Topline Phase 2b data expected in June 2024

预计2024年6月的顶级阶段2b数据

More than 120 sites around the world actively recruiting in the Phase 3 portion of the IMPAHCT trial

在IMPAHCT试验的第三阶段，全球有120多个站点积极招募

WALTHAM, Mass., Nov. 20, 2023 (GLOBE NEWSWIRE) -- Aerovate Therapeutics, Inc. (Nasdaq: AVTE), a clinical stage biopharmaceutical company focused on developing drugs that meaningfully improve the lives of patients with rare cardiopulmonary disease, today announced the Phase 2b portion of the Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial (IMPAHCT) Phase 2b/Phase 3 study evaluating AV-101, a novel dry powder inhaled formulation of imatinib for the treatment of pulmonary arterial hypertension (PAH), has completed enrollment at 202 adult patients.

2023年11月20日（GLOBE NEWSWIRE）-Aerovate Therapeutics，Inc。（纳斯达克股票代码：AVTE），一家临床阶段的生物制药公司，专注于开发有意义地改善罕见心肺疾病患者生活的药物，今天宣布吸入伊马替尼肺动脉高压临床试验（IMPAHCT）2b期/3期研究的2b期部分评估AV-101，一种用于治疗肺动脉高压（PAH）的新型干粉吸入伊马替尼制剂，已完成202名成年患者的入组。

Aerovate expects to report topline data from the Phase 2b portion of the trial in June 2024. In addition, the first patient has been enrolled in the Phase 3 portion of IMPAHCT..

Aerovate预计将于2024年6月报告试验阶段2b部分的顶级数据。另外，第一名患者已经参加了IMPAHCT的第三阶段。。

“The completion of enrollment in the Phase 2b portion of the trial marks an exciting milestone for Aerovate and for people living with PAH,” said Tim Noyes, Chief Executive Officer at Aerovate Therapeutics. “Because of our innovative adaptive Phase 2b/Phase 3 trial design for IMPAHCT, we were also able to rapidly enroll our first patient in the Phase 3 portion of the study evaluating our novel investigational antiproliferative drug candidate, AV-101, for the treatment of PAH.

Aerovate Therapeutics首席执行官Tim Noyes说：“试验阶段2b部分的入学完成标志着Aerovate和PAH患者的一个令人兴奋的里程碑。“由于我们针对IMPAHCT的创新适应性阶段2b/阶段3试验设计，我们还能够在研究的阶段3部分快速招募我们的第一位患者，评估我们的新型研究性抗增殖候选药物AV-101用于治疗PAH。

I am extremely grateful for the commitment of our clinical investigators and their patients, and proud of the Aerovate team’s dedication and ongoing execution of the trial, as this furthers our goal of developing meaningful therapeutic options for PAH patients as quickly as possible.”.

我非常感谢我们的临床研究人员及其患者的承诺，并为Aerovate团队的奉献精神和持续执行试验感到自豪，因为这进一步推动了我们为PAH患者开发有意义的治疗方案的目标“。

IMPAHCT is a Phase 2b/Phase 3, randomized, double-blind, placebo-controlled multi-national trial evaluating the safety, efficacy, tolerability, pharmacokinetics (PK) and pharmacodynamics of AV-101 in adults with PAH. The IMPAHCT trial was structured with a seamless adaptive Phase 2b/Phase 3 design to allow for an efficient development timeline, thus enabling the immediate enrollment of patients in the Phase 3 portion of the study upon the completion of enrollment in the Phase 2b portion.

IMPAHCT是评估AV-101在PAH成人中的安全性，有效性，耐受性，药代动力学（PK）和药效学的2b期/3期随机，双盲，安慰剂对照多国试验。IMPAHCT试验采用无缝自适应阶段2b/阶段3设计，以实现有效的开发时间表，从而在完成阶段2b部分注册后立即将患者纳入研究的阶段3部分。

Currently, more than 120 sites are open and actively recruiting patients in the Phase 3 portion of the trial..

目前，在试验的第三阶段，有120多个地点开放并积极招募患者。。

The Phase 2b primary endpoint of IMPAHCT is the placebo corrected change from baseline in pulmonary vascular resistance (PVR). Following the results of the Phase 2b portion of the study, an optimal dose will be selected and all subsequent patients enrolled in IMPAHCT will be randomized to either placebo or the optimal dose.

IMPAHCT的2b期主要终点是安慰剂校正的肺血管阻力（PVR）基线变化。根据研究的阶段2b部分的结果，将选择最佳剂量，并且所有随后参加IMPAHCT的患者将被随机分配到安慰剂或最佳剂量。

The Phase 3 primary endpoint will be the placebo corrected change in 6-minute walk distance (6MWD) after 24 weeks of treatment with the optimal dose..

第3阶段的主要终点是用最佳剂量治疗24周后6分钟步行距离（6MWD）的安慰剂校正变化。。

AV-101, an investigational, proprietary dry powder inhaled formulation of imatinib, is being developed by Aerovate for the treatment of PAH, a rare, progressive disease which involves abnormal cellular proliferation and resistance to apoptosis in the pulmonary vasculature. AV-101 has been formulated for self-administration in which patients will perform two inhalations twice daily with a discreet, pocket-sized device designed for easy use..

