AzurRx BioPharma Announces Completion of Enrollment in Phase 2b OPTION 2 Clinical Trial of MS1819 in Cystic Fibrosis

AzurRx BioPharma宣布完成MS1819治疗囊性纤维化2b期方案2临床试验的注册

2021-03-16 21:30:07 BioSpace

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Top-line data expected by end of Q1 2021 DELRAY BEACH, Fla., March 16, 2021 (GLOBE NEWSWIRE) -- AzurRx BioPharma, Inc. (NASDAQ: AZRX), (“AzurRx” or the “Company”), a clinical stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced it has completed enrollment for its Phase 2b OPTION 2 clinical trial to investigate MS1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI). The trial enrolled 30 CF patients and top line data from the study is anticipated by the end of Q1 2021. The Phase 2b multi-center study is designed to investigate the safety, tolerability and efficacy of MS1819 in enteric capsules) in a head-to-head comparison against the current porcine enzyme replacement therapy (PERT) standard of care. The primary efficacy endpoint is the coefficient of fat absorption (CFA), with secondary endpoints of stool weight, signs and symptoms of malabsorption and coefficient of nitrogen absorption (CNA). The trial also includes an extension arm that uses an immediate release MS1819 capsule, allowing the Company to compare data from the existing arm that uses enteric (delayed release) capsules with data from the new arm, and ultimately select the optimal delivery method. “Completion of enrollment in our Phase 2b OPTION 2 trial is an important milestone in the clinical development of MS1819 as an EPI monotherapy for patients with cystic fibrosis,” said James Sapirstein, CEO, President and Chairman of AzurRx. “Data from this trial will inform the optimal dose for a potential pivotal Phase 3 study, and we are on target to report topline results from this study at the end of the first quarter of 2021.” Dr. James Pennington, Chief Medical Officer of AzurRx, added, “The overarching goal of our MS1918 program is to provide a safe and effective therapy to control EPI and improve upon the current standard of care, PERT, which is porcine-derived and requires patients to take upwards of 40 capsules per day to control symptoms. MS1819 is a synthetic lipase that does not contain any animal products and offers the potential to substantially reduce the daily pill burden currently required to manage EPI.” Additional information about this clinical trial can be found at: https://www.clinicaltrials.gov/ct2/show/NCT04375878 Phase 2 OPTION 2 Trial Design The Phase 2b OPTION 2 multi-center trial is designed to investigate the safety, tolerability and efficacy of MS1819 (2.2 and 4.4 gram doses in enteric capsules) in a head-to-head comparison versus the current standard of care, porcine pancreatic enzyme replacement therapy pills. The OPTION 2 trial is an open-label, crossover study, conducted in 15 sites in the U.S. and Europe. A total of 30 CF patients 18 years or older are enrolled. MS1819 will be administered in enteric capsules to provide gastric protection and allow optimal delivery of enzyme to the duodenum. Patients will first be randomized into two cohorts: to either the MS1819 arm, where they receive a 2.2 gram daily oral dose of MS1819 for three weeks; or to the PERT arm, where they receive their pre-study dose of PERT pills for three weeks. After three weeks, stools will be collected for analysis of coefficient of fat absorption. Patients