PGN-EDO51

The FDA granted breakthrough therapy designation to STK-001 for Dravet syndrome, highlighting its potential to improve upon current treatments by restoring NaV1.1 protein levels.

The FDA has placed a clinical hold on PepGen's Phase 2 CONNECT2-EDO51 trial for PGN-EDO51 in Duchenne muscular dystrophy (DMD) due to regulatory questions.

• The FDA has approved Neurocrine's Crenessity as an adjunct therapy for classic congenital adrenal hyperplasia, offering patients a way to reduce steroid treatment burden. • Checkpoint Therapeutics received FDA approval for Unloxcyt to treat advanced cutaneous squamous cell carcinoma, targeting a $1 billion market opportunity. • Galderma's Nemluvio gains FDA approval for moderate-to-severe atopic dermatitis in patients 12 and older, with projected peak annual sales exceeding $2 billion.

PepGen's PGN-EDO51 Phase 2 study shows promising initial data with mean exon skipping in biceps tissue of 2.15% at week 13 for Duchenne muscular dystrophy.

PepGen anticipates releasing preliminary data from its Phase II CONNECT1-EDO51 trial of PGN-EDO51 in mid-2024, offering a potential treatment for Duchenne muscular dystrophy (DMD).