Briquilimab

REDWOOD CITY, Calif., Aug. 13, 2025 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a clinical stage biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU) and asthma, today reported results for the fiscal quarter ended June 30, 2025 and provided a corporate update. “The compelling results we are generating in both CSU and CIndU continue to reinforce our belief that briquilimab has the potential to be a highly differentiated therapy in mast cell-driven diseases,” said Ronald Martell, President and Chief Executive Officer of Jasper. “We continue to generate strong data, with briquilimab driving complete responses in 89% of patients across the 240 mg and 360 mg single-dose cohorts in the BEACON study in CSU, 73% of CSU patients at 12 weeks in the open-label extension dosed at 180mg Q8W, and 92% of CIndU patients in the 180mg SPOTLIGHT cohort. With the favorable safety and tolerability profile we continue to observe, we believe briquilimab has the potential to be an important treatment option in chronic urticarias, and we remain well-positioned to deliver multiple catalysts over the coming quarters, which we believe can drive meaningful benefits for patients and long-term value for stockholders.” “Our investigation into the 240mg Q8W and the 240mg/180mg Q8W cohorts of the BEACON study, which did not demonstrate the rapid onset and deep symptom control at levels we have observed in previous cohorts at varying dose levels, remains ongoing,” said Daniel Adelman, M.D., Interim Chief Medical Officer of Jasper. “We are pursuing a number of avenues of investigation and expect to provide an update on the status of the investigation later this year. Meanwhile, the lack of any observed dose-limiting safety signals in these two cohorts enables us to also proceed with redosing those patients with drug product from a different lot, as well as enrolling an additional 10-12 new patients across those cohorts. We plan to report data from both the new patients added and the redosed patients in late 2025.” Highlights for Second Quarter 2025 and Recent Weeks Presented updated data from the BEACON Phase 1b/2a of subcutaneous briquilimab in adult participants with CSU, as well as from CSU patients in the related open-label extension study, both of which demonstrated robust efficacy: 89% (8 of 9) of participants in the 240mg and 360mg single-dose cohorts achieved complete response (UAS7=0) following a single dose, with 7 of those patients achieving an initial clinical response by week 2. 73% (8 of 11) patients in the open-label extension study at 180 mg Q8W maintained a complete response at 12 weeks, and 82% of patients (9 of 11) demonstrated well-controlled disease at 12 weeks. Data from an additional 10-12 new patients being enrolled across the 240mg Q8W and the 240mg/180mg Q8W BEACON cohorts as well as additional data from the open-label extension study are anticipated later this year. Briquilimab continued to be well-tolerated with no dose limiting toxicities observed, and any safety observations potentially related to KIT blockade were infrequent and generally limited to low grade events, none of which resulted in discontinuations or dose delays and the majority of which resolved during repeat dosing. 