The trial aims to evaluate PGN-EDO51 across up to three different dose levels in DMD patients. Credit: Kateryna Kon / Shutterstock.

PepGen has reported positive data from the first dose cohort (5mg/kg) of the ongoing CONNECT1-EDO51 Phase II clinical trial of PGN-EDO51 for the treatment of Duchenne muscular dystrophy (DMD).

PGN-EDO51 is an investigational therapy that is designed for patients whose DMD mutations are amenable to an exon 51-skipping approach.

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The open-label, multiple ascending dose study aims to evaluate PGN-EDO51 across up to three different dose levels.

The starting dose of 5mg/kg is administered intravenously every four weeks over a 12-week period.

Safety, dystrophin production, and exon skipping are the primary endpoints of this trial. Multiple pharmacokinetic parameters are also being evaluated as part of the study protocol.

According to the findings, PGN-EDO51 showed higher levels of exon skipping compared to other oligonucleotide therapies at similar dose levels in DMD patients.

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In addition, the therapy achieved a mean muscle-adjusted dystrophin level of 1.49% of normal and a 0.70% change from baseline following four doses.

A mean absolute dystrophin level of 0.61% of normal was reported, with a 0.26% change from baseline, as measured by Western blot analysis at week 13.

The 5mg/kg dose was well tolerated by all trial participants through to week 13, without any cases of discontinuations, dose interruptions, or reductions recorded.

The only related treatment-emergent adverse event was mild and resolved without complications.

There were also no significant changes in kidney biomarkers, electrolytes, and hepatic function, as well as no cases of anaemia or thrombocytopenia.

All three patients in the low-dose cohort are continuing treatment with PGN-EDO51 at 5mg/kg in the long-term extension phase of the trial, maintaining a good tolerance to the drug as of 29 July 2024.

The trial has also begun dosing participants at 10mg/kg, with initial results from this cohort expected in early 2025.

PepGen CEO and president James McArthur said: “We are encouraged by the early data from our CONNECT1 clinical trial of PGN-EDO51 in people with DMD.

“In three months, the starting monthly dose of 5mg/kg achieved high levels of exon skipping and all patients showed increases in dystrophin.

“PGN-EDO51 produced meaningfully higher levels of exon skipped transcript at lower doses and in a shorter time period, compared to other exon 51 therapies approved and in development, indicating that our Enhanced Delivery Oligonucleotide technology is delivering higher levels of oligonucleotide to the nuclei.”