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2021-11-29

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Krystal Biotech shares Skyrocketed 140% in premarket trading<blockquote>Krystal Biotech股价在盘前交易中飙升140%</blockquote>

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{"i18n":{"language":"zh_CN"},"detailType":1,"isChannel":false,"data":{"magic":2,"id":600719938,"tweetId":"600719938","gmtCreate":1638197242380,"gmtModify":1638197243895,"author":{"id":3568258797592691,"idStr":"3568258797592691","authorId":3568258797592691,"authorIdStr":"3568258797592691","name":"Joykong","avatar":"https://static.laohu8.com/default-avatar.jpg","vip":1,"userType":1,"introduction":"","boolIsFan":false,"boolIsHead":false,"crmLevel":12,"crmLevelSwitch":0,"individualDisplayBadges":[],"fanSize":6,"starInvestorFlag":false},"themes":[],"images":[],"coverImages":[],"extraTitle":"","html":"<html><head></head><body>Good</body></html>","htmlText":"<html><head></head><body>Good</body></html>","text":"Good","highlighted":1,"essential":1,"paper":1,"likeSize":1,"commentSize":1,"repostSize":0,"favoriteSize":0,"link":"https://laohu8.com/post/600719938","repostId":1164587624,"repostType":4,"repost":{"id":"1164587624","kind":"news","weMediaInfo":{"introduction":"Providing stock market headlines, business news, financials and earnings ","home_visible":1,"media_name":"Tiger Newspress","id":"1079075236","head_image":"https://static.tigerbbs.com/8274c5b9d4c2852bfb1c4d6ce16c68ba"},"pubTimestamp":1638195795,"share":"https://www.laohu8.com/m/news/1164587624?lang=zh_CN&edition=full","pubTime":"2021-11-29 22:23","market":"us","language":"en","title":"Krystal Biotech shares Skyrocketed 140% in premarket trading<blockquote>Krystal Biotech股价在盘前交易中飙升140%</blockquote>","url":"https://stock-news.laohu8.com/highlight/detail?id=1164587624","media":"Tiger Newspress","summary":"Krystal Biotech Inc. shares more than doubled in premarket trading after Dystrophic EB trial meets o","content":"Krystal Biotech Inc. shares more than doubled in premarket trading after Dystrophic EB trial meets objectives.<blockquote>在营养不良EB试验达到目标后，Krystal Biotech Inc.的股价在盘前交易中上涨了一倍多。</blockquote> <img src=\"https://static.tigerbbs.com/9a6cac27a6ef602da5ab0bfd20f69c16\" tg-width=\"852\" tg-height=\"621\" referrerpolicy=\"no-referrer\"><blockquote></blockquote> Krystal Biotech, Inc.,, the leader in redosable gene therapies for rare diseases, today announced positive topline results from the pivotal GEM-3 trial of investigational beremagene geperpavec (B-VEC), now known as VYJUVEKTM, for the treatment of dystrophic Epidermolysis Bullosa (dystrophic EB).<blockquote>罕见疾病可重复剂量基因疗法的领导者郑秀晶生物技术公司今天宣布了研究性beremagene geperpavec（B-VEC）（现称为VYJUVEKTM）治疗营养不良性大疱性表皮松解症（营养不良性EB）的关键GEM-3试验的积极顶线结果。</blockquote> The primary endpoint of the trial evaluated complete wound healing of topical VYJUVEKTMcompared to placebo at six-month timepoints and met statistical significance. VYJUVEKTMis the first non-invasive, topical and redosable gene therapy in development, and the only genetically corrective approach to treat dystrophic EB that has successfully completed a double blinded Phase 3 trial.<blockquote>该试验的主要终点评估了与安慰剂相比，局部VYJUVEKTM在六个月时间点的伤口完全愈合情况，并符合统计学显著性。VYJUVEKTMis是第一种正在开发的非侵入性、局部和可重复给药的基因疗法，也是唯一一种成功完成双盲3期试验的治疗营养不良型EB的基因矫正方法。