PepGen To Voluntarily Pause Mid-Stage Duchenne Muscular Dystrophy Study, Stock Falls

On Tuesday, PepGen Inc. PEPG announced its voluntary decision to temporarily pause the Phase 2 CONNECT2-EDO51 study of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD) until the company can review results from the 10 mg/kg cohort.

The first two cohorts of the CONNECT1 study are fully enrolled and data from the 10 mg/kg cohort are expected during the third quarter of 2025. No new safety issues related to PGN-EDO51 have been observed since the company’s last safety update as of January 23, 2025.

Also Read: Capricor Therapeutics Cell Therapy Goes Under Priority FDA Review For Duchenne Associated-Heart Condition

“With our 10 mg/kg cohort of CONNECT1 study fully enrolled and data expected later this year, we decided to pause CONNECT2 until we are able to review results from the 10 mg/kg cohort in patients with DMD. This will allow us to gather additional safety data, assess the impact of this dose of PGN-EDO51 on dystrophin levels, and potentially improve the design of CONNECT2,” said James McArthur, president and CEO of PepGen.

“This decision enables us to focus resources on completing CONNECT1, as well as rapidly advancing our FREEDOM studies in myotonic dystrophy type 1 with PGN-EDODM1, in which we recently reported encouraging initial clinical data from the Phase 1 FREEDOM-DM1 study,” McArthur added.

In February, PepGen announced early data from its FREEDOM-DM1 trial for myotonic dystrophy.

The study showed significant dose-dependent splicing correction, with mean corrections of 12.3% at 5 mg/kg and 29.1% at 10 mg/kg at 28 days post-dosing.

Price Action: PEPG stock is down 21% at $2.22 at the last check Tuesday.

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