89% (8 of 9) of participants in the 240mg and 360mg single-dose cohorts achieved complete response (UAS7=0) following a single dose, with 7 of those patients achieving an initial clinical response by week 2. 73% (8 of 11) patients in the open-label extension study at 180 mg Q8W maintained a complete response at 12 weeks, and 82% of patients (9 of 11) demonstrated well-controlled disease at 12 weeks. Data from an additional 10-12 new patients being enrolled across the 240mg Q8W and the 240mg/180mg Q8W BEACON cohorts as well as additional data from the open-label extension study are anticipated later this year. Briquilimab continued to be well-tolerated with no dose limiting toxicities observed, and any safety observations potentially related to KIT blockade were infrequent and generally limited to low grade events, none of which resulted in discontinuations or dose delays and the majority of which resolved during repeat dosing. Commenced an investigation to identify the root cause of an atypical absence of UAS7 reduction in 11 of the 13 patients enrolled in the 240mg Q8W and 240mg/180mg Q8W cohorts of the BEACON study. Among other factors being examined, Jasper is assessing potential product lot variability in one lot of drug product first introduced into the BEACON study in those two cohorts, as all 10 patients dosed with drug supply from that lot failed to show reductions in their reported UAS7 scores. Jasper expects to complete the investigation in the second half of 2025, and in the near-term has provided new clinical drug supply from a different lot for ongoing dosing of existing patients, and is enrolling an additional 10-12 new patients in aggregate across those two cohorts. Jasper expects that the additional data on these cohorts, expected to be reported in late 2025, should be adequate to complete dose selection for the planned Phase 2b CSU study, which is now expected to commence mid-2026. Presented data from the 180mg cohort in the SPOTLIGHT study demonstrating 92% (11 of 12) complete response rate, 100% (12 of 12) clinical response, no serious or grade or higher adverse events, and a rapid onset of action with responses observed as early as one week-post treatment. Implemented a corporate restructuring, including a workforce reduction of approximately 50%, to focus resources on its urticaria programs and preserve capital. As part of the reorganization, Dr. Edwin Tucker stepped down as Chief Medical Officer effective August 1, 2025. He was succeeded in an interim capacity by