Dive Brief:

简报：

San Diego-based Protego Biopharma has raised $130 million in a Series B round that will propel an experimental drug for amyloid light-chain, or AL, amyloidosis into late-stage clinical testing, the company said Monday.

总部位于圣地亚哥的Protego生物制药公司在周一表示，该公司在B轮融资中筹集了1.3亿美元，这将推动一种用于治疗轻链型或AL型淀粉样变性的实验性药物进入晚期临床试验。

The drug, called PROT-001, is designed to bind to and stabilize a protein that misfolds and toxically accumulates in people with AL amyloidosis. That effect is believed to impact disease progression, rather than only symptoms, though it hasn’t yet been proven in testing. An early-stage study

这种名为PROT-001的药物旨在结合并稳定一种在AL型淀粉样变性患者体内错误折叠并有毒性积累的蛋白质。 这种作用被认为会影响疾病进展，而不仅仅是症状，尽管尚未在测试中得到证实。 一项早期研究

began in the second quarter,

开始于第二季度，

with results expected next year. A Phase 2/3 trial should start in the second half of 2026.

预计明年会出结果。二期/三期试验应该会在2026年下半年开始。

Protego claimed in a statement that the approach may also be applicable to other diseases characterized by protein misfolding. Its Series B round was led by Novartis Venture Fund and Forbion, with additional backing from firms such as Omega Funds, Vida Ventures and MPM BioImpact.

Protego在一份声明中称，这种方法也可能适用于其他以蛋白质错误折叠为特征的疾病。其B轮融资由诺华风险基金和Forbion领投，Omega Funds、Vida Ventures和MPM BioImpact等公司也提供了额外支持。

Dive Insight:

深入洞察：

In AL amyloidosis, plasma cells errantly make abnormal antibody parts known as “light chains,” which then misfold, clump up in tissues and damage organs. While there are certain options available, like a stem cell transplant, or a cocktail of cancer chemotherapies and the drug Darzalex, they each have limitations..

在AL型淀粉样变性中，浆细胞错误地产生称为“轻链”的异常抗体部分，这些轻链随后发生错误折叠，在组织中聚集并损害器官。虽然有一些可用的治疗选项，如干细胞移植、癌症化疗药物组合以及药物Darzalex（达拉图单抗），但它们各自都存在局限性。

Bringing new prospects to market has proven frustrating for some of Protego’s peers, however. In May, disappointing study results led

然而，对于 Protego 的一些同行来说，将新前景推向市场一直令人沮丧。今年五月，令人失望的研究结果导致

Prothena to shelve a therapy

Prothena将搁置一种疗法

it had long been working on. Two months later, AstraZeneca said a drug it had acquired in a 2021 buyout

它一直在研发。两个月后，阿斯利康表示，其在 2021 年收购中获得的一种药物

fell short in a late-stage study, too

在晚期研究中也未达到预期效果

.

。

Brent Warner is the CEO of Protego Biopharma, which is developing a drug for light chain amyloidosis.

布伦特·华纳是Protego生物制药公司的首席执行官，该公司正在开发一种用于轻链淀粉样变性的药物。

Permission granted by Protego Biopharma

普罗特戈生物制药公司授权许可

Both of those drugs are designed to clear toxic deposits of amyloid from the body. Protego believes it’ll have better luck by intervening beforehand and preventing them from accumulating in the first place. Those failures “give us a lot of conviction on our approach,” said CEO Brent Warner.

这两种药物都旨在清除体内的淀粉样蛋白毒性沉积。普罗特戈公司认为，通过提前干预，防止这些沉积物首先积累起来，会取得更好的效果。首席执行官布伦特·华纳表示，那些失败“让我们对自己的方法充满信心”。

“There is very clear data,” he added, that “toxic light chain is really what's driving a lot of the mortality in this disease.”

“有非常明确的数据，”他补充说，“毒性轻链确实是导致该疾病大量死亡的原因。”

Protego’s work is built on the research of

Protego的工作建立在以下研究的基础上

Jeffery Kelly

杰弗里·凯利

, one of Protego’s co-founders and a Scripps Research professor. Kelly, an expert in protein misfolding, was a cofounder of FoldRx, which discovered the protein stabilizing drug now known as

，Protego的联合创始人之一，也是Scripps研究所的教授。凯利是蛋白质错误折叠方面的专家，曾共同创立FoldRx，该公司发现了现在被称为蛋白质稳定药物

Vyndamax

维恩达麦克斯

. That treatment was the first medicine in the U.S. approved for a form of the protein misfolding disease transthyretin amyloidosis that affects the heart. It’s now owned by Pfizer and generates billions of dollars in sales each year.

。该药物是美国首个获批用于治疗影响心脏的蛋白质错误折叠疾病——转甲状腺素蛋白淀粉样变性的一种药物。它现在归辉瑞公司所有，每年销售额达数十亿美元。

Protego hopes PROT-001 can prove to be a similar leap forward. The goal of the commonly used Darzalex regimen is to essentially wipe out the plasma cells making misfolded proteins. Patients relapse, though, because some of those cells return. Adding a protein stabilizer to the mix could prompt a “very drastic, fast and deeper response,” Warner said..

Protego希望PROT-001能够证明是一个类似的飞跃。常用Darzalex方案的目标是基本上清除制造错误折叠蛋白的浆细胞。然而，患者会复发，因为其中一些细胞又回来了。Warner表示，添加蛋白质稳定剂可能会促使“非常剧烈、快速且更深层次的反应”。

“In a disease like this, you may not necessarily need a sledgehammer, you just may need a sharper knife to cut it,” Warner said.

“在这种疾病中，你不一定需要一把大锤，你可能只需要一把更锋利的刀来切割它，”华纳说。