STOCKHOLM, April 23, 2024 /PRNewswire/ --Medivir AB(NASDAQ: MVIR) (STO: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, announced today an update from a Type C meeting with the FDA, regarding the company's plans for a global phase 2b study and opening of an IND for fostroxacitabine bralpamide (fostrox) in the US..

斯德哥尔摩，2024年4月23日/PRNewswire/--Medivir AB（纳斯达克：MVIR）（STO：MVIR）是一家专注于在医疗需求未得到满足的领域开发创新癌症治疗方法的制药公司，今天宣布了与FDA举行的C型会议的最新消息，该公司计划在美国进行全球2b期研究，并在美国开设福斯特罗西他滨-布拉帕胺（fostroxacitabine-bralpamide，fostrox）的IND。。

The key outcome of the Type C meeting was that Medivir's preparations for the randomized phase 2b study, comparing fostrox + Lenvima® with Lenvima alone, continues with two key adjustments to the study design.

C型会议的关键成果是，Medivir为随机2b期研究进行的准备工作，将fostrox+Lenvima®与Lenvima单独进行比较，继续对研究设计进行两个关键调整。

Firstly, as Medivir plans to use the improved capsule formulation of fostrox in the upcoming phase 2b study, an initial dose run-in as the first part of the study will be added. This means including 20-25 additional patients in an arm with a lower dose, in addition to the 30 mg dose used in the currently ongoing phase 1b/2a study.

首先，由于Medivir计划在即将到来的2b期研究中使用改进的fostrox胶囊制剂，因此将添加初始剂量作为研究的第一部分。这意味着除了目前正在进行的1b/2a期研究中使用的30毫克剂量外，还包括20-25名额外的低剂量患者。

This change will also further strengthen the study design in relation to FDA's Project Optimus..

这一变化也将进一步加强与FDA Optimus项目相关的研究设计。。

Secondly, Objective Response Rate (ORR) is a validated surrogate endpoint for Overall Survival. In prior HCC studies, ORR has supported accelerated approvals. Therefore, the primary endpoint in the enhanced study design will be changed to ORR with key secondary endpoints, including duration of response, progression free survival and overall survival..

其次，客观缓解率（ORR）是总体生存的有效替代终点。在之前的HCC研究中，ORR支持加速批准。因此，增强研究设计的主要终点将改为ORR，并具有关键的次要终点，包括反应持续时间，无进展生存期和总生存期。。

The aim of the study, as well as size and study length, remain similar as previously planned and communicated phase 2b design.

该研究的目的以及规模和研究长度与之前计划和传达的2b期设计相似。

The FDA guidance has been discussed with Scientific Advisory Council members and regulatory experts. The next step is to finalize the study design and study protocol before submitting an IND in the US to enable study start in the beginning of 2025.

FDA指南已经与科学咨询委员会成员和监管专家进行了讨论。下一步是在美国提交IND之前完成研究设计和研究方案，以便在2025年初开始研究。

- 'With the outcome of the Type C meeting, we are confidently moving forward with the enhanced phase 2b study design, including the adjustments informed by the discussion with FDA. Our current phase 1b/2a study, in a similar patient population, is still ongoing and patients are staying on treatment longer than expected.

-“随着C型会议的结果，我们有信心推进增强的2b期研究设计，包括与FDA讨论后的调整。我们目前在类似患者人群中进行的1b/2a期研究仍在进行中，患者的治疗时间比预期的要长。

With the planned phase 2b study, we are excited to move forward with the development of a promising treatment alternative to second line HCC patients,' says Pia Baumann, CMO at Medivir..

Medivir首席医学官皮亚·鲍曼（PiaBaumann）说，随着计划中的2b期研究的进行，我们很高兴能够开发出一种有前途的二线肝癌患者替代治疗方案。。

- 'We continue to accelerate the development plan for fostrox. Engaging with FDA, to ensure best possible study design for the next phase, is critical for the program and is an important element in partnering discussions. Clarity on study design for phase 2b also allows us to move forward and perform study feasibility with speed as well as finalize CMC preparations,' says Jens Lindberg, CEO at Medivir..

-“我们继续加快fostrox的开发计划。与FDA合作，以确保下一阶段的最佳研究设计，对于该计划至关重要，也是合作讨论的重要因素。Medivir首席执行官延斯·林德伯格（Jens Lindberg）说，第二阶段研究设计的清晰性也使我们能够快速推进和执行研究可行性，并最终确定CMC的准备工作。。

Medivir will provide additional details regarding the updated study design at the Quarterly Results webcast on April 30.

