Mereo BioPharma, the Osteogenesis Imperfecta Federation Europe and the Osteogenesis Imperfecta Foundation announces completion of the IMPACT Survey enrollment

Mereo生物制药、欧洲成骨不全联合会和成骨不全基金会宣布完成影响调查登记

2021-10-14 23:00:06 BioSpace

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Living With Osteogenesis IMPerfecta: UnderstAnding Experiences Based On Community InsighT and Evidence Survey, the IMPACT Survey More than 2200 individual responses collected over a 3-month period from some 65 countries in 8 languages Set to be the largest global gathering of data to date about the impact that Osteogenesis Imperfecta (OI) has on people with OI, families and caregivers LONDON and REDWOOD CITY, Calif., Oct. 13, 2021 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO), “Mereo” or the “Company”, a clinical stage biopharmaceutical company focused on oncology and rare diseases, the Osteogenesis Imperfecta Federation Europe (OIFE) and the Osteogenesis Imperfecta Foundation (OIF) announced completion of enrollment in the largest global gathering of data to date about the impact that OI has on people with OI, their families and caregivers. The IMPACT Survey closed with more than 2200 individual responses collected over a 3-month period from some 65 countries. The survey results will be used to support future collaborative work on better diagnosis, treatment, and care and to support the timely evaluation and availability of potential new treatments for OI. The primary results will be published in 2022. “The importance of these data cannot be overstated. The learnings directly from the OI community will help shape and improve care in a truly meaningful manner,” commented Dr Frank Rauch, Professor of Pediatrics at McGill University, and Chair of the IMPACT Survey Steering Committee. OI is a rare genetic condition caused by a collagen defect, which results in fragile bones and reduced bone mass resulting in bones that break easily. In severe cases patients may experience hundreds of fractures in a lifetime. In addition, people with OI often suffer muscle weakness, early hearing loss, fatigue, curved bones, scoliosis, respiratory and digestive problems, and short stature, leading to significant impacts on overall health and quality of life. Despite its severity, the full impact on people with OI, their families and caregivers are not broadly understood or well-documented. This is similar to many rare diseases, especially those, like OI, where there is no approved therapy. The IMPACT Survey was developed based on the findings from a systematic literature review by Mereo which was presented at the American Society for Bone and Mineral Research (ASBMR) 2021 and is expected to be fully published in 2022. The IMPACT Survey was made possible through a close collaboration and partnership between Mereo, Ultragenyx (partners for the development of setrusumab for the treatment of OI) and the OI community, led by the umbrella organizations OIFE and OIF. “The IMPACT Survey was originally a company suggestion, but the OI organizations embraced and took ownership of it. With expert support, a unique survey that felt relevant to people with OI and parents was developed. Relevance and good collaboration were success criteria. The OI community understood the importance of big data and took to completing this survey in record numbers,” observed Ingunn Westerheim, President of OIFE. “We are thrilled to have collected the largest global gathering of data about the impact that osteogenesis imperfecta has on people with OI, families, and caregivers. This is a remarkable number of responses, and we eagerly await the first data publications,” added Tracy Hart, CEO of OIF. The project is being overseen by a Steering Committee comprising