Q32 Bio在2025年美国皮肤病学会会议上展示了SIGNAL-AA A部分临床试验的结果，该试验评估了Bempikibart在斑秃患者中的效果-动脉网

Q32 Bio Presents Results from SIGNAL-AA Part A Clinical Trial Evaluating Bempikibart in Patients with Alopecia Areata at the 2025 American Academy of Dermatology Meeting

CISION 等信源发布 2025-03-09 00:00

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可切换为仅中文

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-- Results presented in AAD late-breaker demonstrate bempikibart's encouraging improvement on SALT reduction at week 24 and continued effects after dosing cessation in patients with severe and very severe alopecia areata (AA) --

-- 在AAD年会上公布的最新结果显示，bempikibart在第24周对严重和极重度斑秃（AA）患者的SALT评分降低展现出令人鼓舞的改善，并在停药后持续有效 --

-- Durable, ongoing responses in multiple patients through week 36 follow-up period and beyond to week 55, despite only 24 weeks of dosing, suggestive of potential for remittive effect; multiple inbound patient requests to re-initiate dosing --

-- 在36周随访期及至55周期间，尽管仅进行了24周的给药，但多名患者表现出持久、持续的反应，显示出潜在的缓解效果；同时收到多位患者主动要求重新开始给药的请求 --

-- In the Phase 2a clinical trial, bempikibart was observed to be safe and well-tolerated, with PK supporting subcutaneous dosing and receptor occupancy data demonstrating desired target engagement; clinical biomarkers showed changes in Th2 biomarkers, and expected on-mechanism changes in T-cells, indicative of potent IL-7 and TSLP inhibition --.

-- 在2a期临床试验中，观察到bempikibart是安全且耐受性良好的，药代动力学（PK）支持皮下给药，受体占位数据显示目标结合达到预期；临床生物标志物显示出Th2生物标志物的变化，以及T细胞中预期的机制相关变化，表明对IL-7和TSLP的有效抑制 --。

-- Bempikibart development program remains on track with open-label extension study to initiate in 1H'25; SIGNAL-AA Part B remains on track for initiation of dosing in 1H'25, with topline data expected in 1H'26 --

-- Bempikibart开发项目按计划进行，开放标签扩展研究将于2025年上半年启动；SIGNAL-AA B部分仍按计划于2025年上半年开始给药，预计2026年上半年获得初步数据 --

WALTHAM, Mass.

马萨诸塞州沃尔瑟姆

，

March 8, 2025

2025年3月8日

/PRNewswire/ -- Q32 Bio Inc. (Nasdaq:

/PRNewswire/ -- Q32 Bio Inc.（纳斯达克：

QTTB

) ('Q32 Bio'), a clinical stage biotechnology company focused on developing biologic therapeutics to restore immune homeostasis, today announced additional results from Part A of its SIGNAL-AA Phase 2a clinical trial of bempikibart in patients with alopecia areata (AA) at the 2025 American Academy of Dermatology (AAD) Meeting in .

) ('Q32 Bio')，一家专注于开发恢复免疫稳态的生物治疗药物的临床阶段生物技术公司，今天在其于2025年美国皮肤病学会（AAD）会议上公布了其针对斑秃（AA）患者进行的SIGNAL-AA 2a期临床试验A部分的更多结果。

Orlando, FL.

奥兰多，佛罗里达州。

Bempikibart is a fully human anti-IL-7Rα antibody that re-regulates adaptive immune function by blocking IL-7 and TSLP signaling that is in development for the treatment of AA and currently being evaluated in a Phase 2 program.

Bempikibart 是一种全人源抗 IL-7Rα 抗体，通过阻断 IL-7 和 TSLP 信号重新调节适应性免疫功能，目前正开发用于治疗 AA，并在 2 期项目中进行评估。

'We are excited to share the results from our late-breaker at AAD which includes completed 36-week data and extended follow-up of patients in long-term response who voluntarily re-consented for further assessments based on their maintenance or further deepening of responses after the end of the trial,' said .

