Daratumumab/Hyaluronidase-fihj

昨天，临床阶段生物技术公司Protego Biopharma宣布完成超额认购的1.3亿美元B轮融资。本轮融资由诺华风险基金和Forbion联合领投，新加入的投资方包括Omega Funds、Droia Ventures、YK Bioventures及Digitalis Ventures，Vida Ventures、MPM BioImpact、Lightspeed Venture Partners等现有投资方亦积极跟投。募集资金将重点用于推动其主导候选药物PROT-001进入AL淀粉样变性的关键临床试验阶段。 值得关注的是，Protego Biopharma的创始团队堪称“传奇班底”——正是这支团队一手打造了辉瑞重磅心肌病药物Vyndaqel（维万心，通用名氯苯唑酸）。该公司成立于2017年，由Scripps研究所教授Jeffery W. Kelly博士、Xing Jiang博士和Richard Labaudinière博士于2017年共同创立的。其中Jeffery Kelly与Richard Labaudinière是Vyndaqel的核心研发者。 作为改变转甲状腺素蛋白淀粉样变性治疗格局的重磅药物，Vyndaqel如今已成为辉瑞的核心营收支柱之一。据辉瑞2024年财报显示，Vyndaqel系列产品（含Vyndaqel、Vyndamax等）销售额高达54.51亿美元，同比增长64%。 此次，他们将研发重心投向了另一种致命罕见病——AL型淀粉样变性（AL Amyloidosis）。该病由错误折叠的免疫球蛋白轻链在心脏、肾脏等器官中异常积聚所致，可引发心力衰竭、器官衰竭甚至死亡，临床预后极差，轻链型淀粉样变心肌病中位生存期仅0.3年。目前市场上的标准疗法如强生的Darzalex Faspro（兆珂®，通用名：daratumumab，达雷妥尤单抗），核心机制是通过杀灭浆细胞减少异常蛋白产生，属于“源头减量”的对症治疗，无法从根本上解决蛋白质错误折叠问题。 Protego的治疗策略根植于人类遗传学和独特的药理学伴侣机制。这些小分子作为细胞“导引”，确保蛋白质正确折叠。通过稳定免疫球蛋白轻链并防止淀粉样蛋白堆积，PROT-001不仅仅管理症状，更关注疾病的根本原因。这一方法不仅代表AL淀粉样变性，也为多种严重未被满足的蛋白质错折叠疾病带来了潜在的范式转变。 PROT-001的I期首次人体试验已于2025年第二季度（5月）在澳大利亚启动，计划招募约122名健康成年受试者，主要评估药物在单次及多次递增剂量给药后的安全性、耐受性、药代动力学及药效学特征，核心研究结果预计于2026年初公布。根据规划，该药物的II/III期关键性临床试验将在2026年下半年正式启动。 “凭借此次募集的资金，我们已做好准备推进PROT-001进入关键性试验阶段，朝着推出首个AL型淀粉样变性疾病修饰疗法的目标迈进，为目前面临毁灭性后果的患者带来新的希望。”Protego相关负责人Warner表示。依托团队在小分子蛋白稳定剂领域的成熟技术路径与成功经验，PROT-001的研发进展备受行业关注，有望为蛋白质错误折叠疾病治疗领域开辟新的赛道。  推 荐 阅 读

A San Diego biotech hoping to crack into a rare protein misfolding disease has drummed up $130 million in investor support to get into a potential pivotal trial next year. Protego Biopharma, from the creators of Pfizer’s amyloidosis medicine Vyndaqel, has secured a Series B from lead investors Novartis Venture Fund and Forbion. Omega Funds, Droia Ventures, Vida Ventures, MPM BioImpact and others also chipped into the round, the company said on Monday morning. The biotech, founded in 2017 , is developing small molecules to reprogram protein folding. Its lead drug is for amyloid light chain amyloidosis, or AL amyloidosis. The rare disease is marked by a buildup of misfolded proteins in the heart and other organs. The toxic proteins can damage organs and lead to heart failure, sometimes resulting in death. Protego’s funding arrives just a few weeks after the FDA stamped full approval onto Johnson & Johnson’s Darzalex Faspro for the disease. The treatment received accelerated approval in January 2021 . Meanwhile, two high-profile clinical failures occurred earlier this year at Prothena and for AstraZeneca’s anselamimab , dealing a blow to the UK pharma’s $3 billion projections for the antibody. Protego’s candidate, nicknamed PROT-001, works to help proteins fold correctly, the company said. Essentially, Protego doesn’t want to address symptoms, but rather the main cause of the disease. “The standard of care is shutting down the plasma cells or trying to eradicate most of them. Having a light chain stabilizer at its source that can capture the availability of this misfolded light chain should lead to significant improvements in morbidity and mortality,” Protego CEO Brent Warner said in an interview with Endpoints News . PROT-001 is in a Phase 1 healthy volunteer trial in Australia, Warner said. The biotech anticipates collecting the traditional single-ascending and multiple-ascending dose data in the first half of 2026 and then filing for a Phase 2/3 trial later in the year, the CEO said. “Even with standard of care, patients who’ve been on it, even post-treatment, still have some of those plasma cells available that is continuously pumping out toxic light chain, and even a small amount of that toxic misfolded light chain is detrimental to cells,” Warner said. “Having a stabilizer that would be available to neutralize that toxicity should lead to no further formation of aggregates and allow those patients to get back into more of a native, natural state.” The biotech was co-founded by Scripps Research professor Jeffery Kelly, Xin Jiang and Richard Labaudinière. Kelly’s research previously led to the creation of FoldRx Pharmaceuticals, which was led by Labaudinière and bought by Pfizer in 2010 . FoldRx developed the drug tafamidis, or Vyndaqel. That treatment is approved for patients with transthyretin amyloid cardiomyopathy. Protego still has to meet with regulators to determine whether it could also get an accelerated approval like J&J did four years ago. “The regulatory environment is ever changing, particularly in rare disease,” Warner said. “Further discussions need to occur, which we anticipate having next year.” The startup has under 20 employees today and aims to have about 30 or so full-time staffers over the next year, Warner said.