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ELIMINATE-B Phase 1 dose finding study for chronic Hepatitis B executing on schedule with completion of first dose administration for cohort 1 (n=3 patients)

ELIMINATE-B 第1阶段慢性乙型肝炎剂量探索研究按计划执行，已完成第1组（n=3名患者）的首次剂量给药。

PBGENE-HBV, the first LNP gene editing technology studied for Hepatitis B, was safe and well tolerated

PBGENE-HBV，首个用于乙型肝炎研究的LNP基因编辑技术，安全且耐受性良好。

PBGENE-HBV demonstrated substantial antiviral activity measured by reduction of Hepatitis B surface antigen (HBsAg) after one administration at the lowest dose level

PBGENE-HBV 在最低剂量水平下，单次给药后通过减少乙型肝炎表面抗原 (HBsAg) 显示出显著的抗病毒活性。

First clinical proof-of-concept in chronic Hepatitis B for a unique editing modality designed to directly eliminate and inactivate the root cause of Hepatitis B virus from covalently closed circular DNA (cccDNA) and integrated DNA

慢性乙型肝炎中，首个临床概念验证，用于一种独特的编辑方式，旨在直接消除和灭活乙型肝炎病毒的根本原因，即共价闭合环状DNA（cccDNA）和整合DNA。

These PBGENE-HBV data mark the second clinical validation for ARCUS in vivo gene editing in 2025

这些PBGENE-HBV数据标志着ARCUS在2025年进行的第二次体内基因编辑临床验证。

DURHAM, N.C.

杜伦，北卡罗来纳州

--(BUSINESS WIRE)--Feb. 19, 2025--

--（商业资讯）--2025年2月19日--

Precision BioSciences, Inc.

精准生物科学公司

(Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop

(Nasdaq: DTIL)，一家临床阶段的基因编辑公司，利用其新型专有的ARCUS®平台进行开发

in vivo

体内

gene editing therapies for high unmet need diseases, today announced initial results from the first administration of PBGENE-HBV in cohort 1, the lowest dose level of the ELIMINATE-B trial. The ELIMINATE-B trial is designed to investigate PBGENE-HBV at multiple ascending dose levels with three dose administrations per dose level in patients afflicted with chronic Hepatitis B who are HBeAg-negative..

针对高未满足需求疾病的基因编辑疗法，今日宣布了ELIMINATE-B试验中第1组（最低剂量水平）首次施用PBGENE-HBV的初步结果。ELIMINATE-B试验旨在研究PBGENE-HBV在多个递增剂量水平上的效果，每个剂量水平进行三次施用，针对的是HBeAg阴性的慢性乙型肝炎患者。

PBGENE-HBV, which comprises an ARCUS-encoding mRNA encapsulated in a lipid nanoparticle (LNP), was safe and well tolerated in all three participants in cohort 1 after the first administration of a 0.2 mg/kg dose. The planned dosing schedule in ELIMINATE-B allows for two additional administrations at this dose level while in parallel investigating the next higher dose level.

PBGENE-HBV由封装在脂质纳米颗粒（LNP）中的ARCUS编码mRNA组成，在队列1中所有三名参与者首次接受0.2 mg/kg剂量后，显示安全且耐受性良好。ELIMINATE-B的计划给药方案允许在此剂量水平进行两次额外给药，同时平行研究下一个更高剂量水平。

The participants treated in cohort 1 possessed different baseline characteristics: age of infection, duration of infection and level of HBsAg. Across the three participants dosed, none experienced a Grade ≥2 treatment-related adverse event or serious adverse event..

队列1中的受试者具有不同的基线特征：感染年龄、感染持续时间和HBsAg水平。在三名接受给药的参与者中，没有人经历≥2级的治疗相关不良事件或严重不良事件。

“This exciting initial safety data set provides evidence that ARCUS encapsulated in a LNP was well tolerated in chronic Hepatitis B patients upon first dose administration at dose level 1. When studying novel technologies and drug mechanisms it is important to monitor safety closely, and we believe the extensive preclinical safety experiments Precision conducted along with several rounds of mRNA optimization were critical steps to ensure patient safety,” said .

