Dive Brief:

简报：

The biotechnology company Rallybio is discontinuing its lead candidate for a rare blood disorder in pregnant women after disappointing data from a mid-stage study, sending shares down sharply Tuesday morning.

生物技术公司Rallybio在一项中期研究数据令人失望后，决定停止其针对孕妇罕见血液病的主要候选药物研发，导致周二早盘股价急剧下跌。

The drug, dubbed RLYB212, was in testing to treat the disorder known as fetal and neonatal alloimmune thrombocytopenia, or FNAIT, which has

这种药物名为RLYB212，正在测试用于治疗胎儿和新生儿同种免疫性血小板减少症（FNAIT）。

no approved

未批准

treatment available in the U.S. Development was also

美国可获得的治疗。也得到了发展

supported

支持的

from Johnson & Johnson, which provided Rallybio with

来自强生公司，该公司为Rallybio提供了

an equity investment of $6.6 million,

660万美元的股权投资，

an upfront payment of $500,000 and additional funding of up to $3.7 million to support a natural history study.

预先支付50万美元，并提供高达370万美元的额外资金，以支持一项自然历史研究。

Rallybio said Tuesday the drug did not meet expectations in a Phase 2 pharmacokinetic study and all screening of trial participants has stopped. The biotech will now focus on development of another candidate, RLYB116, and other preclinical programs.

Rallybio 表示，在周二的一项二期药代动力学研究中，该药物未达预期，所有试验参与者的筛查工作已停止。这家生物技术公司现在将专注于另一款候选药物 RLYB116 以及其他临床前项目的开发。

Dive Insight:

深入洞察：

Rallybio, spearheaded by former Alexion Pharmaceuticals leaders Steve Uden and Martin Mackay, has already faced tough decisions over the years including

由前Alexion制药公司高管史蒂夫·乌登和马丁·麦凯领导的Rallybio近年来已经面临了多项艰难决策，包括

switching lead candidates

切换主要候选人

and

和

cutting its workforce by half

裁员一半

to stay afloat.

保持漂浮。

RLYB212 was viewed as a major opportunity, however. After completing an epidemiological analysis of the risk of FNAIT across diverse ancestries, Rallybio estimated the drug’s commercial opportunity was

然而，RLYB212 被视为一个重要的机会。在完成对不同血统 FNAIT 风险的流行病学分析后，Rallybio 估计该药物的商业机会为

greater than $1.6 billion.

超过16亿美元。

In FNAIT, the mother’s immune cells are not compatible with the fetus’ platelets, so allo-antibodies can attack blood-clotting cells in the fetus leading to thrombocytopenia, or low platelet levels. The disease can be life-threatening or lead to miscarriages or neurological disabilities of the infant.

在FNAIT中，母亲的免疫细胞与胎儿的血小板不兼容，因此同种抗体可以攻击胎儿的凝血细胞，导致血小板减少症，即血小板水平低下。这种疾病可能危及生命，或导致流产或婴儿的神经系统残疾。

Platelet transfusion is typically first line of treatment, but there are no other preventions available..

血小板输注通常是首选的治疗方法，但没有其他可用的预防措施。

RLYB212 is a monoclonal antibody targeting “HPA-1a,” a protein thought to cause alloimmunization. Diagnostic tests for the disease include

RLYB212是一种针对“HPA-1a”的单克隆抗体，这种蛋白质被认为会导致同种免疫。该疾病的诊断测试包括

testing the mother for anti-HPA antibodies.

检测母亲的抗HPA抗体。

The Phase 2 trial assessed pharmacokinetics and safety in pregnant women at higher risk of HPA-1a alloimmunization and FNAIT. In testing, the drug did not achieve predicted target concentration levels, nor did it meet the bar set for efficacy. The company said it did not seem feasible to adjust the dose..

二期试验评估了妊娠期妇女在高风险HPA-1a同种免疫和FNAIT中的药代动力学和安全性。在测试中，该药物未能达到预期的目标浓度水平，也未达到疗效设定的标准。公司表示，调整剂量似乎并不可行。

Rallybio said it believes HPA-1a antigen expression on the placenta could have impacted plasma concentrations of the antibody.

Rallybio 表示，其认为胎盘上的 HPA-1a 抗原表达可能已经影响了抗体的血浆浓度。

The company will now shift focus to RLYB116, an antibody mimetic fusion protein designed to inhibit C5, which is set to begin an early-stage study in the second quarter.

该公司现在将把重点转移到RLYB116上，这是一种旨在抑制C5的抗体模拟融合蛋白，预计将在第二季度开始早期研究。