Entrada Therapeutics (TRDA) Corporate presentation summary

Strategic overview and pipeline

• Five clinical-stage programs in DMD and DM1, plus two inherited retinal disease candidates projected by year-end 2026.

• Four clinical data catalysts in DMD and DM1 achieved or expected in 2026, including key readouts from ENTR-601-44, ENTR-601-45, and VX-670.

• Proprietary EEV platform and novel PMO conjugates drive differentiation, with best-in-class preclinical and early clinical data.

• Cash runway extends into Q3 2027, supported by up to $485M in DM1 milestones and Vertex partnership royalties.

• U.S. DMD market opportunity estimated at $5B annually, with significant upside in untapped global markets.

Clinical milestones and data highlights

• ENTR-601-44 Cohort 1 demonstrated favorable safety and statistically significant functional benefit at 6 mg/kg, with no serious adverse events.

• Cohort 2 dose escalation to 12 mg/kg is ongoing, with higher plasma and muscle exposure expected to drive increased dystrophin expression.

• Time to Rise Velocity (TTRV) improvement was ~3.5x above the minimal clinically important difference, outperforming other therapies.

• Renal markers remained within normal range, supporting dose escalation and de-risking future DMD programs.

• Vertex's VX-670 for DM1 is in global Phase 1/2, with results expected in H2 2026 and up to $485M in milestones.

Mechanistic and platform differentiation

• EEV platform enables 25-50-fold improvement in endosomal escape and lower whole drug requirements versus antibody-based therapies.

• EEV-PMO conjugates show enhanced satellite cell uptake, supporting muscle regeneration and durability of response.

• Proprietary PMO sequences and optimized EEVs deliver superior exon skipping and dystrophin restoration in preclinical models.

• Predictive PK modeling and juvenile NHP data indicate higher dystrophin levels and functional gains at increased doses.

• All neuromuscular programs leverage the same EEV platform, streamlining development and regulatory strategy.