</blockquote> Highlights of Topline Results from the GEM-3 Trial<blockquote>GEM-3试验的主要结果要点</blockquote> <ul> <li>31 patients (31 primary matched-wound pairs) were enrolled and evaluable for safety and efficacy per the primary intent-to-treat (ITT) analysis</li> <li>67% of wounds treated with VYJUVEKTMachieved the primary endpoint of investigator assessed complete wound healing at the six-month timepoints as compared to 22% of wounds treated with placebo (absolute difference (95% CI): 45.8% (23.6%-68.0%); p&lt;0.005)</li> <li>71% of wounds treated with VYJUVEKTMachieved the secondary endpoint of investigator assessed complete wound healing at the three-month timepoints as compared to 20% of wounds treated with placebo (absolute difference (95% CI): 51.0% (29.3%-72.6%); p&lt;0.005)</li> <li>In an ad-hoc analysis, the trial also demonstrated a statistical difference between the active and placebo groups for wounds that demonstrated complete wound healing at both the three- and six-month timepoints (p&lt;0.005)</li> </ul> <ul> <li>VYJUVEKTMwas well tolerated. No drug-related serious adverse events or discontinuations due to treatment were reported. One mild drug-related adverse event was reported during the trial.</li> <li>The immunogenicity profile of VYJUVEKTM(as measured by anti-HSV-1 and anti-COL7 antibodies) was consistent with the prior GEM-1/2 study where we observed no meaningful change in anti-HSV-1 or anti-COL7 antibodies</li> </ul> “Dystrophic Epidermolysis Bullosa is referred to as ‘the worst disease you’ve never heard of’ because of the incredibly devastating reality that patients with this genetic condition face, and we are thrilled to announce positive results from our pivotal GEM-3 trial of VYJUVEKTMwhich showed that this topical gene therapy led to durable wound healing in dystrophic EB wounds,” said Suma Krishnan, Founder and Chief Operating Officer of Krystal. “With these results in hand, we look forward to advancing discussions with regulatory authorities and will work quickly to bring this potential first-ever treatment to patients with dystrophic EB and their families who are in desperate need.”<blockquote><ul><li>根据主要意向治疗（ITT）分析，纳入了31例患者（31对主要匹配伤口）并可评估安全性和有效性</li><li>67%接受VYJUVEKTMA治疗的伤口在6个月时间点达到研究者评估的伤口完全愈合的主要终点，而接受安慰剂治疗的伤口为22%（绝对差异（95%CI）：45.8%（23.6%-68.0%）；p&lt;0.005）</li><li>71%接受VYJUVEKTMA治疗的伤口在3个月时间点达到研究者评估的伤口完全愈合的次要终点，而接受安慰剂治疗的伤口为20%（绝对差异（95%CI）：51.0%（29.3%-72.6%）；p&lt;0.005）</li><li>在一项特别分析中，该试验还证明了活性组和安慰剂组在3个月和6个月时间点伤口完全愈合的伤口之间存在统计学差异（p&lt;0.005）</li></ul><ul><li>VYJUVEKTM耐受性良好。未报告与药物相关的严重不良事件或因治疗而停药。试验期间报告了1起轻度药物相关不良事件。</li><li>VYJUVEKTM的免疫原性特征（通过抗HSV-1和抗COL7抗体测量）与先前的GEM-1/2研究一致，在先前的GEM-1/2研究中，我们没有观察到抗HSV-1或抗COL7抗体有意义的变化</li></ul>“营养不良性大疱性表皮松解症被称为‘你从未听说过的最严重的疾病’，因为患有这种遗传疾病的患者面临着令人难以置信的毁灭性现实，我们很高兴宣布我们的关键GEM-3试验VyjuvekTM的积极结果表明，这种局部基因疗法可以使营养不良性EB伤口持久愈合，”Krystal创始人兼首席运营官Suma Krishnan说道。“有了这些结果，我们期待与监管机构推进讨论，并将迅速努力，将这种潜在的首次治疗方法带给急需的营养不良型EB患者及其家人。”</blockquote> “Today’s positive B-VEC results represent the culmination of years of study on the molecular basis and genetic correction of this disease. Finally, dystrophic EB patients may have an easily administered genetically targeted therapy which has been shown to promote durable wound healing in this clinical trial. This is a long overdue milestone for patients living with this disease, and one that has potential to drastically change the treatment paradigm,” said Dr. Peter Marinkovich, M.D., Bullous Disease Clinic Director and Associate Professor of Dermatology at Stanford University.<blockquote>“今天的B-VEC阳性结果代表了多年来对这种疾病的分子基础和基因矫正研究的高潮。最后，营养不良型EB患者可能有一种易于管理的基因靶向疗法，该疗法已被证明可以促进持久的伤口愈合。对于患有这种疾病的患者来说，这是一个姗姗来迟的里程碑，也有可能彻底改变治疗范式，”斯坦福大学大疱性疾病诊所主任兼皮肤病学副教授Peter Marinkovich医学博士说。