European Biotech Pharming Group Touts Positive Data From Pivotal Pediatric Study Of FDA-Approved Immune System Disorder Drug
European Biotech Pharming Group Touts Positive Data From Pivotal Pediatric Study Of FDA-Approved Immune System Disorder Drug
Pharming plans to include data from this 4-11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.On Wednesday, Pharming Group N.V. (NASDAQ:PHAR) revealed topline results of data from its Phase 3 trial evaluating leniolisib in children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS).
在周三，Pharming Group N.V.（纳斯达克：PHAR）公布了其评估leniolisib在4至11岁儿童中治疗激活的磷脂酰肌醇3-激酶δ综合症（APDS）的第三阶段试验的顶线数据。
APDS is a genetic disorder that impairs the immune system. Individuals with this condition often have low numbers of white blood cells, particularly certain types of B and T cells. These cells are necessary to recognize and attack foreign invaders, such as viruses and bacteria, to prevent infection.
APDS是一种损害免疫系统的遗传疾病。患有这种控件的人通常白细胞数量较少，尤其是某些类型的b细胞和t细胞。这些细胞对于识别和攻击外来侵入者（如病毒和细菌）以防止感染是必要的。
Leniolisib, marketed under Joenja in the U.S., received FDA approval for APDS in adult and pediatric patients 12 years and older in March 2023.
Leniolisib在美国以Joenja的名义上市，于2023年3月获得FDA对12岁及以上成人和儿童患者APDS的批准。
Pharming plans to include data from this 4-11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.
Pharming计划将这项针对4至11岁儿童的试验数据纳入全球监管提交，以申请leniolisib用于患有APDS的儿童患者的批准，预计于2025年开始。
The study enrolled 21 children with APDS ages 4 to 11 years.
该研究招募了21名年龄在4至11岁的APDS儿童。
The study's primary efficacy endpoints are a reduction in index lymph node size and an increased proportion of naïve B cells out of total B cells from baseline at 12 weeks.
该研究的主要疗效终点是减少指数淋巴结的大小和基线时在12周时naïve b细胞占总b细胞的比例增加。
Secondary endpoints include assessing the ability of leniolisib to modify health-related quality of life.
次要终点包括评估leniolisib对健康相关生活质量的影响。
All 21 patients enrolled completed the 12-week treatment period.
所有21名患者均完成了12周的治疗周期。
Lymphoproliferation improved as measured by a mean reduction in index lesion size, and immunophenotype correction was demonstrated by an increase in the percent of naïve B cells.
淋巴增生通过平均减少指标病灶大小得到了改善，免疫表型纠正通过幼稚b细胞的百分比增加得到了证明。
The improvement in lymphoproliferation and immunophenotype correction was seen across the four dose levels being investigated and was consistent with the improvements previously reported in adolescent and adult patients.
在研究的四个剂量水平上，淋巴增生和免疫表型纠正的改善是一致的，与以前在青少年和成人患者中报告的改善相符。
All treatment-emergent adverse events were reported to be mild to moderate in nature. There were no drug-related serious adverse events, and all patients completed the 12-week treatment period.
所有治疗出现的不良事件均被报告为轻度至中度。没有与药物相关的严重不良事件，所有患者完成了为期12周的治疗期。
In October, Pharming started a Phase 2 proof-of-concept trial evaluating leniolisib in primary immunodeficiencies with immune dysregulation linked to altered PI3Kẟ signaling in lymphocytes.
在十月份，Pharming开始了一项评估leniolisib在与淋巴细胞的PI3Kẟ信号改变相关的免疫功能失调的原发免疫缺陷中的概念验证二期试验。
Price Action: PHAR stock is up 11.6% at $9.75 at last check Wednesday.
价格动态：PHAR股票在周三最后检查时上涨了11.6%，报9.75美元。
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