In after-hours trading, PepGen Inc. (NASDAQ: PEPG) stock value jumped 10.29% to $2.68, a substantial rise. It closed at $2.43 during regular trading hours, following an incredible 58.82% increase. The increase in investor confidence is due to the company’s recent revelation of good clinical trial findings for PGN-EDODM1, an investigational therapy for myotonic dystrophy type 1 (DM1).
Important Findings from the FREEDOM-DM1 Trial
PepGen said that the 5 mg/kg and 10 mg/kg dose groups have shown promising preliminary findings from the FREEDOM-DM1 Phase 1 clinical investigation, which is now in progress. The research assesses the possibility of PGN-EDODM1 in treating DM1, a crippling neuromuscular condition. Following a single treatment, the data showed substantial splicing repair, exceeding expectations.
Given that mis-splicing is the primary cause of DM1 disease, these discoveries represent a significant advancement in the possible creation of therapeutics. The splicing correction shown 28 days after delivery at 10 mg/kg was remarkably better than those seen in multi-dose clinical studies that lasted up to nine months.
With this result, PepGen’s Enhanced Delivery Oligonucleotide (EDO) technology shows promise in efficiently delivering therapeutic oligonucleotides to target cells, providing patients without authorized therapy alternatives with fresh hope.
Future Development and Expanded Study Plans
The initial data provide strong validation of PGN-EDODM1’s capability to selectively bind to pathogenic CUG-repeat DMPK RNA. Encouraged by these promising results, PepGen plans to expand its evaluation through the FREEDOM2-DM1 multiple ascending dose study. The company aims to explore additional dose levels over extended durations to enhance therapeutic benefits and functional improvements for DM1 patients.
Although single-dose studies have yet to demonstrate significant functional improvements, early trends suggest potential benefits in some outcome measures. Repeated dosing could yield meaningful functional gains over time, reinforcing the importance of continued research and development.
Encouraging Safety Profile and Future Outlook
PGN-EDODM1 has shown a favorable emerging safety profile in both the 5 mg/kg and 10 mg/kg cohorts. PepGen anticipates releasing results from the FREEDOM 15 mg/kg cohort in the latter half of 2025 and from the FREEDOM2 5 mg/kg cohort in early 2026. These forthcoming milestones could further cement PGN-EDODM1’s role in addressing DM1, offering new hope for patients and investors alike.