“这一令人兴奋的初步安全数据集提供了证据，表明在慢性乙型肝炎患者中，首次以剂量水平1给药时，封装在LNP中的ARCUS耐受性良好。在研究新颖的技术和药物机制时，密切监测安全性非常重要，我们认为Precision进行的广泛临床前安全实验以及多轮mRNA优化是确保患者安全的关键步骤，”该人士表示。

Murray A. Abramson

默里·A·艾布拉姆森

, MD, MPH, Head of Clinical Development. “We are proud to share this first in human proof-of-concept data with the Hepatitis B community as we plan additional administrations at this dose level and escalating dose levels.”

，医学博士，公共卫生硕士，临床开发主管。“我们很自豪能与乙型肝炎社区分享这一首个人体概念验证数据，因为我们计划在此剂量水平和递增剂量水平进行更多的给药。”

In addition to safety, PBGENE-HBV demonstrated a substantial reduction in Hepatitis B surface antigen (HBsAg) in two of the three participants following the first administration at dose level 1. The ELIMINATE-B protocol is designed for three dose administrations at each dose level, with the goal to maximize cumulative viral editing to achieve undetectable levels of HBsAg.

除了安全性之外，PBGENE-HBV 在首次以剂量等级 1 给药后，三名参与者中的两名显示乙肝表面抗原 (HBsAg) 显著减少。ELIMINATE-B 方案设计为在每个剂量等级进行三次给药，目标是最大化累计病毒编辑，以实现 HBsAg 达到无法检测的水平。

With a well-tolerated safety profile and early antiviral activity established at pre-specified timepoints, Precision will complete subsequent administrations in all cohort 1 patients..

具有良好耐受性的安全特性，并在预先指定的时间点确立了早期抗病毒活性，Precision 将完成队列 1 所有患者的后续给药。

“The ELIMINATE-B global investigators are enthusiastic about the initial safety and activity profile of PBGENE-HBV and look forward to treating additional patients globally. Patient interest in this trial remains very high, and these data are re-assuring to me for my patients with chronic Hepatitis B,” said Alina Jucov, MD, PhD, Principal Investigator, .

“ELIMINATE-B 全球研究者对 PBGENE-HBV 的初步安全性和活性特征感到非常兴奋，并期待在全球范围内治疗更多的患者。患者对这项试验的兴趣仍然非常高，这些数据让我对我的慢性乙型肝炎患者感到放心，”Alina Jucov 医学博士、哲学博士，主要研究者表示。

Arensia Research Clinic

阿伦西亚研究诊所

,

，

Moldova

摩尔多瓦

.

。

“These data excite the entire Precision team, and we hope it instills confidence among the patients who are courageously embarking in our clinical trial. Progress against this wide-spread and devastating disease would not be possible without their participation,” said

“这些数据令整个Precision团队感到兴奋，我们希望它能给那些勇敢参与我们临床试验的患者带来信心。没有他们的参与，就无法取得对抗这种广泛且破坏性极强的疾病的进展，”

Michael Amoroso

迈克尔·阿莫罗索

, President and Chief Executive Officer of

，总裁兼首席执行官

Precision BioSciences

精准生物科学

. “This marks an important step forward for Precision in a large patient population and the second clinical validation of ARCUS

“这标志着Precision在大规模患者群体中的一个重要进步，也是ARCUS的第二次临床验证。

in vivo

体内

gene editing following the recent clinical data from the OTC-HOPE study being conducted by our partner iECURE in a dire rare disease.”

“基因编辑，根据我们合作伙伴iECURE在一项严重罕见病的OTC-HOPE研究中获得的近期临床数据。”

The ELIMINATE-B study is currently enrolling HBeAg-negative chronic Hepatitis B patients at world-class sites in

ELIMINATE-B 研究目前正在世界级的站点招募 HBeAg 阴性的慢性乙型肝炎患者，

Moldova

摩尔多瓦

,

，

Hong Kong

香港特别行政区

, and

，以及

New Zealand

新西兰

. Investigators accrued the first cohort of patients within a month. The company is on schedule to provide additional administrations at this dose level and subsequently plans to escalate to higher dose levels to define the optimal dose and number of dose administrations for safely eliminating cccDNA and inactivating integrated HBV DNA.

调查人员在一个月内累积了第一批患者。公司按计划在此剂量水平上提供额外的给药，并随后计划提升到更高的剂量水平，以确定最佳剂量和给药次数，从而安全地消除cccDNA并使整合的HBV DNA失活。

Precision expects to expand the study to the .

Precision 预计将把研究扩展到。

U.S.

