Financial Data and Key Metrics Changes - The company has progressed significantly since its IPO five years ago, moving from no programs in the clinic to three programs in late-stage development [3][4] - The company plans to file three Biologics License Applications (BLAs) within a 12-month period, starting with the DMD Delzeta program for accelerated approval in the U.S. [4][24] Business Line Data and Key Metrics Changes - The Delzeta program has shown a 25% increase in dystrophin levels in patients, with a significant decrease in creatine kinase levels [11][12] - The company is focused on delivering first-in-class and best-in-class therapies across its product lines, with a strong emphasis on muscle delivery [4][10] Market Data and Key Metrics Changes - There are approximately 900 boys and young men in the U.S. with DMD exon 44, with about 50% of them treated at the same 40 centers of excellence [33] - The company anticipates that its therapies will be the first and best available options for DMD patients [34] Company Strategy and Development Direction - The company is preparing for commercialization with a foundational commercial infrastructure that can be leveraged across all three drug opportunities [9][10] - The company is exploring partnerships and M&A opportunities as it approaches the commercial phase with potential blockbuster drugs [8][9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in executing their plans and emphasized the importance of focusing on upcoming approvals and launches [9][10] - The company is optimistic about the regulatory landscape and believes its data package will be compelling for the FDA [30][41] Other Important Information - The company has removed biopsy requirements for new participants in the Explore 44 trial, focusing on dystrophin and creatine kinase as key markers for the BLA filing [23][25] - The company is actively working on global trial enrollment for FSHD, with strong community support [46][47] Q&A Session Summary Question: Can you provide an overview of the company's platform and key programs? - The company has three programs in late-stage development and aims to improve lives through RNA therapy [3] Question: What is the commercial strategy and business development approach? - The company is building a commercial infrastructure and is open to exploring M&A opportunities [8][9] Question: Can you discuss the Delzeta program and its data? - The Delzeta program has shown a 25% increase in dystrophin levels and will present functional data ahead of schedule [11][12][16] Question: What is the market opportunity for DMD exon 44? - There are about 900 DMD exon 44 patients in the U.S., and the company plans to target this population effectively [33] Question: What are the key filing requirements for the BLA? - The primary requirements include demonstrating dystrophin levels and safety data, with a focus on creatine kinase as a critical marker [23][25] Question: Will there be a pre-BLA meeting? - Yes, a pre-BLA meeting is planned to ensure the data package meets FDA expectations [44] Question: What are the expectations for the Harbor study's primary endpoint? - The primary endpoint is video hand opening time, and the company is confident in meeting this goal [51]

Financial Data and Key Metrics Changes - The company has progressed significantly since its IPO five years ago, moving from no programs in the clinic to three late-stage development programs [3] - The company is on track to file three Biologics License Applications (BLAs) within a 12-month period, starting with the DMD Delzeta program for accelerated approval in the U.S. [4] Business Line Data and Key Metrics Changes - The Delzeta program for DMD has shown a 25% increase in dystrophin levels, rising from a baseline of 7% to 32%, and a decrease in creatine kinase (CK) to near-normal levels [11][12] - The company plans to present functional data for Delzeta ahead of schedule, indicating strong progress in its clinical trials [16] Market Data and Key Metrics Changes - There are approximately 900 boys and young men in the U.S. diagnosed with DMD exon 44, with about 50% of them treated at the same 40 centers of excellence [33] - The company anticipates being the first and best therapy available for DMD patients, leveraging its established patient services and field force [34] Company Strategy and Development Direction - The company is focused on building a foundational commercial infrastructure that can be leveraged across all three drug opportunities [10] - The strategy includes exploring partnerships and M&A opportunities as the company approaches the commercial phase with potential blockbuster drugs [7][9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in executing the upcoming approvals and launches, emphasizing the importance of maintaining focus on their current programs [9] - The company is optimistic about the regulatory landscape, having aligned with the FDA on the requirements for accelerated approval based on dystrophin and CK data [24][26] Other Important Information - The company has initiated a biomarker study for the Delbrax program, which is expected to provide critical data for the accelerated approval pathway [40] - The company is actively working to ensure global trial enrollment for its FSHD program, indicating strong community interest and support [47] Q&A Session Summary Question: What drove the decision to report the data ahead of schedule? - The company aims to exceed expectations and found that the data was ready sooner than anticipated [16] Question: Can you give us a sense of patient number, follow-up time, and functional endpoints? - The upcoming data will involve 17 patients from the Explore 44 trial, with a focus on various functional measures [18][19] Question: Could you talk about the market opportunity for exon 44 and your go-to-market strategy? - The company sees a significant opportunity in the DMD market, with a well-established infrastructure to support patient access [33] Question: Will there be a pre-BLA meeting, and what will be the focus? - A pre-BLA meeting is planned to ensure the submission package meets FDA expectations [43] Question: What is the expectation for the primary endpoint in the Harbor study? - The company is confident in meeting the primary endpoint and is focused on a comprehensive data package for regulatory approval [52]

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.

