A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)

The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
The primary purpose of the MAD period is to evaluate the safety and tolerability and levels of dystrophin after multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). During the MAD period, participants will be randomized to either receive PGN-EDO51 or placebo in a 3:1 fashion, meaning that participants have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during this period. The primary purpose of the open-label LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. All participants who roll-over into the LTE will receive PGN-EDO51 (no placebo in the LTE).

开始日期2024-12-17

申办/合作机构

PepGen Inc.

NCT06079736

/ Active, not recruiting临床2期

A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)

The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne Muscular Dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period.

开始日期2024-01-08

申办/合作机构

PepGen Inc.

100 项与 PGN-EDO51 相关的临床结果

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100 项与 PGN-EDO51 相关的转化医学

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100 项与 PGN-EDO51 相关的专利（医药）

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项与 PGN-EDO51 相关的新闻（医药）

2025-07-23

·PRNewswire

SHAREHOLDER REMINDER: Faruqi & Faruqi, LLP Investigates Claims on Behalf of Investors of PepGen

Faruqi & Faruqi, LLP Securities Litigation Partner James (Josh) Wilson Encourages Investors Who Suffered Losses Exceeding $50,000 In PepGen To Contact Him Directly To Discuss Their Options If you suffered losses exceeding $50,000 in PepGen between March 7, 2024 and March 3, 2025 and would like to discuss your legal rights, call Faruqi & Faruqi partner Josh Wilson directly at 877-247-4292 or 212-983-9330 (Ext. 1310). [You may also click here for additional information] NEW YORK, July 23, 2025 /PRNewswire/ -- Faruqi & Faruqi, LLP, a leading national securities law firm, is investigating potential claims against PepGen Inc. ("PepGen" or the "Company") (NASDAQ: PEPG) and reminds investors of the August 11, 2025 deadline to seek the role of lead plaintiff in a federal securities class action that has been filed against the Company. Continue Reading Faruqi & Faruqi Logo (PRNewsfoto/Faruqi & Faruqi, LLP) Faruqi & Faruqi is a leading national securities law firm with offices in New York, Pennsylvania, California and Georgia. The firm has recovered hundreds of millions of dollars for investors since its founding in 1995. See . As detailed below, the complaint alleges that the Company and its executives violated federal securities laws by making false and/or misleading statements and/or failing to disclose that: (i) PGN-EDO51 was less effective and safe than Defendants had led investors to believe; (ii) the CONNECT2 study was dangerous or otherwise deficient for purposes of U.S. Food and Drug Administration ("FDA") approval; (iii) as a result of all the foregoing, PepGen was likely to halt the CONNECT2 study, and PGN-EDO51's clinical, regulatory, and commercial prospects were overstated; and (iv) as a result, Defendants' public statements were materially false and misleading at all relevant times. On July 30, 2024, PepGen issued a press release announcing purported "positive clinical data from the first dose cohort (5 mg/kg) of PGN-EDO51" in its ongoing CONNECT1 study. Among other results, the Company reported that "PGN-EDO51 achieved a mean absolute dystrophin level of 0.61% of normal and a 0.26% change from baseline after 4 doses, measured at week 13 by Western blot analysis." However, as subsequently noted by a Stifel analyst, "the magnitude of dystrophin increase was below what [PepGen] anticipated, which is disappointing[.]" Likewise, a