PepGen(US:PEPG)2025-11-10 17:30Summary of PepGen Conference Call Company Overview - Company: PepGen - Event: Guggenheim's 2025 Healthcare Innovations Conference - Key Presenters: James McArthur (President and CEO), Paul Strak (EVP and Head of R&D) [1][2] Industry Focus - Industry: Neuromuscular diseases - Technology: EDO platform technology for delivering therapeutic oligonucleotides [3] Core Points and Arguments 1. Clinical Study Results: - Reported a 54% splicing improvement at the top dose of 15 mg/kg in the Freedom clinical study, significantly higher than the previous 22% improvement achieved with a different approach [3][4] - The study showed that the treatment was generally safe, with no serious adverse events reported [4] 2. Future Expectations: - Anticipates reporting results from a multiple ascending dose study in Q1 2024, aiming to build upon the 12% splicing improvement seen with a single dose [4][9] - Plans to report data from a second cohort at 10 mg/kg in the second half of 2024, expecting to improve upon the 29% splicing improvement previously reported [4] 3. Market Potential: - The DM1 patient population is significantly larger than that of SMA, with no approved therapies currently available for DM1 [5][18] 4. Therapeutic Index: - Observed a better-than-dose-dependent increase in muscle concentration and splicing, with mild transient changes in renal function noted [4][5] 5. Preclinical Data: - In a mouse model, a three-and-a-half-fold increase in oligo concentration in muscle was observed with multiple doses, leading to nearly complete splicing improvement [6] 6. Safety Monitoring: - Safety is monitored in real-time during trials, with a focus on ensuring no adverse effects from the treatment [7] 7. Regulatory Pathways: - The company is considering both accelerated approval pathways and a single large phase three study, depending on the outcomes of the ongoing studies [16] 8. Differentiation from Competitors: - Emphasizes the unique targeting approach of the EDO platform, which focuses on pathogenic RNA, potentially leading to better outcomes compared to existing therapies [13][18] 9. Secondary Endpoints: - Plans to monitor additional endpoints such as 10-meter walk/run and hand grip strength, with optimism for improvements in larger muscle groups [14] 10. Future Indications: - While currently focused on DM1, the company is exploring other neuromuscular indications like Charcot-Marie-Tooth for future development [20] Additional Important Points - Delivery Method: Currently focused on IV formulation, with no immediate plans to shift to subcutaneous delivery [19] - Patient Enrollment: Plans to enroll patients both in the U.S. and internationally for ongoing studies [22] - Timeline for Data: Expected data for the 5 mg/kg dose in Q1 2024 and for the 10 mg/kg dose in the second half of 2024 [21][22]