Itolizumab

▎药明康德内容团队编辑 Equillium今日宣布其潜在“first-in-class”疗法itolizumab用于治疗中重度溃疡性结肠炎（UC）的临床2期研究获得积极结果。分析显示，itolizumab展现与现有标准治疗药物相当的治疗效果。 这项双盲、安慰剂及活性药物对照的2期临床研究评估了itolizumab在中重度活动性UC患者中的安全性和有效性，这些患者未曾接受过生物制品治疗。共有90名患者按1：1：1比例，随机分配接受每两周一次的itolizumab（固定剂量140毫克）、安慰剂或标准疗法adalimumab治疗，初始治疗期为12周。研究的主要终点为达成临床缓解（clinical remission）的患者比率，定义为患者在第12周时总体Mayo评分≤2且各单项评分均不大于1。次要终点则包括在第12周和第24周时达到临床应答（clinical response，按总体Mayo评分评估）和内镜缓解（endoscopic remission，由中央内镜中心评估）的受试者比例。 第12周时，主要终点的分析显示，itolizumab组患者的临床缓解率为23.3%，活性药物对照组为20.0%，安慰剂组为10.0%。次要终点分析显示，itolizumab组临床应答率为63.3%，活性药物组为60.0%，安慰剂组为46.7%。此外，itolizumab组的内镜缓解率为16.7%，活性药物组为16.7%，安慰剂组为6.7%。Itolizumab总体耐受性良好，未观察到安全性信号。 ▲Itolizumab作用机制（图片来源：Equillium公司官网） Itolizumab是一种在研、潜在“first-in-class”的CD6靶向单克隆抗体，可选择性靶向CD6-ALCAM信号通路，选择性抑制致病性效应T细胞，同时保留对维持平衡免疫反应至关重要的调节性T细胞。T细胞可驱动多种免疫炎症疾病，该通路在调节T细胞的活性和运输方面发挥着核心作用。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Equillium Announces Positive Data from Phase 2 Study Evaluating Itolizumab in Patients with Moderate to Severe Ulcerative Colitis. Retrieved February 6, 2025 from https://www.businesswire.com/news/home/20250206241338/en/Equillium-Announces-Positive-Data-from-Phase-2-Study-Evaluating-Itolizumab-in-Patients-with-Moderate-to-Severe-Ulcerative-Colitis 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

Equillium retains rights to itolizumab following substantial funding from Ono partnership Evaluating accelerating Phase 3 EQUATOR study in aGVHD to completion in Q1 2025 Phase 2 ulcerative colitis study recently completed; topline data expected Q1 2025 LA JOLLA, Calif.--(BUSINESS WIRE)-- Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders, today announced financial results for the third quarter 2024 and provided corporate and clinical updates. “At the end of October we announced that our partnership with Ono Pharmaceutical had ended following substantial non-dilutive financing that has fully funded itolizumab research and development since July 2022. This funding enabled us to advance the program through two recent milestones – positive interim review from the Phase 3 EQUATOR study in acute graft-versus-host disease and positive topline data from the EQUALISE study in lupus nephritis. Ono informed us that their decision was strategic in nature, and that the data we have delivered from these studies met expectations with no observed or reported safety concerns,” said Bruce Steel, chief executive officer at Equillium. “We have enrolled over 150 patients in EQUATOR, and as of the end of October have temporarily paused enrollment to review clinical options for the program, including the potential to accelerate our timeline to topline data to the first quarter of 2025 while preserving registrational integrity of the study. We maintain orphan drug and fast track designations for first-line acute graft-versus-host disease, where currently no drugs are approved. In addition, in collaboration with Equillium, our partner Biocon has recently completed a robust placebo-controlled Phase 2 study of itolizumab in biologic-naïve patients with moderate to severe ulcerative colitis, and we expect topline data from this study also in the first quarter of 2025. We remain enthusiastic about the therapeutic potential and commercial opportunity for itolizumab, and we are very pleased to maintain our rights to the program as we approach the two most important data events in Equillium’s history.” Highlights Since the Beginning of the Third Quarter of 2024: Retained rights to itolizumab following Ono partnership Positive interim analysis of Phase 3 EQUATOR study of itolizumab in acute graft-versus-host disease subjects Phase 2 ulcerative colitis study completed by partner Biocon Anticipated Upcoming Milestones: Itolizumab: Topline data from Phase 2 study in ulcerative colitis – Q1 2025 Itolizumab: Topline data from Phase 3 EQUATOR study in aGVHD – potential acceleration to Q1 2025 Third Quarter 2024 Financial Results Revenue for the third quarter of 2024 was $12.2 million, compared to $8.9 million during the same period in 2023. Revenue in the third quarters of 2024 and 2023 consisted entirely of itolizumab development funding and amortization of the upfront payment related to our former partnership with Ono. Research and development (R&D) expenses for the third quarter of 2024 were $9.6 million, compared to $9.0 million for the same period in 2023. The increase was primarily due to greater expenses associated with chemistry, manufacturing and controls (CMC) activities related to itolizumab, greater non-clinical research expenses, and an increase in employee compensation expenses, which were partially offset by lower clinical study expenses primarily driven by the EQUALISE and EQ101 clinical studies, partially offset by greater expenses for our EQUATOR clinical study. General and administrative (G&A) expenses for the third quarter of 2024 were $3.3 million, compared with $3.5 million for the same period in 2023. The decrease was primarily driven by lower legal, audit and tax professional fees. Net loss for the third quarter of 2024 was approximately $7,000, or $(0.00) per basic and diluted share, compared with a net loss of $3.7 million, or $(0.11) per basic and diluted share, for the same period in 2023. The decrease in net loss was primarily attributable to greater revenue related to the Ono partnership, lower income tax expense, and other income, which were partially offset by the increase in R&D expense. Cash, cash equivalents and short-term investments totaled $25.9 million as of September 30, 2024, compared to $33.3 million as of June 30, 2024. Equillium believes that its cash, cash equivalents and short-term investments are sufficient to