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Savara宣布罕见肺部疾病——自身免疫性肺泡蛋白沉积症(aPAP)在Lifetime®电视上播出的新一集《平衡法案》中得到了强调

Savara Announces the Rare Lung Disease, Autoimmune Pulmonary Alveolar Proteinosis (aPAP), is Highlighted on a New Episode of “The Balancing Act ® ” Airing on Lifetime ® TV

businesswire 等信源发布 2024-10-22 18:05

可切换为仅中文


LANGHORNE, Pa.--(BUSINESS WIRE)--Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, announced that a new episode of The Balancing Act®, sponsored by Savara and featuring autoimmune Pulmonary Alveolar Proteinosis (aPAP), is airing on Lifetime TV’s Behind the Mystery™ a recurring series on The Balancing Act® television show devoted to advocating for people with rare and genetic diseases.

宾夕法尼亚州兰格霍恩(商业新闻短讯)--萨瓦拉公司(Nasdaq:SVRA)(该公司)是一家专注于罕见呼吸系统疾病的临床阶段生物制药公司,宣布由萨瓦拉赞助并以自身免疫性肺泡蛋白沉着症(aPAP)为特色的平衡法案(Balancing Act®)的新一集将在终身电视节目《神秘背后》(Behind the Mystery™)上播出,这是平衡法案(Balancing Act®)电视节目上反复播出的一集,致力于为患有罕见疾病和遗传疾病的人提供支持。

The episode centers on the diagnostic journey of an aPAP patient and includes insights from Bruce Trapnell, M.D., Professor of Medicine and Pediatrics, University of Cincinnati College of Medicine and the International Coordinating Investigator for Savara’s IMPALA-2 Phase 3 clinical trial of molgramostim inhalation solution (molgramostim) in aPAP..

本集以aPAP患者的诊断过程为中心,包括来自辛辛那提大学医学院医学和儿科教授、医学博士布鲁斯·特拉普内尔(BruceTrapnell)的见解,以及萨瓦拉(Savara)IMPALA-2 molgramostim吸入溶液(molgramostim)在aPAP的3期临床试验的国际协调研究者。。

The episode provides a look into the real-life story of an aPAP patient—highlighting his journey to diagnosis, disease management, and need for improved treatment options. A replay of the episode can be viewed at https://bit.ly/480OPyy.

这一集讲述了一名aPAP患者的真实故事,强调了他的诊断之旅,疾病管理以及对改进治疗方案的需求。这一集的重播可以在https://bit.ly/480OPyy.

“We are pleased to partner with The Balancing Act to highlight aPAP, a rare respiratory disease with no currently approved medicines in the United States or Europe and a high unmet need for patients,” said Matt Pauls, Chair and CEO, Savara. “Educating people about the hallmark signs and symptoms of aPAP, as well as the simple, accurate, non-invasive blood test to help diagnose the disease, may increase awareness for this debilitating condition and provide a quicker path to diagnosis for patients.

。“教育人们了解aPAP的标志性体征和症状,以及简单,准确,无创的血液检测来帮助诊断疾病,可能会提高人们对这种衰弱状况的认识,并为患者提供更快的诊断途径。

We are committed to the global aPAP community and continue to work towards our goal of potentially providing an effective therapy for patients.”.

我们致力于全球aPAP社区,并继续努力实现为患者提供有效治疗的目标。”。

The Company expects to complete submission of the Biologics License Application (BLA) for molgramostim in aPAP with the U.S. Food and Drug Administration (FDA) in 1H 2025.

该公司预计将于2025年上半年在aPAP向美国食品和药物管理局(FDA)提交molgramostim的生物制剂许可证申请(BLA)。

About aPAP

关于aPAP

Autoimmune PAP (aPAP) is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli (or air sacs) of the lungs. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages.

自身免疫性PAP(aPAP)是一种罕见的肺部疾病,其特征是肺泡(或气囊)中表面活性剂的异常积聚。表面活性剂由蛋白质和脂质组成,是一种重要的生理物质,排列在肺泡上以防止其塌陷。在健康的肺中,过量的表面活性剂被称为肺泡巨噬细胞的免疫细胞清除和消化。

Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in autoimmune PAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas exchange, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue.

肺泡巨噬细胞需要被粒细胞巨噬细胞集落刺激因子(GM-CSF)刺激才能正确清除表面活性剂,但在自身免疫性PAP中,GM-CSF被抗GM-CSF的抗体中和,使巨噬细胞无法充分清除表面活性剂。因此,过量的表面活性剂积聚在肺泡中,导致气体交换受损,导致呼吸急促的临床症状,通常伴有咳嗽和频繁疲劳。

Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary lung infection develops. In the long-term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant..

