Eli Lilly & Co.

Alzheimer’s disease has afflicted Hannah Richardson’s family for generations. A rare genetic mutation, passed down from her great-grandmother, has struck her relatives with the first signs of memory loss at an average age of 39. Last year, her uncle died with dementia at 42. Her mother, now the same age, is in the moderate stages of the disease. Richardson, 24, has joined a clinical trial hoping to avoid the same fate. She will be one of the youngest people ever to receive an experimental drug intended to prevent dementia-linked amyloid plaques from building up in the first place. “I knew immediately I wanted to be a part of it,” she told Endpoints News in an interview. The study, helmed by Washington University School of Medicine in St. Louis, will enroll people who carry genes for inherited forms of Alzheimer’s, but who aren’t expected to develop their first symptoms for another 11 to 25 years. “Amyloid pathology can be identified one to two decades before people develop symptoms,” Eric McDade, a professor of neurology who is leading the study, told Endpoints. “What we’re trying to do is actually show that we can prevent that amyloid pathology from developing.” They’ll use an experimental Eli Lilly drug called remternetug. It is similar to the Indianapolis drugmaker’s drug Kisunla, which was approved last summer, but is administered as a quarterly injection instead of a monthly infusion. The trial is expected to last at least six years and cost more than $130 million. Most of the funding, an estimated $98.3 million, will come from the National Institutes of Health. If successful, it could provide one of the most dramatic tests yet of the much-debated amyloid hypothesis. “This is really going to be a landmark study,” Howard Fillit, chief science officer of the Alzheimer’s Drug Discovery Foundation, told Endpoints. “I believe it is the earliest treatment trial that has ever been conducted.” For years, drugmakers failed to make a dent in Alzheimer’s, either because they went after the wrong form of amyloid or tested the drugs in patients too deep in the throes of the disease. Two approved drugs, Eisai and Biogen’s Leqembi and Lilly’s Kisunla, are marketed for mild cognitive impairment, the earliest symptomatic stage of Alzheimer’s where brainpower noticeably begins to fade. But the effects are modest, and the companies have now started identifying patients with blood tests and brain scans, but no outward signs of memory loss, to start treatment earlier. Those tests are now underway. Results from Lilly’s trial could come as soon as 2027, and Eisai’s study is scheduled to start wrapping up in 2028. But the WashU Medicine study aims to treat people even earlier, before there’s almost any progression of the disease in the brain. It’s a bet that they might be able to prevent, not just slow, the disease. “Most of the people that will start this study won’t have any amyloid in their brain that we can detect,” McDade said. “We’re calling it primary prevention.” Fillit compared the approach to using statins in young people with high cholesterol before they develop coronary artery disease. “Except these are people that don’t even have the biomarker evidence,” he added. Last week, Richardson went in to get her blood and spinal fluid collected and her brain scanned. The tests will provide a baseline for doctors to track her progress in the years to come. “I’ve been aware of Alzheimer’s in my family since a young age, so I’ve had a long time to kind of sit with that,” Richardson said. “But now, being the one going through the tests and the imaging and receiving the first dose of the medication in a few weeks, it definitely is eye-opening.” The study is part of the Dominantly Inherited Alzheimer Network Trials Unit, or DIAN-TU, a closely-watched effort at WashU Medicine to investigate drugs in people with rare mutations who are almost guaranteed to develop the disease. Richardson’s mom and uncle participated in another DIAN-TU study when they were in their early 30s. Most people in the study already had amyloid plaques by the time they got an experimental Roche drug called gantenerumab. Although the drug seemed to lower amyloid, it didn’t slow cognitive decline. McDade, the trial leader, originally planned to use gantenerumab again in a primary prevention study, but after it failed in Roche’s own trials in 2022, he looked for another option. He landed on remternetug, which seemed like a simpler alternative to Leqembi, infused every two weeks, and Kisunla, infused every month. “If we really, truly have to treat people for a decade or more, you certainly don’t want them coming in for infusions every two weeks,” McDade said. Richardson and about 240 other participants in the study will get an injection of remternetug or a placebo every three months for two years. “What we can’t do, kind of ethically and realistically, is run a placebo-controlled trial for 20 years,” McDade said. As a comparison, the study will include family members who don’t carry the Alzheimer’s risk genes. Measuring amyloid on brain scans is the primary outcome. After the initial two years, people with the mutations can continue to get injections of the drug in an open-label extension for another four years. Beyond that, McDade expects that people will need less frequent dosing, but the details of how often that will be are to be determined. Richardson is clear-eyed about her family’s medical history. “I hope there is benefit,” she said. “But even if that is not the outcome, just collecting this data is still so important for helping people down the line not suffer the way that people are suffering now, and that’s really important to me.”