AV-101是伊马替尼研究性专有干粉吸入制剂，目前正在开发用于治疗PAH，PAH是一种罕见的进行性疾病，涉及异常细胞增殖和肺血管系统细胞凋亡抵抗。AV-101已配制用于自我管理，患者将每天两次使用一个谨慎的口袋大小的装置进行两次吸入，以便于使用。。

About PAH

关于PAH

PAH is a rare, progressive orphan disease with unmet medical need that affects approximately 70,000 people in the United States and Europe. PAH can cause strain on the heart, leading to limitation of physical activity, heart failure and reduced life expectancy.

PAH是一种罕见的进行性孤儿疾病，医疗需求未得到满足，影响了美国和欧洲约70000人。PAH可导致心脏紧张，导致身体活动受限，心力衰竭和预期寿命缩短。

About AV-101

关于AV-101

AV-101 is an investigational, proprietary dry powder inhaled formulation of the antiproliferative drug imatinib. Developed specifically for pulmonary arterial hypertension (PAH), AV-101 targets cellular hyperproliferation and resistance to apoptosis, driven by improper signaling in cells of the distal pulmonary arteries.

AV-101是抗增殖药物伊马替尼的研究性专有干粉吸入制剂。AV-101专门针对肺动脉高压（PAH）开发，其靶向细胞过度增殖和抗凋亡，这是由远端肺动脉细胞中的不正确信号驱动的。

By targeting the proliferation and accumulation of cells in the arteries of the lungs, we believe AV-101 has the potential to provide meaningful improvements for patients beyond the capabilities of currently approved therapies. AV-101 is designed for delivery by an easy-to-use dry powder inhaler, directly into the lungs to maximize potential clinical benefit and limit systemic adverse effects.

通过靶向肺动脉中细胞的增殖和积累，我们相信AV-101有可能为超出当前批准疗法能力的患者提供有意义的改善。AV-101设计用于通过易于使用的干粉吸入器直接进入肺部，以最大限度地发挥潜在的临床益处并限制全身不良反应。

Phase 1 results published in ERJ Open Research showed that AV-101 delivered by dry powder inhalation was generally well-tolerated by healthy adult volunteers with no serious adverse events reported. Aerovate has completed enrollment in the Phase 2b portion of the IMPAHCT clinical trial and is currently enrolling patients in the Phase 3 portion to evaluate the safety and efficacy of AV-101 in adults with PAH..

在ERJ Open Research上发表的第1阶段结果显示，通过干粉吸入递送的AV-101通常被健康的成年志愿者良好耐受，没有报告严重的不良事件。Aerovate已经完成IMPAHCT临床试验的2b期部分的入选，目前正在招募3期部分的患者，以评估AV-101在成人PAH中的安全性和有效性。。

About the IMPAHCT Trial

关于IMPAHCT试验

IMPAHCT (Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial) is a multi-national, placebo-controlled Phase 2b/Phase 3 trial in adults with PAH that continuously enrolled patients from Phase 2b to Phase 3. The Phase 2b portion of the trial will evaluate three doses of AV-101 over 24 weeks, compared to placebo, to identify an optimal dose based on the primary endpoint, change in pulmonary vascular resistance (PVR), and safety, tolerability, and other clinical measures.

IMPAHCT（吸入伊马替尼肺动脉高压临床试验）是一项针对PAH成人的多国安慰剂对照2b/3期临床试验，该试验持续招募2b期至3期患者。与安慰剂相比，试验的2b期部分将在24周内评估三剂AV-101，以根据主要终点，肺血管阻力（PVR）的变化以及安全性，耐受性和其他临床措施。

The Phase 3 portion of the trial will compare patients taking the optimal dose of AV-101, selected from the Phase 2b data, to placebo. The primary endpoint of the Phase 3 portion of the trial will be change in six-minute walk distance (6MWD) over 24 weeks versus placebo. More information about this trial is available at https://clinicaltrials.gov/ct2/show/NCT05036135..

试验的第3阶段部分将比较从2b阶段数据中选择的最佳剂量AV-101的患者与安慰剂。与安慰剂相比，试验的第3阶段部分的主要终点将在24周内以6分钟步行距离（6MWD）改变。有关此试用版的更多信息，请访问https://clinicaltrials.gov/ct2/show/NCT05036135..

About Aerovate Therapeutics, Inc.

关于Aerovate Therapeutics，Inc。

Aerovate is a clinical stage biopharmaceutical company focused on developing drugs that meaningfully improve the lives of patients with rare cardiopulmonary disease. Aerovate's initial focus is on advancing AV-101, its proprietary dry powder inhaled formulation of the drug imatinib for the treatment of patients with PAH.