representatives from both OIFE and OIF, specialized physicians treating both children and adults affected by OI and representatives from Mereo and Ultragenyx. A Data Management Committee will be responsible for ownership and management of the data and will decide on future requests from any party for use of the anonymized data. “We wish to thank OIFE, OIF and the OI organizations around the world for supporting, leading and driving this initiative. We would also like to thank all the volunteers who supported the development, translation and promotion of the survey and, finally, the more than 2200 participants who took the time to complete the IMPACT Survey. We could not have achieved this outstanding outcome without all of you,” stated Arun Mistry, Vice President, Therapeutic Area Head for Setrusumab at Mereo BioPharma. About the IMPACT Survey Mereo financially supported the development and delivery of the IMPACT Survey, a joint initiative with the OIFE and the OIF. Mereo provided full financial sponsorship to the third-party scientific agency conducting the work. About Mereo BioPharma Mereo BioPharma is a biopharmaceutical company focused on the development and commercialization of innovative therapeutics that aim to improve outcomes for oncology and rare diseases. The Company has developed a portfolio of six clinical stage product candidates. Mereo’s lead oncology product candidate, etigilimab (Anti-TIGIT), has recently advanced into an open label Phase 1b/2 basket study evaluating Anti-TIGIT in combination with an anti-PD-1 in a range of tumor types including three rare tumors and a number of gynecological carcinomas including cervical, ovarian and endometrial carcinomas. The Company’s second oncology product, navicixizumab, for the treatment of late line ovarian cancer, has completed a Phase 1 study and has been partnered with OncXerna Therapeutics, Inc., formerly Oncologie, Inc. The Company has two rare disease product candidates: alvelestat for the treatment of severe Alpha-1 antitrypsin deficiency (AATD), which is being investigated in an ongoing Phase 2 proof-of-concept study in the U.S. and Europe, for which the Company expects to provide an update in the fourth quarter of 2021, and setrusumab for the treatment of osteogenesis imperfecta (OI). In September 2020, the FDA granted Rare Pediatric Disease designation to setrusumab for the treatment of OI. In December 2020, the Company signed a license and collaboration agreement for setrusumab in OI with Ultragenyx Pharmaceutical Inc. Forward-Looking Statements This press release contains “forward-looking statements.” All statements other than statements of historical fact contained in this press release are forward-looking statements within the meaning of Section 27A of the United States Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the United States Securities Exchange Act of 1934, as amended (the “Exchange Act”). Forward-looking statements usually relate to future events and anticipated revenues, earnings, cash flows or other aspects of our operations or operating results. Forward-looking statements are often identified by the words “believe,” “expect,” “anticipate,” “plan,” “intend,” “foresee,” “should,” “would,” “could,” “may,” “estimate,” “outlook” and similar expressions, including the negative thereof. The absence of these words, however, does not mean that the statements are not forward-looking. These forward-looking statements are based on the Company’s current expectations, beliefs and assumptions concerning future developments and business conditions and their potential effect on the Company. While management believes that these forward-looking statements are reasonable as and when made, there can be no assurance that future