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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基因療法在早期研究中顯示出令人鼓舞的作用，股價飆升
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.
  • 果園治療公司(納斯達克：ORTX)作為對以下情況的反應，售前收益積極的中期數據來自一項概念驗證研究，評估了OTL-203在I型粘多糖病(MPS-I)中的基因治療。所有8名接受OTL-203治療的患者在治療後的多個數據點都顯示出穩定的認知功能、運動功能和在正常範圍內的生長。
  • OTL-203治療顯示出快速和持續的代謝糾正，所有患者在12個月時都在幹血斑樣本中實現了超生理性IDUA的表達(主要療效終點)。
  • FDA和歐洲藥品管理局(EMA)已經接受了Orchard的平行科學建議的請求，以便在今年年底之前開始OTL-203的註冊研究之前尋求反饋。
  • 該公司還宣佈了在三名MPS-IIIA(MPS-IIIA)患者中用於OTL-201的令人鼓舞的初步生物標誌物數據，在最初三個月的隨訪期內顯示出良好的耐受性、植入性和生物標誌物數據。最近，第四名患者參加了這項研究。註冊可能在今年完成(n=5)，預計今年晚些時候會有更多的中期數據。
  • IR網絡研討會定於今天下午4：30舉行。ET審查OTL-203和OTL-201數據。
  • 去年12月，該公司收到了首次產品審批在歐洲為符合條件的早發性異染性腦白質營養不良患者提供Libmeldy。
  • 價格行動：週二盤前交易中，ORTX跳漲10%，報8.31美元.

譯文內容由第三人軟體翻譯。


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