Beyond Air® Reports Positive Interim Results for LungFit® GO Pilot Study Using High Concentration Inhaled Nitric Oxide Self-Administered, At-Home for Nontuberculous Mycobacterial Lung Disease

Beyond Air®报告了LungFit®GO试点研究的积极中期结果,该研究使用高浓度吸入一氧化氮在家自我给药治疗非结核性分枝杆菌性肺病

2021-10-21 18:00:07 BioSpace

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Interim results show that 250 parts per million nitric oxide was well-tolerated with no study discontinuations or treatment-related serious adverse events observed At the time of data cutoff, 8 subjects were successfully titrated up to 250 ppm NO with none having titrated down while in the study LungFit® GO is the first NO generator and delivery system safely used in a clinical trial in the home setting with patients self-administering high concentration NO treatment GARDEN CITY, N.Y., Oct. 20, 2021 (GLOBE NEWSWIRE) -- Beyond Air, Inc. (NASDAQ: XAIR), a clinical-stage medical device and biopharmaceutical company focused on developing inhaled nitric oxide (NO) for the treatment of patients with respiratory conditions, including serious lung infections and pulmonary hypertension, and gaseous NO (gNO) for the treatment of solid tumors, today announced positive interim data from the ongoing LungFit® GO pilot study in Australia. In this study, patients self-administered high concentration inhaled NO at home to treat severe nontuberculous mycobacterial (NTM) lung disease. The Company expects complete safety and efficacy results to be reported in 2022. At the time of the data cutoff on September 6, 2021, a total of 8 subjects were enrolled in the pilot study. The mean age of subjects was 56.6 years (range: 22–73 years) with the majority female (87.5%), a distribution consistent with real-world NTM disease, and occurring at a higher rate in older adult women than men1. At baseline some subjects were diagnosed with more than one strain of NTM. Interim data showed that high concentration inhaled NO was well tolerated with no study discontinuations and no treatment-related serious adverse events. All 8 subjects were successfully titrated to 250 ppm NO in the hospital setting, and none have required dose reductions during the subsequent at-home portion of the study. Methemoglobin and NO2 concentrations remained within acceptable ranges in all subjects during NO treatment, and below the safety thresholds of 10% and 5 ppm, respectively. The study continues to enroll patients, and the totality of the data will be used to evaluate efficacy measures including quality of life, physical function, and sputum bacteria as compared to baseline measurements. “We believe these data show that safe self-administration of nitric oxide at concentrations up to 250 ppm in the home setting is now a reality. This therapeutic advance was made possible by the Beyond Air engineering team’s ability and dedication to produce the LungFit® system. We believe that LungFit® GO will ultimately offer freedom to NTM patients, and others suffering from chronic refractory severe lung infections, by allowing them to self-administer treatment in the comfort of their own homes or anywhere they choose with a standard electric outlet available,” said Steve Lisi, Chairman and Chief Executive Officer of Beyond Air. “The Beyond Air team continues to work with our Australian colleagues, who have been outstanding in a difficult COVID-19 lockdown environment, to complete this study. We are encouraged by these interim data and look forward to reporting the complete efficacy and safety results, which are expected to be announced in 