Entrada Therapeutics (TRDA) Goldman Sachs 45th Annual Global Healthcare Conference summary

EEV platform and scientific differentiation

• EEV (endosomal escape vehicle) platform enables targeting of previously inaccessible intracellular targets using cyclic cell-penetrating peptides.

• Early preclinical data in mice and non-human primates showed strong muscle penetration and dystrophin production, including in cardiac muscle, supporting efficacy in DMD.

• EEV PMOs demonstrated superior exon skipping in skeletal and cardiac muscle compared to naked PMOs and PPMOs in preclinical models.

• Platform allows for repeat dosing, dose titration, and therapy cessation, addressing gene therapy limitations in DMD and DM1.

Clinical progress and upcoming milestones

• ENTR-601-44, the lead DMD asset, is in a healthy volunteer study in the UK, with key October data to focus on safety, tolerability, and muscle concentration.

• Exon skipping will be measured as a biomarker of target engagement, though not a direct efficacy indicator in healthy volunteers.

• No hypomagnesemia or immune-mediated myositis observed in preclinical models; safety profile remains a top priority.

• Data from the UK study and ongoing research will be submitted to regulatory agencies in Q4, aiming for global phase 2 trial initiation early next year.

Portfolio strategy and collaborations

• The same EEV technology underpins all neuromuscular programs, enabling predictable PK/PD and efficacy across the portfolio.

• Collaboration with Vertex for DM1: Entrada leads preclinical, Vertex leads clinical, regulatory, and commercial activities; 7 sites open for enrollment in Canada, UK, and Australia.

• Partnership with Vertex includes $226M upfront, $24M equity, up to $485M in milestones, and royalties; $75M milestone already achieved.

• Business development remains a focus, with openness to new partnerships beyond Vertex to accelerate disease targeting.