PEPGEN

• Phase I/II Deliver trial demonstrates sustained functional improvements in DMD patients treated with DYNE-251, showing mean absolute dystrophin expression of 8.72% above baseline at six months with the 20mg/kg dose. • The therapy, designed for patients amenable to exon 51 skipping, showed improvements across multiple functional endpoints including mobility assessments, positioning Dyne for potential accelerated FDA approval submission in early 2026. • If approved, DYNE-251 could generate significant market impact, with GlobalData projecting revenue growth from $5 million in 2025 to $129 million by 2030 in the competitive DMD treatment landscape.

Initial results from FREEDOM-DM1 trial demonstrate significant splicing correction of 29.1% at 10 mg/kg dose in patients with Myotonic Dystrophy Type 1, showing potential therapeutic efficacy.

• 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. • Tolebrutinib received breakthrough therapy designation for non-relapsing secondary progressive multiple sclerosis based on phase 3 trial results showing delayed disability progression. • The FDA placed a clinical hold on PepGen’s PGN-EDO51 phase 2 study for Duchenne muscular dystrophy, pending further clarification from the agency.

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.

• The myotonic dystrophy therapeutic landscape is experiencing a surge in R&D, with over 20 companies actively involved in developing 22+ novel therapies. • Key players like Avidity Biosciences, Dyne Therapeutics, and Vertex Pharmaceuticals are advancing promising drugs such as AOC 1001, DYNE-101, and VX-670 through clinical trials. • Regulatory designations, including Breakthrough Therapy and Fast Track, have been granted to several pipeline drugs, expediting their development for myotonic dystrophy. • Recent clinical trial initiations and data presentations highlight the progress in addressing unmet needs in myotonic dystrophy treatment, fostering hope for improved patient outcomes.

• 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. • FREEDOM-DM1 Phase 1 study data for myotonic dystrophy type 1 patients, assessing safety, tolerability, and splicing correction, is expected by the end of Q1 2025. • PepGen anticipates current funds will sustain operations into 2026, while acknowledging the necessity for additional capital through various financial avenues. • The company faces challenges including financial losses, clinical trial risks, and reliance on third parties, as detailed in its recent SEC 10-Q report.

• 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). • The CONNECT1-EDO51 trial assesses the safety, exon 51 skipping, and dystrophin protein production in male patients with DMD amenable to exon 51 skipping. • PGN-EDO51 utilizes PepGen's Enhanced Delivery Oligonucleotide (EDO) technology to target the genetic root cause of DMD, a muscle-wasting disease. • PepGen is also advancing PGN-EDODM1 for myotonic dystrophy type 1 (DM1), with Phase I trial data expected this year, alongside preclinical DMD programs.