Dyne Therapeutics (DYN) Morgan Stanley 23rd Annual Global Healthcare Conference summary

Strategic positioning and platform innovation

• Positioned as a pure-play neuromuscular company with two late-stage programs (DMD and DM1) in accelerated approval pathways, targeting commercialization in 2027.

• FORCE platform enables efficient delivery of genetic medicines to muscle and potentially CNS, overcoming previous industry hurdles.

• Management team built for capital raising, allocation, and near-term revenue generation.

• Pipeline includes preclinical and IND-enabling programs, aiming for a fully integrated neuromuscular portfolio.

DM1 program clinical progress and regulatory strategy

• ACOS study showed 40% improvement in hand function (VHOT), 20% muscle strength gain at 12 months, and significant CNS-related benefits.

• Shifted registrational cohort primary endpoint from splicing correction to VHOT after FDA feedback; cohort expanded to 60 patients for statistical significance.

• Received breakthrough designation for DM1; confirmatory phase 3 trial planned for Q1 next year.

• Phase 3 will focus on clinically meaningful endpoints like time function tests and CNS subscales.

DMD program highlights and commercial outlook

• DMD program targets Exon 51 mutation, representing 13% of US patients; current standard of care is inadequate.

• Early data show sustained functional improvement and significant dystrophin increases (up to 8.7% at 6 months).

• Pursuing accelerated approval with dystrophin as the biomarker; received breakthrough designation.

• Commercial launch preparations underway, leveraging experienced leadership and anticipated rapid market uptake.