Core Insights - Avidity Biosciences' shares have increased by 36% in the past month due to reports of Novartis' interest in acquiring the company, although discussions are still in early stages with no guarantees of a deal [1][7] - Avidity is a clinical-stage biotech focused on RNA therapeutics, with key programs targeting rare muscular diseases and expanding into precision cardiology [2][3] - Year-to-date, Avidity's shares have surged by 59%, significantly outperforming the industry average growth of 4% [6] Company Overview - Avidity Biosciences is developing RNA therapeutics for conditions such as myotonic dystrophy type 1, facioscapulohumeral muscular dystrophy, and Duchenne muscular dystrophy [2] - The company has established partnerships with major pharmaceutical companies like Bristol Myers and Eli Lilly to develop therapies for cardiovascular and immunology indications [3] Market Context - Novartis is reportedly interested in acquiring Avidity to mitigate potential revenue losses from generic competition affecting its top-selling drugs, such as Entresto, Gleevec, and Diovan [7][8] - The acquisition would enhance Novartis' pipeline in rare muscular diseases and strengthen its position in the cardiovascular market [8]

Core Viewpoint - Novartis plans to acquire Avidity Biosciences, a leader in the AOC field with a market value of nearly $6 billion, to strengthen its pipeline in the rare disease treatment sector [3]. Group 1: Market Dynamics - The small nucleic acid drugs, including siRNA, ASO, and miRNA, are designed to regulate gene expression for disease treatment, offering breakthrough therapies for genetic, rare, and chronic diseases [4]. - Novartis faces a "patent cliff" with several blockbuster drugs, including Entresto, set to lose patent protection between 2025 and 2030, necessitating the acquisition of new pipeline assets to ensure future growth [5]. - Major pharmaceutical companies, including Sanofi, Pfizer, and Eli Lilly, have been actively acquiring and partnering in the small nucleic acid drug space to capture the potential market [6]. Group 2: AOC Development and Commercialization - AOC (Antibody-Oligonucleotide Conjugates) represents an innovative delivery method for small nucleic acid drugs, combining the targeting ability of monoclonal antibodies with the gene regulation capabilities of oligonucleotides [10]. - Avidity Biosciences is a leading player in the AOC field, with three pipelines in Phase III clinical trials, including a drug for Duchenne Muscular Dystrophy (DMD) that has shown promising results [12]. - The commercialization of AOC drugs is expected to accelerate, with several biotech companies entering late-stage clinical trials, aiming for market entry around 2026-2027 [11]. Group 3: Investment and Policy Support - The Chinese government continues to support the development of RNA and small nucleic acid drugs through funding initiatives aimed at overcoming core bottlenecks in drug creation [14]. - Recent financing activities in the small nucleic acid drug sector indicate growing interest and investment, with several companies securing significant funding [17]. - Novartis's acquisition of Avidity, if completed, would mark a significant milestone in the global AOC drug market, highlighting the increasing focus on innovative therapies [15].

Investment Rating - The report indicates a strong interest in the small nucleic acid drug sector, particularly through acquisitions and partnerships, highlighting the potential for significant growth in this area [3][5][15]. Core Insights - Novartis is focusing on expanding its pipeline in rare disease treatments by acquiring Avidity Biosciences, a leader in the Antibody-Oligonucleotide Conjugates (AOC) field, which is valued at nearly $6 billion [3][11]. - The small nucleic acid drugs, including siRNA, ASO, and miRNA, are gaining traction due to their ability to target traditionally "undruggable" disease-causing genes, offering breakthrough treatment options for genetic, rare, and chronic diseases [4][9]. - The report emphasizes the importance of delivery technologies for small nucleic acid drugs, particularly the need for effective delivery systems to target tissues outside the liver, as 90% of disease-causing genes are expressed in non-liver tissues [8][9]. Summary by Sections Acquisition and Market Dynamics - Novartis has been actively acquiring companies to fill the pipeline gap created by upcoming patent expirations of key drugs, such as Entresto, which is projected to generate $7.822 billion in sales in 2024 [5][6]. - The report outlines several significant acquisitions in the small nucleic acid space, including Novartis's $9.7 billion acquisition of The Medicines Company and a $1.7 billion deal for Regulus Therapeutics [6][12]. Delivery Technology and Challenges - The report discusses the challenges faced by small nucleic acid drugs in terms of delivery, particularly the degradation by nucleases and the difficulty in penetrating the blood-brain barrier [7][8]. - Innovations in delivery systems, such as the FALCON platform by DTx Pharma, are highlighted as crucial for advancing the application of RNA therapies beyond liver-targeted treatments [8][10]. Competitive Landscape - The competitive landscape is characterized by major pharmaceutical companies, including Sanofi, Pfizer, and Eli Lilly, making significant investments in small nucleic acid drug technologies to capture the emerging market [6][10]. - Avidity Biosciences is noted as a key player with multiple clinical-stage AOC pipelines, including a breakthrough therapy for Duchenne Muscular Dystrophy [12][13]. Regulatory and Funding Environment - The report mentions supportive policies from the Chinese government aimed at promoting RNA and small nucleic acid drug development, including funding initiatives for innovative research [14][15]. - Recent financing activities in the small nucleic acid drug sector indicate growing investor interest, with several companies securing substantial funding to advance their research and development efforts [16].