Dr. Adelman, a member of Jasper’s scientific advisory board and an industry veteran with a strong track record of advancing therapies for allergy and immunology indications. Halted its non-mast cell focused clinical and preclinical programs, including ongoing investigator sponsored trials and the SCID clinical program, to concentrate fully on briquilimab development in mast-cell driven diseases such as CSU and CIndU. Jasper also halted enrollment in the ETESIAN study in asthma and expects to report data from that study, as well as determine next steps in asthma, once the investigation into the anomalous BEACON cohorts is completed, which is expected to be in the second half of 2025. Second Quarter Fiscal 2025 Financial Results Cash and cash equivalents as of June 30, 2025, totaled $39.5 million. Research and development expense for the three months ended June 30, 2025, was $21.2 million. General and administrative expense for the three months ended June 30, 2025, was $5.9 million. Jasper reported a net loss of $26.7 million, or basic and diluted net loss per share attributable to common stockholders of $1.74 for the three months ended June 30, 2025. About Jasper Jasper is a clinical-stage biotechnology company focused on developing briquilimab as a therapeutic for chronic mast cell diseases. Briquilimab is a targeted aglycosylated monoclonal antibody that blocks stem cell factor from binding to the cell-surface receptor KIT, thereby inhibiting signaling through the receptor. This inhibition disrupts the critical survival signal, leading to the depletion of the mast cells via apoptosis which removes the underlying source of the inflammatory response in mast cell driven diseases such as chronic urticaria and asthma. Jasper is currently conducting clinical studies of briquilimab as a treatment in patients with CSU, CIndU, and asthma. Briquilimab has a demonstrated efficacy and safety profile in patients and healthy volunteers, with positive clinical outcomes in both CSU and CIndU. For more information, please visit us at www.jaspertx.com. Forward-Looking Statements Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding briquilimab’s potential, including with respect to its potential in mast cell driven diseases such as CSU and CIndU, its potential to be a highly differentiated therapy in mast cell-driven diseases, its potential to be an important treatment option in chronic urticarias; Jasper’s ability to deliver multiple catalysts over the coming quarters, and the possibility such catalysts may drive meaningful benefits for patients and long term value for stockholders; Jasper’s focus of its resources on the development of briquilimab in mast-cell driven diseases such as CSU and CIndU, including halting its other clinical and preclinical programs; the expected timing of announcing additional data from the BEACON and ETESIAN studies; the expected timing for initiating the planned Phase 2b CSU study; and the expected timing for completing the investigation regarding an atypical lack of UAS7 reduction in certain cohorts and for providing an update on such investigation. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that prior test, study and trial results may not be replicated in continuing or future studies and trials; the risk that Jasper’s investigation into the drug product lot may be inconclusive or may not lead to the anticipated conclusion; the risk that Jasper may be unable to raise capital to continue its operations and continue the BEACON study; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that prior study results may not be replicated; the risk that Jasper’s product candidates may not be beneficial to patients or successfully commercialized; patients’ willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper’s business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper’s business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jasper’s filings with the SEC, including its Annual Report on Form 10-K for the year ended December 31, 2024 and subsequent Quarterly Reports on Form 10-Q. If any of these risks materialize or Jasper’s assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jasper’s assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements. Contacts: Alex Gray (investors) Jasper Therapeutics 650-549-1454 agray@jaspertx.com Joyce Allaire (investors) LifeSci Advisors 617-435-6602 jallaire@lifesciadvisors.com Molly Devlin (media) Real Chemistry 443-416-6675 mdevlin@realchemistry.com

2025年7月7日，Jasper Therapeutics报告Briquilimab治疗慢性自发性荨麻疹的临床数据更新。 “在240毫克和360毫克单剂量队列中接受治疗的9名参与者中，有8名（89%）实现了完全缓解。 在180毫克每8周一次的开放标签扩展研究中，11名参与者中有8名（73%）在12周时实现了完全缓解。 240毫克每8周一次以及240毫克后接180毫克每8周一次队列的结果似乎受到其中一个药物批次问题的干扰。 由于同样的药物批次问题，哮喘领域的ETESIAN研究将被停止。 未报告3级或以上与治疗相关的不良事件。” 图片来自企业官网 Briquilimab的作用机制 Briquilimab Briquilimab，这是一种针对KIT（CD117）的新型抗体疗法，用于治疗由肥大细胞驱动的疾病，如慢性自发性荨麻疹（CSU）和慢性诱导性荨麻疹（CIndU）。 肥大细胞与KIT的关系 KIT（CD117）是一种受体酪氨酸激酶，其配体是干细胞因子（SCF）。SCF/KIT 信号通路在肥大细胞的存活、增殖和激活中起关键作用。 肥大细胞是多种过敏性和特应性疾病（包括荨麻疹、哮喘、食物过敏等）的主要驱动因素。而KIT抑制是唯一被证明可以显著减少肥大细胞的治疗机制，能够实现持久且耐受良好的疾病控制，并且已在多种肥大细胞介导的疾病中展示了临床概念验证。 慢性荨麻疹 慢性荨麻疹是一种严重且使人衰弱的疾病，会对患者的生活质量产生负面影响。它是一种持续6周或更长时间的皮肤炎症性疾病，其特征是出现瘙痒性风团（荨麻疹）、血管性水肿或两者兼有，分为自发性（CSU）和诱导性（CIndU）两种。肥大细胞脱颗粒导致组胺和其他炎症介质的释放，是引起严重瘙痒、荨麻疹和血管性水肿的关键因素。慢性荨麻疹患者在身体和心理上都承受着痛苦，严重的疾病对生活质量（QoL）的负面影响与银屑病或特应性皮炎相似。 治疗现状与机制 肥大细胞在多种免疫和炎症疾病中起着驱动炎症的核心作用，而目前批准的治疗方法依赖于抑制肥大细胞激活的单一通路，疗效有限且反应持续时间较短。由于皮肤中肥大细胞的恢复需要时间，这可能带来持久的疾病控制。抑制SCF/KIT信号通路已被证明可以防止肥大细胞激活并导致肥大细胞耗竭，Briquilimab直接抑制SCF/KIT信号通路，通过受控的凋亡途径实现肥大细胞耗竭，实现双重疾病控制。 Briquilimab的作用机制 Briquilimab的初步疗效 Briquilimab 的疗效和安全性已在慢性自发性荨麻疹（CSU，BEACON 研究）和慢性诱导性荨麻疹（CIndU，SPOTLIGHT 研究）的 Phase 1b/2a 临床研究中得到验证。Briquilimab 的设计允许较长的剂量间隔（例如每 8 周一次），这有助于在减少肥大细胞数量的同时，避免对其他表达 KIT 的细胞类型（如造血干细胞）产生持续的抑制作用而带来的不良影响。 