</blockquote> Next Steps<blockquote>后续步骤</blockquote> Based on these results, Krystal intends to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) in the first half of 2022 as the first step in executing its global regulatory and commercialization strategy to bring this investigational therapy to patients in need. The Company expects to submit a Marketing Authorization Application (MAA) in Europe shortly after the BLA. Exploration of the potential regulatory path forward in other geographies, including Japan, is underway. Krystal will continue to manufacture VYJUVEKTMusing the commercial scale process at its in-house cGMP manufacturing facility, ANCORIS, which was designed to support potential launch. The Company is currently constructing its second, larger, facility ASTRA, which is expected to come on-line in 2022 to help support a potential global launch and the pipeline.<blockquote>基于这些结果，Krystal打算向美国提交生物制品许可申请（BLA）。美国食品和药物管理局(FDA)将于2022年上半年作为执行其全球监管和商业化战略的第一步，将这种研究疗法带给有需要的患者。该公司预计将在BLA后不久在欧洲提交营销授权申请（MAA）。正在探索包括日本在内的其他地区的潜在监管路径。Krystal将继续在其内部cGMP制造工厂ANCORIS采用商业规模工艺生产Vyjuvektmus，该工厂旨在支持潜在的发布。该公司目前正在建设第二个更大的设施ASTRA，预计将于2022年上线，以帮助支持潜在的全球发布和管道。</blockquote> “We founded Krystal less than six years ago with the goal of developing a non-invasive, genetically corrective therapy for dystrophic EB. We offer our deepest gratitude to the patients, caregivers, investigators, and of course to the broader Krystal team who worked tirelessly to help us reach this exciting moment in the progression of the VYJUVEKTMprogram,” said Krish Krishnan, Chairman and CEO of Krystal. “These pivotal data provide important validation of our redosable gene delivery technology, emboldening us to expand our pipeline to address other genetic skin diseases, continue to explore the potential in genetic lung diseases, and invest in growing the platform capability to address new organ systems as well.”<blockquote>“不到六年前，我们创立了Krystal，目标是开发一种针对营养不良型EB的非侵入性基因矫正疗法。我们向患者、护理人员、研究人员，当然还有更广泛的Krystal团队表示最深切的感谢，他们不知疲倦地工作帮助我们在VyjuveKTM计划的进展中达到这一激动人心的时刻，”Krystal董事长兼首席执行官Krish Krishnan说道。“这些关键数据为我们的可重复剂量基因递送技术提供了重要验证，使我们有勇气扩大我们的管道以解决其他遗传性皮肤病，继续探索遗传性肺病的潜力，并投资提高解决新器官系统的平台能力以及。”</blockquote>","collect":0,"html":"<!DOCTYPE html>\n<html>\n<head>\n<meta http-equiv=\"Content-Type\" content=\"text/html; charset=utf-8\" />\n<meta name=\"viewport\" content=\"width=device-width,initial-scale=1.0,minimum-scale=1.0,maximum-scale=1.0,user-scalable=no\"/>\n<meta name=\"format-detection\" content=\"telephone=no,email=no,address=no\" />\n<title>Krystal Biotech shares Skyrocketed 140% in premarket trading<blockquote>Krystal Biotech股价在盘前交易中飙升140%</blockquote></title>\n<style type=\"text/css\">\na,abbr,acronym,address,applet,article,aside,audio,b,big,blockquote,body,canvas,caption,center,cite,code,dd,del,details,dfn,div,dl,dt,\nem,embed,fieldset,figcaption,figure,footer,form,h1,h2,h3,h4,h5,h6,header,hgroup,html,i,iframe,img,ins,kbd,label,legend,li,mark,menu,nav,\nobject,ol,output,p,pre,q,ruby,s,samp,section,small,span,strike,strong,sub,summary,sup,table,tbody,td,tfoot,th,thead,time,tr,tt,u,ul,var,video{ font:inherit;margin:0;padding:0;vertical-align:baseline;border:0 }\nbody{ font-size:16px; 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}\n.meta {color:#5e5c6d;font-size:13px;margin:0 0 .5em; }\na{text-decoration:none; color:#2a4b87;}\n.meta .head { display: inline-block; overflow: hidden}\n.head .h-thumb { width: 30px; height: 30px; margin: 0; padding: 0; border-radius: 50%; float: left;}\n.head .h-content { margin: 0; padding: 0 0 0 9px; float: left;}\n.head .h-name {font-size: 13px; color: #eee; margin: 0;}\n.head .h-time {font-size: 12.5px; color: #7E829C; margin: 0;}\n.small {font-size: 12.5px; display: inline-block; transform: scale(0.9); -webkit-transform: scale(0.9); transform-origin: left; -webkit-transform-origin: left;}\n.smaller {font-size: 12.5px; display: inline-block; transform: scale(0.8); -webkit-transform: scale(0.8); transform-origin: left; -webkit-transform-origin: left;}\n.bt-text {font-size: 12px;margin: 1.5em 0 0 0}\n.bt-text p {margin: 0}\n</style>\n</head>\n<body>\n<div class=\"wrapper\">\n<header>\n<h2 class=\"title\">\nKrystal Biotech shares Skyrocketed 140% in premarket trading<blockquote>Krystal Biotech股价在盘前交易中飙升140%</blockquote>\n</h2>\n<h4 class=\"meta\">\n<a class=\"head\" href=\"https://laohu8.com/wemedia/1079075236\">\n\n<div class=\"h-thumb\" style=\"background-image:url(https://static.tigerbbs.com/8274c5b9d4c2852bfb1c4d6ce16c68ba);background-size:cover;\"></div>\n\n<div class=\"h-content\">\nTiger Newspress \n2021-11-29 22:23\n</div>\n</a>\n</h4>\n</header>\n<article>\nKrystal Biotech Inc. shares more than doubled in premarket trading after Dystrophic EB trial meets objectives.<blockquote>在营养不良EB试验达到目标后，Krystal Biotech Inc.的股价在盘前交易中上涨了一倍多。</blockquote> <img src=\"https://static.tigerbbs.com/9a6cac27a6ef602da5ab0bfd20f69c16\" tg-width=\"852\" tg-height=\"621\" referrerpolicy=\"no-referrer\"><blockquote></blockquote> Krystal Biotech, Inc.,, the leader in redosable gene therapies for rare diseases, today announced positive topline results from the pivotal GEM-3 trial of investigational beremagene geperpavec (B-VEC), now known as VYJUVEKTM, for the treatment of dystrophic Epidermolysis Bullosa (dystrophic EB).<blockquote>罕见疾病可重复剂量基因疗法的领导者郑秀晶生物技术公司今天宣布了研究性beremagene geperpavec（B-VEC）（现称为VYJUVEKTM）治疗营养不良性大疱性表皮松解症（营养不良性EB）的关键GEM-3试验的积极顶线结果。</blockquote> The primary endpoint of the trial evaluated complete wound healing of topical VYJUVEKTMcompared to placebo at six-month timepoints and met statistical significance. VYJUVEKTMis the first non-invasive, topical and redosable gene therapy in development, and the only genetically corrective approach to treat dystrophic EB that has successfully completed a double blinded Phase 3 trial.<blockquote>该试验的主要终点评估了与安慰剂相比，局部VYJUVEKTM在六个月时间点的伤口完全愈合情况，并符合统计学显著性。VYJUVEKTMis是第一种正在开发的非侵入性、局部和可重复给药的基因疗法，也是唯一一种成功完成双盲3期试验的治疗营养不良型EB的基因矫正方法。</blockquote> Highlights of Topline Results from the GEM-3 Trial<blockquote>GEM-3试验的主要结果要点</blockquote> <ul> <li>31 patients (31 primary matched-wound pairs) were enrolled and evaluable for safety and efficacy per the primary intent-to-treat (ITT) analysis</li> <li>67% of wounds treated with VYJUVEKTMachieved the primary endpoint of investigator assessed complete wound healing at the six-month timepoints as compared to 22% of wounds treated with placebo (absolute difference (95% CI): 45.8% (23.6%-68.0%); p&lt;0.005)</li> <li>71% of wounds treated with VYJUVEKTMachieved the secondary endpoint of investigator assessed complete wound healing at the three-month timepoints as compared to 20% of wounds treated with placebo (absolute difference (95% CI): 51.0% (29.3%-72.6%); p&lt;0.005)</li> <li>In an ad-hoc analysis, the trial also demonstrated a statistical difference between the active and placebo groups for wounds that demonstrated complete wound healing at both the three- and six-month timepoints (p&lt;0.005)</li> </ul> <ul> <li>VYJUVEKTMwas well tolerated. No drug-related serious adverse events or discontinuations due to treatment were reported. One mild drug-related adverse event was reported during the trial.