Avidity Biosciences (RNA) 7th Annual Evercore ISI HealthCONx Healthcare Conference summary

Key achievements and strategic updates

• Achieved consistent, positive data across all three neuromuscular programs in 2024, positioning for registrational trials in each.

• Expanded into precision cardiology, launching a new therapeutic franchise and broadening the platform.

• Advanced next-generation technology to improve delivery, potency, and durability of therapeutics.

• Reported $1.6 billion in cash, providing runway through mid-2027 to support trial progression and commercialization.

• Initiated commercial infrastructure buildout in preparation for product launches.

Clinical program progress and timelines

• HARBOR trial for DM1 is on track for global enrollment, expected to complete by mid-2025.

• Pivotal DM1 study uses a randomized, double-blind, placebo-controlled design with efficacy data cut at week 30 for filing; safety follow-up continues to week 54.

• No safety signals observed across neuromuscular programs; long-term data show favorable tolerability.

• FSHD program (del-brax) showed >50% DUX4 reduction and functional improvements; phase 3 dose set at 2 mg/kg every six weeks.

• DMD program (del-zota) 10 mg/kg data to be presented at a 2025 medical conference; accelerated regulatory pathway remains open for all exon skipping.

Regulatory and commercial outlook

• DM1 trial will disclose primary and key secondary endpoint results at 30 weeks, with granular data at later scientific meetings.

• FSHD and DMD programs targeting accelerated approval; further regulatory and data updates expected at JP Morgan and other 2025 conferences.

• Commercial team expansion underway to support anticipated product launches in 2025.