Dyne Therapeutics (DYN) Piper Sandler 36th Annual Healthcare Conference summary

Platform technology and innovation

• FORCE platform enables targeted delivery of oligonucleotides and biologics to muscle, heart, and CNS tissues, using a fragment antibody for deep tissue distribution.

• Plug-and-play design allows for various payloads, including ASOs, PMOs, siRNAs, and enzymes, tailored to specific genetic defects.

• Demonstrated ability to cross the blood-brain barrier, addressing neuromuscular diseases with CNS involvement.

• Preclinical and clinical data show broad and deep tissue distribution in both animal models and humans.

Clinical progress in DMD (DYNE-251)

• At 40 mg/kg, serious adverse events (SAEs) were observed, leading to a reduction to 20 mg/kg as the go-forward dose.

• Registration cohort enrolling 32 patients at 20 mg/kg, with robust functional improvements seen in multiple endpoints, including SV95C.

• EMA and FDA recognize SV95C as a primary endpoint; improvements exceeded the minimum clinically important difference.

• Basket trial approach planned to accelerate development for additional exons using the same platform.

Clinical progress in DM1 (DYNE-101)

• DYNE-101 targets the underlying splicing defect in myotonic dystrophy type 1, correcting splicing by over 25% at effective doses.

• Safety profile in the ACHIEVE study is favorable, with no concerning adverse events at doses up to 6.8 mg/kg.

• Registration cohort to be initiated at a dose between 3.4 and 6.8 mg/kg, aiming for accelerated approval based on CASI and functional endpoints.

• Patient-reported outcomes and CNS effects are being evaluated to address the broad impact of the disease.