</li> <li>The immunogenicity profile of VYJUVEKTM(as measured by anti-HSV-1 and anti-COL7 antibodies) was consistent with the prior GEM-1/2 study where we observed no meaningful change in anti-HSV-1 or anti-COL7 antibodies</li> </ul> “Dystrophic Epidermolysis Bullosa is referred to as ‘the worst disease you’ve never heard of’ because of the incredibly devastating reality that patients with this genetic condition face, and we are thrilled to announce positive results from our pivotal GEM-3 trial of VYJUVEKTMwhich showed that this topical gene therapy led to durable wound healing in dystrophic EB wounds,” said Suma Krishnan, Founder and Chief Operating Officer of Krystal. “With these results in hand, we look forward to advancing discussions with regulatory authorities and will work quickly to bring this potential first-ever treatment to patients with dystrophic EB and their families who are in desperate need.”<blockquote><ul><li>根据主要意向治疗（ITT）分析，纳入了31例患者（31对主要匹配伤口）并可评估安全性和有效性</li><li>67%接受VYJUVEKTMA治疗的伤口在6个月时间点达到研究者评估的伤口完全愈合的主要终点，而接受安慰剂治疗的伤口为22%（绝对差异（95%CI）：45.8%（23.6%-68.0%）；p&lt;0.005）</li><li>71%接受VYJUVEKTMA治疗的伤口在3个月时间点达到研究者评估的伤口完全愈合的次要终点，而接受安慰剂治疗的伤口为20%（绝对差异（95%CI）：51.0%（29.3%-72.6%）；p&lt;0.005）</li><li>在一项特别分析中，该试验还证明了活性组和安慰剂组在3个月和6个月时间点伤口完全愈合的伤口之间存在统计学差异（p&lt;0.005）</li></ul><ul><li>VYJUVEKTM耐受性良好。未报告与药物相关的严重不良事件或因治疗而停药。试验期间报告了1起轻度药物相关不良事件。</li><li>VYJUVEKTM的免疫原性特征（通过抗HSV-1和抗COL7抗体测量）与先前的GEM-1/2研究一致，在先前的GEM-1/2研究中，我们没有观察到抗HSV-1或抗COL7抗体有意义的变化</li></ul>“营养不良性大疱性表皮松解症被称为‘你从未听说过的最严重的疾病’，因为患有这种遗传疾病的患者面临着令人难以置信的毁灭性现实，我们很高兴宣布我们的关键GEM-3试验VyjuvekTM的积极结果表明，这种局部基因疗法可以使营养不良性EB伤口持久愈合，”Krystal创始人兼首席运营官Suma Krishnan说道。“有了这些结果，我们期待与监管机构推进讨论，并将迅速努力，将这种潜在的首次治疗方法带给急需的营养不良型EB患者及其家人。”</blockquote> “Today’s positive B-VEC results represent the culmination of years of study on the molecular basis and genetic correction of this disease. Finally, dystrophic EB patients may have an easily administered genetically targeted therapy which has been shown to promote durable wound healing in this clinical trial. This is a long overdue milestone for patients living with this disease, and one that has potential to drastically change the treatment paradigm,” said Dr. Peter Marinkovich, M.D., Bullous Disease Clinic Director and Associate Professor of Dermatology at Stanford University.<blockquote>“今天的B-VEC阳性结果代表了多年来对这种疾病的分子基础和基因矫正研究的高潮。最后，营养不良型EB患者可能有一种易于管理的基因靶向疗法，该疗法已被证明可以促进持久的伤口愈合。对于患有这种疾病的患者来说，这是一个姗姗来迟的里程碑，也有可能彻底改变治疗范式，”斯坦福大学大疱性疾病诊所主任兼皮肤病学副教授Peter Marinkovich医学博士说。</blockquote> Next Steps<blockquote>后续步骤</blockquote> Based on these results, Krystal intends to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) in the first half of 2022 as the first step in executing its global regulatory and commercialization strategy to bring this investigational therapy to patients in need. The Company expects to submit a Marketing Authorization Application (MAA) in Europe shortly after the BLA. Exploration of the potential regulatory path forward in other geographies, including Japan, is underway. Krystal will continue to manufacture VYJUVEKTMusing the commercial scale process at its in-house cGMP manufacturing facility, ANCORIS, which was designed to support potential launch. The Company is currently constructing its second, larger, facility ASTRA, which is expected to come on-line in 2022 to help support a potential global launch and the pipeline.<blockquote>基于这些结果，Krystal打算向美国提交生物制品许可申请（BLA）。