Group 1: Core Insights - Goldman Sachs' latest report focuses on "2Q25 EPS updates," analyzing biotech companies such as Amylyx (AMLX.US), CG Oncology (CGON.US), GPCR (GPCR.US), and Ideaya Biosciences (IDYA.US), concluding that operational data largely meets expectations with minor adjustments to target prices due to cash flow or clinical milestones [1] Group 2: Amylyx (AMLX.US) - In Q2, Amylyx reported operating expenses of approximately $43 million, slightly above market consensus of $41 million and Goldman Sachs' estimate of $40 million, primarily due to higher R&D costs of $27 million compared to the expected range of $22 million to $25 million [2] - The management reiterated the timeline for the Phase III clinical trial for post-bariatric hypoglycemia (PBH), aiming for enrollment completion in 2025, data readout in the first half of 2026, and potential market entry in 2027, with an estimated patient population of around 160,000 [2] - Goldman Sachs maintains a "Buy" rating with a target price of $10 [2] Group 3: CG Oncology (CGON.US) - CG Oncology's BOND-003 Phase III trial data showed a complete response rate of 75.5% for its bladder cancer gene therapy, with 12-month and 24-month sustained response rates of 50.7% and 42.3%, respectively [3] - The company has $661 million in cash, sufficient to support operations until mid-2028, and Goldman Sachs maintains a "Buy" rating with minor model adjustments [3] Group 4: GPCR (GPCR.US) - GPCR's Q2 R&D expenses were approximately $54 million, significantly higher than market expectations of $36 million and Goldman Sachs' estimate of $44 million, attributed to workforce expansion and increased trial costs [4] - The company has $787 million in cash, expected to last until 2027, and plans to read out Phase 2b ACCESS data in 2025 [4] Group 5: Ideaya Biosciences (IDYA.US) - Ideaya reported Q2 R&D expenses of about $74 million, slightly above market expectations of $71 million and Goldman Sachs' estimate of $68 million, with cash reserves of $992 million projected to last until 2029 [5] - The core pipeline includes darovasertib in combination with crizotinib for HLA-A2 negative metastatic uveal melanoma, with Phase 2/3 results expected by year-end [5] Group 6: Terns Pharmaceuticals (TERN.US) and RNA (RNA.US) - Terns Pharmaceuticals plans to release preliminary data for its leukemia drug TERN-701 and obesity drug TERN-601 in Q4 2025, with cash reserves of $315 million sufficient until 2028 [6] - RNA has signed a manufacturing agreement with Lonza to ensure product supply stability from 2026 to 2028, with BLA submissions expected for its treatments by the end of 2025 and mid-2026 [6] Group 7: AI in Biotech - Goldman Sachs emphasizes the core role of biotechnology R&D, highlighting collaborations with institutions like OpenAI to drive innovation, while also focusing on the pricing potential of rare disease drugs and opportunities for indication expansion [7] Group 8: Overall Industry Outlook - The biotech sector is expected to see over 15 key clinical data readouts from late 2025 to early 2026, with a focus on companies like AMLX, CGON, and RNA for their potential breakthroughs [8] - Despite some companies facing increased short-term losses due to R&D spending, pipeline progress remains in line with expectations, and cash flow is generally secure [8]

PART I – FINANCIAL INFORMATION [Item 1. Condensed Consolidated Financial Statements (unaudited)](index=3&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements%20(unaudited)) This section presents unaudited condensed consolidated financial statements, including balance sheets, statements of operations, equity, cash flows, and detailed notes [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) This section provides a summary of the company's financial position at specific dates Condensed Consolidated Balance Sheet Highlights (in thousands) | Metric | June 30, 2025 | December 31, 2024 | | :-------------------------- | :------------ | :---------------- | | Cash and cash equivalents | $243,907 | $219,868 | | Marketable securities | $939,237 | $1,281,629 | | Total current assets | $1,250,560 | $1,542,290 | | Total assets | $1,368,926 | $1,563,895 | | Total current liabilities | $135,061 | $98,018 | | Total liabilities | $176,262 | $138,936 | | Total stockholders' equity | $1,192,664 | $1,424,959 | - Total assets decreased by **$195.0 million** from December 31, 2024, to June 30, 2025, primarily due to a reduction in marketable securities[11](index=11&type=chunk) - Total liabilities increased by **$37.3 million**, while total stockholders' equity decreased by **$232.3 million** during the six-month period[11](index=11&type=chunk) [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) This section details the company's revenues, expenses, and net loss over specific periods Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Collaboration revenue | $3,847 | $2,045 | $5,420 | $5,588 | | Research and development expenses | $138,125 | $63,940 | $237,615 | $130,772 | | General and administrative expenses | $36,864 | $20,731 | $70,464 | $34,629 | | Loss