NEW HAVEN, Conn.--(BUSINESS WIRE)--Rallybio Corporation (Nasdaq: RLYB), a clinical-stage biotechnology company translating scientific advances into transformative therapies for patients with devastating rare diseases, announced today that nonclinical data demonstrating ENPP1 inhibition as a therapeutic approach for the treatment of patients with hypophosphatasia (HPP) was presented at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting.

康涅狄格州纽黑文讯（商业新闻短讯）--临床阶段生物技术公司Rallybio Corporation（纳斯达克：RLYB）今天宣布，非临床数据显示ENPP1抑制作为治疗低磷血症（HPP）患者的治疗方法已在美国骨与矿物质研究学会（ASBMR）2024年会上发表。

ASBMR is being held September 27 – 30, 2024 in Toronto, Canada..

ASBMR将于2024年9月27日至30日在加拿大多伦多举行。。

“From my team’s earlier work published in 2002 and 2005, we knew that ENPP1 could be a targetable molecule to modulate TNAP’s all-important substrate PPi. I am delighted that we have now been able to demonstrate the efficacy of this principle in our mouse model of later-onset HPP,” said José Luis Millán, Ph.D., Professor, Human Genetics Program at Sanford Children’s Health Research Center, Sanford Burnham Prebys Medical Discovery Institute, and author of the study..

“从我的团队在2002年和2005年发表的早期工作中，我们知道ENPP1可能是调节TNAP至关重要的底物PPi的可靶向分子。我很高兴我们现在能够在我们的晚发性HPP小鼠模型中证明这一原理的有效性，”桑福德·伯纳姆·普雷比斯医学发现研究所桑福德儿童健康研究中心人类遗传学项目教授、该研究的作者何塞·路易斯·米兰博士说。。

HPP is a rare, genetic, metabolic disorder characterized by poor bone mineralization. The disease has a broad spectrum of symptoms and severity ranging from the life-threatening perinatal- and infantile-onset form to the less severe juvenile-onset form that can manifest with frequent bone fractures, significant joint and bone pain, and joint swelling.

HPP是一种罕见的遗传性代谢疾病，其特征是骨矿化不良。该疾病具有广泛的症状和严重程度，从危及生命的围产期和婴儿期发作形式到较轻的青少年发作形式，可表现为频繁的骨折，明显的关节和骨痛以及关节肿胀。

HPP is caused by loss-of-function mutations in the gene that encodes tissue-nonspecific alkaline phosphatase (TNAP), whose deficiency results in the accumulation of extracellular inorganic pyrophosphate (PPi), an inhibitor of bone mineralization..

HPP是由编码组织非特异性碱性磷酸酶（TNAP）的基因中的功能丧失突变引起的，该突变的缺乏会导致细胞外无机焦磷酸盐（PPi）的积累，PPi是骨矿化的抑制剂。。

“The data presented at ASBMR supports ENPP1 inhibition as a therapeutic approach for patients with hypophosphatasia,” said Stephen Uden, MD, Chief Executive Officer of Rallybio. “There is significant unmet patient need in HPP, particularly in adults. We believe this data with an early lead ENPP1 inhibitor from our joint venture with Exscientia is very promising and gives us enthusiasm that the development candidate we expect to nominate in the fourth quarter could be a safe and effective treatment to meaningfully improve the lives of patients suffering from HPP.”.

Rallybio首席执行官斯蒂芬·乌登（Stephen Uden）医学博士表示：“ASBMR上提供的数据支持ENPP1抑制作为低磷血症患者的治疗方法。HPP患者的需求尚未得到满足，尤其是成年人。我们相信，我们与Exscientia合资企业的早期领先ENPP1抑制剂的数据非常有希望，并使我们充满热情，认为我们预计在第四季度提名的开发候选人可能是一种安全有效的治疗方法，可以显着改善HPP患者的生活。”。

This nonclinical study was designed to assess whether ENPP1 could be a druggable target to treat the non-lethal forms of HPP using an early lead ENPP1 inhibitor and the Alpl-/Prx1 mouse, which is a model of later-onset HPP.

这项非临床研究旨在评估ENPP1是否可以成为使用早期铅ENPP1抑制剂和Alpl-/Prx1小鼠治疗非致命形式HPP的药物靶标，Alpl-/Prx1小鼠是晚期HPP的模型。

Results indicate that oral dosing of an early lead ENPP1 inhibitor, REV101, to adult HPP mice lowered PPi by 30%, leading to improvements in mineralization of long and vertebrate bones. Furthermore, data showed that ENPP1 inhibition was safe and well-tolerated, and, for the first time, showed that ENPP1 is a druggable target for later-onset HPP..