注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。 2025年1月10日，临床阶段生物技术公司Mediar Therapeutics（以下简称“Mediar”），宣布与全球制药巨头礼来（Eli Lilly and Company）达成全球许可协议，双方将MTX-463携手推进至针对特发性肺纤维化（IPF）的II期临床试验。MTX-463是一种首创的人源化IgG1抗体，旨在中和WISP1介导的纤维化信号通路，用于治疗多种致残性疾病。 I期研究近期已经在健康受试志愿者中完成，显示MTX-463在所有给药剂量下有良好的耐受性，并成功与WISP1结合。该IPF的II期研究旨在评估患者的安全性、药代动力学及疗效，计划于2025年上半年由Mediar负责开展。在II期临床研究完成后，礼来将有权主导该项目的后续临床试验开发及商业化工作。 药融云数据，www.pharnexcloud.com；改名后为摩熵医药数据 根据本次BD&L协议条款，Mediar将获得总计9900万美元的款项，包括首付款和近期里程碑付款(upfont+near-term)。Mediar还可获得高达6.87亿美元的潜在注册开发和商业化里程碑付款。此外，Mediar有资格根据未来可能的产品销售获得从高个位数到低双位数的特许权使用费以及净销售里程碑付款。本次交易合计最多达7.86亿美元。 MTX-463是一种针对WNT1诱导信号通路蛋白1（WISP1）开发的全球首创人源化IgG1抗体。WISP1是一种分泌型细胞外基质蛋白，与纤维化进展密切相关，可通过人类血液测量，其水平与疾病严重程度相关。初步数据显示，MTX-463可中和WISP1介导的多种纤维化信号，在体外实验和小鼠模型中显著减少纤维化。Mediar计划于2025年上半年启动MTX-463治疗IPF的II期研究。 药融圈获悉：除了上述全球领先的生物医药企业和相关基金（礼来制药，辉瑞风险基金，百时美施贵宝，小野制药风险投资等），来自中国的维亚生物对其也做过早期投资。 参考： NMPA/CDE； 药融云数据，www.pharnexcloud.com；改名后为摩熵医药数据； FDA/EMA/PMDA； 相关公司公开披露； https://www.mediartx.com/；https://www.mediartx.com/wp-content/uploads/2025/01/MediarTx-PRESS-RELEASE_1-10-25.pdf；等等。 此文仅用于向医疗卫生专业人士提供科学信息，不代表平台立场，不作任何用药推荐。更多药企信息交流可后台留下名片。

Plus, news about Immunome, 89bio and Valneva: Tectonic’s $185M private placement: After a hectic week of stock swings, Tectonic Therapeutic disclosed plans for a large private placement. The Boston-area biotech will raise the money from Adage Capital Partners, Ally Bridge Group, Deep Track Capital, EcoR1 Capital and others. Last week, Eli Lilly’s decision to terminate a mid-stage heart failure program tanked Tectonic’s stock $TECX . The company is working on a similar relaxin program. Days later, the biotech released early-stage data for that drug that led to a major rebound in its share price. Its stock is up 12% over the last month. — Kyle LaHucik Acelyrin returns rights to drug: After ending development of izokibep in November, the Los Angeles biotech is officially handing back the rights to the dermatology drug candidate to Affibody. “It is clear that Acelyrin has not been able to fully capitalize on the potential demonstrated by izokibep’s best-in-class Phase 3 data,” Affibody CEO David Bejker said in a statement . Acelyrin’s stock price $SLRN is down 73% over the past 12 months. — Kyle LaHucik Immunome upsized its public offering, bringing in $172.5 million. — Jaimy Lee 89bio raised a total of $287.5 million in its public offering. — Jaimy Lee Valneva’s contract with the DoD: The French drugmaker said last week it will now supply at least $32.8 million of Ixiaro, its Japanese encephalitis vaccine, to the US. — Jaimy Lee