from operations | $(171,142) | $(82,626) | $(302,659) | $(159,813) | | Interest income | $14,478 | $11,949 | $30,657 | $20,382 | | Net loss | $(157,315) | $(70,793) | $(273,088) | $(139,648) | | Net loss per share, basic and diluted | $(1.21) | $(0.65) | $(2.11) | $(1.44) | | Weighted-average shares outstanding | 129,622 | 106,928 | 129,428 | 97,070 | - Net loss significantly increased for both the three-month period (from **$(70.8 million)** to **$(157.3 million)**) and the six-month period (from **$(139.6 million)** to **$(273.1 million)**) year-over-year[12](index=12&type=chunk) - Research and development expenses more than doubled for both periods, driving the increased net loss[12](index=12&type=chunk) [Condensed Consolidated Statements of Stockholders' Equity](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders'%20Equity) This section outlines changes in the company's equity, including common stock and accumulated deficit Changes in Stockholders' Equity (in thousands) | Metric | December 31, 2024 | March 31, 2025 | June 30, 2025 | | :-------------------------------- | :---------------- | :------------- | :------------ | | Common Stock Shares | 119,893 | 120,512 | 120,778 | | Additional Paid-in Capital | $2,315,111 | $2,334,784 | $2,357,029 | | Accumulated Other Comprehensive Income | $2,902 | $2,766 | $1,777 | | Accumulated Deficit | $(893,066) | $(1,008,839) | $(1,166,154) | | Total Stockholders' Equity | $1,424,959 | $1,328,723 | $1,192,664 | - Total stockholders' equity decreased by **$232.3 million** from December 31, 2024, to June 30, 2025, primarily due to the accumulated net loss[15](index=15&type=chunk) - Additional paid-in capital increased due to stock option exercises, ESPP, and stock-based compensation[15](index=15&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) This section presents the company's cash inflows and outflows from operating, investing, and financing activities Condensed Consolidated Statements of Cash Flows (in thousands, Six Months Ended June 30) | Activity | 2025 | 2024 | | :------------------------------------------ | :--------- | :--------- | | Net cash used in operating activities | $(324,495) | $(135,384) | | Net cash provided by (used in) investing activities | $342,673 | $(307,616) | | Net cash provided by financing activities | $5,710 | $836,169 | | Net increase in cash, cash equivalents and restricted cash | $24,042 | $393,169 | | Cash, cash equivalents and restricted cash at end of period | $246,705 | $578,546 | - Net cash used in operating activities increased significantly from **$135.4 million** in 2024 to **$324.5 million** in 2025[20](index=20&type=chunk) - Investing activities shifted from using **$307.6 million** in 2024 to providing **$342.7 million** in 2025, primarily due to proceeds from maturities of marketable securities[20](index=20&type=chunk) [Notes to Unaudited Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) This section provides detailed explanations and disclosures for the unaudited condensed consolidated financial statements [1. Description of Business and Basis of Presentation](index=9&type=section&id=1.%20Description%20of%20Business%20and%20Basis%20of%20Presentation) This note describes the company's biopharmaceutical business and the basis for financial statement presentation - Avidity Biosciences is a biopharmaceutical company developing Antibody Oligonucleotide Conjugates (AOCs) to target the root cause of previously untreatable diseases[22](index=22&type=chunk) - As of June 30, 2025, the company had an accumulated deficit of **$1.2 billion** and cash, cash equivalents, and marketable securities of **$1.2 billion**[23](index=23&type=chunk) - Existing cash, cash equivalents, and marketable securities are believed to be sufficient to fund operations for at least 12 months from the Form 10-Q filing date[24](index=24&type=chunk) [2. Summary of Significant Accounting Policies](index=10&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) This note outlines the key accounting policies used in preparing the condensed consolidated financial statements - No significant changes to accounting policies occurred during the six months ended June 30, 2025[29](index=29&type=chunk) - The company is evaluating the impact of new FASB ASUs 2023-09 (Income Tax Disclosures) and 2024-03 (Expense Disaggregation Disclosures) on its financial statements[33](index=33&type=chunk)[34](index=34&type=chunk) Common Stock Equivalent Securities Not Included in Diluted Net Loss Per Share (in thousands) | Security Type | June 30, 2025 | June 30, 2024 | | :-------------------------------- | :------------ | :------------ | | Common stock options | 14,394 | 12,728 | | Restricted stock units | 2,812 | 1,526 | | Performance stock units | 810 | 562 | | ESPP shares pending issuance | 3 | 4 | | **Total** | **18,019** | **14,820** | [3. Fair Value Measurements](index=11&type=section&id=3.