Biotechnology enterprise PepGen Inc. (PEPG) announced on Monday that it has received a clinical hold notification from the U.S. Food and Drug Administration (FDA) concerning its Investigational New Drug (IND) application for initiating the CONNECT2-EDO51 clinical trial involving patients with Duchenne muscular dystrophy (DMD).
The FDA has communicated that an official clinical hold letter will be delivered to the company within 30 days.
CONNECT2 is PepGen's Phase 2 international, double-blind, placebo-controlled trial, involving multiple ascending doses over a 25-week period. This clinical study of PGN-EDO51 in DMD patients is already underway in the United Kingdom.
PepGen operates as a clinical-stage biotechnology firm that is advancing the development of next-generation oligonucleotide therapies, aiming to revolutionize treatment for severe neuromuscular and neurological disorders.
PGN-EDO51, PepGen's therapeutic candidate for DMD, employs the company’s proprietary Enhanced Delivery Oligonucleotide (EDO) technology to administer a therapeutic phosphorodiamidate morpholino oligomer (PMO). This approach is tailored to address the underlying cause of this profoundly impactful disease.
