Entrada Therapeutics (TRDA) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Strategic momentum and clinical pipeline

• Four phase I/II multiple ascending dose (MAD) studies in DMD and DM1 are expected by the end of 2025, with global studies and regulatory applications for 44 and 45 programs submitted late last year.

• Vertex partnership is advancing the DM1 program, with the MAD portion of the VX-670 phase I/II study initiated.

• 50 program regulatory filings expected in H2 2025, with phase I study starting Q4 2025; 51 program filings planned for early 2026.

• Preclinical pipeline is expanding beyond neuromuscular diseases, with new programs generating data and initial focus on ocular and metabolic diseases.

• Financial position is strong, ending 2024 with approximately $420M in cash and a runway extending into Q2 2027.

Technology platform and pipeline expansion

• Endosomal Escape Vehicle (EEV) technology enables efficient intracellular delivery, with up to 50% endosomal escape and ~90% cellular uptake, supporting broad biodistribution and consistent pharmacokinetics.

• EEV platform is used across initial programs, with unique chemistry and a budding mechanism that conserves endosomal integrity.

• Platform supports expansion into gene editing, mRNA delivery, antibody/protein therapeutics, and metabolic diseases.

• 75% of disease targets are intracellular, and the platform is designed for broad applicability.

• Vertex partnership and positive phase I data in DMD validate the EEV approach, supporting best-in-class potential.

Clinical and preclinical data highlights

• ENTR-601-44 phase I showed no treatment-related adverse effects up to 6 mg/kg, robust target engagement, and flexible six-week dosing.

• Dose-dependent plasma and muscle concentrations observed, supporting efficacy at low doses without proportional toxicity risk.

• Statistically significant exon skipping and muscle concentration were observed at 6 mg/kg, supporting initiation of phase I/II MAD studies.

• Preclinical data for ENTR-601-45, ENTR-601-50, and ENTR-601-51 show robust, dose-dependent dystrophin restoration and functional correction in mouse models.

• All future exon programs to go direct to patient studies, leveraging healthy volunteer data from 44 program.