Avidity Biosciences (NasdaqGM:RNA) FY Conference Transcript

Avidity Biosciences FY Conference Summary Company Overview - Company: Avidity Biosciences (NasdaqGM:RNA) - Industry: Biotechnology, focusing on neuromuscular diseases Key Points and Arguments Pipeline and Upcoming Catalysts - Avidity has transitioned from a preclinical company to having three late-stage clinical trials within five years since going public [4] - Anticipates three Biologics License Applications (BLAs) within a 12-month period, starting with Delzeta for Duchenne muscular dystrophy (DMD) exon 44 by the end of 2025, with a launch expected in 2026 [5] - Additional BLAs for Delzeta in myotonic dystrophy and Delbrax for facioscapulohumeral muscular dystrophy (FSHD) are expected in the second half of 2026 [5] Clinical Trial Data and Efficacy - Upcoming data readouts include: - Functional data from the Delzeta trial this month, focusing on participants who have been on the drug for 12 months [5] - Data from the Marine Long-Term Extension (LLE) trial for participants on the drug for 24 months in Q4 2025 [6] - 30-week efficacy data from the Harbor trial for myotonic dystrophy in Q2 2026 [6] - Avidity is currently dosing approximately 30 patients per week across its platform, with over 250 patient years of data collected [10] Product Design and Safety - Avidity's AOC™ (Antibody Oligonucleotide Conjugate) platform is utilized across its clinical programs, with siRNA used for FSHD and myotonic dystrophy, and PMO for DMD [9] - The company has learned that more frequent dosing is necessary for efficacy, with current dosing intervals set at every six to eight weeks [11] Regulatory Strategy and Market Engagement - The Harbor trial is the first global phase three study in myotonic dystrophy, with endpoints designed based on extensive input from the patient and physician communities [15] - Avidity is actively engaging with patient advocacy groups to enhance awareness and diagnosis rates for the diseases it targets [28] Commercial Strategy - Avidity has built a commercial organization in preparation for the launch of Delzeta, including patient services and a sales force targeting specialists who treat these rare diseases [44] - The company plans to leverage relationships developed during the Delzeta launch for subsequent launches of Delbrax and other products [46] Financial Position - Avidity has a strong cash position of $1.4 billion, providing a runway to mid-2027, with expectations to reach profitability quickly post-launch of Delzeta and Delbrax [48] Additional Important Insights - The company is focusing on a biomarker approach for FSHD, with a circulating biomarker (CDUX) identified that correlates with disease severity [30] - Avidity is preparing for a global launch strategy, initially targeting the U.S., Europe, and Japan, with significant enthusiasm from the patient community [29] - The company emphasizes the importance of demonstrating functional benefits alongside biomarker data to support its BLA submissions [43]