Medivir将在4月30日的季度结果网络广播中提供有关更新研究设计的更多详细信息。

For additional information, please contact

有关更多信息，请联系

Magnus Christensen, CFO, Medivir AB

Magnus Christensen，Medivir AB首席财务官

Telephone: +46 8 5468 3100

电话：+46 8 5468 3100

E-mail: magnus.christensen@medivir.com

电子邮件：magnus.christensen@medivir.com

About fostrox

关于fostrox

Fostrox is a type of smart chemotherapy that delivers the cell-killing compound selectively to the tumor while minimizing the harmful effect on normal cells. This is achieved by coupling an active chemotherapy (troxacitabine) with a prodrug tail. The prodrug design enables fostrox to be administered orally and travel directly to the liver where the active substance is released locally in the liver.

Fostrox是一种智能化学疗法，可选择性地将细胞杀伤化合物递送至肿瘤，同时最大程度地减少对正常细胞的有害影响。这是通过将活性化疗（曲西他滨）与前药尾部偶联来实现的。前药设计使fostrox能够口服给药，并直接进入肝脏，活性物质在肝脏局部释放。

With this unique mechanism, fostrox has the potential to become the first liver-targeted, orally administered drug that can help patients with various types of liver cancer. A phase 1b monotherapy study with fostrox has been completed and a phase 1b/2a combination study in HCC is ongoing..

凭借这种独特的机制，fostrox有可能成为第一种针对肝脏的口服药物，可以帮助各种类型的肝癌患者。fostrox的1b期单药治疗研究已经完成，HCC的1b/2a期联合研究正在进行中。。

About primary liver cancer

关于原发性肝癌

Primary liver cancer is the third leading cause of cancer-related deaths worldwide and hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver. Although existing therapies for advanced HCC can extend the lives of patients, treatment benefits are insufficient and death rates remain high.

原发性肝癌是全球癌症相关死亡的第三大原因，肝细胞癌（HCC）是肝脏中最常见的癌症。虽然现有的晚期肝癌治疗方法可以延长患者的寿命，但治疗效果不足，死亡率仍然很高。

There are approximately 660,000 patients diagnosed with primary liver cancer per year globally and current five-year survival is less than 20 percent1). HCC is a heterogeneous disease with diverse etiologies, and lacks defining mutations observed in many other cancers. This has contributed to the lack of success of molecularly targeted agents in HCC.

全球每年约有66万名患者被诊断患有原发性肝癌，目前的五年生存率低于20%1）。HCC是一种病因多样的异质性疾病，缺乏在许多其他癌症中观察到的明确突变。这导致分子靶向药物在HCC中缺乏成功。

The limited overall benefit, taken together with the poor overall prognosis for patients with intermediate and advanced HCC, results in a large unmet medical need..

总体效益有限，加上中晚期HCC患者总体预后较差，导致大量医疗需求未得到满足。。

About Medivir

关于Medivir

Medivir develops innovative drugs with a focus on cancer where the unmet medical needs are high. The drug candidates are directed toward indication areas where available therapies are limited or missing and there are great opportunities to offer significant improvements to patients. Medivir is focusing on the development of fostroxacitabine bralpamide (fostrox), a smart, targeted chemotherapy designed to selectively treat liver cancer cells and to minimize side effects.

Medivir开发创新药物，重点关注未满足医疗需求的癌症。候选药物针对可用疗法有限或缺失的适应症领域，并且有很大的机会为患者提供显着改善。Medivir专注于开发fostroxacitabine bralpamide（fostrox），这是一种智能的靶向化疗，旨在选择性治疗肝癌细胞并尽量减少副作用。

Collaborations and partnerships are important parts of Medivir's business model, and the drug development is conducted either by Medivir or in partnership. Medivir's share (ticker: MVIR) is listed on Nasdaq Stockholm's Small Cap list. www.medivir.com..

合作和伙伴关系是Medivir商业模式的重要组成部分，药物开发由Medivir或合伙进行。Medivir的股票（股票代码：MVIR）在纳斯达克斯德哥尔摩的小盘股名单上上市。www.medivir.com。。

Rumgay et al.,European Journal of Cancer 2022 vol.161, 108-118.

Rumgay等人，《欧洲癌症杂志2022》，第161卷，108-118页。

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SOURCE Medivir

来源Medivir

Company Codes: Bloomberg:MVIR@SS, ISIN:SE0020181014, RICS:MVIR.ST, Stockholm:MVIR

公司代码：彭博社：MVIR@SS，ISIN:SE0020181014，RICS:MVIR。斯德哥尔摩ST：MVIR