“我们很高兴在AAD会议上分享我们的最新突破性结果，其中包括完成的36周数据，以及对长期响应患者延长随访的结果，这些患者在试验结束后基于维持或进一步加深的响应自愿重新同意接受进一步评估，”他表示。

Jodie Morrison

乔迪·莫里森

, Chief Executive Officer of Q32 Bio. 'These findings demonstrate for the first time in patients the potential of an IL-7Rα antagonist approach to deliver durable and sustained activity and recapitulate over a decade of nonclinical research highlighting the potential of this type of sustained response in multiple animal disease models.

Q32 Bio的首席执行官表示：“这些研究结果首次在患者中展示了IL-7Rα拮抗剂方法具有提供持久且持续活性的潜力，并且重现了十多年来非临床研究的成果，突显了这种类型的持续反应在多种动物疾病模型中的潜力。

Given these exciting findings, we have committed to advancing bempikibart as a potentially differentiated therapy for alopecia areata patients who have had limited treatment choices and, to date, no biologic option available.'.

鉴于这些令人兴奋的发现，我们已致力于推进bempikibart作为一种潜在的差异化疗法，用于治疗选择有限且迄今为止尚无生物制剂可用的斑秃患者。

'The responses of patients with longstanding and severe disease, not only at 24 weeks but several weeks after treatment withdrawal, is very provocative,' said

“患有长期严重疾病的患者不仅在24周时，甚至在治疗停止后的数周内都有反应，这非常令人振奋，”

Brett King

布雷特·金

, M.D., Ph.D., of Dermatology Physicians of

医学博士，皮肤病学医师

Connecticut

康涅狄格州

, and former Associate Professor of Dermatology,

，皮肤科前副教授，

Yale University

耶鲁大学

School of Medicine. 'If the activity of bempikibart, including the potential to induce a durable, long-term response, and the safety profile are confirmed in upcoming clinical trials, bempikibart has the potential to change the treatment paradigm of alopecia areata.'

医学院。'如果在接下来的临床试验中证实了bempikibart的活性，包括诱导持久长期反应的潜力及其安全性，bempikibart有潜力改变斑秃的治疗模式。'

Results from SIGNAL-AA Part A Phase 2a Clinical Trial:

SIGNAL-AA 第A部分 2a期临床试验结果：

SIGNAL-AA Part A is a Phase 2a, randomized, double-blind, placebo-controlled, multi-center clinical trial evaluating bempikibart in adult patients with severe and very severe AA (baseline Severity of Alopecia Tool (SALT) scores of 50-100) treated over 24 weeks, with follow-up through 36 weeks. The trial is being conducted to evaluate the efficacy and safety of bempikibart 200 mg administered subcutaneously (SC), every-other-week (Q2W) compared to placebo.

SIGNAL-AA 第A部分是一项2a期、随机、双盲、安慰剂对照、多中心临床试验，评估bempikibart在患有严重和极严重AA（基线脱发严重程度工具(SALT)评分为50-100）的成年患者中的疗效，治疗持续24周，并随访至36周。该试验旨在评估每两周一次皮下注射(SC) 200 mg bempikibart与安慰剂相比的疗效和安全性。

SIGNAL-AA Part A comprised of 41 patients in the modified intent-to-treat population and 27 in the per protocol population, with a primary endpoint of the mean relative percent change in SALT score at 24 weeks compared with baseline, with follow-up in a 12-week post-treatment period through week 36.

SIGNAL-AA A部分包括41名改良意向治疗人群中的患者和27名符合方案人群中患者，主要终点为与基线相比，第24周时SALT评分的平均相对百分比变化，并在治疗后12周期间（直至第36周）进行随访。

Additional data has been collected on patients after week 36, with follow-up on multiple patients through week 55 to date, and additional long-term follow-up ongoing..