美国

and

和

U.K.

英国

and continue accelerating recruitment and evaluation of a genetically diverse patient population in the Phase 1 study. Precision plans to share detailed clinical data throughout 2025.

并在 1 期研究中继续加速招募和评估遗传多样性患者群体。Precision 计划在 2025 年全年分享详细的临床数据。

“Prior to commencing the ELIMINATE-B clinical trial, we conducted numerous preclinical studies with PBGENE-HBV to understand the pharmacokinetics, safety, and impact on viral markers at various dose levels and following multiple dose administrations. Importantly, the early data in the first cohort of patients is consistent with the safety and HBsAg reductions observed in our preclinical models,” said .

“在开始ELIMINATE-B临床试验之前，我们进行了多项PBGENE-HBV的临床前研究，以了解其在不同剂量和多次给药后的药代动力学、安全性和对病毒标志物的影响。重要的是，第一组患者中的早期数据与我们在临床前模型中观察到的安全性和HBsAg降低结果一致。”

Cassie Gorsuch

凯西·戈尔苏奇

, PhD, Chief Scientific Officer. “The safety and early reduction of HBsAg suggests that PBGENE-HBV is doing what no previous treatment has been able to accomplish, eliminating the source of viral replication in cccDNA and inactivating integrated disease.”

，博士，首席科学官。“PBGENE-HBV 的安全性和早期降低 HBsAg 的效果表明，它实现了以往任何治疗方法都无法做到的事情，即消除 cccDNA 中的病毒复制源并使整合的病毒失活。”

About PBGENE-HBV

关于PBGENE-HBV

(Viral Elimination Program)

（病毒消除程序）

:

：

PBGENE-HBV is Precision’s wholly owned

PBGENE-HBV 是 Precision 完全拥有的

in vivo

体内

gene editing program under investigation in a global first-in-human clinical trial, which is designed to potentially cure chronic hepatitis B virus (HBV) infection. Currently, it is estimated that 300 million people worldwide are afflicted with chronic hepatitis B. PBGENE-HBV is the first and only potentially curative gene editing program to enter clinical investigation that is specifically designed to eliminate cccDNA and inactivate integrated HBV DNA..

基因编辑项目正在全球首个临床试验中进行调查，该试验旨在潜在治愈慢性乙型肝炎病毒（HBV）感染。据估计，目前全球有3亿人患有慢性乙型肝炎。PBGENE-HBV是首个也是唯一一个进入临床研究的潜在治愈性基因编辑项目，专门设计用于消除cccDNA并使整合的HBV DNA失活。

About the OTC Program

关于OTC计划

(Gene Insertion Program): Led by iECURE, ECUR-506 is an ARCUS-mediated in vivo gene editing program currently in a first-in-human phase 1/2 trial (OTC-HOPE) evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. In

（基因插入程序）：由iECURE主导，ECUR-506是一个基于ARCUS的体内基因编辑项目，目前正处于首次人体1/2期试验（OTC-HOPE），评估ECUR-506作为治疗新生儿发病型鸟氨酸氨甲酰转移酶（OTC）缺乏症的潜在疗法。

January 2025

2025年1月

, iECURE reported clinical efficacy and safety data in the first patient dosed showing a complete clinical response from three months post exposure to the end of study (six months post exposure). ECUR-506 was generally well tolerated with no significant clinical safety concerns.

，iECURE报告了首个接受治疗的患者的临床疗效和安全性数据，数据显示从暴露后三个月到研究结束（暴露后六个月）出现了完全的临床反应。ECUR-506通常耐受性良好，没有显著的临床安全问题。

About

关于

Precision BioSciences, Inc.

精准生物科学公司

Precision BioSciences, Inc.

精准生物科学公司

is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes.

是一家临床阶段的基因编辑公司，致力于通过其新颖且专有的ARCUS®基因组编辑平台改善生活（DTIL），该平台在切割方式、更小的尺寸和更简单的结构上不同于其他技术。ARCUS核酸酶的关键能力和差异化特性可能有助于实现更多预期的、明确的治疗效果。

Using ARCUS, the Company’s pipeline is comprised of .