will then be crossed over for another three weeks of the alternative treatment. After three weeks of cross-over therapy, stools will again be collected for analysis of CFA. A parallel group of patients will be randomized and studied in the same fashion, using a 4.4 gram daily dose of MS1819. All patients will be followed for an additional two weeks after completing both crossover treatments for post study safety observation. Patients will be assessed using descriptive methods for efficacy, comparing CFA between MS1819 and PERT arms, and for safety. About MS1819 MS1819 is a recombinant lipase enzyme for the treatment of exocrine pancreatic insufficiency associated with cystic fibrosis and chronic pancreatitis. MS1819, supplied as an oral non-systemic biologic capsule, is derived from the Yarrowia lipolytica yeast lipase and breaks up fat molecules in the digestive tract of EPI patients so that they can be absorbed as nutrients. Unlike the standard of care, the MS1819 synthetic lipase does not contain any animal products. About Exocrine Pancreatic Insufficiency EPI is a condition characterized by deficiency of the exocrine pancreatic enzymes, resulting in a patient’s inability to digest food properly, or maldigestion. The deficiency in this enzyme can be responsible for greasy diarrhea, fecal urge and weight loss. There are more than 30,000 patients in the U.S. with EPI caused by cystic fibrosis according to the Cystic Fibrosis Foundation and approximately 90,000 patients in the U.S with EPI caused by chronic pancreatitis according to the National Pancreas Foundation. Patients are currently treated with porcine pancreatic enzyme replacement pills. About AzurRx BioPharma, Inc. AzurRx BioPharma, Inc. (NASDAQ: AZRX) is a clinical stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases. The Company has a pipeline of three gut-restricted GI assets. The lead therapeutic candidate is MS1819, a recombinant lipase for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis and chronic pancreatitis, currently in two Phase 2 clinical trials. AzurRx is launching two clinical programs using proprietary formulations of niclosamide, a pro-inflammatory pathway inhibitor, FW-420, for grade 1 Immune Checkpoint Inhibitor Colitis and diarrhea in oncology patients and FW-1022, for COVID-19 gastrointestinal infections. The Company is headquartered in Delray Beach, Florida with clinical operations in Hayward, California. For more information, visit www.azurrx.com. Forward-Looking Statement This press release may contain certain statements relating to future results which are forward-looking statements. It is possible that the Company’s actual results and financial condition may differ, possibly materially, from the anticipated results and financial condition indicated in these forward-looking statements, depending on factors including whether results obtained in preclinical and nonclinical studies and clinical trials will be indicative of results obtained in future clinical trials; whether preliminary or interim results from a clinical trial will be indicative of the final results of the trial; and the impact of the coronavirus (COVID-19) pandemic on the Company’s operations and current and planned clinical trials, including potential delays in clinical trial recruitment and participation. Additional information concerning the Company and its business, including a discussion of