在第36周后收集了更多患者的数据，迄今为止已对多名患者进行了第55周的随访，目前仍在进行额外的长期随访。

Highlights on the per protocol basis from the 2025 AAD late-breaking presentation include:

2025年AAD最新突破性报告中的每协议基础亮点包括：

During the on-treatment window:

在治疗窗口期间：

At week 24: Patients with a SALT score of 50-100 treated with bempikibart (n=23) showed a mean reduction in SALT score of 16% vs a reduction of 2% in the placebo group (n=4). A

在第24周：SALT评分为50-100的患者使用bempikibart（n=23）治疗后，SALT评分平均降低了16%，而安慰剂组（n=4）则降低了2%。

Wilcoxon Rank Sum

威尔科克森秩和检验

test yielded a p-value of 0.045.

测试得到的p值为0.045。

At week 26: Patients with severe and very severe disease treated with bempikibart showed a mean reduction in SALT score of 18% in the bempikibart group vs a reduction of 2% in the placebo group.

在第26周：接受bempikibart治疗的重度和极重度患者，bempikibart组的SALT评分平均降低了18%，而安慰剂组降低了2%。

At week 24: 9% of bempikibart patients with severe and very severe disease achieved a SALT score less than or equal to 20 compared to 0% in placebo.

在第24周：9%的接受bempikibart治疗的重度和极重度患者达到了SALT评分小于或等于20，而安慰剂组为0%。

At week 26: 14% of bempikibart patients with severe and very severe disease achieved a SALT score less than or equal to 20 compared to 0% in placebo.

在第26周：14%的重度和极重度疾病患者使用bempikibart后，SALT评分小于或等于20，而安慰剂组为0%。

In the subset of patients with severe disease (baseline SALT 50-95):

在重度疾病患者子集（基线 SALT 50-95）中：

At week 24: patients treated with bempikibart (n=15) showed a mean reduction in SALT score of 25% improving to 27% at week 26.

在第24周：接受bempikibart治疗的患者（n=15）SALT评分平均降低了25%，到第26周时改善至27%。

At week 24: 13% of bempikibart patients achieved a achieved a SALT score less than or equal to 20, compared to 0% in placebo, improving to 21% at week 26 vs 0% in the placebo group.

在第24周时，13%的bempikibart患者达到了SALT评分小于或等于20，而安慰剂组为0%，到第26周时这一比例提高到21%，而安慰剂组仍为0%。

During the post-treatment follow-up period:

治疗后随访期间：

Despite only 24 weeks of treatment across bempikibart treated patients, a deepening response, as measured by mean SALT improvement, was observed following dosing cessation (week 24) through the post-treatment follow-up period (week 36), a paradigm believed to be associated with IL-7 on-mechanism modulation of rebalancing T effector memory cells and T regulatory function..

尽管在使用bempikibart治疗的患者中仅进行了24周的治疗，但在停药后（第24周）至治疗后随访期（第36周）期间，观察到通过平均SALT改善评估的反应加深，这一现象被认为与IL-7机制调节有关，即重新平衡T效应记忆细胞和T调节功能。

At week 36, across patients treated with bempikibart, a mean reduction in SALT score of 20% was observed. In the subset of patients with severe disease, at week 36, the mean reduction in SALT score was 28%.

在第36周，接受bempikibart治疗的患者中，观察到SALT评分平均降低了20%。在患有严重疾病的患者子集中，第36周时，SALT评分平均降低了28%。

Additional post-treatment data collection remains ongoing, including longer-term follow-up of patients following the completion of the trial (post 36 weeks). Outreach was made to patients regarding the post-treatment experience and patients willing to participate were re-consented.

额外的治疗后数据收集仍在进行中，包括试验完成后（36周后）对患者的长期随访。已联系患者了解治疗后的体验，并重新获得了愿意参与的患者的同意。

Amongst patients responding to outreach that completed the treatment period and showed a SALT response during the trial (n=12), all achieved maintenance of response or further hair growth in the post treatment period (post 24 weeks), including after the end of the trial (post 36 weeks).