%20Fair%20Value%20Measurements) This note details the fair value measurements of financial instruments, primarily marketable securities Fair Value Measurements of Marketable Securities (in thousands, as of June 30, 2025) | Asset Type | Total | Level 1 (Quoted Prices) | Level 2 (Other Observable Inputs) | Level 3 (Unobservable Inputs) | | :----------------------- | :------ | :---------------------- | :-------------------------- | :------------------------ | | U.S. Treasury securities | $939,237 | $939,237 | $0 | $0 | - All marketable securities are measured at fair value using Level 1 inputs, indicating readily available quoted prices in active markets[35](index=35&type=chunk) [4. Marketable Securities](index=11&type=section&id=4.%20Marketable%20Securities) This note provides information on the company's marketable securities, including their fair value and maturity Marketable Securities (in thousands, as of June 30, 2025) | Maturity | Amortized Cost | Unrealized Gains | Unrealized Losses | Estimated Fair Value | | :--------------- | :------------- | :--------------- | :---------------- | :------------------- | | U.S. Treasury securities (1 year or less) | $753,666 | $1,350 | $(94) | $754,922 | | U.S. Treasury securities (1-2 years) | $183,924 | $435 | $(44) | $184,315 | | **Total** | **$937,590** | **$1,785** | **$(138)** | **$939,237** | - Unrealized losses on marketable securities are primarily due to interest rate increases, not credit quality, and no allowance for credit losses was recorded[37](index=37&type=chunk) - The company does not intend to sell these investments before maturity and expects to recover their amortized cost bases[37](index=37&type=chunk) [5. Collaboration, License and Research Agreements](index=12&type=section&id=5.%20Collaboration,%20License%20and%20Research%20Agreements) This note details revenue and deferred revenue from collaboration, license, and research agreements Collaboration Revenue (in thousands) | Agreement | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Bristol Myers Squibb Company (BMS) | $3,847 | $2,045 | $5,420 | $4,500 | | Eli Lilly and Company (Lilly) | $0 | $0 | $0 | $1,100 | | **Total Collaboration Revenue** | **$3,847** | **$2,045** | **$5,420** | **$5,600** | - Revenue from the BMS Collaboration Agreement increased for both the three and six months ended June 30, 2025[39](index=39&type=chunk) - Deferred revenue related to collaboration agreements decreased from **$58.9 million** at December 31, 2024, to **$53.5 million** at June 30, 2025[41](index=41&type=chunk) [6. Composition of Certain Consolidated Financial Statement Items](index=13&type=section&id=6.%20Composition%20of%20Certain%20Consolidated%20Financial%20Statement%20Items) This note provides a breakdown of specific balance sheet items, including prepaid assets and other current assets Prepaid and Other Current Assets (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :-------------------------- | :------------ | :---------------- | | Prepaid assets | $15,976 | $12,571 | | Interest receivable | $8,540 | $9,447 | | Other current assets | $42,900 | $18,775 | | **Total** | **$67,416** | **$40,793** | - Other current assets increased significantly, including reimbursable tenant improvements rising from **$7.1 million** to **$31.1 million**[42](index=42&type=chunk) - The company recorded approximately **$87.0 million** in nonrefundable reservation fees with a Contract Manufacturing Organization (CMO) for future production batches (2026-2028)[44](index=44&type=chunk) [7. Commitments and Contingencies](index=14&type=section&id=7.%20Commitments%20and%20Contingencies) This note discloses the company's future lease commitments and any material litigation matters - The company has future lease commitments of approximately **$80.0 million** for its corporate headquarters sublease (payments starting Q3 2025) and **$53.7 million** for an amended sublease for an adjacent building (payments starting April 2026)[46](index=46&type=chunk)[47](index=47&type=chunk) - A **$2.5 million** letter of credit is maintained for the benefit of the sublandlord[48](index=48&type=chunk) - No material litigation matters are currently outstanding for which liabilities have been accrued[49](index=49&type=chunk) [8. Stockholders' Equity](index=15&type=section&id=8.%20Stockholders'%20Equity) This note details changes in stockholders' equity and stock-based compensation expenses Stock-Based Compensation Expense (in thousands) | Expense Type | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development | $9,490 | $6,529 | $18,609 | $12,266 | | General and administrative | $8,169 | $6,283 | $16,786 | $10,852 | | **Total** | **$17,659** | **$12,812** | **$35,395** | **$23,118** | - Unrecognized compensation cost as of June 30, 2025, includes **$121.0 million** for time-based options, **$68.0 million** for restricted stock units, and **$34.2 million** for performance stock units[51](index=51&type=chunk) - The company issued 92,348 shares under the Employee Stock Purchase Plan (ESPP) during the six months ended June 30, 2025[52](index=52&type=chunk) [9. Segment Information](index=16&type=section&id=9.