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）

2025-06-24

·PRNewswire

August 8, 2025 Deadline: Contact Levi & Korsinsky to Join Class Action Suit Against PEPG

NEW YORK, June 24, 2025 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in PepGen Inc. ("PepGen Inc." or the "Company") (NASDAQ: PEPG) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of PepGen Inc. investors who were adversely affected by alleged securities fraud between March 7, 2024 and March 3, 2025. Follow the link below to get more information and be contacted by a member of our team: PEPG investors may also contact Joseph E. Levi, Esq. via email at [email protected] or by telephone at (212) 363-7500. CASE DETAILS: The filed complaint alleges that defendants made false statements and/or concealed that: (i) the Company's lead product candidate, PGN-EDO51, was less effective and safe than defendants had led investors to believe; (ii) phase two, 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. WHAT'S NEXT? If you suffered a loss in PepGen Inc. during the relevant time frame, you have until August 8, 2025 to request that the Court appoint you as lead plaintiff. Your ability to share in any recovery doesn't require that you serve as a lead plaintiff. NO COST TO YOU: If you are a class member, you may be entitled to compensation without payment of any out-of-pocket costs or fees. There is no cost or obligation to participate. WHY LEVI & KORSINSKY: Over the past 20 years, the team at Levi & Korsinsky has secured hundreds of millions of dollars for aggrieved shareholders and built a track record of winning high-stakes cases. Our firm has extensive expertise representing investors in complex securities litigation and a team of over 70 employees to serve our clients. For seven years in a row, Levi & Korsinsky has ranked in ISS Securities Class Action Services' Top 50 Report as one of the top securities litigation firms in the United States. CONTACT: Levi & Korsinsky, LLP Joseph E. Levi, Esq. Ed Korsinsky, Esq. 33 Whitehall Street, 17th Floor New York, NY 10004 [email protected] Tel: (212) 363-7500 Fax: (212) 363-7171 SOURCE Levi & Korsinsky, 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2期

2025-06-12

·PRNewswire

PEPG Investor Notice: Robbins LLP Reminds Investors of the Class Action Lawsuit Against PepGen Inc.

SAN DIEGO, June 12, 2025 /PRNewswire/ -- Robbins LLP reminds stockholders that a class action was filed on behalf of investors who purchased or otherwise acquired PepGen Inc. (NASDAQ:PEPG) securities between March 7, 2024 and March 3, 2025. PepGen is a clinical-stage biotechnology company that focuses on the development of oligonucleotide therapeutics for use in the treatment of severe neuromuscular and neurologic diseases. The Company's lead product candidate was PGN-EDO51, a proprietary enhanced delivery oligonucleotide ("EDO") peptide for the treatment of Duchenne muscular dystrophy ("DMD"). For more information, submit a form, email attorney Aaron Dumas, Jr., or give us a call at (800) 350-6003. The Allegations: Robbins LLP is Investigating Allegations that PepGen Inc. (PEPG) Misled Investors Regarding the Viability of its Drug Candidate According to the complaint, defendants failed 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. The complaint alleges that 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." On this news, PepGen's stock price fell $0.40 per share, or 21.74%, to close at $1.44 per share on January 30, 2025. Then, 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. What Now: You may be eligible to participate in the class action against PepGen Inc. Shareholders who want to serve as lead plaintiff for the class must file a motion for lead plaintiff by August 8, 2025. The lead plaintiff is a representative party who acts on behalf of other class members in directing the litigation. You do not have to participate in the case to be eligible for a recovery. If you choose to take no action, you can remain an absent class member. For more information, click here. All representation is on a contingency fee basis. Shareholders pay no fees or expenses. About Robbins LLP: A recognized leader in shareholder rights litigation, the attorneys and staff of Robbins LLP have been dedicated to helping shareholders recover losses, improve corporate governance structures, and hold company executives accountable for their wrongdoing since 2002. To be notified if a class action against PepGen Inc. settles or to receive free alerts when corporate executives engage in wrongdoing, sign up for Stock Watch today. Attorney Advertising. Past results do not guarantee a similar outcome. SOURCE Robbins LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