developments affecting the Company will be those that it anticipates. All of the Company’s forward-looking statements involve known and unknown risks and uncertainties some of which are significant or beyond its control and assumptions that could cause actual results to differ materially from the Company’s historical experience and its present expectations or projections. You should carefully consider the foregoing factors and the other risks and uncertainties that affect the Company’s business, including those described in the “Risk Factors” section of its latest Annual Report on Form 20-F, reports on Form 6-K and other documents furnished or filed from time to time by the Company with the Securities and Exchange Commission. The Company wishes to caution you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof. The Company undertakes no obligation to publicly update or revise any of our forward-looking statements after the date they are made, whether as a result of new information, future events or otherwise, except to the extent required by law. Mereo BioPharma Contacts:
与成骨不全症生活在一起:基于社区洞察和证据调查的理解经验,影响调查 在3个月的时间里,从大约65个国家以8种语言收集了2200多份个人答复 这将是迄今为止最大的关于成骨不全(OI)对OI患者、家庭和护理人员影响的全球数据收集 伦敦和加州红木城,2021年10月13日(环球新闻)--梅雷奥生物制药集团(纳斯达克代码:MREO),“梅雷奥”或“公司”,一家专注于肿瘤学和罕见疾病的临床阶段生物制药公司,欧洲成骨不全联合会(OIFE)和成骨不全基金会(OIF)宣布完成迄今为止最大的关于OI对OI患者、他们的家人和护理人员影响的全球数据收集。影响调查结束时,在3个月的时间里,从大约65个国家收集了2200多份个人答复。调查结果将用于支持未来关于更好的诊断、治疗和护理的合作工作,并支持及时评估和提供潜在的OI新治疗方法。初选结果将于2022年公布。 “这些数据的重要性怎么强调都不为过。麦吉尔大学儿科教授、影响调查指导委员会主席弗兰克·劳奇博士评论说:“直接从OI社区学习将有助于以真正有意义的方式塑造和改善护理。” OI是一种罕见的遗传疾病,由胶原蛋白缺陷引起,导致骨骼脆弱和骨量减少,导致骨骼容易断裂。在严重的情况下,患者一生中可能会经历数百次骨折。此外,患有OI的人经常出现肌肉无力、早期听力损失、疲劳、骨骼弯曲、脊柱侧弯、呼吸和消化问题以及身材矮小,从而对整体健康和生活质量产生重大影响。尽管它很严重,但对患有OI的人、他们的家人和护理人员的全部影响并没有得到广泛的理解或充分的记录。这与许多罕见的疾病相似,尤其是像OI这样没有批准治疗的疾病。 这项影响调查是基于Mereo在2021年美国骨与矿物研究学会(ASBMR)上提交的系统文献综述的发现而开发的,预计将于2022年完全发表。在伞式组织OIFE和OIF的领导下,Mereo、Ultragenyx(开发setrusumab治疗OI的合作伙伴)和OI社区之间的密切合作和伙伴关系使影响调查成为可能。 “影响调查最初是公司的建议,但OI组织接受并拥有它。在专家的支持下,开发了一项独特的调查,该调查认为与OI患者和父母相关。相关性和良好的合作是成功的标准。OI社区理解大数据的重要性,并开始以创纪录的数量完成这项调查,“OIFE总裁Ingunn Westerheim观察到。 “我们很高兴能收集到关于成骨不全对OI患者、家庭和护理人员影响的全球最大数据。OIF首席执行官特雷西·哈特补充说:“这是一个惊人的回应数量,我们急切地等待第一批数据的发布。” 该项目由一个指导委员会监督,该委员会由来自OIFE和OIF的代表、治疗受OI影响的儿童和成人的专业医生以及Mereo和UltraGenyx的代表组成。一个数据管理委员会将负责数据的所有权和管理,并将决定任何一方今后使用匿名数据的请求。 “我们要感谢世界各地的OIFE、OIF和OI组织支持、领导和推动这项倡议。我们还要感谢所有支持调查的制定、翻译和推广的志愿者,最后还要感谢花时间完成影响调查的2200多名参与者。没有你们所有人,我们不可能取得这一杰出的结果,“梅雷奥生物制药公司Setrusumab治疗领域负责人副总裁阿伦·米斯特里说。 关于影响调查 Mereo为影响调查的制定和实施提供了财政支助,这是一项与地方选举办公室和法语国家组织的联合倡议。Mereo向开展这项工作的第三方科学机构提供了全额财政赞助。 关于Mereo生物制药 Mereo BioPharma是一家生物制药公司,专注于创新疗法的开发和商业化,旨在改善肿瘤学和罕见疾病的结果。该公司已经开发了六个临床阶段的候选产品组合。Mereo的主要肿瘤候选产品etigilimab(Anti-TIGIT)最近进入了一项开放标记1B/2阶段篮子研究,该研究评估了抗Tigit与抗PD-1结合在一系列肿瘤类型中的作用,包括三种罕见肿瘤和包括宫颈癌、卵巢癌和子宫内膜癌在内的许多妇科癌症。该公司的第二个肿瘤学产品navicixizumab用于治疗晚期卵巢癌,已经完成了1期研究,并与OncXerna Therapeutics,Inc.(前Oncologie,Inc.)合作。该公司有两个罕见疾病候选产品:用于治疗严重α-1抗胰蛋白酶缺乏症(AATD)的alvelestat,该产品正在美国和欧洲进行的2期概念验证研究中进行研究,该公司预计将在2021年第四季度提供更新,以及用于治疗成骨不全症(OI)的setrusumab。2020年9月,美国食品和药物管理局批准了setrusumab治疗OI的罕见儿科疾病指定。2020年12月,该公司与Ultragenyx Pharmaceutical Inc.签署了setrusumab在OI的许可和合作协议。 前瞻性陈述 本新闻稿包含“前瞻性陈述”。除历史事实陈述外,本新闻稿中包含的所有陈述均为符合经修订的1933年美国证券法第27A条(“证券法”)和经修订的1934年美国证券交易法第21E条(“交易法”)含义的前瞻性陈述。前瞻性陈述通常与未来事件和预期收入、收益、现金流量或我们经营或经营成果的其他方面有关。前瞻性陈述通常由“相信”、“预期”、“预期”、“计划”、“打算”、“预见”、“应该”、“将会”、“可能”、“可能”、“估计”、“展望”等词和类似的表达来识别,包括其否定。然而,没有这些词语并不意味着这些陈述没有前瞻性。这些前瞻性陈述是基于本公司目前对未来发展和业务状况及其对本公司潜在影响的预期、信念和假设。虽然管理层认为这些前瞻性陈述在作出时是合理的,但不能保证影响本公司的未来发展将是其预期的发展。 本公司的所有前瞻性陈述都涉及已知和未知的风险和不确定性,其中一些是重大的或超出其控制范围的风险和不确定性,以及可能导致实际结果与本公司的历史经验及其目前的预期或预测大不相同的假设。您应仔细考虑上述因素以及影响本公司业务的其他风险和不确定因素,包括其最新年度报告中关于表格20-F的“风险因素”一节、关于表格6-K的报告以及本公司不时向证券交易委员会提供或提交的其他文件中描述的风险和不确定因素。本公司希望提醒您不要过分依赖任何前瞻性陈述,这些陈述仅限于本报告所述日期。除法律规定的范围外,本公司没有义务在我们的任何前瞻性陈述发表后公开更新或修改这些陈述,无论是由于新的信息、未来事件或其他原因。 Mereo生物制药联系人:

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