使用ARCUS，公司的管道由以下组成。

in vivo

体内

gene editing candidates designed to deliver lasting cures for genetic and infectious diseases where no adequate treatments exist. For more information about

基因编辑候选药物旨在为那些尚无足够治疗方法的遗传性和传染性疾病提供持久的治疗方案。欲了解更多信息，请访问

Precision BioSciences

精密生物科学

, please visit

，请访问

www.precisionbiosciences.com

www.precisionbiosciences.com

.

。

The ARCUS® platform is being used to develop

ARCUS® 平台正被用于开发

in vivo

体内

gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV)..

复杂的基因编辑疗法，包括基因插入（将DNA插入基因以引起表达/添加功能）、消除（移除基因组，例如病毒DNA或突变的线粒体DNA）和切除（通过单个AAV递送两个ARCUS核酸酶来移除大部分有缺陷的基因）。

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. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the well-tolerated safety profile and substantial antiviral activity established after the first administration at dose level 1 of PBGENE-HBV; the clinical development and demonstrated, potential and expected safety, efficacy and benefit of PBGENE-HBV, our other product candidates and those being developed by partners including ECUR-506; the unique design of PBGENE-HBV to eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures; the expected timing of regulatory processes (including filings such as IND’s and CTA’s and studies for PBGENE-HBV and the acceptance of these filings by regulatory agencies); the suitability of PBGENE-HBV for the treatment of hepatitis and the targeting of the root cause of the disease; the safety, tolerability and efficacy signals observed through preclinical evaluation in non-human primates (NHPs), transgenic and episomal mouse models, human cell models of HBV and primary human hepatocytes; the translatability of preclinical models to human clinical trials; the key advantages of ARCUS and its key capabilities and differentiating characteristics ; expectations about operational initiatives, strategies, and further development of PBGENE-HBV; plans to provide additional administrations of PBGENE-HBV at the first dose level; plans to escalate to higher dose levels in the ELIMINATE-B clinical trial to define the optimal dose and number of dose administrations for safely .

本新闻稿包含1995年《私人证券诉讼改革法案》意义上的前瞻性声明。本新闻稿中所有不涉及历史事实的声明均应被视为前瞻性声明，包括但不限于以下声明：关于PBGENE-HBV在剂量水平1首次给药后表现出的良好耐受性安全性特征和显著抗病毒活性；PBGENE-HBV、我们的其他候选产品以及合作伙伴（包括ECUR-506）正在开发的产品的临床开发及其展示出的潜在和预期的安全性、有效性和益处；PBGENE-HBV的独特设计旨在以高特异性消除cccDNA并使整合的HBV DNA失活，可能实现功能性治愈；监管流程（包括IND和CTA等文件提交及针对PBGENE-HBV的研究）的预期时间安排以及监管机构对这些文件的接受情况；PBGENE-HBV适用于治疗乙肝并针对疾病根本原因的适用性；通过非人类灵长类动物（NHP）、转基因和游离小鼠模型、HBV人细胞模型及原代人肝细胞的临床前评估观察到的安全性、耐受性和有效性信号；临床前模型向人体临床试验的可转化性；ARCUS的关键优势及其主要能力和差异化特征；关于运营计划、战略及PBGENE-HBV进一步开发的预期；计划在第一剂量水平提供额外的PBGENE-HBV给药；计划在ELIMINATE-B临床试验中提升至更高剂量水平，以确定安全的最佳剂量和给药次数。

the United States

美国

and

和

United Kingdom

英国

; expectations around acceleration of recruitment of the ELIMINATE-B clinical trial and plans to evaluate a genetically diverse patient population in the Phase 1 study. Precision plans to share detailed clinical data throughout 2025; expectations about achievement of key milestones; and anticipated timing of patient dosing and clinical data for PBGENE-HBV and ECUR-506.

；对ELIMINATE-B临床试验招募加速的期望，以及计划在第一阶段研究中评估基因多样化患者群体。Precision公司计划在2025年全年分享详细的临床数据；对实现关键里程碑的期望；以及PBGENE-HBV和ECUR-506的患者给药和临床数据预期时间。

In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “design,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “suggest,” “target,” “will,” “would,” or the negative thereof and similar words and expressions..

在某些情况下，您可以通过诸如“目标”、“预期”、“方法”、“相信”、“考虑”、“可能”、“设计”、“已设计”、“估计”、“期望”、“目的”、“意图”、“展望”、“或许”、“使命”、“计划”、“可能”、“潜力”、“预测”、“项目”、“追求”、“应该”、“努力”、“建议”、“针对”、“将”、“会”或其否定形式以及类似词语和表达来识别前瞻性陈述。

Forward-looking statements are based on management’s current expectations, beliefs, and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ or other licensees’ ability to identify, develop and commercialize product candidates; pending and potential product liab.