寡核苷酸

2025-06-01

·药事纵横

寡核苷酸biotech：研究失败，终止全部DMD项目开发

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。2025年5月28日，PepGen，致力于推进下一代寡核苷酸疗法的临床阶段生物技术公司，宣布根据其针对杜氏肌营养不良症（DMD）的寡核苷酸药物PGN-EDO51在CONNECT1-EDO51研究中表现不佳，公司决定专注于推进目前处于2期临床开发阶段的强直性肌营养不良症1型（DM1）项目PGN-EDODM1。并且，公司自愿停止PGN-EDO51的开发，并计划逐步结束所有与DMD相关的研发活动。研究结果不佳在CONNECT1研究的10 mg/kg队列（n=4）中，PGN-EDO51使外显子51跳跃转录本增加至4.26%（平均增加3.5%），然而，肌营养不良蛋白水平仅升高至正常水平的0.59%（平均增加0.36%）。PGN-EDO51的安全性总体上仍然良好，所有与治疗相关的不良事件均为轻度。研究中未报告任何严重不良事件。早在2024年12月，FDA曾暂停了PGN-EDO51的II期CONNECT2-EDO51在美国的启动。次月，PepGen披露10毫克/千克剂量组中的两名患者报告了无症状镁缺乏症，但在服用口服镁补充剂后，他们的镁水平已恢复到基线水平。 PepGen随后于 2025年3月自愿决定暂停PGN-EDO51的II期CONNECT2-EDO51试验 (NCT06833931)。除了PGN-EDO51，PepGen在DMD治疗领域原本还有另外三个临床前项目PGN-EDO53、PGN-EDO45和PGN-EDO44正在开发。但随着此次的终止决定，PepGen的DMD研发管线将全面清空。PepGen公司总裁兼首席执行官James McArthur博士表示：“我们对CONNECT1研究中10 mg/kg剂量队列观察到的肌营养不良蛋白结果感到失望，因为我们原本希望我们能够以更深远的方式改善患者的现有疗法。在我们逐步结束DMD项目之际，我们想感谢患者、家属、护理人员、研究人员和研究团队对这项研究的支持和参与。我也要感谢我们团队为推进DMD患者潜在新疗法所付出的努力和承诺。”研发重心转移至DM1项目展望未来PepGen公司将集中资源推进DM1临床项目PGN-EDODM1以及研发管线。EDODM1目前正处于两项临床试验阶段，一项为1期临床试验，另一项为2期临床试验。PGN-EDODM1利用PepGen公司专有的增强型寡核苷酸递送（EDO）技术，恢复MBNL1的正常剪接功能。MBNL1是一种关键的RNA剪接蛋白。PGN-EDODM1旨在直接应对肌营养不良蛋白激酶（DMPK）转录本中胞嘧啶-尿嘧啶-鸟嘌呤（CUG）重复序列扩增的有害影响。该药物通过结合DMPK转录本中存在的致病性CUG三核苷酸重复序列扩增，破坏CUG重复序列扩增与MBNL1之间的结合。截至2025年2月24日的最新安全性更新，该药物在患者中单剂量10 mg/kg后已显示出强大的靶点参与度，平均纠正错配剪接的比例达到29%，并且安全性良好。错配剪接是DM1病理的主要驱动因素，目前患者尚无获批的治疗方案来应对这种致残且缩短寿命的疾病。PepGen强调了PGN-EDODM1 的开发时间表，表示预计将于今年下半年公布I期 FREEDOM-DM1 15毫克/千克剂量组的数据。该研究的关键结果包括安全性、28 天剪接校正和功能获益终点。此外，该公司预计II期FREEDOM2-DM1研究的5 毫克/千克剂量组数据将于2026年第一季度公布。然而，PGN-EDODM1在进入临床之前也遇到了不少麻烦。2023年5月，FDA对 FREEDOM-DM1 临床试验的 IND 申请发布了临床暂停令，似乎与药物剂量安全性有关。PepGen仍在其他司法管辖区进行了试验，并在10月份FDA审查了安全数据并批准了剂量递增策略后恢复了试验。参考资料：公司官网

临床2期寡核苷酸临床1期临床终止

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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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	醖鹹鏇簾鏇鏇築顧壓襯(糧糧衊膚鑰願蓋醖簾鏇) = 夢壓襯艱範鬱壓築糧獵範襯醖艱餘網艱鏇獵鹹 (獵鏇淵觸網選窪鹽製膚 ) 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	壓餘鬱範顧糧繭夢廠廠(壓願窪襯窪構淵齋鬱憲) = 壓衊壓餘艱願壓醖顧鹹廠餘繭鹹憲鏇糧壓壓鹹 (夢齋艱艱鑰積蓋蓋糧構 ) 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	網廠觸選願選糧憲顧積(獵淵願製廠製衊淵選鏇) = 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 廠築鏇蓋獵壓築積獵鹹 (憲醖夢簾醖網鹽鹹製廠 )	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	淵選遞襯蓋獵廠壓選淵(範壓鬱選襯憲獵蓋鏇製) = 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. 衊鬱窪鬱襯選醖醖構遞 (餘廠簾簾衊積廠鬱繭憲 ) 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	夢選顧艱積夢鹹廠糧襯(膚廠夢蓋網膚鹽觸簾艱) = 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 構窪鹽繭齋襯襯壓遞壓 (醖艱糧鹽鏇簾醖糧憲鹽 )	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	衊鏇鹹蓋製構艱鬱蓋構(遞膚艱夢蓋鏇糧繭壓簾) = mild and resolved without any intervention 衊製衊願齋憲願獵襯醖 (鑰獵壓膚鏇餘鑰構夢鬱 ) 更多	积极	2022-09-28
				Placebo