Pictured: Exterior view of Pfizer's office in Belgium/iStock, Alexandros Michailidis

图为辉瑞（Pfizer）驻比利时办事处的外部视图/亚历山大·米切利迪斯（AlexandrosMichailidis）伊斯托克（iStock）

Pfizer has discontinued one of its two Phase III studies evaluating the anti-P-selecting antibody inclacumab in sickle cell disease, according to an update on the ClinicalTrials.gov webpage.

根据ClinicalTrials.gov网页上的最新消息，辉瑞已经停止了其评估镰状细胞病中抗P选择抗体inclacumab的两项III期研究之一。

According to the online database, Pfizer terminated the late-stage study “due to poor accrual and associated recruitment challenges.” The trial was launched in December 2021. However, at the time of Monday’s update, the study had only enrolled 78 patients out of its target size of 280 participants.

根据在线数据库，辉瑞终止了后期研究，“由于应计不良和相关的招聘挑战”。该试验于2021年12月启动。然而，在周一更新时，该研究仅招募了280名参与者中的78名患者。

A Pfizer spokesperson confirmed to Fierce Biotech that the company ended the study “due to slow recruitment,” while adding that the pharma is still eyeing an approval for inclacumab in sickle cell disease by 2026.

辉瑞的一位发言人向威猛生物科技证实，该公司“由于招聘缓慢”结束了这项研究，同时补充说，该制药公司仍在期待2026年前批准镰状细胞病的inclacumab。

Originally developed by Global Blood Therapeutics (GBT), Inclacumab is a fully human monoclonal antibody that works by selectively blocking the P-selectin protein, which is known to play a role in cell adhesion and is a clinically validated target for reducing pain associated with vaso-occlusive crises (VOC) in patients with sickle cell disease (SCD)..

Inclacumab最初由全球血液治疗学（GBT）开发，是一种完全人类单克隆抗体，通过选择性阻断P-选择素蛋白发挥作用，P-选择素蛋白已知在细胞粘附中起作用，是临床验证的减轻镰状细胞病（SCD）患者血管闭塞性危象（VOC）相关疼痛的靶点。。

Inclacumab in June 2022 secured the FDA’s Orphan Drug and Rare Pediatric Disease designations for SCD treatment due to its best-in-class potential as well as its quarterly dosing regimen instead of monthly infusions.

2022年6月，Inclacumab获得了FDA的孤儿药和罕见儿科疾病指定用于SCD治疗，因为它具有同类最佳的潜力，并且每季度给药方案而不是每月输注。

In August 2022, Pfizer moved to acquire GBT in a $5.4 billion deal for the biotech’s portfolio of SCD therapies, including the FDA-approved Oxbryta (voxelotor), an oral hemoglobin S polymerization inhibitor indicated for patients four years of age and older. Inclacumab was also part of the acquisition..

2022年8月，辉瑞公司以54亿美元的价格收购了GBT，收购了生物技术公司的SCD治疗组合，其中包括FDA批准的Oxbryta（voxelotor），一种口服血红蛋白s聚合抑制剂，适用于4岁及以上的患者。Inclacumab也是收购的一部分。。

Following the buyout, Pfizer took over the Phase III THRIVE program, which comprised two studies—including the subsequently terminated trial—designed to evaluate the efficacy and safety of inclacumab at reducing VOCs in SCD patients. The other trial remains active and has completed recruitment at 240 patients.

收购后，辉瑞公司接管了III期THRIVE计划，该计划包括两项研究，包括随后终止的试验，旨在评估inclacumab在减少SCD患者挥发性有机化合物方面的疗效和安全性。另一项试验仍在进行中，已完成240名患者的招募。

According to its ClinicalTrials.gov page, a readout is expected later this year..

根据ClinicalTrials.gov页面，预计今年晚些时候会有读数。。

The termination of Pfizer’s late-stage study follows back-to-back landmark approvals in the SCD space. In December 2023, the FDA on the same day gave its nod to CRISPR Therapeutics and Vertex Pharmaceuticals’ CRISPR-based Casgevy (exagamglogene autotemcel) and bluebird bio’s lentiviral delivery-based Lyfgenia (lovotibeglogene autotemcel)..

辉瑞公司后期研究的终止是在SCD领域连续获得里程碑式批准之后进行的。2023年12月，FDA在同一天批准了CRISPR Therapeutics和Vertex Pharmaceuticals基于CRISPR的Casgevy（exagamglogene autotemcel）和bluebird bio基于慢病毒递送的Lyfgenia（lovotibeglogene autotemcel）。。

Casgevy and Lyfgenia are cell-based gene therapies. Both products are made from the patients’ own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion. Casgevy has a U.S. list price of $2.2 million per patient, while bluebird set the wholesale acquisition cost of Lyfgenia in the U.S.

Casgevy和Lyfgenia是基于细胞的基因疗法。这两种产品都是由患者自己的血液干细胞制成的，这些干细胞经过修饰，并作为一次性单剂量输注给患者。卡西维（Casgevy）在美国的标价为每位患者220万美元，而蓝鸟（bluebird）在美国设定了Lyfgenia的批发收购成本。

at $3.1 million..

310万美元。。

In January 2024, the Centers for Medicare and Medicaid Services announced that it would seek outcomes-based agreements with the developers of these SCD gene therapies to help curb healthcare costs.

2024年1月，医疗保险和医疗补助服务中心宣布，将寻求与这些SCD基因疗法的开发人员达成基于结果的协议，以帮助控制医疗保健成本。

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，总部位于菲律宾大马尼拉。在LinkedIn上联系他，或发电子邮件给他tristan@tristanmanalac.com或tristan.manalac@biospace.com.