Leerink Partners analyst noted that the low dose missed PepGen's expectations of 1% or greater dystrophin expression. On this news, PepGen's stock price fell $5.55 per share, or 32.69%, to close at $11.43 per share on July 31, 2024. On December 16, 2024, PepGen issued a press release announcing that it had received a clinical hold notice from the FDA regarding an Investigational New Drug ("IND") application "to initiate the [CONNECT2] clinical trial in patients with [DMD]" in the U.S. Notably, the FDA's issuance of a clinical hold notice for the IND application indicated that the FDA had concerns regarding risks posed to patients in the CONNECT2 study and/or there were other deficiencies associated with the study. On this news, PepGen's stock price fell $0.17 per share, or 3.63%, to close at $4.51 per share on December 16, 2024. On January 29, 2025, PepGen issued a press release providing updates regarding safety concerns observed in the CONNECT1 study and the FDA's concerns regarding the CONNECT2 study. With respect to the CONNECT1 study, the press release stated, inter alia, that "[d]osing of one of the[] . . . participants [in the 10 mg/kg cohort] was paused due to a reduction of his estimated glomerular filtration rate[.]" In addition, PepGen "ha[d] received communication from Health Canada . . . request[ing] additional information from the Company to address Health Canada's safety concerns before any further dose escalation or enrollment of any additional participants at the current dose levels." With respect to the CONNECT2 study, the same press release stated, in relevant part, that "[t]he Company is working with the FDA to address its questions regarding supportive data for the dosing levels planned for the patient population." Following these disclosures, PepGen's stock price fell $0.40 per share, or 21.74%, to close at $1.44 per share on January 30, 2025. On March 4, 2025, PepGen issued a press release "announc[ing] its voluntary decision to temporarily pause the [CONNECT2] study . . . until the Company can review results from the 10 mg/kg cohort in the ongoing [CONNECT1] study." On this news, PepGen's stock price fell $0.53 per share, or 18.86%, to close at $2.28 per share on March 4, 2025. Then, on May 28, 2025, PepGen issued a press release announcing that "PGN-EDO51 did not achieve target dystrophin levels" in the CONNECT1 study and had chosen to discontinue development of its DMD programs. The court-appointed lead plaintiff is the investor with the largest financial interest in the relief sought by the class who is adequate and typical of class members who directs and oversees the litigation on behalf of the putative class. Any member of the putative class may move the Court to serve as lead plaintiff through counsel of their choice, or may choose to do nothing and remain an absent class member. Your ability to share in any recovery is not affected by the decision to serve as a lead plaintiff or not. Faruqi & Faruqi, LLP also encourages anyone with information regarding PepGen's conduct to contact the firm, including whistleblowers, former employees, shareholders and others. To learn more about the PepGen class action, go to or call Faruqi & Faruqi partner Josh Wilson directly at 877-247-4292 or 212-983-9330 (Ext. 1310). Follow us for updates on LinkedIn, on X, or on Facebook. Attorney Advertising. The law firm responsible for this advertisement is Faruqi & Faruqi, LLP (). Prior results do not guarantee or predict a similar outcome with respect to any future matter. We welcome the opportunity to discuss your particular case. All communications will be treated in a confidential manner. SOURCE Faruqi & Faruqi, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床申请