fund operations into the fourth quarter of 2025, assuming certain operational changes including accelerating the completion of the Phase 3 EQUATOR study based on reduced enrollment and early unblinding of the study, pausing further development activities related to EQ101 and EQ302, the elimination of certain positions and the reduction of certain discretionary expenditures, as well as assuming no further repurchases under our stock repurchase program. About Equillium Equillium is a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders with high unmet medical need. The company’s pipeline consists of the following novel first-in-class immunomodulatory assets and product platform targeting immuno-inflammatory pathways. Itolizumab: a monoclonal antibody that targets the CD6-ALCAM signaling pathway which plays a central role in the modulation of effector T cells that drive a number of immuno-inflammatory diseases; currently under evaluation in a Phase 3 clinical study of patients with acute graft-versus-host disease (aGVHD) and announced positive data from a Phase 1b clinical study of patients with lupus/lupus nephritis in April 2024. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited, who also provides commercial manufacturing for the product. EQ101: a selective tri-specific cytokine inhibitor targeting IL-2, IL-9, and IL-15; recently announced positive results from a Phase 2 proof-of-concept clinical study of patients with alopecia areata. EQ302: an orally delivered, selective bi-specific cytokine inhibitor targeting IL-15 and IL-21 at pre-clinical stage. For more information, visit www.equilliumbio.com. Forward Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking Statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words such as “anticipate”, “believe”, “could”, “continue”, “expect”, “estimate”, “may”, “plan”, “outlook”, “future”, “potential” and “project” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These statements include, but are not limited to, statements regarding Equillium’s plans and strategies with respect to developing itolizumab, including repositioning itolizumab as the top priority in the pipeline, anticipated upcoming milestones, timelines for topline data for itolizumab, the temporary pausing and potential acceleration of the EQUATOR study, the pausing of further activities related to EQ101 and EQ302, Equillium’s cash runway and related assumptions, and the potential benefits of Equillium’s product candidates. Because such statements are subject to risks and uncertainties, many of which are outside of Equillium’s control, actual results may differ materially from those expressed or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: Equillium’s ability to execute its plans and strategies; risks related to performing clinical and pre-clinical studies; whether the results from clinical and pre-clinical studies will validate and support the safety and efficacy of Equillium’s product candidates; changes in the competitive landscape; changes in Equillium’s strategic plans; uncertainties related to Equillium’s capital requirements and ability to obtain sufficient financing to fund Equillium’s strategic plans; the potential impact of the registrational integrity that would be caused by Equillium’s decision to accelerate the completion of EQUATOR; and having to use cash in ways or on timing other than expected and the impact of market volatility on cash reserves. These and other risks and uncertainties are described more fully under the caption "Risk Factors" and elsewhere in Equillium's filings and reports, which may be accessed for free by visiting the Securities and Exchange Commission’s website and on Equillium’s website under the heading “Investors.” Investors should take such risks into account and should not rely on forward-looking statements when making investment decisions. All forward-looking statements contained in this press release speak only as of the date on which they were made. Equillium undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Equillium, Inc. Condensed Consolidated Balance Sheets (In thousands) (unaudited) September 30, December 31, 2024 2023 Assets Cash, cash equivalents and short-term investments $ 25,877 $ 40,866 Accounts receivable 5,009 3,735 Prepaid expenses and other assets 3,098 5,133 Operating lease right-of-use assets 473 796 Total assets $ 34,457 $ 50,530 Current liabilities Accounts payable and other current liabilities $ 8,989 $ 11,844 Current portion of deferred revenue 2,068 15,729 Total current liabilities 11,057 27,573 Long-term operating lease liabilities 223 384 Total liabilities 11,280 27,957 Total stockholders' equity 23,177 22,573 Total liabilities and stockholders' equity $ 34,457 $ 50,530 Equillium, Inc. Condensed Consolidated Statements of Operations (In thousands, except share and per share data) (unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Revenue $ 12,161 $ 8,870 $ 36,703 $ 26,873 Operating expenses: Research and development 9,562 8,974 30,113 27,855 General and administrative 3,278 3,519 10,161 10,340 Total operating expenses 12,840 12,493 40,274 38,195 Loss from operations (679 ) (3,623 ) (3,571 ) (11,322 ) Total other income, net 672 409 1,298 893 Loss before income taxes (7 ) (3,214 ) (2,273 ) (10,429 ) Income tax expense - 496 - 564 Net loss $ (7 ) $ (3,710 ) $ (2,273 ) $ (10,993 ) Net loss per share, basic and diluted $ (0.00 ) $ (0.11 ) $ (0.06 ) $ (0.32 ) Weighted-average number of common shares outstanding, basic and diluted 35,424,388 34,878,700 35,324,092 34,582,574 Investor Contact Michael Moore Vice President, Investor Relations & Corporate Communications 619-302-4431 ir@equilliumbio.com