Phase 2/3 pivotal trial design and protocol for HB-200 in combination with pembrolizumab for the first-line treatment of patients with HPV16+ recurrent or metastatic OPSCC aligned with FDA feedback

HB-200与pembrolizumab联合用于HPV16+复发或转移性OPSCC患者一线治疗的2/3期关键试验设计和方案符合FDA反馈

HB-200 accepted for oral abstract presentation at ASCO 2024 Annual Meeting with data from approximately 40 patients treated with HB-200 in combination with pembrolizumab

HB-200在ASCO 2024年会上接受了口头摘要介绍，其中有大约40名接受HB-200联合pembrolizumab治疗的患者的数据

Two additional abstracts for HB-200 and HB-700 accepted for the ASCO 2024 Annual Meeting

ASCO 2024年会接受了HB-200和HB-700的另外两份摘要

Company to host investor call at 8:00 a.m. ET on Thursday, April 25, 2024, to highlight path to potential registration; participant details below

公司将于2024年4月25日（星期四）美国东部时间上午8:00召集投资者，重点介绍潜在注册途径；参与者详细信息如下

NEW YORK and VIENNA, Austria, April 25, 2024 (GLOBE NEWSWIRE) -- HOOKIPA Pharma Inc. (NASDAQ: HOOK, HOOKIPA or the Company), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, today announced its final pivotal Phase 2/3 trial design for HB-200 in combination with pembrolizumab.

2024年4月25日，纽约和维也纳，奥地利（环球通讯社）--HOOKIPA Pharma Inc.（纳斯达克：HOOK，HOOKIPA或该公司），一家基于其专有的沙粒病毒平台开发新型免疫治疗剂的公司，今天宣布了其HB-200与pembrolizumab联合的最终关键2/3期试验设计。

The Phase 2/3 trial design and protocol are based on alignment with the U.S. Food and Drug Administration (FDA) following the Company’s Type C meeting with the FDA..

2/3期试验设计和方案基于与美国食品和药物管理局（FDA）的C型会议后与美国食品和药物管理局（FDA）的一致性。。

The seamless Phase 2/3 trial is for the investigational product HB-200 in combination with pembrolizumab for the treatment of patients with Human Papillomavirus 16-positive (HPV16+) recurrent/metastatic PD-L1 CPS ≥ 20 oropharyngeal squamous cell carcinoma (OPSCC) in the first line setting. The Company anticipates the first patient will be enrolled in the fourth quarter of 2024..

无缝2/3期试验是针对研究产品HB-200联合pembrolizumab治疗人乳头瘤病毒16阳性（HPV16+）复发/转移性PD-L1 CPS≥20口咽鳞状细胞癌（OPSCC）患者的一线治疗。该公司预计第一名患者将于2024年第四季度登记。。

The Company also announced acceptance of its HB-200 study abstract as an oral presentation at the American Society of Clinical Oncology (ASCO) 2024 Annual Meeting in the head and neck cancer session to be held on June 4, 2024. The presentation will include data for approximately 40 head and neck cancer patients treated with HB-200 in combination with pembrolizumab..

该公司还宣布接受其HB-200研究摘要，作为2024年6月4日举行的美国临床肿瘤学会（ASCO）2024年头颈癌年会的口头报告。该演讲将包括大约40名接受HB-200联合派姆单抗治疗的头颈部癌症患者的数据。。

“It has been a dream of mine—and my HOOKIPA colleagues—to translate our science into a product that will make an impact for patients and help them fight cancer and infectious diseases. I am happy to say that we are taking a very big step forward to making this dream a reality,” said Joern Aldag, Chief Executive Officer of HOOKIPA.

HOOKIPA首席执行官乔恩·奥尔达格（Joern Aldag）表示：“我和我的HOOKIPA同事一直梦想着将我们的科学转化为一种对患者产生影响并帮助他们抗击癌症和传染病的产品。我很高兴地说，我们正在朝着实现这一梦想迈出一大步。”。

“We believe our data is best-in-class and puts us in the lead position for OPSCC in the first line setting. We have alignment with the FDA on our pivotal trial design and protocol, as well as PRIME designation for HB-200 in combination with pembrolizumab for the treatment of patients with OPSCC in the first line setting from the European Medicines Agency.

