NEW HAVEN, Conn.--(BUSINESS WIRE)--Rallybio Corporation (Nasdaq: RLYB) today announced preliminary Phase 1 multiple ascending dose (MAD) data for RLYB116, an innovative, long-acting, low volume subcutaneously injected inhibitor of complement component 5 (C5), in development for patients with complement-mediated diseases..

康涅狄格州纽黑文（商业新闻短讯）--Rallybio Corporation（纳斯达克：RLYB）今天宣布了RLYB116的初步第一阶段多重递增剂量（MAD）数据，RLYB116是一种创新的，长效的，低容量皮下注射的补体成分5（C5）抑制剂，正在为补体介导的疾病患者开发中。。

The Phase 1 MAD study for RLYB116 evaluated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous RLYB116 in healthy participants with multiple dose administration. The MAD study utilized an adaptive design and included four cohorts of 12 participants receiving doses of up to 200 mg per week of RLYB116 or placebo, with a four-week treatment duration and a 10-week follow-up period..

RLYB116的1期MAD研究评估了多次给药的健康参与者皮下RLYB116的安全性，耐受性，药代动力学（PK）和药效学（PD）。MAD研究采用适应性设计，包括四组12名参与者，每周接受高达200毫克的RLYB116或安慰剂，治疗时间为四周，随访期为10周。。

The preliminary results showed:

初步结果显示：

A 100 mg low volume (1 mL) once-a-week dose of subcutaneously administered RLYB116 achieved sustained mean reductions in free C5 of greater than 93%, including at Day 29 with measurement prior to the last dose. The reduction in free C5 at 24 hours after the first dose of 100 mg was greater than 99%.

每周一次皮下注射100毫克低容量（1毫升）剂量的RLYB116，游离C5的持续平均减少率大于93%，包括在第29天，在最后一次剂量之前进行测量。第一剂100 mg后24小时游离C5的减少大于99%。

These data and additional work we have conducted with RLYB116 reinforce our confidence that RLYB116 has the potential to be an effective treatment for patients with certain complement-mediated diseases, including generalized myasthenia gravis (gMG)..

这些数据和我们对RLYB116进行的其他工作增强了我们的信心，即RLYB116有可能成为某些补体介导疾病（包括全身性重症肌无力（gMG））患者的有效治疗方法。。

RLYB116 also demonstrated low inter-subject variability and consistent increases in exposure relative to dose. The mean estimated elimination half-life for RLYB116 was >300 hours.

RLYB116还表现出较低的受试者间变异性和相对于剂量的暴露持续增加。RLYB116的平均估计消除半衰期>300小时。

In comparison to 100 mg weekly administration, higher concentrations of RLYB116 were observed in a cohort with 100 mg administered twice per week and were associated with a greater than 97% mean reduction in free C5.

与每周100 mg给药相比，在每周两次100 mg给药的队列中观察到更高浓度的RLYB116，并且游离C5的平均减少率大于97%。

RLYB116 administered as a 100 mg once-a-week dose was observed to be generally well tolerated. The most common adverse event (AE) in the cohort was injection site reaction (ISR), which occurred in 60% of the participants in the cohort. All AEs during subcutaneous administration with the 100 mg weekly dose were mild in severity..

观察到RLYB116以100 mg每周一次的剂量给药，通常耐受性良好。队列中最常见的不良事件（AE）是注射部位反应（ISR），发生在队列中60%的参与者中。每周100 mg皮下给药期间的所有AE严重程度较轻。。

The ISR rate for all participants in the 4 cohorts was 59% and all were mild in severity. There were no serious AEs reported for participants receiving study treatment.

4个队列中所有参与者的ISR率为59%，严重程度均较轻。接受研究治疗的参与者没有报告严重的AE。

A participant with a history of hepatitis A receiving the 150 mg dose experienced liver enzyme test elevation that resulted in discontinuation and a reduction in the dose for the 3rd cohort from 150 mg to 125 mg.

有甲型肝炎病史的参与者接受150毫克剂量的肝酶试验升高，导致第三组患者停药，剂量从150毫克降至125毫克。

The measurement of anti-drug antibody (ADA) formation in the study did not demonstrate an effect on PK or PD parameters and did not appear to be associated with an effect on AE incidence or severity.

