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Orchard Therapeutics' MPS-I Gene Therapy Shows Encouraging Action In Early Study, Shares Spike

Orchard Therapeutics' MPS-I Gene Therapy Shows Encouraging Action In Early Study, Shares Spike

Orchard Treatetics的MPS-I基因疗法在早期研究中显示出令人鼓舞的作用，股价飙升
Benzinga Real-time News ·  2021/02/09 21:19
  • Orchard Therapeutics Plc (NASDAQ: ORTX) gains in premarket in reaction to positive interim data from a proof-of-concept study evaluating its gene therapy OTL-203 in Mucopolysaccharidosis type I (MPS-I). All eight patients treated with OTL-203 showed stable cognitive function, motor function, and growth within the normal range at multiple data points post-treatment.
  • OTL-203 treatment demonstrated rapid and sustained metabolic correction with all patients achieving supra-physiological IDUA expression in dried blood spot samples at 12 months (a primary efficacy endpoint).
  • FDA and European Medicines Agency (EMA) have accepted Orchard's request for parallel scientific advice to seek feedback in advance of initiating a registrational study for OTL-203 by this year-end.
  • The company also announced encouraging preliminary biomarker data for OTL-201 in three Mucopolysaccharidosis type IIIA (MPS-IIIA) patients, showing promising tolerability, engraftment, and biomarker data over the initial three-month follow-up period. Recently, a fourth patient was enrolled in the study. The enrollment could complete (n=5) this year, and additional interim data expected later this year.
  • IR webinar is scheduled today at 4:30 p.m. ET to review OTL-203 and OTL-201 data.
  • Last year in December, the company received its first product approval for Libmeldy in Europe for eligible patients with early-onset metachromatic leukodystrophy.
  • Price Action: ORTX jumped 10% at $8.31 in premarket trading on the last check Tuesday.
  • 果园治疗公司(纳斯达克：ORTX)作为对以下情况的反应，售前收益积极的中期数据来自一项概念验证研究，评估了OTL-203在I型粘多糖病(MPS-I)中的基因治疗。所有8名接受OTL-203治疗的患者在治疗后的多个数据点都显示出稳定的认知功能、运动功能和在正常范围内的生长。
  • OTL-203治疗显示出快速和持续的代谢纠正，所有患者在12个月时都在干血斑样本中实现了超生理性IDUA的表达(主要疗效终点)。
  • FDA和欧洲药品管理局(EMA)已经接受了Orchard的平行科学建议的请求，以便在今年年底之前开始OTL-203的注册研究之前寻求反馈。
  • 该公司还宣布了在三名MPS-IIIA(MPS-IIIA)患者中用于OTL-201的令人鼓舞的初步生物标志物数据，在最初三个月的随访期内显示出良好的耐受性、植入性和生物标志物数据。最近，第四名患者参加了这项研究。注册可能在今年完成(n=5)，预计今年晚些时候会有更多的中期数据。
  • IR网络研讨会定于今天下午4：30举行。ET审查OTL-203和OTL-201数据。
  • 去年12月，该公司收到了首次产品审批在欧洲为符合条件的早发性异染性脑白质营养不良患者提供Libmeldy。
  • 价格行动：周二盘前交易中，ORTX跳涨10%，报8.31美元.

译文内容由第三方软件翻译。


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