“我们相信我们的数据是同类中最好的，使我们在一线环境中处于OPSCC的领先地位。我们在关键的试验设计和方案上与FDA保持一致，并将HB-200与pembrolizumab联合用于治疗欧洲药品管理局一线环境中的OPSCC患者。

We believe our trial design and alignment with our primary regulators can help us reach a potential registration more quickly. Further, we have been accepted for an oral abstract presentation at ASCO in June 2024 where we will present an update from our Phase 1/2 trial with approximately 40 patients treated with the combination of HB-200 and pembrolizumab.”.

我们相信，我们的试验设计以及与主要监管机构的协调可以帮助我们更快地获得潜在的注册。此外，我们已于2024年6月在ASCO接受了口头摘要介绍，我们将介绍我们的1/2期试验的最新情况，其中约有40名患者接受了HB-200和pembrolizumab联合治疗。”。

HB-200 in combination with pembrolizumab pivotal Phase 2/3 trial design summary

HB-200联合pembrolizumab关键2/3期试验设计总结

The trial will treat patients with HPV16+ recurrent/metastatic PD-L1 CPS ≥ 20 oropharyngeal squamous cell carcinoma in the first line setting.

该试验将在一线治疗HPV16+复发/转移性PD-L1 CPS≥20口咽鳞状细胞癌患者。

The trial is expected to enroll approximately 250 patients across the seamless Phase 2/3 design.

该试验预计将在无缝2/3期设计中招募约250名患者。

Patients will be randomized one-to-one for HB-200 plus pembrolizumab or placebo plus pembrolizumab.

患者将被一对一随机分配HB-200加pembrolizumab或安慰剂加pembrolizumab。

The primary endpoints are objective response rate for the Phase 2 portion and overall survival for the Phase 3 portion.

主要终点是第二阶段部分的客观缓解率和第三阶段部分的总体生存率。

The Company may seek accelerated approval based on data from the Phase 2 portion of the trial, from approximately half of the Phase 2/3 study patients, if favorable.

如果有利的话，公司可能会根据试验第二阶段部分的数据，从大约一半的2/3期研究患者那里寻求加速批准。

Phase 2 primary analysis expected in 2026 with potential subsequent filing for accelerated approval.

第二阶段初步分析预计在2026年进行，随后可能会提交加速批准。

The Company will host a conference call today where HOOKIPA’s Executive Team will discuss the full details of the trial design, and the Company’s clinical development strategy for oncology.

HOOKIPA公司将于今天召开电话会议，HOOKIPA的执行团队将讨论试验设计的全部细节以及公司的肿瘤学临床发展战略。

Call Details:

通话详细信息：

HOOKIPA HB-200 Phase 2/3 Clinical Trial Update

HOOKIPA HB-200 2/3期临床试验更新

Thursday, April 25, 2024, 8:00 a.m. ET

2024年4月25日星期四，美国东部时间上午8:00

Webcast Registration

网络广播注册

Dial-in Registration

拨号登记

Abstract details: ASCO 2024 Annual Meeting

摘要细节：ASCO 2024年会

HB-200:

HB-200：

Title: HB-200 arenavirus-based immunotherapy plus pembrolizumab as first-line treatment of patients with recurrent/metastatic HPV16-positive head and neck cancer: Updated results

标题：基于HB-200沙粒病毒的免疫疗法加pembrolizumab作为复发/转移性HPV16阳性头颈癌患者的一线治疗：最新结果

Presenter: Dr. Alan L. Ho, Head and Neck Oncologist at Memorial Sloan Kettering Cancer Center and a trial investigator

演示者：Dr。AlanL.Ho，纪念斯隆·凯特琳癌症中心头颈肿瘤学家兼试验研究员

Abstract Type: Oral abstract

摘要类型：口头摘要

Session Name: Head and Neck Cancer

课程名称：头颈癌

Session Date and Time: June 4, 2024; 9:45 AM-12:45 PM CDT

会议日期和时间：2024年6月4日；9： CDT上午45:00-下午12:45

Abstract Number: 6005

摘要编号：6005

Title: Neoadjuvant HPV16-specific arenavirus-based immunotherapy HB-200 plus chemotherapy followed by response-stratified de-intensification in HPV16+ oropharyngeal cancer: TARGET-HPV

标题：基于HPV16特异性沙粒病毒的新辅助免疫疗法HB-200加化疗，然后在HPV16+口咽癌中进行反应分层去强化：TARGET-HPV

Presenter: Dr. Ari Rosenberg, Principal Investigator, TARGET-HPV Trial, University of Chicago Medicine

