Clinical trial Safety Review Committee (SRC) approved opening of Cohort 4 based on favorable safety data from the three patients comprising Cohort 3

临床试验安全审查委员会 (SRC) 根据第三组三名患者的良好安全性数据，批准了第四组的开放。

No significant safety or dose limiting toxicities reported in Cohorts 1, 2, or 3

在队列1、2或3中未报告显著的安全性或剂量限制性毒性

Patients currently being evaluated for Cohort 4 eligibility

目前正在评估符合队列4资格的患者

PK and PD data from patients in Cohort 1 and Cohort 2 consistent with preclinical results and results from Phase 0 clinical trial

第1队列和第2队列患者的PK和PD数据与临床前结果及0期临床试验结果一致

BOSTON

波士顿

,

，

March 13, 2025

2025年3月13日

/PRNewswire/ -- TransCode Therapeutics, Inc. (NASDAQ:

/PRNewswire/ -- TransCode Therapeutics, Inc.（纳斯达克：

RNAZ

RNAZ

), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced that the Safety Review Committee (SRC) monitoring its Phase I clinical trial has unanimously approved opening of the fourth cohort of patients based on the SRC's favorable review of Cohort 3 safety data.

)，这家致力于使用RNA疗法更有效治疗癌症的RNA肿瘤学公司，今天宣布，监督其I期临床试验的安全审查委员会 (SRC) 根据对第三组患者安全数据的良好评估，一致批准开放第四组患者。

The therapeutic candidate being evaluated, TTX-MC138, is a first-in-class therapeutic designed to treat multiple metastatic cancers using antisense technology.  The dose administered to the fourth cohort, as originally planned in the clinical protocol, will be approximately fifty percent higher than the dose administered in the third cohort..

正在评估的治疗候选药物 TTX-MC138 是一种首创的治疗方法，旨在利用反义技术治疗多种转移性癌症。按照临床方案的最初计划，给予第四组的剂量将比第三组的剂量高出约百分之五十。

Under the clinical protocol, patients may remain on study absent safety events or disease progression. Out of 9 patients treated with TTX-MC138 in the first three cohorts, 6 remain on study for continued treatment since there have been no dose limiting toxicities or disease progression with these patients.

根据临床方案，患者在没有安全性事件或疾病进展的情况下可以继续留在研究中。在前三个队列中接受TTX-MC138治疗的9名患者中，有6名患者因未出现剂量限制性毒性或疾病进展而仍在研究中继续接受治疗。

The patient that has remained on study the longest has, to date, received 7 doses each approximately 28 days apart over the approximately 7 months that this patient has been on study. In addition to approving opening the fourth cohort, the SRC approved enrollment of additional patients in Cohort 3 to build upon the safety profile of TTX-MC138.  Further, Cohort 1 and 2 data analysis for both pharmacokinetic (PK) and pharmacodynamic (PD) activity is ongoing and suggests that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode's Phase 0 clinical trial.  Preliminary results from Cohort 2 confirmed observations from the Phase 0 trial and from Cohort 1 that the drug candidate shows evidence of activity in patient blood.

在研究中停留时间最长的患者迄今为止已经接受了7次给药，每次给药间隔大约28天，该患者在约7个月的研究期间内持续接受治疗。除了批准开放第四组外，SRC还批准在第三组中招募更多患者，以进一步完善TTX-MC138的安全性数据。此外，第一和第二组的药代动力学（PK）和药效学（PD）活动数据分析正在进行中，结果显示TTX-MC138的PK/PD特性与临床前结果以及TransCode的0期临床试验结果一致。第二组的初步结果证实了0期试验和第一组的观察结果，即该候选药物在患者血液中显示出活性迹象。

Preliminary PK analysis suggests that dose levels 0.8-1.6 mg/kg, the levels administered to Cohorts 1 and 2, could represent an efficacious range..

初步PK分析表明，剂量水平为0.8-1.6 mg/kg（即第1组和第2组接受的剂量）可能代表一个有效的范围。

'SRC approval to open the fourth cohort and expand enrollment in Cohort 3 is an important advancement for the clinical trial. It will provide an opportunity to obtain additional safety and PK/PD data, inform the dose expansion stage of the clinical trial and may allow us to obtain initial evidence of clinical activity' commented .

“SRC批准开设第四批患者队列并扩大第三批患者的入组，是该临床试验的重要进展。这将提供一个机会，以获取更多的安全性和药代动力学/药效学数据，为临床试验的剂量扩展阶段提供信息，并可能让我们获得临床活性的初步证据。”

Sue Duggan

苏·杜根

, TransCode's Senior Vice President of Operations. Duggan added, 'Enrollment into the study continues based on the cumulative safety data review. Eligible subjects may now be screened and scheduled in Cohort 4 for treatment with the next dose level of TTX-MC138 while preliminary data analysis continues.'     .

，TransCode运营高级副总裁。Duggan补充道：‘基于累积的安全数据审查，研究的招募工作仍在继续。符合条件的受试者现在可以进行筛选并安排进入第4组，接受下一个剂量水平的TTX-MC138治疗，同时初步数据分析正在进行中。’

About TTX-MC138

关于TTX-MC138

TTX-MC138 is a first-in-class therapeutic candidate that targets microRNA-10b, a micro-RNA widely believed to be a driver of metastatic disease. TransCode's 2023 Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.  .

TTX-MC138 是一种首创的治疗候选药物，靶向微小RNA-10b，这种微小RNA被广泛认为是转移性疾病的驱动因素。TransCode公司在2023年进行的0期临床试验提供了将放射性标记版本的TTX-MC138递送至转移病灶并展现药效学活性的证据，甚至在该候选药物的微量剂量下也显示出效果，表明TTX-MC138具有广泛的治疗窗口。

About the Trial

关于试验

TransCode's Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study, designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers.  While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138.