美国食品和药物管理局(FDA)将于2022年上半年作为执行其全球监管和商业化战略的第一步，将这种研究疗法带给有需要的患者。该公司预计将在BLA后不久在欧洲提交营销授权申请（MAA）。正在探索包括日本在内的其他地区的潜在监管路径。Krystal将继续在其内部cGMP制造工厂ANCORIS采用商业规模工艺生产Vyjuvektmus，该工厂旨在支持潜在的发布。该公司目前正在建设第二个更大的设施ASTRA，预计将于2022年上线，以帮助支持潜在的全球发布和管道。</blockquote> “We founded Krystal less than six years ago with the goal of developing a non-invasive, genetically corrective therapy for dystrophic EB. We offer our deepest gratitude to the patients, caregivers, investigators, and of course to the broader Krystal team who worked tirelessly to help us reach this exciting moment in the progression of the VYJUVEKTMprogram,” said Krish Krishnan, Chairman and CEO of Krystal. “These pivotal data provide important validation of our redosable gene delivery technology, emboldening us to expand our pipeline to address other genetic skin diseases, continue to explore the potential in genetic lung diseases, and invest in growing the platform capability to address new organ systems as well.”<blockquote>“不到六年前，我们创立了Krystal，目标是开发一种针对营养不良型EB的非侵入性基因矫正疗法。我们向患者、护理人员、研究人员，当然还有更广泛的Krystal团队表示最深切的感谢，他们不知疲倦地工作帮助我们在VyjuveKTM计划的进展中达到这一激动人心的时刻，”Krystal董事长兼首席执行官Krish Krishnan说道。“这些关键数据为我们的可重复剂量基因递送技术提供了重要验证，使我们有勇气扩大我们的管道以解决其他遗传性皮肤病，继续探索遗传性肺病的潜力，并投资提高解决新器官系统的平台能力以及。”</blockquote>\n</article>\n</div>\n</body>\n</html>\n","type":0,"thumbnail":"","relate_stocks":{"KRYS":"Krystal Biotech Inc."},"is_english":true,"share_image_url":"https://static.laohu8.com/e9f99090a1c2ed51c021029395664489","article_id":"1164587624","content_text":"Krystal Biotech Inc. shares more than doubled in premarket trading after Dystrophic EB trial meets objectives.\n\nKrystal Biotech, Inc.,, the leader in redosable gene therapies for rare diseases, today announced positive topline results from the pivotal GEM-3 trial of investigational beremagene geperpavec (B-VEC), now known as VYJUVEKTM, for the treatment of dystrophic Epidermolysis Bullosa (dystrophic EB).\nThe primary endpoint of the trial evaluated complete wound healing of topical VYJUVEKTMcompared to placebo at six-month timepoints and met statistical significance. VYJUVEKTMis the first non-invasive, topical and redosable gene therapy in development, and the only genetically corrective approach to treat dystrophic EB that has successfully completed a double blinded Phase 3 trial.\nHighlights of Topline Results from the GEM-3 Trial\n\n31 patients (31 primary matched-wound pairs) were enrolled and evaluable for safety and efficacy per the primary intent-to-treat (ITT) analysis\n67% of wounds treated with VYJUVEKTMachieved the primary endpoint of investigator assessed complete wound healing at the six-month timepoints as compared to 22% of wounds treated with placebo (absolute difference (95% CI): 45.8% (23.6%-68.0%); p<0.005)\n71% of wounds treated with VYJUVEKTMachieved the secondary endpoint of investigator assessed complete wound healing at the three-month timepoints as compared to 20% of wounds treated with placebo (absolute difference (95% CI): 51.0% (29.3%-72.6%); p<0.005)\nIn an ad-hoc analysis, the trial also demonstrated a statistical difference between the active and placebo groups for wounds that demonstrated complete wound healing at both the three- and six-month timepoints (p<0.005)\n\n\nVYJUVEKTMwas well tolerated. No drug-related serious adverse events or discontinuations due to treatment were reported. One mild drug-related adverse event was reported during the trial.