Group 1 - Avidity Biosciences is presenting the first functional data from the EXPLORE44 open-label extension study of Del-zota, targeting boys and young men living with Duchenne Muscular Dystrophy (DMD) [1] - Katja Lange serves as the Chief Business Officer at Avidity Biosciences and is leading the presentation [1] - The presentation is part of a broader effort to share advancements in treatment options for DMD [1] Group 2 - The presentation includes forward-looking statements that involve risks and uncertainties, which may lead to actual results differing from those anticipated [2] - Stakeholders are advised to refer to detailed cautionary language and risk factors in recent SEC filings [2]

Group 1 - Avidity Biosciences is presenting the first functional data from the EXPLORE44 open-label extension study of Del-zota, targeting boys and young men living with Duchenne Muscular Dystrophy (DMD) [1] - Katja Lange serves as the Chief Business Officer at Avidity Biosciences and is leading the presentation [1] - The presentation is part of a broader effort to share advancements in treatment options for DMD [1] Group 2 - The presentation includes forward-looking statements that involve risks and uncertainties, which may lead to actual results differing from those anticipated [2] - Stakeholders are advised to refer to detailed cautionary language and risk factors in recent SEC filings [2]

Summary of Avidity Biosciences Update / Briefing (September 10, 2025) Company Overview - **Company**: Avidity Biosciences (NasdaqGM:RNA) - **Focus**: Development of RNA therapeutics, particularly for neuromuscular diseases such as Duchenne Muscular Dystrophy (DMD), Facioscapulohumeral Muscular Dystrophy (FSHD), and Myotonic Dystrophy [2][3] Key Points and Arguments Industry and Product Development - Avidity is preparing to launch three drugs in the neuromuscular space: DELBRAX for FSHD, Aldosterone for Myotonic Dystrophy, and Delzota for DMD [3][4] - The company aims to be the first to receive global approval for treatments targeting these rare diseases [4] DMD Focus - DMD is a severe condition affecting young boys, leading to loss of mobility and reduced life expectancy [5] - Approximately 900 boys in the US and Europe are amenable to exon 44 skipping treatment [5] Clinical Data and Efficacy - The EXPLORER 44 study showed unprecedented functional improvements in patients treated with Delzota, with significant reductions in creatinine kinase (CK) levels, indicating muscle health [6][22] - At the one-year mark, about 50% of participants had CK levels within the normal range, demonstrating long-term muscle protection [22][70] - Delzota treatment resulted in a 25% increase in dystrophin levels, approaching levels associated with a normal phenotype [18][21] Functional Improvements - Delzota-treated patients showed significant improvements in functional endpoints compared to matched natural history controls, with absolute improvements of over two seconds in various mobility tests [26][30] - The improvements are unprecedented in the context of DMD treatment, indicating a potential reversal of disease progression [34] Safety Profile - The safety profile of Delzota remains favorable, with most adverse events being mild or moderate [15][16] - Only one serious adverse event was deemed related to the treatment, highlighting the overall tolerability of the drug [16] Regulatory and Commercial Strategy - Avidity plans to submit its first Biologics License Application (BLA) by the end of 2025, with subsequent submissions for the other two drugs within a year [8][45] - The company is building a commercial infrastructure that leverages synergies across its product launches in the same therapeutic area [46][47] Future Outlook - Avidity anticipates continued functional improvements as long-term muscle protection is maintained [41] - The company is also exploring platform designation for future exon-skipping therapies, which could expedite development timelines [66] Additional Important Information - The company emphasizes the importance of delivering RNA therapeutics effectively to achieve significant clinical outcomes [40][73] - Avidity is committed to redefining possibilities for boys and young men living with DMD, aiming for a future where they can engage in normal activities [36][37] This summary encapsulates the critical insights from the Avidity Biosciences briefing, highlighting the company's innovative approach to treating neuromuscular diseases and the promising data supporting its lead product, Delzota.

