Memo

全文共2385字，共1图预计阅读时间：6分钟在新药研发的漫漫征途中，临床试验是至关重要的关卡。每一期临床试验都承载着对药物安全性和有效性的严格检验，而能否顺利通过这些关卡，往往决定了药物能否走向市场、造福患者。近期，瑞士新药研发公司Memo Therapeutics在其肾脏移植药物potravitug的2期临床试验中未能达到主要终点，却仍计划推进至3期，这一决策引发了广泛的关注与讨论。Memo的2期试验：希望与挑战并存Memo的2期试验原本寄予厚望，旨在验证potravitug对抗BK多瘤病毒在肾脏移植受者中的疗效。然而，结果显示，在第20周时，治疗组与安慰剂组相比，在血液中病毒水平无法检测到的比例上并无统计学上的显著差异，这意味着试验的主要目标未能达成。这无疑是给Memo的研发团队当头一棒，因为在新药研发领域，主要终点的达成与否常常被视为药物是否有效的关键指标。但事情并非全无转机。Memo在其他指标上看到了希望的曙光。治疗组在病毒反应上表现出显著的改善，活检证实的BK多瘤病毒肾病（BKPyVAN）得到了一定程度的解决，其比例从基线时的51.2%下降到第20周的31.6%，而安慰剂组则毫无变化。这一结果表明，potravitug在组织学层面上对BKPyVAN有着积极的影响，似乎暗示着药物对潜在疾病的缓解作用。此外，Memo还报告称未出现与治疗相关的严重不良事件，也未因不良事件导致退出，这在一定程度上证明了药物的安全性。然而，这是否足以支撑Memo推进至3期的决策呢？我们不妨从多个角度来剖析。2期折戟，3期之路如何走？首先，从药物研发的科学逻辑来看，2期临床试验的主要终点是经过精心设计和论证的，它通常反映了药物在关键疗效指标上的表现。未能达到主要终点，可能意味着药物在当前的剂量、给药方案或患者群体中，未能充分展现出预期的疗效。尽管其他指标有所改善，但这些指标是否能够真正代表药物的临床价值，还需要更多的证据来支持。例如，病毒水平的降低虽然在一定程度上反映了药物的抗病毒活性，但能否转化为患者的长期临床获益，如降低移植肾失功的风险、提高患者的生存率等，仍是一个未知数。其次，从资源投入和风险收益的角度来看，推进至3期临床试验意味着需要投入大量的资金、人力和时间。对于Memo这样的生物技术公司而言，这是一笔不小的开支。如果最终3期试验仍未能取得令人满意的结果，那么这些投入可能会付诸东流。而且，目前市场上尚无针对BK多瘤病毒的特效药物获批，虽然这为potravitug提供了潜在的市场机会，但竞争对手也并非毫无动作。Vera Therapeutics的MAU868虽然在2期试验后进展停滞，但未来仍有可能卷土重来；AlloVir的posoleucel也曾有过一定的研发进展，尽管其3期试验失败，但技术积累和经验教训也可能为未来的研发提供借鉴。Memo在推进3期试验的同时，需要面对来自同行的竞争压力，以及市场对新药的期待和不确定性。再者，从监管机构的角度来看，FDA对新药的审批有着严格的标准和流程。尽管potravitug已获得快速通道指定，但这并不意味着3期试验的结果就能得到保证。监管机构在评估药物的有效性和安全性时，会综合考虑多个方面的因素，而不仅仅是单一的指标。Memo需要在3期试验中提供更加充分、有力的证据，来证明potravitug相较于现有治疗方案的优势和价值，否则很难获得批准上市。Memo的3期之路：失败中寻找合理性那么，Memo 推进至3期的决策是否就完全没有合理性呢？也未必。从积极的方面来看，2期试验的其他数据确实为药物的进一步开发提供了一定的依据。在新药研发过程中，有时候即使未能达到主要终点，但其他指标的改善也可能提示药物在某些方面具有潜在的疗效，值得进一步探索。而且，Memo可能也在试验过程中积累了宝贵的经验，对药物的作用机制、患者群体的特征等有了更深入的理解，这些都可能为3期试验的设计和实施提供指导。此外，与监管机构的沟通也至关重要。如果Memo能够在与FDA的交流中，充分阐述药物的潜力和价值，争取到监管机构的支持和认可，那么推进至3期试验或许还有一线生机。结 语Memo在potravitug的2期临床试验中未能达到主要终点，却仍计划推进至3期，这一决策无疑充满了风险与挑战。从科学逻辑、资源投入、监管审批等多方面来看，这一选择都需要谨慎权衡。但同时，我们也应该看到，药物研发本身就是一个充满不确定性和探索性的过程。Memo的决策或许是在综合考虑了各种因素后，基于对药物潜力的信心和对患者需求的重视所做出的尝试。无论最终结果如何，这一过程都将为整个行业提供宝贵的经验和教训。对于Memo而言，未来的路虽然艰难，但也充满了希望。我们期待在3期试验中能够看到更加令人振奋的结果，为肾脏移植患者带来新的治疗选择。药时代朋友们，您怎么看这家biotech的计划？如果您的公司遇到这种情况，会做出怎样的决定呢？参考资料：1. Memo pushes kidney drug to phase 3 despite primary endpoint miss3. 各公司官网4. 其它公开资料图片来源：豆包AI8.56亿美元！礼来抢占 “分子门”2025-07-26市值130亿港元！创始人敲响了中国TCE的第一股2025-07-252025 WCLC：复宏汉霖3项口头报告，10项肺癌研究入选，多核心数据首次发布！2025-07-25科济炸场后，实体瘤CAR-T东风再起！2025-07-24版权声明/免责声明本文为原创文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢!药时代官方网站:www.drugtimes.cn联系方式:电话:13651980212微信:27674131邮箱:contact@drugtimes.cn点击这里，查看更多精彩！

