Summary of AVIDITY Biosciences Conference Call Company Overview - **Company**: AVIDITY Biosciences - **Focus**: Development of RNA therapeutics targeting muscle diseases, with three late-stage programs: Dalzosia (DMD), Delbrex (FSHD), and Daldistarone (myotonic dystrophy) [4][5][10] Core Points and Arguments Vision and Strategy - AVIDITY aims to revolutionize the RNA space and significantly impact patients' lives, with a focus on muscle diseases [3][4] - The company is on track to file its first Biologics License Application (BLA) for Dalzosia by the end of the year [4][17] Drug Development Progress - **Dalzosia**: Targeting boys and young men with DMD amenable to exon 44 skipping; enrollment completed, with data readout expected in Q2 next year [4][12] - **Delbrex**: For FSHD, with alignment on primary endpoint for accelerated approval; data readout also expected in Q2 next year [4][12][54] - **Daldistarone**: For myotonic dystrophy, with a focus on significant unmet medical needs [4][48] Delivery Mechanism - AVIDITY's platform allows for high delivery of RNA therapeutics to muscle tissue, achieving 3-5 times higher delivery compared to other methods [7][8][40] - The use of antibody oligonucleotide conjugates (AOCs) targets the transferrin receptor, enhancing muscle delivery [6][7] Market Potential and Commercialization - Anticipated rapid uptake for Dalzosia, with approximately 900 boys and young men in the US eligible for treatment [27] - The company has established a commercial infrastructure, including patient services and payer engagement teams, to support drug launches [22][49] Regulatory Environment - Positive interactions with the FDA, with no significant impact from leadership changes; the same division reviews all three programs, ensuring consistency [20][21] - Plans for BLA submissions for FSHD and myotonic dystrophy in the second half of next year [57] Additional Important Insights - The company is preparing for potential acquisition interest due to the value of its upcoming drug launches, with two drugs expected to be multi-billion dollar opportunities [10][11] - The myotonic dystrophy patient population is estimated at 40,000 in the US, with around 10,000 already diagnosed, indicating a significant market opportunity [45][46] - AVIDITY has secured a global commercial supply agreement with Lonza for manufacturing, ensuring readiness for market demand [49] Conclusion AVIDITY Biosciences is positioned for significant growth with its innovative RNA therapeutics targeting high-need muscle diseases. The company is on track for multiple drug launches, backed by a strong regulatory strategy and a well-prepared commercial infrastructure.

华福证券 2025 年 09 月 01 日 策 略 研 究 基因细胞治疗领域把握：前沿疗法为未来医疗蓄 力（2025.8.25—2025.8.29） 投资要点： 近期观点 1、医药指数本周关注的 6 个子行业仅有 1 个录得正收益，表现不 佳。 策 略 定 期 报 告 2、根据《Gene, Cell, & RNA Therapy Landscape Repor,Q2 2025 Quarterly Data Report》报告指出，2025 年第二季度，全球基因、细胞 与 RNA 疗法领域发展迅速，共有三款新疗法获批，包括美国 FDA 批 准的 Zevaskyn 和 mNexspike，以及中国 NMPA 批准的首款血友病 B 基 因疗法 BBM-H901。全球研发管线共计 4,469 项，其中基因疗法占比 49%，RNA 疗法占 29%，细胞疗法占 22%。肿瘤和罕见病仍是研发重 点，基因编辑和递送技术成为关键创新动力。本季度行业并购活跃， 交易总额超 60 亿美元，但初创公司融资下降 35%。未来多个疗法将迎 来监管决策，行业长期前景广阔。 团队成员 分析师： 赵月(S0210524050016) zy30 ...