2022.” “I am very pleased with the safety profile of 250 ppm NO to date, as this is the highest concentration of inhaled NO that has ever been tested in a clinical trial with patient self-administration in the home. The bactericidal effects of high concentration NO on the Mycobacterium abscessus strain of NTM have been well documented in preclinical in vitro studies, along with its broad-spectrum activity against multiple bacterial and fungal pathogens at 200 ppm NO,” commented Andrew Colin, M.D., Batchelor Family Professor of Cystic Fibrosis and Pediatric Pulmonology Director, Division of Pediatric Pulmonology, Miller School of Medicine, University of Miami. “Refractory NTM lung infection is notoriously difficult to treat, and patients often suffer side effects and require complex dosing schedules to complete the 6-to-18-month combination antibiotic regimens that are the current standard of care. Given these new interim safety and tolerability data, with methemoglobin and nitrogen dioxide levels remaining at acceptable levels, the future for the LungFit® GO device is promising. I look forward to the final analyses of the full data set and hope to see results to support a move to a larger, definitive study in NTM patients. If successful, such a study could prove transformative for the lives of NTM patients.” ________________________ 1 Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). The Voice of the Patient. Non-Tuberculous Mycobacterial (NTM) Lung Infection. Public Meeting: October 15, 2015. Report Date: April, 2016. accessed 10/4/2021 at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM496941.pdf LungFit® GO NTM Trial Design The 12-week, multi-center, open-label clinical trial is taking place in Australia and will enroll approximately 20 adult subjects with chronic refractory NTM lung disease. The trial is enrolling both cystic fibrosis (CF) and non-CF subjects chronically infected with Mycobacterium avium complex (MAC), Mycobacterium abscessus complex (MABSC) or other strains of NTM. The trial consists of a run-in period followed by two treatment phases. The run-in period provides a baseline for the efficacy endpoints, such as patient physical function and bacterial load. The first treatment phase takes place over a two-week period and begins in the hospital setting where subjects are titrated from 150 ppm NO up to 250 ppm NO over several days. During this first treatment phase subjects receive NO for 40 minutes, four times per day while methemoglobin and nitrogen dioxide (NO2) levels are monitored. Subjects are trained to use LungFit® GO and subsequently discharged to complete the remaining portion of this two-week treatment period at their home administering the highest tolerated NO concentration. For the second treatment phase, a 10-week maintenance phase, the inhalation treatments are administered twice daily. The study evaluates safety, quality of life, physical function, and bacterial load among other parameters, as compared to baseline measurements. About Beyond Air, Inc. Beyond Air, Inc. is a clinical-stage medical device and biopharmaceutical company developing a revolutionary NO Generator and Delivery System, LungFit®, that uses NO generated from ambient air to deliver precise amounts of NO to the lungs for the potential treatment of a variety of pulmonary diseases. The LungFit® can generate up to 400 ppm of NO, for delivery either continuously or for a fixed amount of time and has the ability to either titrate dose on demand or maintain a constant dose. The Company is currently applying its