在对推广活动有响应并完成治疗期且在试验期间显示出SALT响应的患者（n=12）中，所有患者在治疗后阶段（24周后）都保持了响应或实现了进一步的头发生长，包括在试验结束之后（36周后）。

All 12 were confirmed by SALT assessment by the investigator, with a median follow-up of 41 weeks to date (17 weeks post last treatment) with additional follow-up ongoing.

所有12例均经研究者通过SALT评估确认，截至目前中位随访时间为41周（最后一次治疗后17周），并且仍在继续进行后续随访。

Of these, seven patients (7/12) showed additional hair growth by SALT assessment post-treatment, with median follow-up of 44 weeks to date (20 weeks post last treatment) with additional follow-up ongoing.

其中，七名患者（7/12）在治疗后的SALT评估中显示出额外的毛发生长，截至目前中位随访时间为44周（最后一次治疗后20周），并且仍在继续随访。

At week 55: two patients demonstrated improved and ongoing responses approximately seven months following dosing cessation, supporting the potential for remittive effect and durability of response with bempikibart.

在第55周：两名患者在停止给药约七个月后显示出改善且持续的反应，这支持了bempikibart可能具有缓解效果和反应持久性的潜力。

Bempikibart demonstrated a well-tolerated safety and tolerability profile, with no Grade 3 or higher adverse events related to treatment. Further, no related viral infections were reported in the bempikibart group.

Bempikibart 表现出良好的安全性和耐受性，未出现与治疗相关的 3 级或更高级别的不良事件。此外，Bempikibart 组未报告相关的病毒感染。

In addition, in the Phase 2a clinical trial, bempikibart at 200mg Q2W SC demonstrated favorable pharmacokinetics (PK) and target engagement as demonstrated by substantial reductions in biomarkers of Th2 and expected modulation of T-cells. The reduction in Th2 biomarkers included TARC, IgE and eosinophils.

此外，在 2a 期临床试验中，bempikibart 以 200mg Q2W SC 的剂量显示出良好的药代动力学 (PK) 和靶点结合，这一点通过 Th2 生物标志物的显著减少以及 T 细胞的预期调节得到了证明。Th2 生物标志物的减少包括 TARC、IgE 和嗜酸性粒细胞。

CD3.

CD3。

加号

T-cells were reduced as expected with target engagement and IL-7Rα blockade. Q32 Bio believes these results demonstrate that bempikibart is a potent inhibitor of both TSLP and IL-7.

随着靶点结合和IL-7Rα的阻断，T细胞按预期减少。Q32 Bio认为这些结果表明bempikibart是TSLP和IL-7的有效抑制剂。

'In addition to the meaningful mean SALT reductions through week 24, we observed deepening responses throughout the follow-up period through week 36 and longer, despite dosing only through 24 weeks, including two patients with continued response at week 55, approximately seven months following dosing cessation,' said .

“除了在第24周观察到显著的平均SALT降低外，我们在整个随访期间，包括第36周及更长时间内，观察到反应持续加深，尽管仅在前24周进行了给药，其中包括两名在第55周仍保持反应的患者，大约是在停止给药七个月后。”

Jason Campagna

杰森·坎帕尼亚

, M.D., Ph.D., Chief Medical Officer of Q32 Bio. 'Our development program for bempikibart is designed to expand on these results, first with our open-label extension allowing for longer term dosing and follow-up, and second, with SIGNAL-AA Part B which introduces a loading dosing regimen, longer dosing period and longer follow up.

医学博士，Q32 Bio公司的首席医学官表示：“我们针对bempikibart的开发计划旨在扩展这些结果。首先是通过我们的开放标签延长期试验，允许更长时间的给药和随访；其次是SIGNAL-AA B部分，引入了负荷剂量方案、更长的给药周期以及更长时间的随访。”

SIGNAL-AA Part B is intended to support advancement into pivotal trials upon completion, pending review of the results.'.