该研究中抗药物抗体（ADA）形成的测量未显示对PK或PD参数的影响，并且似乎与AE发生率或严重程度的影响无关。

“The preliminary results from this Phase 1 multiple ascending dose study of RLYB116 support continued development in patients with gMG,” said Eric Watsky, M.D., RLYB116 Program Lead for Rallybio. “We are encouraged by the free C5 reductions demonstrated by RLYB116 as well as the exposures achieved with subcutaneous administration.

Rallybio RLYB116项目负责人Eric Watsky医学博士说：“RLYB116第一阶段多剂量递增研究的初步结果支持gMG患者的持续发展。”。“我们对RLYB116证明的免费C5减少以及皮下给药获得的暴露感到鼓舞。

Through enhancements in the manufacturing process, we believe we have the opportunity to increase the dose of RLYB116 and improve the tolerability thereby opening up the opportunity to treat a wider range of complement mediated diseases. Our market research is consistent with our belief that an effective, once-a-week, well-tolerated therapy that can be rapidly self-administered with an autoinjector would be an attractive alternative for patients.”.

通过改进制造过程，我们相信我们有机会增加RLYB116的剂量并提高耐受性，从而为治疗更广泛的补体介导的疾病开辟了机会。我们的市场研究与我们的信念是一致的，即一种有效的，每周一次，耐受性良好的治疗方法，可以通过自动注射器快速自我管理，对患者来说是一种有吸引力的替代方法。”。

RLYB116 Near-Term Development Plans and Cash Runway

RLYB116近期发展计划和现金跑道

The preliminary data from the Phase 1 MAD study confirm improvements made to date in the manufacturing process will enable the Company to advance RLYB116 into studies in patients. Rather than immediately proceed to a Phase 2 study in gMG, the Company intends to prioritize near-term investments in RLYB116 in the manufacturing process.

第一阶段MAD研究的初步数据证实，迄今为止在制造过程中取得的改进将使该公司能够将RLYB116推进患者研究。该公司不打算立即在gMG进行第二阶段研究，而是打算在制造过程中优先考虑对RLYB116的短期投资。

The Company expects that the additional manufacturing work will improve tolerability at higher doses with a low injection volume and infrequent subcutaneous administration. The Company believes such enhancements will enable higher exposure to RLYB116 and potentially increase C5 reduction, which can result in treating a broader range of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria and antiphospholipid syndrome.

该公司预计，额外的制造工作将提高低注射量和不频繁皮下给药的高剂量耐受性。该公司认为，这种增强将使RLYB116的暴露率更高，并可能增加C5的减少，从而可以治疗更广泛的补体介导的疾病，包括阵发性夜间血红蛋白尿和抗磷脂综合征。

In addition, this will allow the Company to direct available cash resources to advance RLYB212, its product candidate for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT)..

此外，这将使该公司能够将可用的现金资源用于推进RLYB212，RLYB212是其预防胎儿和新生儿同种免疫性血小板减少症（FNAIT）的候选产品。。

The Company is also updating its cash runway guidance and now expects its current cash, cash equivalents and marketable securities to extend the runway into the third quarter of 2025.

该公司还正在更新其现金跑道指南，目前预计其现有现金、现金等价物和有价证券的跑道将延长至2025年第三季度。

“We are pleased to see substantial reductions in free C5 with once weekly subcutaneous dosing of RLYB116,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “The Phase 1 MAD data show us that RLYB116 can be a potential therapeutic to treat gMG and other complement mediated diseases. In the spirit of managing our cash runway to realize the most value from our portfolio, we have decided to focus our RLYB116 investments on the manufacturing process with a goal of expanding the scope of therapeutic indications and addressing unmet medical need.

Rallybio首席执行官斯蒂芬·乌登（StephenUden）医学博士说：“我们很高兴看到每周一次皮下注射RLYB116的免费C5大幅减少。”。“第一阶段MAD数据显示，RLYB116可能是治疗gMG和其他补体介导疾病的潜在治疗药物。本着管理现金跑道以实现投资组合最大价值的精神，我们决定将RLYB116投资集中在制造过程中，目标是扩大治疗指征的范围并解决未满足的医疗需求。

In parallel, we continue to advance our lead program, RLYB212, and have extended our runway into the third quarter of 2025.”.