therapeutic expertise to develop treatments for pulmonary hypertension in various settings, in addition to treatments for respiratory tract infections that are not effectively addressed with current standards of care. Beyond Air is currently advancing its revolutionary LungFit® for clinical trials for the treatment of severe lung infections such as acute viral pneumonia (including COVID-19) and nontuberculous mycobacteria (NTM). Additionally, Beyond Air is using ultra-high concentrations of NO with a proprietary delivery system to target certain solid tumors in the pre-clinical setting. For more information, visit www.beyondair.net. About the LungFit®* Beyond Air’s is a portable device that weighs only 20 lbs. and operates with a standard electrical outlet (120-240 volts). Since NO is generated from ambient air that flows through a reaction chamber, there is an unlimited supply. Beyond Air’s proprietary nitrogen dioxide (NO2) filters are required for the system to generate and safely deliver NO. Toxic levels of NO2 can result from high concentrations of NO without proper filtration. The filters also program the system, via an attached RFID chip, with respect to NO concentration, flow rate and duration of therapy. The Company believes this design provides maximum flexibility for NO administration. Filters are single patient use and there are no special requirements for disposal. Alarms monitor system performance. * Beyond Air’s LungFit® is not approved for commercial use. Beyond Air’s LungFit® is for investigational use only. About Nitric Oxide (NO) Nitric Oxide (NO) is a powerful molecule, naturally synthesized in the human body, proven to play a critical role in a broad array of biological functions. In the airways, NO targets the vascular smooth muscle cells that surround the small resistance arteries in the lungs. Currently, exogenous inhaled NO is used in adult respiratory distress syndrome, post certain cardiac surgeries, and persistent pulmonary hypertension of the newborn to treat hypoxemia. Additionally, NO is believed to play a key role in the innate immune system and in vitro studies suggest that NO possesses antimicrobial activity not only against common gram-positive and gram-negative bacteria, but also against other diverse pathogens, including mycobacteria, viruses, fungi, yeast and parasites, and has the potential to eliminate multi-drug resistant strains. About NTM Nontuberculous mycobacteria (NTM) infection is a rare and serious bacterial infection in the lungs causing debilitating pulmonary disease associated with high morbidity and mortality. NTM infection is acquired by inhaling aerosolized bacteria from the environment, and can lead to NTM lung disease, a progressive and chronic condition. According to the Cystic Fibrosis Foundation, 13% of U.S. cystic fibrosis patients had a positive culture for a NTM species in 2017. NTM is an emerging public health concern worldwide because of its multi-drug antibiotic resistance. Current treatment guidelines suggest a combination of multiple antibiotics dosed chronically for as long as two years. These complex, expensive and invasive regimens have a poor record in the treatment of Mycobacterium abscessus complex (MABSC) and refractory Mycobacterium avium complex (MAC) and have the potential to cause severe adverse events. Beyond Air’s system is designed to deliver 150 - 400 ppm NO to the lung, and early data indicate that this range of NO concentrations could have a positive effect on patients infected with NTM. Forward Looking Statements This press release