SIGNAL-AA B部分旨在支持在完成后进入关键试验，待结果审查后。

Bempikibart Phase 2 Development Program:

Bempikibart 第二阶段开发计划：

Q32 Bio is advancing a comprehensive development program evaluating bempikibart in AA. Based on re-consent rates for continued follow-up and strong interest from SIGNAL-AA Part A patients to re-initiate dosing, Q32 Bio plans to initiate an open-label extension (OLE) following the same bempikibart dosing regimen leveraged in Part A to enable longer-term follow-up of patients.

Q32 Bio正在推进一项全面的开发计划，评估bempikibart在AA中的应用。基于持续随访的重新同意率以及SIGNAL-AA A部分患者对重新开始给药表现出的强烈兴趣，Q32 Bio计划在A部分所采用的相同bempikibart给药方案基础上启动一项开放标签扩展（OLE），以实现对患者的长期随访。

Initiation of the OLE remains on track for the first half of 2025..

OLE的启动仍在按计划进行，预计在2025年上半年。

In addition, Q32 Bio is advancing bempikibart in the Part B portion of the SIGNAL-AA Phase 2a clinical trial. SIGNAL-AA Part B is an open-label clinical trial, dosing patients with bempikibart for 36 weeks, with follow-up out to 52 weeks, in approximately 20 evaluable patients with severe or very severe AA.

此外，Q32 Bio正在SIGNAL-AA 2a期临床试验的B部分中推进bempikibart。SIGNAL-AA B部分是一项开放标签临床试验，对约20名重度或极重度AA患者进行为期36周的bempikibart给药，并随访至52周。

Dosing will include an initial loading regimen of 200mg of bempikibart dosed weekly over four weeks, followed by a maintenance dose of 200mg every-other-week over a 32-week period for a total of 36 weeks. Efficacy will be evaluated on the basis of mean percentage change from baseline in SALT scores as well as the proportion of subjects achieving various relative and absolute SALT improvements.at week 36, with follow-up through week 52.

给药方案包括最初四周每周一次200mg的贝匹吉巴特负荷剂量，随后在32周的时间内每两周一次200mg的维持剂量，总共持续36周。疗效将根据SALT评分从基线的平均百分比变化以及达到不同相对和绝对SALT改善的受试者比例进行评估，第36周时进行主要评估，并随访至第52周。

The trial is intended to support advancement into pivotal trials upon completion, pending review of the results. Q32 Bio expects to initiate Part B in the first half of 2025 and report topline data in the first half of 2026..

该试验旨在完成后支持进入关键试验，但需等待结果的审查。Q32 Bio 预计将在 2025 年上半年启动 B 部分，并在 2026 年上半年报告初步数据。

A copy of the AAD late-breaking presentation is available on the

AAD最新发布的演讲副本可在

Presentations and Publications

演示与出版物

page of Q32 Bio's website.

Q32 Bio公司网站的页面。

About Q32 Bio

关于Q32生物

Q32 Bio is a clinical stage biotechnology company whose science targets potent regulators of the adaptive immune system to re-balance immunity in autoimmune and inflammatory diseases.

Q32 Bio是一家临床阶段的生物技术公司，其科学目标是针对适应性免疫系统的有效调节剂，以在自身免疫和炎症性疾病中重新平衡免疫力。

Q32 Bio is advancing bempikibart (ADX-914), a fully human anti-IL-7Rα antibody that re-regulates adaptive immune function for the treatment of autoimmune diseases, in a Phase 2 program. The IL-7 and TSLP pathways have been genetically and biologically implicated in driving several T cell-mediated pathological processes in numerous autoimmune diseases..

Q32 Bio公司正在推进bempikibart（ADX-914）的二期项目，这是一种全人源抗IL-7Rα抗体，可重新调节适应性免疫功能，用于治疗自身免疫疾病。IL-7和TSLP通路在遗传和生物学上已被证实参与驱动多种自身免疫疾病中由T细胞介导的病理过程。

For more information, visit

欲了解更多信息，请访问

www.Q32Bio.com

。

Availability of Other Information About Q32 Bio

关于Q32 Bio的其他信息的可用性

Investors and others should note that we communicate with our investors and the public using our company website

投资者及其他人士应注意，我们通过公司网站与投资者和公众进行沟通。

www.Q32Bio.com

, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly Twitter) and LinkedIn. The information that we post on our website or on X or LinkedIn could be deemed to be material information.

