US-based biotechnology company PepGen has secured a no objection letter for its clinical trial application from Health Canada to begin a Phase II trial of PGN-EDO51.
The CONNECT1-EDO51 trial will evaluate PGN-EDO51’s ability to treat Duchenne muscular dystrophy (DMD) patients amenable to an exon 51 skipping approach.
It will evaluate dystrophin levels, exon skipping and safety data after different doses of PGN-EDO51 in these patients, as well as the drug’s tolerability, pharmacokinetics and clinical assessments.
The open-label, multiple ascending dose (MAD) trial is due to start dosing patients in the second half of this year.
As part of the trial, PepGen aims to assess PGN-EDO51 in around three cohorts of ambulatory and non-ambulatory boys and young men, beginning at a dose of 5mg/kg.
The firm expects to increase the dose to 10mg/kg and potentially introduce other doses after a review from the Drug Safety Monitoring Board (DSMB).
It expects an initial data readout by mid-next year.
PepGen senior vice-president and clinical development head Michelle Mellion said: “PGN-EDO51 exhibited the highest levels of oligonucleotide delivery and exon 51 skipping in a clinical study following a single dose of 5mg/kg, 10mg/kg and 15mg/kg in healthy volunteers when compared to publicly available clinical data for other exon 51-skipping approaches.
“At these dose levels, the majority of treatment-emergent adverse events were assessed as mild and resolved without any intervention.
“Looking ahead, and based on our nonclinical data, we believe CONNECT1-EDO51 may support a differentiated profile for PGN-EDO51 relative to other investigational and approved therapies based on previously observed meaningful and durable data on dystrophin production, as well as clinical assessments.”
Based in Massachusetts, PepGen is a clinical-stage biotechnology company that aims to develop the next generation of oligonucleotide therapies for severe neuromuscular and neurological diseases.
The company makes use of cell-penetrating peptides to increase the uptake and activity of conjugated oligonucleotide therapeutics.