%20Segment%20Information) This note confirms the company operates as a single segment and provides related financial information - The company operates as a single operating and reportable segment[54](index=54&type=chunk) - The Chief Executive Officer (CODM) uses consolidated net loss to assess performance and allocate resources[54](index=54&type=chunk) Cash, Cash Equivalents and Marketable Securities (in thousands) | Date | Amount | | :----------- | :--------- | | June 30, 2025 | $1,183,144 | | December 31, 2024 | $1,501,497 | [10. Subsequent Events](index=16&type=section&id=10.%20Subsequent%20Events) This note discloses significant events that occurred after the reporting period, including stock sales and new agreements - From July 1, 2025, through August 7, 2025, the company sold 5,646,583 shares of common stock, generating **$185.5 million** in net proceeds[56](index=56&type=chunk) - On August 1, 2025, the company entered into a commercial manufacturing agreement with a CMO, incurring approximately **$620.0 million** in future unconditional purchase obligations from 2026 through 2028[57](index=57&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=16&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's financial condition, operational results, biopharmaceutical pipeline progress, and liquidity [Overview](index=17&type=section&id=Overview) This overview highlights the company's biopharmaceutical focus, clinical trial progress, and strategic advancements - Avidity Biosciences is a biopharmaceutical company developing Antibody Oligonucleotide Conjugates (AOCs) for rare diseases, with three programs in potentially registrational trials[60](index=60&type=chunk) - The company is expanding into precision cardiology with two wholly-owned development candidates: AOC 1072 (PRKAG2 Syndrome) and AOC 1086 (PLN cardiomyopathy)[76](index=76&type=chunk) - Avidity is building global commercial infrastructure for potential product launches for DMD, DM1, and FSHD starting in 2026[77](index=77&type=chunk) [Delpacibart zotadirsen (del-zota) for the treatment of DMD44](index=18&type=section&id=Delpacibart%20zotadirsen%20(del-zota)%20for%20the%20treatment%20of%20DMD44) This section details del-zota's development for DMD44, including clinical trials and FDA Breakthrough Therapy designation - Del-zota, for Duchenne muscular dystrophy (DMD44), is in Phase 2 development (EXPLORE44-OLE™ study) and received FDA Breakthrough Therapy designation in July 2025[60](index=60&type=chunk)[61](index=61&type=chunk)[62](index=62&type=chunk) - Positive top-line data from the Phase 1/2 EXPLORE44 trial showed statistically significant improvements across key biomarkers and favorable safety[63](index=63&type=chunk) - The company plans to submit its first Biologics License Application (BLA) for del-zota at year-end 2025, using data from EXPLORE44 and EXPLORE44-OLE studies[63](index=63&type=chunk)[67](index=67&type=chunk) [Delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1)](index=18&type=section&id=Delpacibart%20etedesiran%20(del-desiran)%20for%20the%20treatment%20of%20myotonic%20dystrophy%20type%201%20(DM1)) This section details del-desiran's development for DM1, including the HARBOR trial and anticipated data - Del-desiran, for myotonic dystrophy type 1 (DM1), is in the global Phase 3 HARBOR™ trial, with enrollment completed in July 2025[60](index=60&type=chunk)[64](index=64&type=chunk)[66](index=66&type=chunk) - Data from the MARINA-OLE™ trial showed reversal of disease progression across multiple endpoints, including hand function and muscle strength[64](index=64&type=chunk) - Topline data from the HARBOR study is anticipated in the second quarter of 2026, with marketing application submissions expected to start in the second half of 2026[72](index=72&type=chunk) [Delpacibart braxlosiran (del-brax) for the treatment of facioscapulohumeral muscular dystrophy (FSHD)](index=19&type=section&id=Delpacibart%20braxlosiran%20(del-brax)%20for%20the%20treatment%20of%20facioscapulohumeral%20muscular%20dystrophy%20(FSHD)) This section details del-brax's development for FSHD, including registrational trials and regulatory pathways - Del-brax, for facioscapulohumeral muscular dystrophy (FSHD), is in the registrational FORTITUDE biomarker cohort, Phase 2 FORTITUDE-OLE™, and Phase 3 FORTITUDE-3™ trials[60](index=60&type=chunk)[68](index=68&type=chunk) - FDA has aligned on accelerated and full approval pathways for del-brax, with positive topline Phase 1/2 FORTITUDE data showing consistent improvement in functional mobility, muscle strength, and rapid reduction in biomarkers[69](index=69&type=chunk)[73](index=73&type=chunk) - The company plans to submit a BLA for accelerated approval in the second half of 2026, supported by data from FORTITUDE trials[69](index=69&type=chunk) [Company Advancements](index=20&type=section&id=Company%20Advancements) This section highlights pipeline expansion into precision cardiology and commercial infrastructure development - Avidity is expanding its AOC pipeline beyond rare neuromuscular disorders into precision cardiology, with candidates AOC 1072 and AOC 1086[76](index=76&type=chunk) - The company is developing a global commercial infrastructure in preparation for potential product launches