contains “forward-looking statements” concerning inhaled nitric-oxide and the Company’s LungFit®† product, including statements with regard to potential regulatory developments, the potential impact on patients and anticipated benefits associated with its use. Forward-looking statements include statements about our expectations, beliefs, or intentions regarding our product offerings, business, financial condition, results of operations, strategies or prospects. You can identify such forward-looking statements by the words “anticipates,” “expects,” “intends,” “impacts,” “plans,” “projects,” “believes,” “estimates,” “likely,” “goal,” “assumes,” “targets” and similar expressions and/or the use of future tense or conditional constructions (such as “will,” “may,” “could,” “should” and the like) and by the fact that these statements do not relate strictly to historical or current matters. Rather, forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to risks and uncertainties that could cause our actual results to differ materially from any future results expressed or implied by the forward-looking statements. These forward-looking statements are only predictions and reflect our views as of the date they are made with respect to future events and financial performance. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements, including risks related to: our approach to discover and develop novel drugs, which is unproven and may never lead to efficacious or marketable products; our ability to fund and the results of further pre-clinical and clinical trials; obtaining, maintaining and protecting intellectual property utilized by our products; our ability to enforce our patents against infringers and to defend our patent portfolio against challenges from third parties; our ability to obtain additional funding to support our business activities; our dependence on third parties for development, manufacture, marketing, sales, and distribution of products; the successful development of our product candidates, all of which are in early stages of development; obtaining regulatory approval for products; competition from others using technology similar to ours and others developing products for similar uses; our dependence on collaborators; our short operating history and other risks identified and described in more detail in the “Risk Factors” section of the Company’s most recent Annual Report on Form 10-K and other filings with the SEC, all of which are available on our website. We undertake no obligation to update, and we do not have a policy of updating or revising, these forward-looking statements, except as required by applicable law. CONTACTS: Maria Yonkoski, Head of Investor Relations Beyond Air, Inc. Myonkoski@beyondair.net Corey Davis, Ph.D. LifeSci Advisors, LLC Cdavis@lifesciadvisors.com (212) 915-2577
中期结果显示,百万分之250的一氧化氮耐受良好,没有研究中断或与治疗相关的严重不良事件观察 在数据截断时,8名受试者成功地滴定了高达250ppm的NO,没有一名受试者在研究中滴定下来 LungFit®GO是第一个在家庭环境中安全用于临床试验的NO发生器和输送系统,患者可以自行给予高浓度NO治疗 纽约花园城,2021年10月20日(环球新闻)--超越空气公司(纳斯达克代码:XAIR)是一家临床阶段医疗设备和生物制药公司,专注于开发用于治疗呼吸系统疾病(包括严重肺部感染和肺动脉高压)患者的吸入性一氧化氮(NO)和用于治疗实体瘤的气态一氧化氮(gNO),今天宣布了正在进行的LungFit®GO试点研究的积极中期数据。在本研究中,患者在家自行给予高浓度吸入NO治疗重症非结核分枝杆菌(NTM)肺病。该公司预计2022年将报告完整的安全性和有效性结果。 在2021年9月6日数据截断时,共有8名受试者参加了试点研究。研究对象的平均年龄为56.6岁(22-73岁),以女性为主(87.5%),这一分布与现实世界的NTM疾病相一致,老年女性的发病率高于男性1。在基线时,一些受试者被诊断患有一种以上的NTM菌株。 中期数据显示,高浓度吸入NO耐受性良好,没有研究中断,也没有与治疗相关的严重不良事件。所有8名受试者在医院环境中都成功地被滴定到250ppm的NO,在随后的家庭研究中没有一人需要减少剂量。高铁血红蛋白和NO2浓度在NO治疗期间均保持在可接受范围内,分别低于10%和5 ppm的安全阈值。该研究继续招募患者,所有数据将用于评估疗效措施,包括与基线测量相比的生活质量、身体功能和痰菌。 “我们相信这些数据表明,在家庭环境中安全地自我服用浓度高达250ppm的一氧化氮现在已经成为现实。