TransCode的1期临床试验是一项多中心、开放标签、剂量递增和剂量扩展研究，旨在生成关键数据，以支持评估TTX-MC138在多种转移性实体瘤患者中的安全性和耐受性。尽管不是终点，但该试验可能提供TTX-MC138临床活性的早期证据。

The trial comprises an initial dose-escalation stage followed by a dose-expansion stage. The primary objective of the dose-escalation stage is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion stage, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types selected based on preliminary results from the dose-escalation stage..

该试验包括一个初始的剂量递增阶段，随后是剂量扩展阶段。剂量递增阶段的主要目的是评估TTX-MC138递增剂量水平的安全性和耐受性。在剂量扩展阶段，将根据剂量递增阶段的初步结果，进一步评估TTX-MC138在某些选定肿瘤类型中的安全性、耐受性和抗肿瘤活性。

Further information is available at

更多信息请访问

www.clinicaltrials.gov

www.clinicaltrials.gov

NCT Identifier: (NCT06260774).

NCT标识符：（NCT06260774）。

About TransCode Therapeutics

关于TransCode Therapeutics

TransCode is a clinical-stage oncology company focused on treating metastatic disease. The company is committed to defeating cancer through the intelligent design and effective delivery of RNA therapeutics based on its proprietary TTX nanoparticle platform. The company's lead therapeutic candidate, TTX-MC138, is focused on treating metastatic tumors which overexpress microRNA-10b, a unique, well-documented biomarker of metastasis.

TransCode是一家临床阶段的肿瘤学公司，专注于治疗转移性疾病。该公司致力于通过其专有的TTX纳米粒子平台，智能设计和有效递送RNA疗法来战胜癌症。该公司的主要治疗候选药物TTX-MC138专注于治疗过表达microRNA-10b的转移性肿瘤，microRNA-10b是一种独特且有充分文献记录的转移生物标志物。

In addition, TransCode has a portfolio of other first-in-class RNA therapeutic candidates designed to overcome the challenges of RNA delivery and thus unlock therapeutic access to a variety of novel genetic targets that could be relevant to treating a variety of cancers. .

此外，TransCode 还拥有其他一系列首创的 RNA 治疗候选药物，旨在克服 RNA 递送的挑战，从而解锁对多种新型遗传靶点的治疗通道，这些靶点可能与治疗多种癌症相关。

Forward-Looking Statements

前瞻性声明

This release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995, including, without limitation, statements concerning the timing, conduct and results of the Phase 1 clinical trial, statements about microRNAs and their involvement in cancer, and statements concerning the therapeutic potential of TransCode's TTX-MC138.

本发布包含1995年《私人证券诉讼改革法案》所指的“前瞻性声明”，包括但不限于关于第一阶段临床试验的时间、实施和结果的声明，关于微小RNA及其在癌症中的作用的声明，以及关于TransCode的TTX-MC138治疗潜力的声明。

Any forward-looking statements in this press release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements.

本新闻稿中的任何前瞻性陈述均基于管理层对未来事件的当前预期，并受多种风险和不确定因素的影响，这些风险和不确定性可能导致实际结果与这些前瞻性陈述中或暗示的结果存在重大且不利的差异。

These risks and uncertainties include, but are not limited to: the risks associated with drug discovery and development; the risk that the results of clinical trials will not be consistent with TransCode's pre-clinical studies or expectations or with results from previous clinical trials; risks associated with the conduct of clinical trials; risks associated with TransCode's financial condition and its need to obtain additional funding to support its business activities, including TransCode's ability to continue as a going concern; risks associated with the timing and outcome of TransCode's planned regulatory submissions; risks associated with obtaining, maintaining and protecting intellectual property; risks associated with TransCode's ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties; risks of competition from other companies developing products for similar uses; risks associated with TransCode's dependence on third parties; and risks associated with geopolitical events and pandemics, including the COVID-19 coronavirus an.

这些风险和不确定性包括但不限于：与药物发现和开发相关的风险；临床试验结果与TransCode的临床前研究、预期或以往临床试验结果不一致的风险；与临床试验实施相关的风险；与TransCode财务状况及其需要获取额外资金以支持其业务活动相关的风险，包括TransCode能否持续经营的风险；与TransCode计划中的监管提交的时间和结果相关的风险；与获取、维护和保护知识产权相关的风险；与TransCode针对侵权者执行专利及捍卫其专利组合免受第三方挑战的能力相关的风险；来自其他开发类似用途产品的公司竞争的风险；与TransCode对第三方依赖相关的风险；以及与地缘政治事件和疫情（包括COVID-19冠状病毒）相关的风险。

December 31, 2023

2023年12月31日

, as well as discussions of potential risks, uncertainties and other important factors in any subsequent TransCode filings with the Securities and Exchange Commission. All information in this press release is as of the date of this release; TransCode undertakes no duty to update this information unless required by law. .

以及在TransCode随后向证券交易委员会提交的文件中讨论的潜在风险、不确定性和其他重要因素。本新闻稿中的所有信息均为本发布日期的信息；除非法律要求，TransCode不承担更新此信息的责任。

SOURCE TransCode Therapeutics, Inc.

源代码：TransCode Therapeutics, Inc.

WANT YOUR COMPANY'S NEWS

想要你公司的新闻吗

FEATURED ON PRNEWSWIRE.COM?

刊登在PRNEWSWIRE.COM上？

440k+

44万+

Newsrooms &

新闻编辑室 &

Influencers

影响者

9k+

9k+

Digital Media

数字媒体

Outlets

插座

270k+

27万+

Journalists

记者

Opted In

已选择加入

GET STARTED

开始使用