Entrada Therapeutics (TRDA) 7th Annual Evercore ISI HealthCONx Conference summary

Clinical program updates

• Healthy volunteer trial in the UK for ENTR-601-44 showed strong data, exceeding expectations, and supports global regulatory filings in Q4 for both ENTR-601-44 and ENTR-601-45, aiming for phase 2b MAD studies and potential accelerated approval.

• The FDA clinical hold did not impact program progress; phase 2 trials are expected to start ex-U.S. first, with U.S. sites following as regulatory timelines allow.

• Patient trials are expected to begin next year, with higher starting doses planned based on non-clinical and phase 1 data, and dose escalation up to four-fold anticipated in patient MAD trials.

• Three DMD programs (exons 44, 45, and 50) are expected to be in phase 2 by the end of next year, with data cadence dependent on regulatory feedback and trial design finalization.

• Cash runway extends into 2027, supporting all three DMD phase 2s and potentially a registrational trial before additional fundraising is needed.

Safety and efficacy insights

• No renal toxicity or related biomarkers were observed in phase 1 at 6 mg/kg; higher excretion rates in humans versus non-human primates suggest a favorable safety profile for the EEV platform.

• The EEV peptide used across neuromuscular programs provides consistent biodistribution and PK profiles, supporting platform efficiency and cross-program learnings.

• Dose-dependent increases in exon skipping were observed in healthy volunteers, with a non-linear response at higher doses; patient-derived myofibers show 20–40x higher exon skipping than normal.

• Timing of muscle biopsies in patient trials will align with industry standards, focusing on dystrophin expression rather than exon skipping.

• Different exons may require modestly different dosing due to biological factors, but not by an order of magnitude; inherent skipping and mRNA levels vary by exon.

Platform and business development

• The EEV platform demonstrates consistent PK and biodistribution regardless of oligo cargo, streamlining trial design and non-clinical planning.

• Learnings from the Vertex DM1 program, which uses the same EEV, reinforce safety and translational potential across indications.

• Business development discussions are ongoing, with openness to partnerships that advance the platform, though decisions are made with discipline to avoid overextending resources.

• The platform is considered cargo and mechanism agnostic, provided biological rationale and control are maintained.