PepGen Inc., a clinical-stage biotechnology company advancing the next-generation of oligonucleotide therapies, today announced that the first patient has been dosed in its CONNECT1-EDO51 Phase 2, open-label multiple ascending dose (MAD) clinical trial evaluating PGN-EDO51 for the treatment of Duchenne muscular dystrophy (DMD) patients amenable to an exon 51 skipping therapy.
PGN-EDO51, PepGen’s lead clinical candidate for the treatment of DMD, utilises the company’s proprietary Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic oligonucleotide that is designed to target the root cause of DMD. PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, and in doing so restores the reading frame of the gene and produces a truncated, yet functional, version of the dystrophin protein.
The phase 1 trials of PGN-EDO51 showed promising results, with a single dose of 10 mg/kg of PGN-EDO51 in healthy volunteers exhibiting peak exon 51 skipping of 3.8% with 100% of subjects demonstrating exon skipping.
“We are pleased to have dosed the first patient in our CONNECT1-EDO51 clinical trial, which marks another milestone in our commitment to developing therapies with the potential to truly improve the lives of people living with DMD,” said James McArthur, Ph.D., President and CEO of PepGen. “Based on the levels of exon skipping achieved following a single dose of PGN-EDO51 in our Phase 1 healthy volunteer trial, we are looking forward to our initial planned data readout in DMD patients at the 5 mg/kg PGN-EDO51 dose level for CONNECT1-EDO51 in the middle of 2024.”
Preliminary data from the 5 mg/kg PGN-EDO51 dose level in the CONNECT1-EDO51 Phase 2 trial are expected mid-2024: including initial safety, exon 51 skipping and dystrophin protein production data
Read more about PepGen and the CONNECT1-EDO51 Phase 2 clinical trials
Would you like to know more about Duchenne muscular dystrophy? Increase your knowledge and understanding of Duchenne with our bite-sized science video series.
Section 1 – Facts about Duchenne muscular dystrophy
Section 2 – Signs and Symptoms of Duchenne muscular dystrophy
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