演示者：Dr。芝加哥大学医学院TARGET-HPV试验首席研究员Ari Rosenberg

Abstract Type: Rapid oral abstract

摘要类型：快速口头摘要

Session Name: Head and Neck Cancer

课程名称：头颈癌

Session Date and Time: June 3, 2024; 8:00 AM-9:30 AM CDT

会议日期和时间：2024年6月3日；8： CDT上午00:00-9:30

Abstract Number: 6017

摘要编号：6017

Trial Sponsor: UChicago Medicine

试验赞助商：UChicago Medicine

HB-700

HB-700

Title: Development of an arenavirus-based immunotherapy for treatment of KRAS mutant cancer

标题：开发基于沙粒病毒的免疫疗法，用于治疗KRAS突变型癌症

Abstract Type: Abstract only

摘要类型：仅摘要

Session Date: May 23, 2024

会议日期：2024年5月23日

Abstract Number: e14672

摘要编号：e14672

About HB-200

关于HB-200

HB-200 is HOOKIPA’s lead oncology candidate engineered with the company’s proprietary replicating arenaviral vector platform. It comprises two single-vector compounds with arenaviral backbones based on lymphocytic choriomeningitis virus (LCMV) and pichinde virus (PICV). Both express the same transgene encoding an E7E6 fusion protein derived from HPV16.

HB-200是HOOKIPA的首席肿瘤学候选者，由该公司专有的复制沙粒病毒载体平台设计。它包含两种具有基于淋巴细胞脉络丛脑膜炎病毒（LCMV）和皮辛德病毒（PICV）的沙粒病毒骨架的单载体化合物。两者均表达编码源自HPV16的E7E6融合蛋白的相同转基因。

HB-200 is an alternating 2-vector immunotherapy designed to further focus the immune response against the encoded antigen..

HB-200是一种交替的双载体免疫疗法，旨在进一步集中针对编码抗原的免疫应答。。

HB-200 in combination with pembrolizumab received Fast Track Designation from the U.S. Food and Drug Administration and PRIME designation from the European Medicines Agency for the treatment of first-line HPV16+ recurrent/metastatic oropharyngeal squamous cell carcinoma. These designations are supported by preliminary clinical evidence from the Phase 1/2, open-label, clinical trial (NCT04180215) evaluating safety, T cell response, and efficacy based on objective response rate (ORR) and disease control rate (DCR) as defined by RECIST 1.1.

HB-200与pembrolizumab联合治疗一线HPV16+复发/转移性口咽鳞状细胞癌获得了美国食品和药物管理局的快速通道指定和欧洲药品管理局的初步指定。这些名称得到了1/2期开放标签临床试验（NCT04180215）的初步临床证据的支持，该试验根据RECIST 1.1定义的客观缓解率（ORR）和疾病控制率（DCR）评估安全性，T细胞反应和疗效。

and iRECIST. As presented at the European Society for Medical Oncology Annual Congress 2023, HB-200 in combination with pembrolizumab showed a 42 percent confirmed ORR and disease control rate DCR of 74 percent across 19 evaluable patients, doubling the 19 percent ORR for pembrolizumab alone.1 .

和iRECIST。正如在2023年欧洲肿瘤内科学会年会上所介绍的那样，HB-200联合pembrolizumab在19名可评估患者中显示42%的确诊ORR和74%的疾病控制率DCR，是单独使用pembrolizumab的19%ORR的两倍。

About HOOKIPA

关于HOOKIPA

HOOKIPA Pharma Inc. (NASDAQ: HOOK) is a clinical-stage biopharmaceutical company focused on developing novel immunotherapies, based on its proprietary arenavirus platform, which are designed to mobilize and amplify targeted T cells and thereby fight or prevent serious disease. HOOKIPA’s replicating and non-replicating technologies are engineered to induce robust and durable antigen-specific CD8+ T cell responses and pathogen-neutralizing antibodies.

HOOKIPA Pharma Inc.（纳斯达克：HOOK）是一家临床阶段的生物制药公司，专注于开发基于其专有沙粒病毒平台的新型免疫疗法，旨在动员和扩增靶向T细胞，从而对抗或预防严重疾病。HOOKIPA的复制和非复制技术旨在诱导强大而持久的抗原特异性CD8+T细胞反应和病原体中和抗体。

HOOKIPA’s pipeline includes its wholly owned investigational arenaviral immunotherapies targeting Human Papillomavirus 16-positive cancers, KRAS-mutated cancers, and other unnamed indications. In addition, HOOKIPA aims to develop functional cures of HBV and HIV in collaboration with Gilead..

