂的研发生产一体化需求的成本优势：多肽药物生产一体化需求高，对服务方依赖性强。公司能够为多肽创新药和仿制药提供从工艺路线设计，到小试、中试、工艺验证和质量研究等药学研究服务，以及药物研发及商业化生产所需原料药、制剂产品的全链条定制生产服务。 3.强大的多肽生产能力 圣诺生物多肽产业化能力位居全国前列，多肽原料药生产车间通过中国、美国、韩国和墨西哥的官方GMP认证，并实现了商业化生产，目前原料药生产线共计15条，仍有13条原料药生产线在建，预计2024年投产，投产后年生产多肽原料药可达1.5吨；多肽制剂生产车间涵盖了固体口服、外用制剂、黏膜给药、注射用产品等生产线，并有独立的高活生产线。 END 免责声明：本文仅作知识交流与分享及科普目的，不涉及商业宣传，不作为相关医疗指导或用药建议。文章如有侵权请联系删除。 IDC2024多肽与多条偶联药物研发与质控论坛圆满结束，点击图片查看会后报告，期待明年再聚~