Entrada Therapeutics (TRDA) investor relations material

Strategic focus and pipeline overview

• Developing genetic medicines for diseases with significant unmet needs, leveraging proprietary modalities and delivery constructs for optimized therapeutic response.

• Advancing five fully owned programs and one partnered program across DMD, DM1, and inherited retinal diseases, with four clinical data catalysts anticipated in 2026.

• Neuromuscular programs utilize the EEV platform and novel PMO sequences to enhance differentiation, safety, and efficacy, supporting lower drug exposure and reduced risk of anti-drug antibody response.

Clinical program highlights

• ELEVATE-44-201 (ENTR-601-44) Phase 1/2 study in DMD demonstrated favorable safety and statistically significant functional benefit at 6 mg/kg, with higher dystrophin levels expected in subsequent cohorts.

• No serious adverse events or discontinuations observed; renal markers remained within normal range, supporting dose escalation.

• Functional improvement measured by Time to Rise and Time to Rise Velocity (TTR, TTRV) was significant versus placebo, with TTRV outperforming other therapies in cross-trial comparisons.

• Cohort 2 (12 mg/kg) and Cohort 3 (≤18 mg/kg) are expected to yield higher dystrophin and functional outcomes, with data readouts in 2027.

Mechanistic and competitive differentiation

• EEV-enabled therapies provide 25-50 fold improvement in endosomal escape and target both mature and progenitor muscle cells, supporting muscle regeneration and durability of response.

• Proprietary PMO sequences and optimized EEV delivery outperform competitive approaches in preclinical models, showing superior exon skipping and dystrophin restoration.

• ENTR-601-45 targets exon 45 DMD patients, with a differentiated dosing regimen and potential to be first-to-market among next-generation conjugate-PMOs.