患者还可能出现发烧,胸痛或咳血的发作,尤其是在发生继发性肺部感染的情况下。。。

About Savara

关于萨瓦拉

Savara is a clinical stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim inhalation solution (molgramostim), is an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune Pulmonary Alveolar Proteinosis (aPAP).

Savara是一家临床阶段的生物制药公司,专注于罕见的呼吸道疾病。。

Molgramostim is delivered via an investigational eFlow® Nebulizer System (PARI Pharma GmbH). Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at www.savarapharma.com.

Molgramostim通过研究性eFlow®雾化器系统(PARI Pharma GmbH)提供。我们的管理团队在罕见呼吸系统疾病和肺部医学方面拥有丰富的经验,能够识别未满足的需求,并有效地推动候选产品获得批准和商业化。有关更多信息,请访问www.savarapharma.com。

(X, formerly known as Twitter: @SavaraPharma, LinkedIn: www.linkedin.com/company/savara-pharmaceuticals/)..

(X,以前称为推特:@SavaraPharma,LinkedIn:www.LinkedIn.com/company/savara pharmaceuticals/)。。

About Behind the Mystery™

关于神秘背后™

Behind the Mystery™ is a recurring series on The Balancing Act® television show devoted to advocating for rare and genetic diseases. The series clarifies the often-complex issues by offering easy-to-understand explanations from top experts in the field through partnerships with foundations and pharmaceutical companies.

《神秘的背后》是一部关于平衡法案(Balancing Act®)电视节目的反复播出的系列剧,致力于倡导罕见疾病和遗传疾病。该系列通过与基金会和制药公司的合作,为该领域的顶级专家提供了易于理解的解释,从而澄清了通常很复杂的问题。

We amplify the voices of patients, families, and advocates affected by rare diseases via our national television platform. Tune in to Behind the Mystery weekdays at 7:30 a.m. (ET/PT) on Lifetime® and find all previously aired episodes on TheBalancingAct.com/rare..

我们通过国家电视平台扩大了受罕见疾病影响的患者、家属和倡导者的声音。在Lifetime®上,早上7:30(东部时间/东部时间)收听《神秘工作日背后》,并在TheBalancingAct.com/rare上找到之前播出的所有剧集。。

Forward-Looking Statements

前瞻性声明

Savara cautions you that statements in this press release that are not a description of historical fact are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances such as “expect,” “intend,” “plan,” “anticipate,” “believe,” and “will,” among others.

萨瓦拉提醒您,本新闻稿中的声明并非对历史事实的描述,而是1995年《私人证券诉讼改革法案》所指的前瞻性声明。前瞻性陈述可以通过使用引用未来事件或情况的词语来识别,例如“预期”、“打算”、“计划”、“预期”、“相信”和“将会”等。

Such statements include, but are not limited to, statements regarding education potentially leading to increased awareness of aPAP and a quicker path to diagnosis, Savara’s goal of potentially providing an effective therapy, and the anticipated timing of Savara’s BLA submission. Savara may not actually achieve any of the matters referred to in such forward-looking statements, and you should not place undue reliance on these forward-looking statements.

这些声明包括但不限于有关教育的声明,这些声明可能会提高对aPAP的认识和更快的诊断途径,Savara可能提供有效治疗的目标以及Savara提交BLA的预期时间。萨瓦拉可能无法实际实现此类前瞻性声明中提及的任何事项,您不应过度依赖这些前瞻性声明。

These forward-looking statements are based upon Savara’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the risks associated with our ability to successfully develop, obtain regulatory approval for, and commercialize molgramostim for aPAP; the actions and decisions of regulatory authorities; the risks and uncertainties related to the impact of widespread health concerns or changing economic or geopolitical conditions; the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations; the availability of sufficient resources for Savara’s operations and to conduct or continue planned clinical development programs;.

这些前瞻性陈述基于萨瓦拉目前的预期,并涉及可能永远不会实现或可能被证明不正确的假设。由于各种风险和不确定性,实际结果和事件发生的时间可能与此类前瞻性声明中的预期有很大不同,其中包括但不限于与我们成功开发、获得监管部门批准以及将aPAP的molgramostim商业化相关的风险;监管机构的行动和决定;与广泛的健康问题或不断变化的经济或地缘政治条件的影响有关的风险和不确定性;预测或有未来负债和业务运营所需的未来现金利用率和准备金的能力;;。