Core Viewpoint - Avidity Biosciences is experiencing significant market interest due to potential acquisition talks with Novartis, leading to a notable increase in its stock price by 26% [3][4]. Group 1: Company Overview - Avidity Biosciences focuses on developing Antibody Oligonucleotide Conjugates (AOCs) for rare diseases, with a current market capitalization of $5.8 billion [4]. - The company has a strong cash position of approximately $1.4 billion, providing funding into mid-2027 [7]. - Avidity is advancing three AOCs in clinical development, targeting Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy Type 1 (DM1), and Facioscapulohumeral Muscular Dystrophy (FSHD) [26][39]. Group 2: Clinical Development and Pipeline - Avidity's lead therapy, del-zota, for DMD has received breakthrough therapy designation from the FDA and is expected to submit a Biologics License Application (BLA) by the end of 2025 [5][7]. - The company is preparing for three planned BLA submissions within 12 months, with ongoing registrational trials [7]. - Avidity's AOC platform has successfully delivered RNA to muscle tissue, marking a significant advancement in RNA therapy [7][39]. Group 3: Recent Developments and Collaborations - Avidity recently entered a collaboration with Bristol Myers Squibb (BMS) for the development of up to five cardiovascular targets, with potential payments totaling $2.3 billion [18][19]. - The collaboration includes an upfront payment of $100 million and milestone payments based on development progress [19]. - Avidity's stock surged by 40% following the announcement of this partnership, reflecting strong market confidence in its pipeline and strategic direction [18][19].

Summary of Avidity Biosciences Conference Call Company and Industry Overview - **Company**: Avidity Biosciences - **Industry**: RNA therapeutics, specifically targeting Facioscapulohumeral Muscular Dystrophy (FSHD) Key Points and Arguments 1. **FDA Confirmation**: Avidity has received confirmation from the FDA regarding the accelerated approval pathway for their DELBRAX program targeting FSHD, which is significant for the FSHD community [5][6][68] 2. **Clinical Study Updates**: The company is presenting twelve-month top-line data from the FORTITUDE study and has initiated a global confirmatory Phase III study named FORWARD [6][8][68] 3. **Primary Endpoint**: The primary endpoint for the FORTITUDE biomarker study is the reduction of circulating biomarkers, specifically CDAS [5][6][68] 4. **Patient Impact**: The presentation highlighted the profound impact of FSHD on patients' lives, emphasizing the urgency of developing effective treatments [4][70] 5. **Study Design**: The FORTITUDE study is a double-blind, placebo-controlled trial with a one-year duration, focusing on safety and pharmacokinetics, while the FORWARD study will last eighteen months [10][58][62] 6. **Safety Profile**: DELBRAX has shown a favorable long-term safety profile, with no serious adverse events related to the study drug reported [13][14] 7. **Biomarker Significance**: The circulating biomarker CDUX is significantly elevated in FSHD patients and is linked to disease progression. DELBRAX treatment led to rapid reductions in CDUX levels [42][43][55] 8. **Patient Reported Outcomes**: Improvements in strength, mobility, and quality of life were reported by patients treated with DELBRAX, indicating its potential effectiveness [38][39][55] 9. **Regulatory Pathway**: The FDA has provided written confirmation that an accelerated approval path is open, with CDUX as the primary endpoint for the FORTITUDE biomarker cohort [56][68] 10. **Commercial Strategy**: Avidity is building its commercial organization in preparation for three successive product launches in the same therapeutic area, which is expected to enhance operational efficiency [85][86] Additional Important Content - **Patient Testimonials**: Personal stories from patients highlighted the importance of preserving function and improving quality of life, showcasing the emotional and physical benefits of the treatment [39][70] - **Future Directions**: The company plans to leverage the insights gained from the ongoing studies to refine their approach and ensure robust data for regulatory submissions [60][66][92] - **Community Engagement**: Avidity emphasizes the importance of engaging with the FSHD community and addressing their needs through effective treatment options [70][94] This summary encapsulates the critical updates and insights shared during the conference call, focusing on the advancements in Avidity Biosciences' DELBRAX program and its implications for patients with FSHD.