，包括但不限于公司披露、投资者简报和常见问题解答、证券交易委员会文件、新闻稿、公开电话会议记录和网络广播记录，以及在X（前身为Twitter）和LinkedIn上发布的信息。我们发布在公司网站、X或LinkedIn上的信息可能被视为重大信息。

As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended..

因此，我们鼓励投资者、媒体和其他感兴趣的人士定期查阅我们在那里发布的信息。我们网站或社交媒体的内容不应被视为在经修订的《1933年证券法》下的任何文件中引用并入。

Forward-Looking Statements

前瞻性声明

This communication contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements contained herein which do not describe historical facts, including, among others, our beliefs, observations, expectations and assumptions regarding the topline data from the SIGNAL-AA Phase 2a and the safety, tolerability, clinical activity, potential efficacy and potential benefits of bempikibart; which involve risks and uncertainties that could cause actual results to differ materially from those discussed in such forward-looking statements..

本通讯包含根据修订后的1995年美国私人证券诉讼改革法案及其他联邦证券法所定义的前瞻性陈述。本文中包含的任何未描述历史事实的陈述，包括但不限于我们对SIGNAL-AA 2a期试验的顶线数据及bempikibart的安全性、耐受性、临床活性、潜在疗效和潜在益处的信念、观察、预期和假设；这些陈述涉及风险和不确定性，可能导致实际结果与此类前瞻性陈述中讨论的内容存在重大差异。

Forward-looking statements are based on management's current beliefs and assumptions, which are subject to risks and uncertainties and are not guarantees of future performance. Such risks and uncertainties include, among others, the risk that additional data, or the results of ongoing data analyses, may not support our current beliefs and expectations for bempikibart, including with respect to the durability of clinical responses, future clinical studies, including that the OLE may not be initiated by the first half of 2025 and that Part B of the SIGNAL-AA Phase 2a clinical trial may not be initiated by the first half of 2025, may not be completed by the first half of 2026 or at all, might be more costly than expected or might not yield anticipated results, and such other risks and uncertainties identified in the Company's periodic, current and other filings with the U.S.

前瞻性声明基于管理层当前的信念和假设，这些信念和假设受风险和不确定性影响，并不保证未来的表现。这些风险和不确定性包括（但不限于）以下风险：额外数据或正在进行的数据分析可能无法支持我们对bempikibart的当前信念和预期，包括关于临床反应持久性、未来临床研究方面的预期，包括OLE可能不会在2025年上半年启动，SIGNAL-AA 2a期临床试验的B部分可能不会在2025年上半年启动、可能无法在2026年上半年或根本无法完成、可能比预期成本更高或可能无法产生预期结果，以及公司在定期报告、当前报告和其他提交给美国的文件中提到的其他风险和不确定性。

Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended .

证券交易委员会，包括其截至季度末的10-Q表季度报告。

September 30, 2024

2024年9月30日

and any subsequent filings with the Commission, which are available at the SEC's website at

以及随后向委员会提交的任何文件，这些文件可在SEC的网站上查阅，网址为

www.sec.gov

. Any such risks and uncertainties could materially and adversely affect the Company's results of operations and its cash flows, which would, in turn, have a significant and adverse impact on the Company's stock price. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made.

任何此类风险和不确定性都可能对公司的经营结果和现金流产生重大不利影响，这反过来又会对公司的股价产生显著的负面影响。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有效。

The Company disclaims 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..

公司否认有任何义务公开更新或修改任何此类声明，以反映任何预期变化或事件、条件或情况的变化，这些声明可能基于这些变化，或者可能影响实际结果与前瞻性声明中所述结果不同的可能性。

Contacts:

联系人：

Investors:

投资者：

Brendan Burns

布伦丹·伯恩斯

Media:

媒体：

Sarah Sutton

萨拉·萨顿

Argot Partners