\nThe immunogenicity profile of VYJUVEKTM(as measured by anti-HSV-1 and anti-COL7 antibodies) was consistent with the prior GEM-1/2 study where we observed no meaningful change in anti-HSV-1 or anti-COL7 antibodies\n\n“Dystrophic Epidermolysis Bullosa is referred to as ‘the worst disease you’ve never heard of’ because of the incredibly devastating reality that patients with this genetic condition face, and we are thrilled to announce positive results from our pivotal GEM-3 trial of VYJUVEKTMwhich showed that this topical gene therapy led to durable wound healing in dystrophic EB wounds,” said Suma Krishnan, Founder and Chief Operating Officer of Krystal. “With these results in hand, we look forward to advancing discussions with regulatory authorities and will work quickly to bring this potential first-ever treatment to patients with dystrophic EB and their families who are in desperate need.”\n“Today’s positive B-VEC results represent the culmination of years of study on the molecular basis and genetic correction of this disease. Finally, dystrophic EB patients may have an easily administered genetically targeted therapy which has been shown to promote durable wound healing in this clinical trial. This is a long overdue milestone for patients living with this disease, and one that has potential to drastically change the treatment paradigm,” said Dr. Peter Marinkovich, M.D., Bullous Disease Clinic Director and Associate Professor of Dermatology at Stanford University.\nNext Steps\nBased on these results, Krystal intends to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) in the first half of 2022 as the first step in executing its global regulatory and commercialization strategy to bring this investigational therapy to patients in need. The Company expects to submit a Marketing Authorization Application (MAA) in Europe shortly after the BLA. Exploration of the potential regulatory path forward in other geographies, including Japan, is underway. Krystal will continue to manufacture VYJUVEKTMusing the commercial scale process at its in-house cGMP manufacturing facility, ANCORIS, which was designed to support potential launch. The Company is currently constructing its second, larger, facility ASTRA, which is expected to come on-line in 2022 to help support a potential global launch and the pipeline.\n“We founded Krystal less than six years ago with the goal of developing a non-invasive, genetically corrective therapy for dystrophic EB. We offer our deepest gratitude to the patients, caregivers, investigators, and of course to the broader Krystal team who worked tirelessly to help us reach this exciting moment in the progression of the VYJUVEKTMprogram,” said Krish Krishnan, Chairman and CEO of Krystal. “These pivotal data provide important validation of our redosable gene delivery technology, emboldening us to expand our pipeline to address other genetic skin diseases, continue to explore the potential in genetic lung diseases, and invest in growing the platform capability to address new organ systems as well.”","news_type":1,"symbols_score_info":{"KRYS":0.9}},"isVote":1,"tweetType":1,"viewCount":2218,"commentLimit":10,"likeStatus":false,"favoriteStatus":false,"reportStatus":false,"symbols":[],"verified":2,"subType":0,"readableState":1,"langContent":"CN","currentLanguage":"CN","warmUpFlag":false,"orderFlag":false,"shareable":true,"causeOfNotShareable":"","featuresForAnalytics":[],"commentAndTweetFlag":false,"andRepostAutoSelectedFlag":false,"upFlag":false,"length":4,"xxTargetLangEnum":"ZH_CN"},"commentList":[],"isCommentEnd":true,"isTiger":false,"isWeiXinMini":false,"url":"/m/post/600719938"}