for DMD, DM1, and FSHD starting in 2026[77](index=77&type=chunk) - Since inception, the company has incurred operating losses, with a net loss of **$273.1 million** for the six months ended June 30, 2025, and an accumulated deficit of **$1.2 billion**[79](index=79&type=chunk) [Components of Results of Operations](index=21&type=section&id=Components%20of%20Results%20of%20Operations) This section explains the key components of the company's revenue and expenses, and their expected trends - Revenue is derived from license and research collaboration agreements; no product sales revenue is expected until regulatory approval[83](index=83&type=chunk)[84](index=84&type=chunk) - Research and development expenses are expected to increase significantly due to ongoing preclinical studies and clinical trials, including external and internal costs[87](index=87&type=chunk)[89](index=89&type=chunk) - General and administrative expenses are also projected to increase to support expanded R&D activities, commercial readiness, and corporate functions[92](index=92&type=chunk) [Results of Operations (Comparison of the Three and Six Months Ended June 30, 2025 and 2024)](index=23&type=section&id=Results%20of%20Operations%20(Comparison%20of%20the%20Three%20and%20Six%20Months%20Ended%20June%2030,%202025%20and%202024)) This section analyzes the company's financial performance for the three and six months ended June 30, 2025 and 2024 Key Financial Results (in thousands) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Revenue | $3,847 | $2,045 | $5,420 | $5,588 | | Research and development expenses | $138,125 | $63,940 | $237,615 | $130,772 | | General and administrative expenses | $36,864 | $20,731 | $70,464 | $34,629 | | Other income | $13,827 | $11,833 | $29,571 | $20,165 | - Research and development expenses increased by **$74.2 million** (three months) and **$106.8 million** (six months) year-over-year, driven by clinical trial progression, manufacturing costs, and personnel[97](index=97&type=chunk) - General and administrative expenses increased by **$16.1 million** (three months) and **$35.8 million** (six months) due to higher personnel costs and professional fees[98](index=98&type=chunk) [Liquidity and Capital Resources](index=24&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the company's cash position, capital requirements, and future funding strategies - As of June 30, 2025, the company had **$1.2 billion** in cash, cash equivalents, and marketable securities, expected to fund operations for at least 12 months[103](index=103&type=chunk) - Future capital requirements are substantial and will depend on the progress of product candidates, manufacturing, regulatory outcomes, and commercialization efforts[104](index=104&type=chunk)[105](index=105&type=chunk) - The company plans to finance future cash needs through equity offerings, debt financings, or collaborations, as product sales revenue is not anticipated in the immediate term[104](index=104&type=chunk) [Critical Accounting Estimates](index=27&type=section&id=Critical%20Accounting%20Estimates) This section addresses the company's critical accounting estimates and any material changes - No material changes to critical accounting estimates were reported as of June 30, 2025, compared to the annual report on Form 10-K for the year ended December 31, 2024[111](index=111&type=chunk) [Contractual Obligations and Commitments](index=27&type=section&id=Contractual%20Obligations%20and%20Commitments) This section outlines the company's significant contractual obligations and commitments - The company has aggregate future lease commitments of approximately **$80.0 million** for its corporate headquarters sublease and **$53.7 million** for an amended sublease for an adjacent building[112](index=112&type=chunk) - No other material changes to contractual obligations were reported as of June 30, 2025, outside the ordinary course of business[112](index=112&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=27&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section states no material changes in market risk as of June 30, 2025, compared to prior annual report disclosures - No material changes in market risk were reported as of June 30, 2025, compared to the disclosures in the annual report on Form 10-K for the year ended December 31, 2024[114](index=114&type=chunk) [Item 4. Controls and Procedures](index=27&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of June 30, 2025, with no material changes in internal control - Management concluded that the company's disclosure controls and procedures were effective at the reasonable assurance level as of June 30, 2025[116](index=116&type=chunk) - There have been no material changes in internal control over financial reporting during the quarter ended June 30, 2025[117](index=117&type=chunk) PART II – OTHER INFORMATION [Item 1. Legal Proceedings](index=28&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any material legal proceedings - The company is not currently subject to any material legal proceedings[119](index=119&type=chunk) [Item 1A. Risk Factors](index=28&type=section&id=Item%201A.