Three In A Row: FibroGen's Investigational Drug Flunks In Another Study For Rare Disease In Young Boys
Three In A Row: FibroGen's Investigational Drug Flunks In Another Study For Rare Disease In Young Boys
Tuesday, FibroGen Inc (NASDAQ:FGEN) announced topline data from the Phase 3 LELANTOS-2 trial of pamrevlumab for ambulatory patients with Duchenne muscular dystrophy (DMD) on background systemic corticosteroids.
星期二， FibroGen公司 纳斯达克股票代码：FGEN）公布了pamrevlumab用于杜兴氏肌营养不良症（DMD）的卧床患者使用背景全身性皮质类固醇进行的 3 期 LELANTOS-2 试验的头条数据。
DMD is an inherited progressive muscular weakness disorder primarily affecting young boys.
DMD 是一种遗传性进行性肌肉无力障碍，主要影响男孩。
The study did not meet the primary endpoint of change in the North Star Ambulatory Assessment (NSAA) total score from baseline to week 52.
该研究未达到从基线到第52周北极星门诊评估（NSAA）总分变化的主要终点。
NSAA measures functional motor abilities in ambulant children with DMD. It is usually used to monitor the progression of the disease and treatment effects.
NSAA 测量患有 DMD 的行走儿童的功能运动能力。它通常用于监测疾病进展和治疗效果。
Secondary endpoints measured by change from baseline at week 52 in 4-stair climb velocity, 10-meter walk/run test, time to stand, time to loss of ambulation, and proportion of patients with greater than 10 seconds in the 10-meter walk/run test were also not met.
通过与第52周基线相比在4楼梯爬坡速度、10米步行/跑步测试、站立时间、失去行走的时间以及10米步行/跑步测试中超过10秒的患者比例的变化来衡量的次要终点也未达到。
Preliminary safety data showed that pamrevlumab was generally safe and well tolerated. The majority of treatment-emergent adverse events were mild or moderate.
初步的安全数据显示，pamrevlumab总体上是安全的，耐受性良好。大多数治疗后出现的不良事件为轻度或中度。
FibroGen is evaluating the totality of the data, including other pre-specified endpoints, to determine the next steps for the program.
FibroGen正在评估全部数据，包括其他预先指定的终点，以确定该计划的下一步行动。
In June, FibroGen's Phase 3 LELANTOS-1 trial of pamrevlumab for non-ambulatory DMD patients with on-background corticosteroids failed to meet the primary endpoint of the Performance of the Upper Limb 2.0 score at week 52 compared to baseline.
6月，FibroGen针对背景皮质类固醇非活动性DMD患者的pamrevlumab进行的3期 LELANTOS-1 试验与基线相比，未能达到第52周上肢表现2.0评分的主要终点。
The company's recently released topline results from its Phase 3 ZEPHYRUS-1 trial of pamrevlumab for idiopathic pulmonary fibrosis also failed to meet the primary endpoint of change in forced vital capacity at week 48.
该公司最近公布的pamrevlumab治疗特发性肺纤维化的3期 ZEPHYRUS-1 试验的头条结果也未能达到第48周强制性肺活量变化的主要终点。
William Blair analysts Andy Hsieh and Alexandra Ramsey say limited data support the Phase 3 LAPIS trial in locally advanced pancreatic cancer. Since a previous interim look at event-free survival prompted the trial to continue to the overall survival primary endpoint, investors do not seem bullish on the trial.
威廉布莱尔 分析师 谢安迪 亚历山德拉·拉姆齐说，有限的数据支持LAPIS针对局部晚期胰腺癌的3期试验。由于先前对无事件生存的中期观察促使该试验继续达到总体存活率的主要终点，因此投资者似乎并不看好该试验。
Given the LELANTOS-1 setback, the failure of LELANTOS-2 is somewhat expected, but the analysts seem to be surprised that the pamrevlumab arm performed numerically worse than the placebo arm.
鉴于 LELANTOS-1 的挫折，LELANTOS-2 的失败在某种程度上是可以预料的，但分析师似乎感到惊讶的是，pamrevlumab组的表现比安慰剂组差得多。
Other pipeline assets are too early to assess their commercial prospects properly.
其他管道资产还为时过早，无法正确评估其商业前景。
Price Action: FGEN shares are down 22.3% at $1.00 during the premarket session on the last check Wednesday.
价格走势: FGEN股价在周三最后一次支票的盘前交易时段下跌22.3％，至1.00美元。