Oligonucleotide Therapeutic Gets Fast Track Status for Myotonic Dystrophy Type 1

The Food and Drug Administration (FDA) has granted Fast Track designation to PGN-EDODM1 for the treatment of myotonic dystrophy type 1 (DM1).

DM1 is a rare genetic disorder caused by a triplet-repeat in the myotonic dystrophy protein kinase (DMPK) gene, resulting in toxic gain of function mRNA. The disease primarily affects skeletal and cardiac muscle and is characterized by muscle weakness, difficulty breathing, and cognitive and behavioral impairment.

PGN-EDODM1 is designed to restore cellular function by delivering a peptide-conjugated antisense oligonucleotide that blocks the toxic DMPK transcript CUG repeats. Early findings showed 4 doses of PGN-EDODM1 reversed 99% of myotonia and corrected 99% of mis-splicing in a DM1 murine model.

The Company is currently investigating PGN-EDODM1 in the randomized, placebo-controlled, phase 1 FREEDOM-DM1 trial (ClinicalTrials.gov Identifier: NCT06204809). The study is expected to enroll 24 adult patients with DM1 and will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of single ascending doses of PGN-EDODM1 administered by intravenous infusion.

“Receiving Fast Track designation from the FDA for PGN-EDODM1 is a significant milestone in our efforts to deliver a potentially transformative therapy to the DM1 community,” said James McArthur, PhD, President and CEO of PepGen. “Patients with DM1 currently have no available treatment options that target the root cause of the disease, which leads to progressive neuromuscular symptoms and reduction in life expectancy. We believe that PGN-EDODM1 has the potential to be disease-modifying and improve outcomes for patients living with DM1.”