结果表明，向成年HPP小鼠口服早期铅ENPP1抑制剂REV101可将PPi降低30%，从而改善长骨和脊椎动物骨骼的矿化。此外，数据显示ENPP1抑制是安全且耐受性良好的，并且首次表明ENPP1是迟发性HPP的可药物靶标。。

Rallybio and Exscientia plc (Nasdaq: EXAI) are developing an ENPP1 inhibitor with improved properties compared with REV101 as a differentiated therapy to address the unmet need in patients with HPP. Rallybio and Exscientia expect to nominate a development candidate in the fourth quarter of 2024.

Rallybio和Exscientia plc（纳斯达克股票代码：EXAI）正在开发一种ENPP1抑制剂，与REV101相比具有更好的特性，作为一种分化疗法，以解决HPP患者未满足的需求。Rallybio和Exscientia预计将在2024年第四季度提名一名发展候选人。

Details of yesterday’s poster presentation:

昨天海报展示的细节：

Title: ENPP1 Inhibition as a Therapeutic Approach for Later-onset Hypophosphatasia

标题：抑制ENPP1作为晚发性低磷血症的治疗方法

Date/Time: September 29, 2024, 2:15pm – 3:45pm EDT

日期/时间：美国东部时间2024年9月29日下午2:15至3:45

Presenter: Dr. José Luis Millán

主持人：JoséLuis Millán博士

Poster Number: Sun-LB 552

海报编号：Sun LB 552

The poster will be available in the Publications & Presentations section of Rallybio’s website following the conclusion of the conference.

会议结束后，海报将在Rallybio网站的出版物和演示部分提供。

About Rallybio

关于Rallybio

Rallybio (NASDAQ: RLYB) is a clinical-stage biotechnology company with a mission to develop and commercialize life-transforming therapies for patients with severe and rare diseases. Rallybio has built a broad pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of maternal fetal health, complement dysregulation, hematology, and metabolic disorders.

Rallybio（纳斯达克股票代码：RLYB）是一家临床阶段生物技术公司，其使命是为患有严重和罕见疾病的患者开发和商业化改变生命的疗法。Rallybio已经建立了一系列有前途的候选产品，旨在解决母胎健康、补体失调、血液学和代谢紊乱等领域医疗需求未得到满足的疾病。

The Company has two clinical stage programs: RLYB212, an anti-HPA-1a antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) and RLYB116, an inhibitor of complement component 5 (C5), with the potential to treat several diseases of complement dysregulation, as well as additional programs in preclinical development.

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Rallybio is headquartered in New Haven, Connecticut. For more information, please visit www.rallybio.com and follow us on LinkedIn and Twitter..

Rallybio总部位于康涅狄格州纽黑文。有关更多信息，请访问www.rallybio.com，并在LinkedIn和Twitter上关注我们。。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements that are based on our management’s beliefs and assumptions and on currently available information. All statements, other than statements of historical facts contained in this press release are forward-looking statements. In some cases, forward-looking statements can be identified by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words.

本新闻稿包含基于管理层信念和假设以及当前可用信息的前瞻性声明。除本新闻稿中包含的历史事实声明外，所有声明均为前瞻性声明。在某些情况下，前瞻性陈述可以通过“可能”、“将会”、“应该”、“期望”、“计划”、“预期”、“可能”、“打算”、“目标”、“项目”、“沉思”、“相信”、“估计”、“预测”、“潜在”或“继续”等术语或这些术语的否定或其他类似表达来识别，尽管并非所有前瞻性陈述都包含这些词语。

Forward-looking statements in this press release include, but are not limited to, statements concerning the timing of development candidate nomination for Rallybio’ ENPP1 inhibitor, whether Rallybio’s small molecule would successfully inhibit ENPP1, or demonstrate efficacy in later-onset HPP, or otherwise be safe when administered to humans.

本新闻稿中的前瞻性声明包括但不限于关于Rallybio'ENPP1抑制剂的开发候选人提名时间的声明，Rallybio的小分子是否能成功抑制ENPP1，或在晚发性HPP中表现出疗效，或者在给人类服用时是否安全。

The forward-looking statements in this press release are only predictions and are based largely on management’s current expectations and projections about future events and financial trends that management believes may affect Rallybio’s business, financial condition and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions, including, but not limited to, our ability to successfully initiate and conduct our planned clinical trials, including the FNAIT natural history study, and the Phase 2 clinical trial for RLYB212, and complete such clinical trials and obtain results on our expected timelines, or at all, whether our cash resources will be sufficient to fund ou.

本新闻稿中的前瞻性陈述仅为预测，主要基于管理层目前对未来事件和财务趋势的预期和预测，管理层认为这些事件和趋势可能会影响Rallybio的业务、财务状况和经营成果。这些前瞻性声明仅在本新闻稿发布之日起发表，并受到许多已知和未知风险、不确定性和假设的影响，包括但不限于我们成功启动和进行计划临床试验的能力，包括FNAIT自然史研究和RLYB212的2期临床试验，并完成此类临床试验并在我们预期的时间表上取得结果，或者我们的现金资源是否足以资助ou。