Entrada Therapeutics Announces Positive Results from the First Cohort of Participants with Duchenne Muscular Dystrophy (DMD) in the Phase 1/2 ELEVATE-45-201 Study of ENTR-601-45

Natarajan Sethuraman, President of Research and Development at Entrada Therapeutics

“Establishing a favorable safety profile in Cohort 1 was our main objective and we are pleased with the outcome. These positive safety results, combined with an increase in dystrophin across treated participants at the initial low dose of 5 mg/kg, are promising. Importantly, ENTR-601-45 is designed to be dosed once every six weeks. For families currently managing a weekly infusion schedule, ENTR-601-45’s current dosing schedule translates to a potential therapy built around approximately nine administrations per year instead of more than fifty”

Natarajan Sethuraman, President of Research and Development at Entrada Therapeutics

“Additionally, an Independent Data Monitoring Committee’s recent recommendation to proceed to the highest dose of 18 mg/kg in Cohort 3 of the ELEVATE-44-201 clinical study supports our confidence in the opportunity to escalate the dose up to 15 mg/kg in Cohort 3 of ELEVATE-45-201, where we expect to see meaningfully higher exposure, dystrophin production and functional benefit.”

Giovanni Baranello, Professor of Paediatric Neuromuscular Disorders at UCL and Honorary Consultant in Paediatric Neuromuscular Diseases at the Great Ormond Street Hospital, London, and principal investigator in the ELEVATE-45-201 clinical study

“People with a confirmed mutation in the DMD gene who are amenable to exon 45 skipping represent a significant portion of the DMD patient population. As one of the most advanced next-generation exon skipping conjugate PMOs in clinical trials, ENTR-601-45 has the potential to offer a well-tolerated, low-frequency of dosing option for a large Duchenne subpopulation with a clear and increasing unmet need”

Giovanni Baranello, Professor of Paediatric Neuromuscular Disorders at UCL and Honorary Consultant in Paediatric Neuromuscular Diseases at the Great Ormond Street Hospital, London, and principal investigator in the ELEVATE-45-201 clinical study

“Importantly, establishing a favorable safety profile at this initial dose in a three dose MAD study is the essential foundation for escalating to higher doses, and seeing any dystrophin produced in participants this young, with such low baseline levels, is an encouraging early signal.”