Entrada Therapeutics (TRDA) TD Cowen 46th Annual Health Care Conference summary

Key program updates and clinical milestones

• Four clinical readouts expected in 2026 from DMD and DM1 programs, with two DMD exon 44 data points in Q2 and year-end, and DM1 phase II MAD study readout mid-year.

• Expansion into ocular diseases, led by the Usher syndrome program, with IND-enabling studies planned for 2026.

• Five clinical-stage programs anticipated by year-end, with ex-U.S. DMD data supporting a U.S. Accelerated Approval pathway.

• Cash runway extends into Q3 2027, excluding up to $485 million in DM1 milestones from Vertex partnership.

• ENTR-601-44 first cohort data at 6 mg/kg expected Q2, second cohort at 12 mg/kg by year-end; 45 program data expected mid-2026.

Differentiation, strategy, and commercial outlook

• Proprietary EEV platform combined with novel PMO conjugates enables best-in-class muscle uptake and safety.

• Lower drug exposure reduces anti-drug antibody risk and cost of goods.

• Double-digit dystrophin production targeted for exon 44; aim to surpass 25% dystrophin at higher doses, exceeding competitors.

• DMD market opportunity estimated at $5 billion in the U.S., with significant unmet need and limited competition.

• Seven-fold improvements in preclinical models over competitors, supporting a differentiated clinical profile.

Regulatory and partnership insights

• Ex-U.S. studies designed with FDA input to harmonize protocols; plan to approach FDA with safety and efficacy data in Q2/Q3.

• Confidence in FDA support for Accelerated Approval based on established safety and anticipated efficacy.

• Vertex partnership for DM1 provides up to $485 million in milestones and clear division of responsibilities.

• Joint Steering Committee ensures ongoing collaboration and communication with Vertex.

• Open to future BD deals if partners are committed and aligned on patient-focused goals.