HOOKIPA的管道包括其全资拥有的针对人乳头瘤病毒16阳性癌症，KRAS突变癌症和其他未命名适应症的研究性沙粒病毒免疫疗法。此外，HOOKIPA旨在与Gilead合作开发HBV和HIV的功能性治疗。。

Find out more about HOOKIPA online at www.hookipapharma.com.

有关HOOKIPA的更多信息，请访问www.hookipapharma.com。

1 Harrington et al. Pembrolizumab with or Without Chemotherapy in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma: Updated Results of the Phase III KEYNOTE-048 Study. Journal of Clinical Oncology. 2023;41(4);790-802.

1 Harrington等人。Pembrolizumab在复发或转移性头颈部鳞状细胞癌中有或没有化疗：III期KEYNOTE-048研究的最新结果。临床肿瘤学杂志。2023年；41（4）；790-802。

Forward Looking Statements

前瞻性声明

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 terms such as “believes,” “expects,” “plans,” “potential,” “will,” “would” or similar expressions and the negative of those terms.

本新闻稿包含1995年《私人证券诉讼改革法案》修订版所指的前瞻性声明。前瞻性陈述可以通过诸如“相信”、“期望”、“计划”、“潜在”、“将”、“将”或类似的表达以及这些术语的负面含义来识别。

Forward-looking statements in this press release include HOOKIPA’s statements regarding the potential of its product candidates to positively impact quality of life and alter the course of disease in the patients it seeks to treat, HOOKIPA’s expectations regarding the design and protocol of the Company’s upcoming pivotal Phase 2/3 clinical trial of HB-200 in combination with pembrolizumab, the timing of patient enrollment in clinical trials and the availability of data therefrom, and the timing o submissions concerning regulatory approval, and other statements that are not historical fact.

本新闻稿中的前瞻性声明包括HOOKIPA关于其候选产品对其寻求治疗的患者的生活质量和改变疾病进程的潜力的声明，HOOKIPA对公司即将进行的关键性HB-200与pembrolizumab联合2/3期临床试验的设计和方案的期望，患者参加临床试验的时间和数据的可用性，以及有关监管部门批准的提交时间，以及其他非历史事实的声明。

Such forward-looking statements involve substantial risks and uncertainties that could cause HOOKIPA’s research and clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the drug development process, including HOOKIPA’s programs’ early stage of development, the process of designing and conducting preclinical and clinical trials, the risk that results of preclinical studies and clinical trials may not be predictive of future results in preclinical studies or clinical trials, the regulatory approval processes, the timing of regulatory filings, the challenges associated with manufacturing drug products, HOOKIPA’s ability to successfully establish, protect and defend its intellectua.

此类前瞻性声明涉及重大风险和不确定性，可能导致HOOKIPA的研究和临床开发计划、未来结果、绩效或成就与前瞻性声明中明示或暗示的存在重大差异。这些风险和不确定性包括药物开发过程中固有的不确定性，包括HOOKIPA项目的早期开发阶段，设计和进行临床前和临床试验的过程，临床前研究和临床试验的结果可能无法预测临床前研究或临床试验的未来结果的风险，监管审批流程，监管备案的时间，与制造药品相关的挑战，HOOKIPA成功建立，保护和捍卫其智力的能力。

Availability of Other Information About HOOKIPA

HOOKIPA其他信息的可用性

Investors and others should note that we announce material financial information to our investors using our investor relations website (https://ir.hookipapharma.com/), SEC filings, press releases, public conference calls and webcasts. We use these channels, as well as social media, to communicate with our members and the public about our company, our services and other issues.

投资者和其他人应注意，我们使用我们的投资者关系网站向投资者公布重大财务信息(https://ir.hookipapharma.com/)，SEC文件、新闻稿、公共电话会议和网络广播。我们利用这些渠道以及社交媒体与我们的成员和公众沟通我们的公司、服务和其他问题。

It is possible that the information we post on social media could be deemed to be material information. Therefore, we encourage investors, the media, and others interested in our company to review the information we post on the U.S. social media channels listed on our investor relations website..

我们在社交媒体上发布的信息可能被视为重要信息。因此，我们鼓励投资者、媒体和其他对我们公司感兴趣的人查看我们在投资者关系网站上列出的美国社交媒体渠道上发布的信息。。

For further information, please contact:

欲了解更多信息，请联系：

Investors and Media

投资者和媒体

Michael Kaiser

迈克尔·凯撒

michael.kaiser@hookipapharma.com

michael.kaiser@hookipapharma.com

+ 1 (917) 984 7537

+ 1 (917) 984 7537