Aerovate是一家临床阶段的生物制药公司，专注于开发有意义地改善罕见心肺疾病患者生活的药物。Aerovate最初的重点是推进AV-101，它是伊马替尼药物专有的干粉吸入制剂，用于治疗PAH患者。

Learn more at aerovatetx.com or follow the Company on X (formerly known as Twitter) and LinkedIn..

在aerovatetx.com了解更多信息，或在X（以前称为Twitter）和LinkedIn上关注公司。。

Available Information

可用信息

Aerovate announces material information to the public about the Company, its products and services, and other matters through a variety of means, including filings with the U.S. Securities and Exchange Commission (SEC), press releases, public conference calls, webcasts, the investor relations section of the Company website at ir.aerovatetx.com, and the Company’s X (formerly known as Twitter) account @AerovateTx in order to achieve broad, non-exclusionary distribution of information to the public and for complying with its disclosure obligations under Regulation FD..

Aerovate通过各种方式向公众公布有关公司，其产品和服务以及其他事项的材料信息，包括向美国证券交易委员会（SEC）提交申请，新闻稿，公开电话会议，网络广播，公司网站的投资者关系部在ir.aerovatetx.com，以及公司的X（以前称为Twitter）帐户@AerovateTx，以实现向公众广泛，非排他性地分发信息并遵守其根据FD法规的披露义务。。

Cautionary Note Regarding Forward-Looking Statements

关于前瞻性陈述的警示

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements can be identified by words such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “future,” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” seek,” “strategy,” “should,” “target,” “will,” “would” and similar expressions regarding future periods.

本新闻稿包含经修订的1995年“私人证券诉讼改革法”含义内的前瞻性声明。前瞻性陈述可以用“预期”，“相信”，“继续”，“可以”，“估计”，“期望”，“未来”，“目标”，“意图”，“期待”，“可能”，“计划”，“潜力”，“预测”，“寻求，“战略”，“应该”，“目标”，“意愿”以及关于未来时期的类似表达。

These forward-looking statements include, but are not limited to, statements regarding the therapeutic potential and clinical benefits of AV-101; our expectations regarding clinical sites and continuing patient enrollment for the Phase 3 portion of our Phase 2b/Phase 3 trial; our anticipated timing for the release of topline data from the Phase 2b portion of our clinical trial; and our business plans and objectives for AV-101, including expectations regarding timing and success of our Phase 2b/Phase 3 clinical trial, potential regulatory submissions and approvals for AV-101..

这些前瞻性陈述包括但不限于关于AV-101的治疗潜力和临床益处的陈述；我们对2b/3期临床试验3期部分临床部位和持续患者入组的期望；我们预计从临床试验的阶段2b部分发布顶线数据的时间；以及我们对AV-101的业务计划和目标，包括对我们2b/3期临床试验的时间和成功的期望，对AV-101的潜在监管提交和批准。。

Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related to the therapeutic potential and clinical benefits of AV-101; the timing associated with the identification and activation of clinical sites, patient enrollment, initiation, delivery of drug supply and continuation of our Phase 2b/Phase 3 trial of AV-101 in PAH patients; the impact of public health crises on our business, clinical trials, operations and goals; positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; regulatory developments in the United States and foreign countries; as well as those risks and uncertainties set forth more fully under the caption “Risk Factors” in our most recent Annual Report on Form 10-K filed with the SEC and subsequent filings with the SEC.

本新闻稿中的任何前瞻性声明均基于管理层当前的期望和信念，并受到许多风险，不确定性和重要因素的影响，这些风险，不确定性和重要因素可能导致实际事件或结果与任何前瞻性声明所表达或暗示的实质性事件或结果大不相同本新闻稿中包含的声明，包括但不限于，这些风险和不确定性与AV-101的治疗潜力和临床益处有关；与确定和激活临床部位，患者入选，开始，提供药物供应以及继续我们在PAH患者中进行AV-101的2b/3期试验相关的时间；公共卫生危机对我们的业务，临床试验，运营和目标的影响；临床研究的阳性结果可能不一定能预测未来或正在进行的临床研究的结果；美国和外国的监管发展；以及这些风险和不确定性，在我们最近提交给SEC的10-K表格年度报告以及随后向SEC提交的“风险因素”标题下更全面地阐述。

We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. We disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

我们提醒您不要过分依赖任何前瞻性陈述，这些陈述仅在其作出之日起发言。我们不承担任何公开更新或修改任何此类陈述的义务，以反映任何此类陈述可能基于的预期或事件，条件或情况的任何变化，或可能影响实际结果与上述结果不同的可能性。在前瞻性声明中。

Any forward-looking statements contained in this press release represent our views only as of the date hereof and should not be relied upon as representing its views as of any subseque.

本新闻稿中包含的任何前瞻性声明仅在本声明发布之日代表我们的观点，不应被视为代表其对任何子项的观点。

Media Contact

媒体联系

Peg Rusconi

Peg Rusconi

peg.rusconi@vergescientific.com

peg.rusconi@vergescientific.com

Investor Contact

投资者联系

IR@Aerovatetx.com

IR@Aerovatetx.com