PepGen报告了在营养不良患者中最高的剪接矫正，股票翻倍-动脉网

PepGen Reports Highest Splicing Correction In Dystrophy Patients, Stock Doubles

benzinga 等信源发布 2025-09-25 09:46

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PepGen Inc.

佩普根公司

(NASDAQ:

（纳斯达克：

PEPG

) stock is skyrocketing on Thursday, with a session volume of 53.04 million compared to the average volume of 943.2 thousand

）股票在周四暴涨，当日成交量为5304万股，而平均成交量为94.32万股。

as per data from Benzinga Pro

根据Benzinga Pro的数据

。

PepGen on Wednesday released clinical data today from the 15 mg/kg dose cohort of its ongoing FREEDOM-DM1 Phase 1 single ascending dose (SAD) study in patients with myotonic dystrophy type 1 (DM1).

PepGen于周三发布了其正在进行的FREEDOM-DM1一期单次递增剂量（SAD）研究中15 mg/kg剂量组的临床数据，该研究针对的是1型肌强直性营养不良（DM1）患者。

Myotonic dystrophy is a rare, inherited genetic disorder characterized by progressive muscle weakness and myotonia, which is the inability of muscles to relax after a contraction.

肌强直性营养不良是一种罕见的遗传性疾病，其特征是进行性肌肉无力和肌强直，即肌肉在收缩后无法放松。

It is a multisystemic condition affecting not only muscles but also the heart, brain, eyes, endocrine system, and other organs.

它是一种多系统疾病，不仅影响肌肉，还影响心脏、大脑、眼睛、内分泌系统和其他器官。

Also Read:

另请阅读：

PepGen Abandons Duchenne Program, Shifts Spotlight To Another Rare Disease Candidate

PepGen放弃杜氏肌营养不良项目，将焦点转向另一种罕见病候选药物

Data

数据

FREEDOM Results for the 15 mg/kg (n=8) dose cohort, splicing and

15 mg/kg（n=8）剂量组的FREEDOM结果，剪接和

muscle concentration data

肌肉浓度数据

：

Mean splicing correction of 53.7% in patients receiving 15 mg/kg of PGN-EDODM1 (n=6), as measured by the 22-gene panel at 28 days post-dosing. The splicing deregulation induces an aberrant protein expression that provokes the loss of cell function and viability.

接受15 mg/kg PGN-EDODM1治疗的患者（n=6）在给药后28天通过22基因面板测量，平均剪接校正率为53.7%。剪接失调诱导异常蛋白表达，导致细胞功能和活力的丧失。

The company previously reported mean splicing correction at 5 mg/kg (n=6) and at 10 mg/kg (n=4) of 12.3% and 29.1%, respectively, demonstrating greater than dose-proportional increases

公司此前报告了在 5 mg/kg（n=6）和 10 mg/kg（n=4）剂量下平均剪接校正率分别为 12.3% 和 29.1%，显示出高于剂量比例的增长。

in splicing correction

在剪接校正中

。

All six patients (100%) receiving a 15 mg/kg dose of PGN-EDODM1 responded to treatment by showing improved splicing correction.

所有六名接受15 mg/kg剂量PGN-EDODM1治疗的患者（100%）均显示出剪接修复改善，对治疗有反应。

In addition, greater than dose-proportional increases in muscle tissue concentrations of PGN-EDODM1 were observed across 5 mg/kg (n=6), 10 mg/kg (n=5) and 15 mg/kg (n=6) at Day 28.

此外，在第28天，观察到PGN-EDODM1在肌肉组织中的浓度增加大于剂量比例，分别为5 mg/kg（n=6）、10 mg/kg（n=5）和15 mg/kg（n=6）。

PGN-EDODM1 was generally well-tolerated at 15 mg/kg, with no serious treatment-related adverse events (AEs).

PGN-EDODM1 在 15 mg/kg 的剂量下通常具有良好的耐受性，未出现严重的治疗相关不良事件 (AEs)。

All treatment-related AEs at 15 mg/kg were mild or moderate, transient, and with the exception of one patient (who received oral OTC antihistamines), did not require intervention.

所有与治疗相关的15 mg/kg剂量下的不良事件（AEs）均为轻度或中度，短暂出现，除了一名患者（接受了口服非处方抗组胺药）外，均无需干预。

There were no electrolyte-related AEs, including an absence of hypomagnesemia AEs.

没有电解质相关的不良事件，包括没有低镁血症的不良事件。

All renal biomarker-related AEs were asymptomatic, transient (~48hrs) and resolved without intervention.

所有与肾脏生物标志物相关的不良事件均无症状、短暂（约48小时），并且无需干预即可恢复。

A transient and reversible kidney biomarker elevation in one patient qualified as a dose-limiting toxicity, as defined by the study protocol, and was classified as a mild AE, which resolved without intervention. The patient remained in the study.

一名患者出现短暂且可逆的肾脏生物标志物升高，根据研究方案定义为剂量限制性毒性，并被归类为轻度不良事件（AE），无需干预即可恢复。该患者继续留在研究中。

What Next?

接下来是什么？

PepGen anticipates reporting results from the FREEDOM2-DM1 MAD study 5 mg/kg cohort in the first quarter of 2026 and also expects to begin dosing its 10 mg/kg cohort in the first quarter of 2026.

PepGen预计将在2026年第一季度报告FREEDOM2-DM1 MAD研究5 mg/kg队列的结果，并预计在2026年第一季度开始对其10 mg/kg队列进行给药。

On Thursday, PepGen priced its offering of 31.25 million shares at $3.20 per share, gross proceeds

周四，PepGen 将其 3125 万股的发行价定为每股 3.20 美元，总收益为

expected to be $100 million

预计将达到1亿美元

。

PEPG Price Action:

PEPG 价格行为：

PepGen shares were up 122.18% at $5.91 at the time of publication on Thursday. The stock is trading within its 52-week range of $0.88 to $9.93

截至周四发布的数据，PepGen的股价上涨了122.18%，达到5.91美元。该股票在其52周范围内的交易区间为0.88美元至9.93美元。

, according to Benzinga Pro data

，根据Benzinga Pro数据