Del-zota Clinical Trial Results - Del-zota demonstrated unprecedented functional improvement on all key measures at one year across EXPLORE44® & EXPLORE44-OLE[18] - Creatine kinase (CK) levels rapidly reduced by >80% compared to baseline and sustained at near normal levels[41] - 50% of participants had CK levels within normal range at 1 year of treatment[41] - Dystrophin increase to up to 58% of normal[36] Safety and Tolerability - Del-zota continues to demonstrate favorable long-term safety and tolerability[27] - In EXPLORE44-OLE, 85% of participants experienced any adverse event (AE), 26% related to the study drug[27] - Serious adverse events occurred in 8% of participants, with 3% related to the study drug[27] - 3% of participants discontinued treatment due to adverse events[27] Regulatory and Commercialization - The company is on track for BLA submission by year end 2025 and launch in 2026[18] - The company is aligned on path for accelerated approval in the U S for DMD44, which affects approximately 900 people in the U S[14, 17]

Core Insights - Avidity Biosciences announced positive data from the EXPLORE44 and EXPLORE44-OLE trials, showing unprecedented improvements in multiple functional measures for Duchenne muscular dystrophy (DMD) patients treated with del-zota [1][3][4] - The company is on track to submit a Biologics License Application (BLA) to the FDA by the end of 2025 for accelerated approval of del-zota [1][6] Clinical Trial Results - Participants treated with del-zota showed a 25% increase in normal dystrophin production and a reduction of creatine kinase (CK) levels by over 80% compared to baseline, maintained near normal levels for up to 16 months [3][4] - Functional improvements included: - 4-Stair Climb (4SC): Improved by 2.1 seconds compared to a decline of 2.7 seconds in the natural history group [4] - 10-Meter Walk/Run Test (10mWRT): Improved by 0.7 seconds compared to a decline of 1.5 seconds in the natural history group [4] - Time to Rise from Floor (TTR): Improved by 3.2 seconds compared to a decline of 1.6 seconds in the natural history group [4] - Performance of Upper Limb (PUL): Improved by 1.5 points compared to a decline of 0.7 points in the natural history group [12] Safety Profile - Del-zota demonstrated a favorable long-term safety and tolerability profile, with most treatment-emergent adverse events being mild or moderate [5] - Common adverse events included upper respiratory tract symptoms, diarrhea, falls, back pain, and headaches [5] Future Plans - Avidity is preparing for a confirmatory study to support full global approval of del-zota [6] - The company plans to present additional data from the EXPLORE44 program at upcoming scientific congresses [1]

Core Insights - Avidity Biosciences, Inc. (NASDAQ:RNA) is highlighted as a top stock to buy for the next three months according to hedge funds, with a reiterated Buy rating and a price target of $96 from Cantor Fitzgerald [1][2] Business Updates - The company reported significant progress in three late-stage neuromuscular programs targeting Duchenne muscular dystrophy, myotonic dystrophy type 1, and facioscapulohumeral muscular dystrophy [2][3] - The FDA granted Breakthrough Therapy designation for Delpacibart Zotadirsen, with plans to submit a Biologics License Application for del-zota by the end of 2025 [3] Financial Performance - Avidity Biosciences, Inc. achieved an 88.12% increase in collaboration revenue, reaching $3.85 billion, surpassing consensus estimates by $2.24 million [4]

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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]

Company Overview - Avidity Biosciences is currently experiencing a significant period of growth, particularly in the execution of three muscle disease programs [2]. Leadership - The company is led by a strong executive team, including Sarah Boyce as President and CEO, Steve Hughes as Chief Medical Officer, and Kat Lange as Chief Business Officer [1].

Core Insights - The company aims to significantly enhance lives through RNA therapy delivery, reflecting a strong commitment to innovation in the healthcare sector [1] Company Development - Since its IPO five years ago, the company has progressed from having no clinical programs to currently having three programs in late-stage development, indicating substantial growth and advancement in its research and development efforts [1]