Briquilimab 的给药剂量设计考量 BEACON：Briquilimab的1b/2a期 临床研究 研究设计 BEACON研究是一项随机、双盲、安慰剂对照的1b/2a期试验，评估皮下注射Briquilimab的多剂量递增方案，作为对高剂量抗组胺药物治疗无效的中重度CSU成年患者的治疗方案。主要终点是Briquilimab的安全性和耐受性，次要终点则侧重于临床活性和药代动力学/药效学（PK/PD），包括测量血清类胰蛋白酶和皮肤肥大细胞。 BEACON研究设计 BEACON试验单剂量队列：240mg单剂量（SD）和360mg单剂量（SD） 240mg单剂量和360mg单剂量的药代动力学（PK）浓度随时间变化情况如下图： 在给药后大约第5至第8天达到快速的药物浓度峰值（C-Max）。这体现了Briquilimab 快速起效的特点，支持了其在肥大细胞介导疾病中的治疗潜力。 血清类胰蛋白酶水平呈剂量依赖性降低，在接受单剂量240mg和360mg的参与者中，有80%（8/10）的个体在第2周时血清类胰蛋白酶水平降至1.6μg/ml以下（见下图）。 [血清类胰蛋白酶是肥大细胞激活的重要标志物，其水平的变化与多种肥大细胞疾病的发生、发展和严重程度密切相关。肥大细胞的激活以及增大会引起血清类胰蛋白酶的水平升高。测血清类胰蛋白酶水平对于诊断、监测和治疗肥大细胞疾病具有重要意义。] BEACON试验的第6队列（240mg单剂量）和第7队列（360mg单剂量）中，89%（9名患者中的8名）在接受单剂量给药后达到完全缓解（见下图）。 UAS7：7天荨麻疹活动评分，通过统计患者在7天内每天的风团（荨麻疹）数量和瘙痒程度来衡量病情的严重程度。 在240毫克单剂量队列中，UAS7评分从基线平均下降了28.3分，而在360毫克单剂量队列中平均下降了22.9分。 在240毫克单剂量队列中接受治疗的5名参与者全部（100%）实现了完全缓解（UAS7 = 0），在360毫克单剂量队列中接受治疗的4名参与者中有3名（75%）实现了完全缓解，且9名参与者中有7名（78%）在8周内维持了良好的疾病控制。 BEACON试验多剂量队列：240mg每8周一次（Q8W）和240mg/180mg每8周一次（Q8W）。 多剂量组受到药物批次问题的影响，主要为： 10名接受一个药物批次（“Lot A34954”）治疗的患者均未显示出UAS7评分的降低，血清类胰蛋白酶水平也仅有适度降低（见下图）。 2名确认使用了不同批次（该批次也在OLE研究中使用）的患者实现了快速的完全缓解和深度的类胰蛋白酶降低。 在这两个队列中，药代动力学（PK）水平达到了合理范围，与240mg单剂量（SD）和360mg单剂量（SD）患者一致。 未观察到新的安全性信号。 针对这一情况，Jasper公司（1）对该批次潜在问题的调查正在进行中；（2）第二批药物运往各研究地点，用于240mg Q8W和240mg/180mg Q8W患者的给药；（3）在这两个队列中额外增加10-12名患者，以提供更多数据。 安全性 Briquilimab耐受性良好，并且显示出良好的安全性（见下图）。 与KIT阻断可能相关的安全性/耐受性观察结果通常仅限于低级别事件。大多数在重复给药过程中得到解决，且没有任何事件导致停药（见下图）。 240mg单剂量和360mg单剂量的中性粒细胞计数总体稳定，可预测地降低后会恢复。平均恢复时间为42天，没有因中性粒细胞计数降低而导致的停药或剂量延迟（见下图）。 慢性自发性荨麻疹（CSU）开放标签扩展研究：剂量180mg，每8周一次（Q8W） Jasper开展慢性荨麻疹开放标签研究（JSP-CP-014）的目的：为慢性自发性荨麻疹（CSU）和慢性诱导性荨麻疹（CIndU）项目提供额外的安全性、疗效和持久性数据，剂量方案为180mg每8周一次（Q8W）。 研究设计预计将从母研究（BEACON和SPOTLIGHT）中增加约80名患者。 初步开放标签扩展（OLE）180mg Q8W结果 早在第1周就实现了快速控制，第12周时平均从基线变化超过25分（见下图）。 此外，该研究的11名参与者中有8名（73%）在第12周评估时实现了完全缓解。 早在第1周评估时，就观察到类胰蛋白酶水平显著下降，且与临床反应的起效相关。在240毫克和360毫克单剂量队列中，10名参与者中有8名（80%）的类胰蛋白酶水平低于定量下限。 在开放标签扩展（OLE）研究中，Briquilimab耐受性良好，并且显示出良好的安全性特征。 开放标签扩展（OLE）研究中与KIT阻断可能相关的安全性/耐受性观察结果。 结语 单剂量方面，Briquilimab继续显示出快速起效和深度临床反应 单剂量（240mg单剂量和360mg单剂量，n=9）在第4周时UAS7评分降低超过25分，完全缓解（CR）率达到78%，疾病得到控制（WC）的比例为89%。 开放标签扩展（OLE）数据中显示出强大的差异化疗效和安全性 在OLE研究中，180mg每8周一次（Q8W）的剂量观察到深度且持续的UAS7降低。 在第12周时，完全缓解（CR）率为73%，疾病得到控制（WC）的比例为82%（n=11）。 安全性数据支持在180mg Q8W剂量下具有积极的差异化安全性特征。 在慢性自发性荨麻疹（CSU）治疗领域，Briquilimab作为一种研究性药物，展现出了显著的潜力。通过针对肥大细胞的耗竭机制，Briquilimab不仅在单剂量给药中迅速降低了症状评分（UAS7），还实现了深度的临床反应和良好的安全性。尽管在多剂量队列中因药物批次问题（Lot A34954）遇到了挑战，但公司已采取积极措施进行调查，并计划通过替换药物批次和增加患者数据来解决这一问题。此外，开放标签扩展（OLE）研究中的初步结果显示，180mg每8周一次（Q8W）的剂量在控制症状方面表现出色，进一步证实了其在长期治疗中的潜力。随着更多数据的积累和研究的推进，Briquilimab有望为CSU患者提供一种新的、有效的治疗选择，改善他们的生活质量。 参考资料来源于企业官网。欢迎感兴趣的朋友进群讨论，群二维码如下： 推文用于传递知识，如因版权等有疑问，请于本文刊发30日内联系医药速览。 原创内容未经授权，禁止转载至其他平台。 有问题可发邮件至yong_wang@pku.edu.cn获取更多信息。 ©2021 医药速览 保留所有权利 往期链接 “小小疫苗”养成记 | 医药公司管线盘点  人人学懂免疫学| 人人学懂免疫学（语音版） 综述文章解读 | 文献略读 | 医学科普|医药前沿笔记 PROTAC技术| 抗体药物| 抗体药物偶联-ADC 核酸疫苗 | CAR技术| 化学生物学 温馨提示 医药速览公众号目前已经有近12个交流群（好学，有趣且奔波于医药圈人才聚集于此）。进群请扫描上方二维码，备注“姓名/昵称-企业/高校-具体研究领域/专业”，此群仅为科研交流群，非诚勿扰。 简单操作即可星标⭐️医药速览，第一时间收到我们的推送 ①点击标题下方“医药速览”   ②至右上角“...”  ③点击“设为星标