前瞻性声明基于管理层当前的预期、信念和假设，以及我们目前可获得的信息。这些声明既不是承诺也不是保证，涉及许多已知和未知的风险、不确定性和假设，实际结果可能因各种重要因素而与前瞻性声明中表达或暗示的结果有重大差异，这些因素包括但不限于：我们实现盈利的能力；我们获取足够资金以推进我们的计划的能力；与我们的资本需求、预期现金跑道、现有债务工具下的要求及限制的影响相关的风险，包括由于市场状况和/或我们的市值而导致的筹集额外资本的能力；我们的运营费用及我们预测这些费用的能力；我们有限的运营历史；我们在其中投入资源的计划和候选产品的进展和成功；我们评估候选产品安全性和有效性的有限能力或无能力；其他基因组编辑技术可能比我们的ARCUS技术提供显著优势的风险；我们对ARCUS技术的依赖；研究和开发活动以及临床前和临床研究（包括临床试验和研究性新药申请）的启动、成本、时间、进展、里程碑的达成和结果；公众对基因组编辑技术及其应用的看法；在基因组编辑、生物制药和生物技术领域的竞争；我们或我们的合作伙伴或其他被许可方识别、开发和商业化候选产品的能力；待定和潜在的产品责任。

U.S.

美国

and foreign regulatory landscape applicable to our and our collaborators’ or other licensees’ development of product candidates; our or our collaborators’ or other licensees’ ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators’ and other licensees’ ability to enroll patients; changes in interim “top-line” and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees’ ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators’ or other licensees’ ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance.

适用于我们及我们的合作者或其他被许可方开发候选产品相关的国内外监管环境；我们或我们的合作者或其他被许可方将候选产品推进至临床试验并成功设计、实施和完成临床试验的能力；与我们任何候选产品的开发或商业化相关的潜在制造问题；我们及我们的合作者和其他被许可方在患者招募方面可能遇到的延迟或困难；我们宣布或发布的中期“初步”数据和初始数据的变化；如果我们的候选产品未能按预期工作或导致不良副作用；与适用的医疗保健、数据保护、隐私和安全法规及其合规相关的风险；我们或我们的被许可方获得孤儿药资格或快速通道资格的能力，以及实现这些资格预期效益的能力；我们或我们的合作者或其他被许可方获得并维持候选产品的监管批准的能力，以及批准产品的标签中可能包含的相关限制、局限性和/或警告；任何候选产品的市场接受度及其速度；我们有效管理运营增长的能力；我们吸引、保留和激励高管及员工的能力；系统故障和安全漏洞的影响；保险费用及未投保责任的风险；税务规则的影响；任何大流行病、流行病或传染病爆发的影响；我们现有合作及其他许可协议的成功，以及我们达成新合作安排的能力；我们当前及未来的合作关系及相关依赖性。

September 30, 2024

2024年9月30日

, as any such factors may be updated from time to time in our other filings with the

，因为任何此类因素可能会在我们向

SEC

证券交易委员会

, which are accessible on the SEC’s website at

，这些文件可以在SEC的网站上访问，网址为

www.sec.gov

www.sec.gov

and the Investors page of our website under SEC Filings at

我们网站的投资者页面下SEC文件部分

investor.precisionbiosciences.com

投资者.precisionbiosciences.com

.

。

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise..

本新闻稿中的所有前瞻性声明仅截至本新闻稿发布之日，除非适用法律要求，我们没有义务更新或修改本文包含的任何前瞻性声明，无论是否由于新信息、未来事件、情况变化或其他原因。

View source version on

查看源版本于

businesswire.com

商业电报网

:

：

https://www.businesswire.com/news/home/20250219034369/en/

https://www.businesswire.com/news/home/20250219034369/zh/

Investor and Media Contact:

投资者和媒体联系人：

Naresh Tanna

纳雷什·坦纳

Vice President, Investor Relations

副总裁，投资者关系

Naresh.Tanna@precisionbiosciences.com

纳雷什.坦纳@精密生物科学.com

Source:

源代码：

Precision BioSciences, Inc.

精准生物科学公司