Memo Therapeutics will share more phase 2 data at the World Transplant Congress next month. \n A phase 2 trial of Memo Therapeutics’ kidney transplant drug candidate has missed its primary endpoint, adding to the list of setbacks in the indication. But the Swiss biotech saw promise in other aspects of the data set and outlined plans to move into phase 3.The phase 2 trial tested Memo’s anti-BK polyomavirus antibody potravitug. Investigators randomized 95 kidney transplant recipients infected with the virus to receive potravitug or placebo. At Week 20, the proportion of patients with undetectable levels of the virus in the blood was statistically no higher in the treatment arm than in the control group, causing the trial to miss its primary endpoint.However, Memo pointed to success against other measures to make the case for further development of the antibody. The treatment group had significantly higher viral response with resolution of biopsy-proven BK polyomavirus nephropathy (BKPyVAN). “This histological improvement in BKPyVAN indicated resolution of the underlying disease, decreasing from 51.2% at baseline to 31.6% at Week 20 in the treatment group, while no change was observed in the placebo group,” Memo said in a statement. Memo reported no treatment-related serious adverse events or withdrawals due to adverse events. The biotech will share more phase 2 data at the World Transplant Congress next month. In parallel, Memo is preparing to talk to regulators later this year about the design of a planned phase 3 program. Potravitug has a fast-track designation from the FDA.No drugs against the virus are approved by the FDA. Vera Therapeutics has a rival candidate, MAU868, but progress stalled after it completed phase 2 back in 2022. The company is focused on bringing another asset, atacicept, to market. AlloVir was developing posoleucel for use against BK virus, wrapping up a phase 2 trial in 2022, but phase 3 failures led the company to enter into a reverse merger last year.

Memo Therapeutics said Friday it is planning to advance its monoclonal antibody for kidney transplant patients with a viral infection into Phase 3 even though the drug didn’t pass a mid-stage test. The candidate, known as potravitug, did not reach statistical significance in the primary endpoint of a Phase 2 study called SAFE KIDNEY. The endpoint looked at the proportion of patients with undetectable levels of an infection marker called BKV DNAemia at around 20 weeks. This marker measures the amount of BK virus DNA present in the bloodstream. Achieving undetectable levels is “a pretty high bar to reach,” Memo CEO Erik van den Berg told Endpoints News in an interview. “We didn’t meet [the endpoint] because I think it’s just not in the end obtainable by any drug.” The company added in an email that it is “notoriously difficult” to pick the right primary endpoint for rare disease trials where little is known about disease pathology. Despite the primary endpoint miss, patients treated with potravitug had “significantly higher” viral response with resolution of disease-related nephropathy (a type of kidney damage) as confirmed by biopsy. In this so-called “predefined endpoint,” the proportion of treated patients with nephropathy declined from 51.2% at baseline to 31.6% at 20 weeks. There was no reduction for placebo. This result is of “huge importance” and “has not been seen before with any other regimen,” van den Berg said. Human polyomavirus 1, also known as the BK virus, infects most people during childhood and remains dormant in the kidneys. But up to half of the more than 100,000 people who get kidney transplants each year can develop a reactivated infection, according to Memo. This can lead to organ damage and nephropathy. SAFE KIDNEY enrolled 95 people who had received a kidney transplant and subsequently developed a reactivated infection. Memo plans to discuss the design of a Phase 3 trial of potravitug with regulators later this year, with an eye to starting the study in 2026. It will also present detailed results from SAFE KIDNEY at the World Transplant Congress in San Francisco next month. Standard of care for BK polyomavirus involves tapering down immunosuppression, allowing the immune system to fight off the infection. But that raises the risk of the patient’s body rejecting the donor kidney, so there’s a “fine balancing act” between treating the infection and saving the organ, van den Berg said. There are no antivirals specifically for the infection. Antivirals for other indications are sometimes used off-label, but they have questionable efficacy and safety risks, van den Berg said.