Pictured: Illustration of red blood cells/iStock, ismagilov

图片：红细胞/iStock，ismagilov的插图

Protagonist Therapeutics on Wednesday announced that its investigational peptide therapy rusfertide met its primary efficacy endpoint in the Phase II REVIVE trial, demonstrating significant erythrocytosis control in patients with polycythemia vera.

Protation Therapeutics周三宣布，其研究性肽疗法rusfertide在II期REVIVE试验中达到了其主要疗效终点，证明真性红细胞增多症患者的红细胞增多得到了显着控制。

The results, published Thursday in The New England Journal of Medicine, showed that mean maximum hematocrit dropped to 44.5% after treatment with rusfertide, compared to 50% during the first 28 weeks of the study, prior to rusfertide administration.

星期四发表在《新英格兰医学杂志》上的研究结果显示，在使用rusfertide治疗后，平均最大血细胞比容降至44.5%，而在研究的前28周，在使用rusfertide之前，平均最大血细胞比容降至50%。

In addition, 60% of patients in the rusfertide arm showed treatment response, defined as hematocrit control below 45% without needing phlebotomy, versus 17% in the placebo group. This effect was statistically significant, with a p-value of 0.002, according to the NEJM paper. Rusfertide also lowered individual symptom scores in patients who presented with moderate or severe symptoms..

此外，60%的rusfertide组患者表现出治疗反应，定义为血细胞比容控制低于45%，无需静脉切开术，而安慰剂组为17%。根据NEJM的论文，这种效应具有统计学意义，p值为0.002。Rusfertide还降低了出现中度或重度症状的患者的个体症状评分。。

Protagonist CMO Arturo Molina in a statement said that REVIVE’s data could position rusfertide as a “novel treatment option” for a wide range of polycythemia vera patients, many of whom can poorly tolerate standard of care treatments.

主角CMO Arturo Molina在一份声明中表示，REVIVE的数据可能将rusfertide定位为一种“新型治疗选择”，适用于各种真性红细胞增多症患者，其中许多患者对标准治疗的耐受性较差。

The company will continue to assess rusfertide’s long-term safety and durability of response in REVIVE. Protagonist is also running the Phase III VERIFY study—a global placebo-controlled study with 250 patients enrolled—which is designed to test the efficacy, symptom burden and safety of a once-weekly subcutaneous, self-administered rusfertide injection.

该公司将继续评估rusfertide在REVIVE中的长期安全性和反应持久性。PROTAGITOR还正在进行III期VERIFY研究，这是一项全球安慰剂对照研究，共有250名患者参加，旨在测试每周一次皮下自我给药的rusfertide注射液的疗效，症状负担和安全性。

Data from VERIFY are expected in 2025, according to Molina..

根据Molina的数据，VERIFY的数据预计将在2025年发布。。

Affecting approximately 160,000 patients in the U.S., polycythemia vera—a type of blood cancer—is a disease characterized by having too many red blood cells. Patients with this condition often suffer from a higher risk of cardiovascular events and other systemic symptoms, including itching, night sweats, fatigue and difficulties concentrating.

真性红细胞增多症（一种血癌）在美国约有160000名患者，是一种以红细胞过多为特征的疾病。患有这种疾病的患者通常患有心血管事件和其他全身症状的风险较高，包括瘙痒，盗汗，疲劳和注意力不集中。

When left unchecked, polycythemia vera can lead to heart attacks, stroke and death..

如果不加以控制，真性红细胞增多症会导致心脏病发作、中风和死亡。。

Currently, there is no cure for polycythemia vera. Patients are often managed with medications and other interventions that keep hematocrit levels below 45%. These include phlebotomies, which can sometimes also lead to iron deficiency, according to Molina.

目前，真性红细胞增多症尚无治愈方法。患者通常接受药物治疗和其他干预措施，使血细胞比容水平保持在45%以下。据Molina介绍，这些包括静脉切开术，有时也会导致缺铁。

Rusfertide is an investigational peptide mimetic of the hormone hepcidin, which is a key player in the regulation of iron equilibrium and healthy development of red blood cells. Its mechanism of action allows rusfertide to lower hematocrit levels to within acceptable ranges without raising the risk of iron deficiency..

Rusfertide是激素hepcidin的研究性肽模拟物，hepcidin是调节铁平衡和红细胞健康发育的关键参与者。它的作用机制允许rusfertide将血细胞比容水平降低到可接受的范围内，而不会增加缺铁的风险。。

Late last month, Takeda bought into the potential of rusfertide and paid Protagonist $300 million upfront for a worldwide license to develop and commercialize the candidate. Protagonist will remain in charge of the Phase III VERIFY trial and through U.S. regulatory approval, while Takeda will have rights for ex-U.S.

上个月底，武田收购了rusfertide的潜力，并向主角预付了3亿美元，获得了开发和商业化该候选人的全球许可证。Protation将继续负责第三阶段验证试验，并通过美国监管部门的批准，而武田将拥有前美国的权利。

development and will lead global commercialization activities..

发展并将领导全球商业化活动。。

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

特里斯坦·马纳拉克（TristanManalac）是一位独立的科学作家，总部位于菲律宾马尼拉。在LinkedIn上联系他，或发电子邮件给他tristan@tristanmanalac.com或tristan.manalac@biospace.com.