2025-07-09

·药事纵横

大冢旗下公司DMD药物III期试验折戟

7月8日，大冢制药旗下太和制药公司（Taiho Pharmaceutical）研发的杜氏肌营养不良症（DMD）治疗药物匹祖格兰司他（pizuglanstat，商品名TAS-205）在III期临床试验中未能达到预期效果。此次开展的REACH-DMD试验，在日本26个研究中心招募了82名年龄在5岁及以上的男性DMD患者，这些患者被随机分配，每日两次接受匹祖格兰司他或安慰剂治疗，为期52周。研究结果显示，对于能够行走的患者群体而言，在从地板起身所需时间这一主要终点指标上，从基线到第52周的平均变化方面，匹祖格兰司他组与安慰剂组相比，并未展现出显著差异。这家日本制药企业表示，该试验的详细结果将在即将召开的医学会议上公布。据太和制药公司介绍，匹祖格兰司他是一种选择性造血前列腺素D合成酶（HPGDS）抑制剂，旨在无论患者DMD的肌营养不良蛋白基因突变类型如何，都能控制其运动功能的衰退。太和制药此次的失败，为这个近期在药物研发上屡遭挫折的治疗领域又添一重阻碍。今年早些时候，Sarepta Therapeutics公司因出现急性肝衰竭致死病例，停止了其在欧盟开展的DMD基因疗法Elevidys（delandistrogene moxeparvovec）的试验，并暂停了对非行走能力患者的药物供应。5月，PepGen公司因其外显子跳跃药物PGN-EDO51在中期研究中结果不佳，停止了该药物的开发。与此同时，美国食品药品监督管理局（FDA）计划于本月召开咨询委员会会议，就Capricor Therapeutics公司的DMD细胞疗法德雷米奥塞尔（deramiocel，商品名CAP-1002）作出决定。背景延伸DMD作为X染色体隐性遗传病，主要影响男性儿童，患者通常因进行性肌肉萎缩在青少年期丧失行走能力，平均寿命不超过30岁。尽管科学界持续探索基因疗法、外显子跳跃、抗炎药物等多种治疗路径，但过去三年已有超过10个临床项目宣告失败，凸显该领域研发的高风险性。目前全球仍有30余个DMD在研项目处于不同阶段，医学界正加紧探索联合疗法等新策略。药事纵横投稿须知：稿费已上调，欢迎投稿

临床3期基因疗法细胞疗法

2025-07-09

·佰傲谷BioValley

日本药企的DMD药物III期失败

日前，日本药企大冢制药（Otsuka Pharmaceutical）旗下子公司Taiho Pharmaceutical宣布，其DMD候选药物TAS-205在关键III期临床试验中，未能达到主要终点。又一DMD在研管线的失败，再一次为DMD新药的开发增添阴霾。TAS-205失败TAS-205（pizuglanstat）是一款选择性造血前列腺素D合酶（HPGDS）抑制剂。TAS-205被开发作为一种DMD治疗方法（无论基因突变类型如何），旨在通过抑制HPGDS来控制DMD患者的运动功能下降，因为HPGDS会加剧DMD患者肌肉的炎症反应。TAS-205的III期研究是一项随机、安慰剂对照、双盲和开放标签拓展研究，旨在评估持续52周每天两次口服TAS-205的疗效。7月8日发布的公告报告了该研究门诊组的数据。TAS-205的III期研究分为两个队列：门诊组和非门诊组。门诊组的主要终点是对比基线从地面站起所需要时间改善。后期疗效数据包括了82名5岁及以上的男性DMD患者，结果显示：TAS-205在52周内未能改善患者从地板上站起来所需的时间，主要终点未达到。这一失败的结果，表明HPGDS抑制剂治疗DMD的机制验证失败，该机制由日本药企创新发现。DMD新药开发多险阻DMD是一种严重的、进展性的X连锁隐性遗传性神经肌肉疾病，其发病率为1.9-3.4/1000000，是一类“不罕见的罕见病”。尽管近年来有多款DMD药物获得监管批准，包括4款外显子跳跃疗法、1款基因治疗、和1款HDAC抑制剂。外显子跳跃治疗局限于特定的基因突变类型、Sarepta的基因治疗也在近期爆出潜在的安全问题，DMD患者还需要新的治疗方法。但是，更多的DMD新药开发还是以失败告终。有相关数据表明，大约有16种DMD在研药物在临床开发阶段遭遇失败。包括Solid的第一代DMD基因治疗药物SGT-001、辉瑞的PF-06939926，这两款当时的明星管线，唯一跑出来的ELEVIDYS也在近期接连爆出两例死亡事件。延伸阅读：DMD基因治疗报道第二例死亡，直接诱因是？明星管线失败，Solid再出发今年5月，美国公司PepGen也因为其DMD候选药物PGN-EDO51在临床研究中的表现不佳，从而终止了PGN-EDO51的开发，并决定清空所有的DMD研发管线。不过，FDA将在本月底召开专家会议，以评审Capricor公司细胞疗法deramiocel的上市申请，用于治疗杜氏肌营养不良症（DMD）相关的心肌病。可能会使得DMD患者迎来新的药物选择。延伸阅读：含microRNA的药物申报上市参考资料：1.https://www.taiho.co.jp/en/release/2025/20250708.html2.https://www.fiercebiotech.com/biotech/taihos-dmd-asset-fails-improve-functional-motor-test-results-phase-3-trial········

