PepGen Announces Positive Phase 2 FREEDOM2-DM1 10 mg/kg Results Demonstrating Highest Reported Splicing Correction and Improvements Across Multiple Functional Measures

James McArthur, President and Chief Executive Officer of PepGen

“Our ambition is to fundamentally change how DM1 is treated through greater targeting of the underlying molecular mechanism of the disease.”

James McArthur, President and Chief Executive Officer of PepGen

“The 10 mg/kg results mark an important milestone in that pursuit, with robust splicing correction accompanied by encouraging improvements across multiple measures of muscle function.”

James McArthur, President and Chief Executive Officer of PepGen

“Importantly, these findings strengthen our confidence not only in PGN-EDODM1’s potential, but in our broader development strategy.”

James McArthur, President and Chief Executive Officer of PepGen

“With our 12.5 mg/kg cohort fully enrolled and the ongoing FREEDOM-OLE study exceeding 135 doses administered, we believe we are well positioned to continue building the evidence needed to advance toward pivotal studies.”

Hanns Lochmüller, Professor of Neurology at the University of Ottawa, Senior Scientist at the CHEO Research Institute, Neurologist at The Ottawa Hospital, and FREEDOM2 study investigator

“DM1 is a multisystemic disease, placing a major burden on patients and their families.”

Hanns Lochmüller, Professor of Neurology at the University of Ottawa, Senior Scientist at the CHEO Research Institute, Neurologist at The Ottawa Hospital, and FREEDOM2 study investigator

“The improvements observed across myotonia, mobility and grip strength are encouraging because they reflect functions that affect patients’ daily lives.”

Hanns Lochmüller, Professor of Neurology at the University of Ottawa, Senior Scientist at the CHEO Research Institute, Neurologist at The Ottawa Hospital, and FREEDOM2 study investigator

“The relationship between splicing correction and improvements in myotonia is particularly interesting, and longer-term follow-up will be important to better understand the durability and clinical relevance of these findings.”

Paul Streck, Executive Vice President, Head of Research and Development of PepGen

“For people living with DM1, the impact extends far beyond any single symptom, underscoring the need for treatments that target the root cause of the disease.”

Paul Streck, Executive Vice President, Head of Research and Development of PepGen

“We are extremely encouraged by the magnitude of splicing correction observed at 10 mg/kg and the dose-dependent improvements across multiple measures of muscle function.”

Paul Streck, Executive Vice President, Head of Research and Development of PepGen

“Together, these findings strengthen our confidence in PGN-EDODM1’s potential to deliver meaningful benefit for patients.”

Paul Streck, Executive Vice President, Head of Research and Development of PepGen

“We are deeply grateful to the FREEDOM2 investigators, study teams, participants, and their families for their dedication and commitment to this study.”