Entrada Therapeutics Presents Breakthrough DMD Data At World Muscle Congress 2024

Entrada Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing treatments for previously unmet intracellular targets, announced the presentation of clinical and preclinical data at the 29th Annual Congress of the World Muscle Society in Prague, Czechia, from October 8 to 12, 2024. The data highlights progress in its Duchenne muscular dystrophy (DMD) program.

Natarajan Sethuraman, PhD, President of R&D at Entrada Therapeutics. Said, “We are excited to present the data that supports the advancement of our Duchenne franchise. Adding to our previously reported positive data from our Phase 1 ENTR-601-44-101 trial, we are presenting further safety data demonstrating that there were no adverse findings or clinically relevant changes to any biomarkers of renal toxicity measured at the highest dose tested during the study. We are also pleased to present new data from preclinical studies of ENTR-601-45, showing compelling in vivo dystrophin production and functional improvement.” 

Dr. Sethuraman continued, “We are on track to submit regulatory applications this quarter to initiate separate global Phase 2 clinical trials for ENTR-601-44 and ENTR-601-45 in patients with Duchenne who are exon 44 skipping and exon 45 skipping amenable, respectively. In addition, we plan to submit regulatory applications in 2025 to initiate a global Phase 2 clinical trial for our third Duchenne candidate, ENTR-601-50, in patients who are exon 50 skipping amenable.”

Dr Natarajan Sethuraman, PhD, President of R&D Entrada Therapeutics, presented a poster on- Therapeutic Potential of ENTR-601-44, an Endosomal Escape Vehicle (EEV™)–Oligonucleotide Conjugate for the Treatment of Exon 44 Skip-Amenable Duchenne Muscular Dystrophy

The key highlights of the poster presentation are as follows-

The Phase 1 study in healthy male volunteers met all objectives, with no adverse events linked to the drug. Urine PK data and kidney function assessments showed no signs of renal toxicity at the highest dose (6 mg/kg). Significant exon skipping and dose-dependent metabolite increases were observed, marking a breakthrough in the safe and effective delivery of oligonucleotide therapies to skeletal muscle.

Dr. Sweta Girgenrath, VP and Head of Cardiovascular and Neuromuscular Therapeutics, presented  Exon 45 Skipping and Dystrophin Production with ENTR-601-45 in Preclinical Models of Duchenne Muscular Dystrophy.

The highlights showed strong dose-dependent exon skipping and dystrophin restoration in both in vitro and in vivo models, with improved muscle function in a DMD mouse model. At the highest dose, dystrophin production and muscle function were comparable to healthy controls, underscoring ENTR-601-45's potential and supporting its further development.