Dyne Therapeutics (DYN) Jefferies Global Healthcare Conference 2026 summary

Company overview and platform differentiation

• Focused on solving drug delivery challenges to CNS and muscle using the FORCE platform, with multiple de-risked programs in DMD, DM1, FSHD, and Pompe supported by human data.

• 2025 is positioned as a year of clinical validation, with 2026 focused on execution and commercialization preparation.

DMD program updates and commercialization plans

• BLA for DMD Exon 51 asset recently filed; approval and launch targeted for Q1 next year.

• DMD Exon 51 targets about 1,600 patients in the U.S., with three main patient groups: currently treated, previously treated, and untreated.

• Product offers monthly dosing and aims for functional improvement, potentially supporting premium pricing near $1 million/year.

• Commercial team in place, with market access and reimbursement strategies underway; launch will leverage concentrated treatment centers.

• Confirmatory phase III FORZETTO study uses Rise from Floor Velocity as the primary endpoint, with 72-week duration.

DM1 program progress and regulatory alignment

• Completed enrollment of 71 patients in the ACHIEVE registration cohort; data readout expected Q1 next year.

• U.S. DM1 patient population estimated at 40,000; pricing guidance not yet provided.

• Primary endpoint is vHOT, powered using MMRM; secondary endpoints include splicing (CASI) and functional measures.

• FDA views vHOT as an intermediate clinical endpoint for accelerated approval; confirmatory HARMONIA study uses Five Times Sit-to-Stand.

• Both phase III studies will support U.S. and ex-U.S. marketing applications.