factors that could materially affect the Company’s financial results are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2019 under the heading “Risk Factors,” as well as the Company’s subsequent filings with the Securities and Exchange Commission. All forward-looking statements included in this press release are made only as of the date of this press release, and we do not undertake any obligation to publicly update or correct any forward-looking statements to reflect events or circumstances that subsequently occur or of which we hereafter become aware. For more information: AzurRx BioPharma, Inc. 1615 South Congress Avenue Suite 103 Delray Beach, Florida 33445 Phone: (646) 699-7855 info@azurrx.com Media contact: Tiberend Strategic Advisors, Inc. Johanna Bennett/Ingrid Mezo (212) 375-2665/(646) 604-5150 jbennett@tiberend.com/imezo@tiberend.com
预计2021年第一季度结束前的顶线数据 佛罗里达州DELRAY BEACH,2021年3月16日(GLOBE NEWSWIRE)--AzurRx BioPharma,Inc.(纳斯达克代码:AZRX)(“AzurRx”或“本公司”)是一家专门开发胃肠道(GI)疾病靶向非全身性疗法的临床阶段生物制药公司,今天宣布它已经完成了2B期选择2临床试验的注册,该试验旨在研究患有外分泌胰腺功能不全(EPI)的囊性纤维化(CF)患者中的MS1819。该试验纳入了30名CF患者,预计2021年第1季度末将获得该研究的最高数据。 第2B期多中心研究旨在研究MS1819在肠溶胶囊中的安全性、耐受性和有效性,并与当前的猪酶替代疗法(PERT)护理标准进行头对头的比较。主要疗效终点是脂肪吸收系数(CFA),次要终点是大便重量、吸收不良的体征和症状以及氮吸收系数(CNA)。该试验还包括使用即时释放MS1819胶囊的延伸臂,允许公司将使用肠溶(延迟释放)胶囊的现有臂的数据与新臂的数据进行比较,并最终选择最优的递送方式。 Azurrx公司首席执行官、总裁兼董事长詹姆斯·萨皮尔斯坦说:“在我们的2B期选项2试验中完成注册是MS1819作为囊性纤维化患者的EPI单药疗法临床发展的一个重要里程碑。“这项试验的数据将为潜在的关键3期研究提供最佳剂量信息,我们将在2021年第一季度末报告这项研究的背线结果。” AzurRx公司的首席医疗官James Pennington博士补充说:“我们MS1918项目的首要目标是提供一种安全有效的疗法来控制EPI并改善目前的护理标准PERT。PERT是猪源性的,要求患者每天服用40粒以上的胶囊来控制症状。MS1819是一种合成脂肪酶,不含任何动物产品,有可能大大减少目前管理EPI所需的每日药丸负担。“ 有关该临床试验的更多信息,请访问:https://www.clinicaltrials.gov/ct2/show/nct04375878 阶段2方案2试验设计 2b期选择2多中心试验旨在研究MS1819(肠溶胶囊中2.2和4.4克剂量)与当前护理标准猪胰酶替代治疗丸的安全性、耐受性和疗效。选择2试验是一项开放的交叉研究,在美国和欧洲的15个站点进行。共纳入30例18岁或18岁以上的CF患者。MS1819将在肠溶胶囊中给药,以提供胃保护,并允许将酶最佳地递送到十二指肠。患者将首先随机分为两组:MS1819组,每天口服2.2克MS1819,持续三周;或者到PERT臂,在那里他们接受他们的研究前剂量的PERT药片三周。三周后收集粪便进行脂肪吸收系数分析。然后,病人将再接受三个星期的替代治疗。交叉治疗三周后,将再次收集粪便进行CFA分析。一个平行的患者组将被随机化,并以同样的方式进行研究,每天使用4.4克的MS1819。所有患者将在完成两个交叉治疗后再随访两周,以进行研究后的安全性观察。将使用描述性方法评估患者的疗效、比较MS1819和PERT两种臂之间的CFA以及安全性。 关于MS1819 MS1819是一种重组脂肪酶,用于治疗囊性纤维化和慢性胰腺炎相关的外分泌胰腺功能不全。MS1819是由Yarrowia lipolytica酵母脂肪酶衍生而成的口服非全身性生物胶囊,在EPI患者消化道中分解脂肪分子,使其作为营养物质被吸收。与护理标准不同,MS1819合成脂肪酶不含任何动物产品。 关于外分泌性胰腺功能不全 EPI是一种以外分泌胰酶缺乏为特征的疾病,导致病人不能正确消化食物,或消化不良。这种酶的缺乏可导致油腻腹泻、排泄和体重下降。 根据囊性纤维化基金会,美国有30,000多名患者患有囊性纤维化引起的EPI,根据国家胰腺基金会,美国约有90,000名患者患有慢性胰腺炎引起的EPI。患者目前使用猪胰酶置换丸治疗。 关于AzurRx BioPharma,Inc. AzurRx BioPharma,Inc.(纳斯达克代码:AZRX)是一家临床阶段生物制药公司,专门从事胃肠道(GI)疾病的靶向非全身疗法的开发。该公司拥有三个肠道受限的GI资产。主要的候选治疗药物是MS1819,一种重组脂肪酶,用于治疗囊性纤维化和慢性胰腺炎患者的外分泌胰腺功能不全(EPI),目前正在进行两个2期临床试验。AzurRx正在启动两个临床项目,使用氯硝柳胺的专有配方,即促炎通路抑制剂FW-420,用于肿瘤患者的1级免疫检查点抑制剂结肠炎和腹泻,以及FW-1022,用于COVID-19胃肠道感染。该公司总部设在佛罗里达州的德尔雷海滩,在加利福尼亚州的海沃德开展临床业务。欲了解更多信息,请访问www.azurrx.com。 前瞻性陈述 本新闻稿可能包含某些与未来业绩相关的前瞻性陈述。公司的实际结果和财务状况可能与这些前瞻性陈述中指出的预期结果和财务状况有很大差异,这取决于以下因素:临床前和非临床研究和临床试验中获得的结果是否表明了未来临床试验中获得的结果;临床试验的初步或中期结果是否表明试验的最终结果;以及冠状病毒(COVID-19)大流行对公司运营以及当前和计划中的临床试验的影响,包括临床试验招募和参与的潜在延迟。有关本公司及其业务的更多信息,包括可能对本公司财务业绩产生重大影响的因素的讨论,载于本公司截至2019年12月31日年度的10-K表格年度报告“风险因素”标题下,以及本公司随后向证券交易委员会提交的文件。本新闻稿中包含的所有前瞻性陈述仅在本新闻稿发布之日起发表,我们不承担任何义务公开更新或更正任何前瞻性陈述,以反映随后发生的或我们此后获悉的事件或情况。 有关更多信息: AzurRx生物制药公司。 南国会大道1615号 103套房 佛罗里达州德尔雷海滩33445 电话:(646)699-7855 @azurrx.com info@azurrx.com 媒体联系人: 泰伯伦战略顾问公司。 Johanna Bennett/Ingrid Mezo (212)375-2665/(646)604-5150 jbennett@tiberend.com/imezo@tiberend.com

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