与此同时，我们继续推进领先项目RLYB212，并将跑道延长至2025年第三季度。”。

Conference Call Information

电话会议信息

Rallybio will host a conference call and webcast today, December 20, 2023 at 8:30 a.m. Eastern Time to discuss the RLYB116 Phase 1 multiple ascending dose (MAD) study. The live webcast and replay may be accessed by visiting Rallybio’s website at http://investors.rallybio.com. In addition, key slides from the RLYB116 Phase 1 MAD study will be discussed on the conference call and are posted to the “Events and Presentations” section of the Rallybio website.

Rallybio将于今天（2023年12月20日）东部时间上午8:30主持电话会议和网络广播，讨论RLYB116第一阶段多重递增剂量（MAD）研究。可以访问Rallybio的网站访问直播和重播http://investors.rallybio.com.此外，RLYB116第一阶段MAD研究的关键幻灯片将在电话会议上讨论，并发布到Rallybio网站的“活动和演示”部分。

A replay of the webcast will be available on the Rallybio website for 30 days following the event..

赛后30天内，Rallybio网站将提供网络直播的重播。。

About RLYB116 Phase 1 Study

关于RLYB116第一阶段研究

RLYB116 is an innovative, long-acting, subcutaneously injected inhibitor of C5 in development for the treatment of patients with complement-mediated diseases. Phase 1 in healthy participants included the study of RLYB116 as a single ascending dose and multiple ascending dose. The multiple ascending dose (MAD) study of RLYB116 included an adaptive single-blind design initiated in the first quarter of 2023 with a 4-week treatment duration to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of subcutaneous RLYB116 in healthy participants with multiple dose administration..

RLYB116是一种创新的长效皮下注射C5抑制剂，正在开发中，用于治疗补体介导的疾病患者。健康参与者的第一阶段包括RLYB116作为单次递增剂量和多次递增剂量的研究。RLYB116的多次递增剂量（MAD）研究包括2023年第一季度开始的适应性单盲设计，治疗时间为4周，以评估皮下RLYB116在多剂量给药的健康参与者中的安全性，耐受性，药代动力学和药效学。。

The MAD study of RLYB116 included 4 cohorts: Cohort 1 (weekly dosing of 100 mg), Cohort 2 (3 doses of 100 mg the first week followed by weekly dosing), Cohort 3 (150 mg weekly dosing reduced to 125 mg weekly dosing) and Cohort 4 (75 mg twice the first week followed by 100 mg twice per week).

RLYB116的MAD研究包括4个队列：队列1（每周给药100毫克），队列2（第一周给药100毫克，然后每周给药3次），队列3（每周给药150毫克，每周给药125毫克）和队列4（第一周两次75毫克，然后每周两次100毫克）。

Post-treatment / study follow-up continued for 10 weeks.

治疗后/研究随访持续10周。

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..

该公司有两个临床阶段计划：用于预防胎儿和新生儿同种免疫性血小板减少症（FNAIT）的抗HPA-1a抗体RLYB212和补体成分5（C5）抑制剂RLYB116，具有治疗几种补体失调疾病的潜力，以及临床前开发中的其他计划。。

Rallybio is headquartered in New Haven, Connecticut with an additional facility at the University of Connecticut’s Technology Incubation Program in Farmington, 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，在康涅狄格州法明顿的康涅狄格大学技术孵化计划中有一个额外的设施。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声明都包含这些词语。

Forward-looking statements in this press release include, but are not limited to, statements concerning results from the Phase 1 MAD study of RLYB116; potential clinical effects and benefits of RLYB116, including for the treatment of gMG; the timing and initiation of future clinical studies for RLBY116; the success cost and timing of our clinical development of our product candidates, including RLYB212 and RLYB116; and statements concerning the Company’s anticipated use of cash and cash runway.

本新闻稿中的前瞻性声明包括但不限于有关RLYB116第一阶段MAD研究结果的声明；RLYB116的潜在临床效果和益处，包括用于治疗gMG；RLBY116未来临床研究的时机和开始；我们候选产品（包括RLYB212和RLYB116）临床开发的成功成本和时机；以及有关公司预期使用现金和现金跑道的报表。

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 studies, and complete such clinical studies and obtain results on our expected timelin.

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的临床研究的能力，以及完成此类临床研究并在我们预期的时间内获得结果的能力。