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#临床/研发

今日 129 条
9月26日周六
  1. The Lancet8

    《The Lancet》更正 TALENTOP 研究图 3 的 p 值

    《The Lancet》对 TALENTOP 研究论文发布更正,图 3 中的 p 值应为 0·23。该研究由 Sun H-C 等人开展,比较阿替利珠单抗联合贝伐珠单抗后肝切除与维持治疗用于局部晚期肝细胞癌的疗效,为多中心、开放标签、随机、3 期试验,论文发表于 Lancet 2026; 408: 699–710。在线版本已于 2026 年 9 月 24 日完成更正。

9月25日周五
  1. Albert Bourla55

    辉瑞在研三特异性抗体tilrekimig的III期项目已完成首例患者给药,该分子由辉瑞实验室发现,距今年早些时候公布的积极II期结果仅数月。这三项研究是辉瑞今年多项关键性启动的一部分,另有一项针对COPD的II/III期研究正在进行中。

    推荐理由:辉瑞三特异性抗体tilrekimig在II期积极数据后数月即启动III期,可观察其研发推进节奏。

  2. Nature 药物发现综述0

    从 N-of-1 治疗到卫生系统实施

    原文正文未能加载,仅可确认这是一篇发表于 Nature 药物发现综述、题为《From N-of-1 therapy to health-system implementation》的文章,讨论从 N-of-1 治疗走向卫生系统实施。具体内容无法核实,暂不提供更多细节。

  3. Matthew Herper17

    好帖。 @antonioregalado: Anthropic 让 950 个智能体分析细菌 DNA 数据,发现了一种重复字母的模式,他们称这些重复序列与 CRISPR 中最后一个 R 所指的"重复序列"相似,而多年前对这些重复序列的检测最终促成了基因编辑工具的开发。 https://www-cdn.anthropic.com/22573675ada52a8ca8a97a1a4b4326b2f208a071.pdf

    引用Antonio Regalado@antonioregalado

    Anthropic set 950 agents loose on bacterial DNA data and found a pattern of repetitive letters, which they say bear similarity to the "repeats" that are the last R in CRISPR and whose detection years ago eventually led to development of the gene-edit tool. https://www-cdn.anthropic.com/22573675ada52a8ca8a97a1a4b4326b2f208a071.pdf

9月24日周四
  1. Adam Feuerstein ✡️58

    Acadia Pharmaceuticals 公布 remlifanserin 治疗阿尔茨海默病精神病(ADP)的 II 期 RADIANT 研究顶线结果,数据被 STAT 记者 Adam Feuerstein 形容为杂乱。60 mg 高剂量组在症状减轻上呈趋势,但未达到统计学显著(p=0.06)。公司仅以该高剂量继续推进两项 III 期研究。

    推荐理由:原文给出 remlifanserin 在阿尔茨海默病精神病 II 期 RADIANT 研究的关键数据与后续 III 期剂量选择,可据此了解该项目的推进依据。

  2. MedCity News71

    罗氏sefaxersen治疗IgA肾病III期中期分析显示尿蛋白显著下降

    罗氏公布其IgA肾病(IgAN)在研药物sefaxersen的III期试验预设中期分析结果,显示尿蛋白水平下降具有统计学显著性和临床意义,详细数据将在学术会议公布并提交监管机构。

    推荐理由:罗氏sefaxersen的III期中期分析显示尿蛋白下降,读者可借此了解IgAN在研ASO与现有补体抑制剂的差异。

9月23日周三
  1. Matthew Herper62

    Matthew Herper 在 X 上实时记录 FDA 顾问委员会对 Grail($GRAL)Galleri 多癌种血液检测的讨论。会上目前仅有公司方陈述,Grail 强调该检测能发现更多癌症并可定位,但未涉及对患者获益的更大问题;多位委员提出质疑,包括这种对医生的服务能否规模化,以及“有治愈意图但缺乏结局数据”。有委员追问为何 Grail 英国随机临床试验的对照组和主要终点未纳入简报文件,Grail 与 FDA 称这是双方协议所致;另有委员追问为何 Grail 另一项大型研究的许多患者未被纳入,Grail 称与数据提交时间有关。FDA 官员表示随访和结局数据超出本次所审评声称的范围。

    引用Matthew Herper@matthewherper

    I’m following the FDA advisory committee on $GRAL’s Galleri blood test for detecting multiple types of cancer today and will be posting my thoughts here. So far, we’ve seen only company presentations, usually the dullest part of an advisory panel. But there have been some interesting tidbits. First, Grail is leaning heavily into simply the idea that they detect more cancers and can localize them, not getting into larger questions about the benefit to patients from doing so. See this slide. Speaking as part of Grail’s presentation, Mylynda Massart, a family medicine at UPMC, argued that the test can fit will with existing cancer screening and is something that many patients are eager to use. The panelists, in their first questions, expressed skepticism. One asked if the incredible, white-glove treatment that Grail gives doctors now can really scale. Another responded a Grail comment: “You’re carefully choosing your words. It is curative intent but you do not have outcome data.” Another panelist asked why the control arm of Grail’s randomized clinical trial in the UK was not included in the briefing document; the primary outcome of that trial was not included, either. Grail and FDA said that based on the agreement they came to with the FDA. Another panelist asked why so many patients from Grail’s other big study were not included in the briefing material; Grail said that had to do with when the data were submitted. What’s interesting is that the FDA seems to be telling the panel to limit the scope of its discussion. An FDA official said that both follow-ups and outcome data were beyond the scope of the claim being considered.

    推荐理由:FDA 顾问委员会对 Galleri 多癌种血液检测的讨论,呈现审评中对结局数据与检测获益的分歧。

  2. Matthew Herper62

    记者 Matthew Herper 在 X 上实时记录 FDA 专家委员会对 Grail($GRAL)Galleri 多癌种血液检测的审议。目前仅公司方陈述,Grail 强调检测能发现更多癌症并定位,未深入讨论患者获益;UPMC 家庭医学医生 Mylynda Massart 称该检测可融入现有癌症筛查。

    推荐理由:记录 FDA 专家委员会对 Galleri 多癌种早筛检测的现场质疑,呈现审评焦点与证据缺口。