这一治疗进展是由超越空气工程团队的能力和献身精神生产的LungFit®系统所实现的。Beyond Air董事长兼首席执行官史蒂夫·利西(Steve Lisi)表示:“我们相信,LungFit®GO最终将为NTM患者和其他患有慢性难治性严重肺部感染的人提供自由,允许他们在舒适的家中或他们选择的任何地方通过标准插座进行自我治疗。”“超越航空团队继续与我们的澳大利亚同事合作,完成这项研究,他们在困难的新冠肺炎封锁环境中表现出色。我们对这些中期数据感到鼓舞,并期待报告完整的疗效和安全性结果,预计将于2022年公布。“ “我对迄今为止250 ppm NO的安全性非常满意,因为这是在临床试验中用患者在家自我给药测试的最高浓度的吸入NO。高浓度NO对NTM脓肿分枝杆菌菌株的杀菌作用,以及在200ppm NO时对多种细菌和真菌病原体的广谱活性,已经在临床前体外研究中得到了很好的证明,“迈阿密大学米勒医学院囊性纤维化Batchelor家族教授和儿科肺系主任Andrew Colin博士评论道。“众所周知,难治性NTM肺部感染很难治疗,患者经常遭受副作用,并需要复杂的给药时间表来完成目前的护理标准--6至18个月的联合抗生素方案。鉴于这些新的临时安全和耐受性数据,高铁血红蛋白和二氧化氮水平保持在可接受的水平,LungFit®GO设备的未来是有希望的。我期待着对完整数据集的最终分析,并希望看到结果来支持对NTM患者进行更大的、明确的研究。如果成功,这样的研究可能会改变NTM患者的生活。“ ______________________ 1美国食品和药物管理局(FDA)药物评价和研究中心(CDER)。病人的声音。非结核分枝杆菌(NTM)肺部感染。公开会议:2015年10月15日。报告日期:2016年4月。10/4/2021年访问http://www.fda.gov/downloads/forindustry/userfees/primptiondruguserfee/ucm496941.pdf LungFit®GO NTM试验设计 这项为期12周、多中心、开放标签的临床试验正在澳大利亚进行,将招募大约20名患有慢性难治性NTM肺病的成人受试者。这项试验包括囊性纤维化(CF)和长期感染鸟分枝杆菌复合体(MAC)、脓肿分枝杆菌复合体(MABSC)或其他NTM菌株的非CF受试者。试验包括一个磨合期,随后是两个治疗阶段。磨合期为疗效终点提供基线,如患者身体功能和细菌负荷。第一个治疗阶段在两周内进行,并在医院环境中开始,受试者在几天内从150 ppm NO滴定到250 ppm NO。在第一个治疗阶段,受试者每天接受4次40分钟的NO治疗,同时监测高铁血红蛋白和二氧化氮(NO2)水平。受试者被训练使用LungFit®GO,随后出院,在家中完成这两周治疗期的剩余部分,服用最高耐受NO浓度。对于第二个治疗阶段,为期10周的维持阶段,吸入治疗每天进行两次。这项研究评估了安全性、生活质量、身体功能和细菌负荷等参数,与基线测量相比较。 关于超越航空公司。 Beyond Air公司是一家临床阶段的医疗设备和生物制药公司,开发了一种革命性的NO发生器和输送系统,LungFit®,该系统利用环境空气中产生的NO向肺部输送精确数量的NO,用于各种肺部疾病的潜在治疗。LungFit®可以产生高达400 ppm的NO,用于连续或固定时间的输送,并有能力根据需求滴定剂量或保持恒定剂量。该公司目前正在应用其治疗专业知识,开发各种环境下肺动脉高压的治疗方法,以及目前护理标准无法有效解决的呼吸道感染的治疗方法。Beyond Air目前正在推进其革命性的LungFit®,用于治疗急性病毒性肺炎(包括新冠肺炎)和非结核分枝杆菌(NTM)等严重肺部感染的临床试验。此外,超越空气正在使用超高浓度的NO和专有的递送系统,在临床前的环境中靶向某些实体瘤。欲了解更多信息,请访问www.beyondair.net。 关于LungFit®* Beyond Air是一款仅重20磅的便携式设备。并使用标准电源插座(120-240伏)。由于NO是从流经反应室的环境空气中产生的,所以有无限量的供应。Beyond Air专有的二氧化氮(NO2)过滤器需要系统产生和安全地输送NO。如果没有适当的过滤,高浓度的NO可能导致有毒的NO2水平。过滤器还通过附加的RFID芯片对系统进行NO浓度、流量和治疗持续时间的编程。该公司认为,这种设计为无管理提供了最大的灵活性。过滤器是单一的病人使用,没有特殊的要求处置。警报监视系统性能。 *Beyond Air的LungFit®不被批准用于商业用途。Beyond Air的LungFit®仅供调查使用。 关于一氧化氮(NO) 一氧化氮(NO)是一种强大的分子,在人体内自然合成,被证明在一系列广泛的生物功能中发挥着关键作用。在气道中,NO的靶点是围绕在肺部小阻力动脉周围的血管平滑肌细胞。目前,外源性吸入NO用于成人呼吸窘迫综合征、某些心脏手术后和新生儿持续性肺动脉高压治疗低氧血症。此外,NO在天然免疫系统中起着重要作用,体外研究表明,NO不仅对常见的革兰氏阳性和革兰氏阴性细菌具有抗菌活性,而且对其他多种病原体,包括分枝杆菌、病毒、真菌、酵母和寄生虫也具有抗菌活性,并有清除多重耐药菌株的潜力。 关于NTM 非结核分枝杆菌(NTM)感染是一种罕见而严重的肺部细菌感染,可导致肺部虚弱性疾病,发病率和死亡率高。NTM感染是通过吸入环境中的雾化细菌获得的,并可导致NTM肺部疾病,一种进行性和慢性疾病。根据囊性纤维化基金会的数据,2017年,13%的美国囊性纤维化患者的NTM种培养呈阳性。NTM是一个新兴的公共卫生问题,因为它的多药抗生素耐药性。目前的治疗指南建议长期服用多种抗生素长达两年。这些复杂、昂贵和侵入性的方案在治疗脓肿分枝杆菌复合体(MABSC)和难治性鸟分枝杆菌复合体(MAC)方面记录不佳,并有可能导致严重的不良事件。Beyond Air的系统被设计为向肺部输送150-400 ppm的NO,早期数据表明,这一范围的NO浓度可能对感染NTM的患者有积极的影响。 前瞻性陈述 本新闻稿包含关于吸入式一氧化氮和公司的LungFit®产品的“前瞻性陈述”,包括关于潜在监管发展、对患者的潜在影响以及与使用相关的预期益处的陈述。前瞻性陈述包括关于我们对产品供应、业务、财务状况、运营结果、战略或前景的期望、信念或意图的陈述。你可以通过“预期”、“预期”、“打算”、“影响”、“计划”、“项目”、“相信”、“估计”、“可能”、“目标”、“假设”、“目标”和类似的表达和/或将来时态或条件结构的使用(如“将”、“可能”、“可能”、“应该”等)以及这些陈述与历史或当前事项没有严格联系的事实来识别这些前瞻性陈述。相反,前瞻性陈述与预期或预期的事件、活动、趋势或结果有关。由于前瞻性陈述涉及尚未发生的事项,这些陈述固有地受到风险和不确定性的影响,这些风险和不确定性可能导致我们的实际结果与前瞻性陈述所表达或暗示的任何未来结果大相径庭。这些前瞻性陈述只是预测,反映了我们对未来事件和财务表现的看法。许多因素可能导致我们的实际活动或结果与前瞻性陈述中预期的活动和结果存在重大差异,包括与以下风险相关的风险:我们发现和开发新药物的方法未经证实,可能永远不会导致有效或可销售的产品;我们的资金能力和进一步临床前和临床试验的结果;获得、维护和保护我们产品所使用的知识产权;我们有能力对侵权人执行我们的专利,并保护我们的专利组合免受第三方的挑战;我们获得额外资金以支持我们业务活动的能力;我们在产品开发、制造、营销、销售和分销方面对第三方的依赖;成功开发我们的候选产品,所有这些产品都处于开发的早期阶段;获得产品的监管批准;与使用与我们相似的技术和开发类似用途产品的他人竞争;我们对合作者的依赖;我们的短期经营历史和其他风险在公司最近的年度报告10-K表和其他提交给SEC的文件的“风险因素”部分中得到了更详细的识别和描述,所有这些都可以在我们的网站上找到。我们不承担更新这些前瞻性陈述的义务,也没有更新或修订这些前瞻性陈述的政策,除非适用法律要求。 联系人: Maria Yonkoski,投资者关系主管 超越航空公司。 myonkoski@beyondair.net 科里·戴维斯博士 LifeSci Advisors有限责任公司 cdavis@lifesciadvisors.com (212)915-2577

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