Dyne Therapeutics (DYN) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Leadership and organizational updates

• Recent executive transitions were proactively communicated, with key leaders remaining through year-end and new talent added for commercial and HR roles.

• Leadership changes reflect a natural evolution as the company moves from early-stage to later-stage development.

• New commercial and HR leaders have joined, strengthening capabilities for upcoming product launches.

• Additional hires, such as a Chief Financial Officer, are anticipated as the company grows.

DMD (Duchenne Muscular Dystrophy) program highlights

• Efficacy and functional data at 20 mg/kg showed significant improvement from baseline within six months, with the highest dystrophin levels observed to date.

• Two serious adverse events (SAEs) at 40 mg/kg were likely infection-related, but the dose was reduced to 20 mg/kg for safety.

• Regulators, including the FDA, are comfortable with the safety approach and ongoing studies.

• Functional benefits at 20 mg/kg are unprecedented in the field, with improvements in NSAA and SV95C endpoints.

• Registration cohorts at 20 mg/kg are being initiated, with regulatory clarity expected by year-end.

Platform and pipeline expansion

• The platform is being extended to additional exons beyond exon 51, with preclinical work underway and plans to move into IND-enabling studies.

• The approach allows for expedited approval of new exon targets due to shared chemistry and delivery mechanisms.

• Early newborn screening for DMD is expanding, opening opportunities for earlier intervention.