基因疗法临床3期

100 项与 PGN-EDO51 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
杜氏肌营养不良症	临床2期	加拿大	PepGen Inc.	2023-05-18

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06079736 (CONNECT1-EDO51, PipelineReview) 人工标引	临床2期	杜氏肌营养不良症 exon 51-skipping	3	PGN-EDO51 5 mg/kg	繭淵築願淵鹽積鹽蓋壓(餘願壓製繭鹹夢網艱窪) = 壓艱廠糧觸壓製艱鹹觸構餘積製獵網鹽簾鑰鹽 (願艱夢襯廠衊蓋繭觸鏇 ) 更多	积极	2024-07-31
CONNECT1-EDO51 and CONNECT2-EDO51 (MDA2024)	临床2期	杜氏肌营养不良症	-	PGN-EDO23	鏇築夢蓋窪齋顧膚選遞(齋窪窪廠選製醖顧餘壓) = 觸憲鑰蓋鹽觸構窪遞夢糧餘繭淵鑰簾壓廠構顧 (選獵鏇鹽鬱夢廠網廠衊 ) 更多	积极	2024-03-03
ASGCT2023	临床1期	杜氏肌营养不良症	-	PGN-EDO51 1 mg/kg	齋廠襯糧鹹鏇憲構淵鏇(膚顧壓壓鏇窪獵廠廠選) = The majority of treatment-emergent adverse events were mild and resolved without intervention, including transient, reversible changes in kidney biomarkers (n=9) and hypomagnesemia (n=2) at the highest doses, with no significant clinical sequelae 壓選艱繭蓋衊積構鬱觸 (製顧鬱醖衊簾鹽鏇壓廠 )	积极	2023-05-01
				PGN-EDO51 5 mg/kg
P3-8.004 (AAN2023)	临床1期	杜氏肌营养不良症	-	PGN-EDO51 1 mg/kg	顧壓範夢觸醖憲壓淵廠(獵構遞積鏇艱鏇齋製窪) = transient, reversible changes in kidney biomarkers (n=9) and hypomagnesemia (n=2) at the highest doses; with no significant clinical sequelae. At 15 mg/kg, one HV received IV hydration after increases in kidney biomarkers with complete resolution. 網鏇網觸鏇鬱願簾鬱憲 (製膚遞築艱製築膚襯鏇 ) 更多	-	2023-04-25
				PGN-EDO51 5 mg/kg
MDA2023	临床1期	杜氏肌营养不良症 kidney biomarkers \| hypomagnesemia	-	PGN-EDO51 1 mg/kg	範製鏇遞衊構鏇鹹觸膚(窪鏇艱壓鬱鹽網齋齋醖) = The majority of treatment-emergent adverse events were mild and resolved without intervention, including transient, reversible changes in kidney biomarkers (n=9) and hypomagnesemia (n=2) at the highest doses, with no significant clinical sequelae 壓窪簾膚簾蓋艱遞選齋 (鏇蓋構艱憲廠鹽糧鹹壓 )	积极	2023-03-19
				PGN-EDO51 5 mg/kg
GlobeNewswire 人工标引	临床1期	杜氏肌营养不良症 Exon 51	32	PGN-EDO51	夢襯壓鑰鬱艱襯簾襯遞(鹽壓築醖衊廠淵齋範鬱) = mild and resolved without any intervention 衊製鑰艱積齋壓憲醖齋 (糧襯憲鑰淵選鏇壓淵醖 ) 更多	积极	2022-09-28
				Placebo