%20Risk%20Factors) This section states no material changes to risk factors previously disclosed in the annual report on Form 10-K - No material changes to the risk factors set forth in the annual report on Form 10-K for the year ended December 31, 2024, were reported[120](index=120&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=28&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales or issuer repurchases of equity securities during the period - No unregistered sales of equity securities occurred during the period[121](index=121&type=chunk) - No issuer repurchases of equity securities occurred during the period[122](index=122&type=chunk) [Item 3. Defaults Upon Senior Securities](index=28&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This item is not applicable to the company - This item is not applicable[123](index=123&type=chunk) [Item 4. Mine Safety Disclosures](index=28&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - This item is not applicable[124](index=124&type=chunk) [Item 5. Other Information](index=28&type=section&id=Item%205.%20Other%20Information) The Chief Human Resources Officer adopted a Rule 10b5-1 trading arrangement during the quarter - Teresa McCarthy, Chief Human Resources Officer, adopted a Rule 10b5-1 trading arrangement on April 11, 2025, to sell 120,000 shares by July 31, 2026[126](index=126&type=chunk) [Item 6. Exhibits](index=29&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed with the Form 10-Q, including corporate governance documents and certifications - Exhibits include Amended and Restated Certificate of Incorporation, Bylaws, Form of Common Stock Certificate, Form of Pre-Funded Warrant, CEO/CFO Certifications (31.1, 31.2, 32.1, 32.2), and Inline XBRL documents[127](index=127&type=chunk) SIGNATURES This section contains the official signatures of the company's executive officers, certifying the report - The report was signed on August 7, 2025, by Sarah Boyce, President, Chief Executive Officer and Director, and Michael F. MacLean, Chief Financial Officer[131](index=131&type=chunk)

[Executive Summary & Recent Highlights](index=1&type=section&id=Executive%20Summary%20%26%20Recent%20Highlights) Avidity Biosciences reported significant advancements in its neuromuscular programs, including regulatory alignments and positive clinical data, positioning the company for multiple Biologics License Application submissions and a strong financial outlook - Avidity is preparing for **three potential BLA submissions** over a 12-month period, with the **first BLA for del-zota expected by year-end 2025**[1](index=1&type=chunk)[2](index=2&type=chunk)[3](index=3&type=chunk) - The company has **aligned with the FDA on accelerated and full approval pathways** for del-brax for FSHD and reported **positive topline data** from the Phase 1/2 FORTITUDE™ trial[1](index=1&type=chunk)[5](index=5&type=chunk) - Avidity maintains a **strong balance sheet** and **cash runway projected to mid-2027**, supporting global commercial launch readiness, with the **first potential U.S. commercial launch in 2026**[1](index=1&type=chunk)[2](index=2&type=chunk)[6](index=6&type=chunk) [Recent Corporate Highlights](index=1&type=section&id=Company%20Highlights) Avidity Biosciences reported significant progress across its late-stage neuromuscular programs, including regulatory alignments, positive clinical data, and initiation of confirmatory studies, with three potential Biologics License Application (BLA) submissions within a 12-month period, with the first anticipated by year-end 2025 [Delpacibart zotadirsen (del-zota) for DMD44](index=1&type=section&id=Delpacibart%20zotadirsen%20(del-zota)%20for%20the%20treatment%20of%20people%20living%20with%20Duchenne%20muscular%20dystrophy%20with%20mutations%20amenable%20to%20exon%2044%20skipping%20(DMD44)) Delpacibart zotadirsen for DMD44 received Breakthrough Therapy designation and is on track for its first Biologics License Application submission by year-end 2025 - In July 2025, the FDA granted **Breakthrough Therapy designation** to del-zota for DMD44[3](index=3&type=chunk) - Avidity remains on track for its **first BLA submission for del-zota by year-end 2025**[3](index=3&type=chunk) - Topline and functional data from the EXPLORE44-OLE™ trial are planned for presentation in **Q4 2025**[5](index=5&type=chunk) [Delpacibart etedesiran (del-desiran) for DM1](index=2&type=section&id=Delpacibart%20etedesiran%20(del-desiran)%20for%20the%20treatment%20of%20myotonic%20dystrophy%20type%201%20(DM1)) Enrollment for the Phase 3 HARBOR™ trial for del-desiran for DM1 is complete, with marketing application submissions anticipated to begin in H2 2026 - Enrollment in the Phase 3 HARBOR™ trial for del-desiran was **completed in July 2025**[5](index=5&type=chunk) - Updates from the MARINA-OLE™ trial, including long-term efficacy and safety data, are expected in **Q4 2025**[5](index=5&type=chunk) - Topline data from the HARBOR™ study is anticipated in **Q2 2026**, with marketing application submissions in the U.S., E.U., and Japan expected to begin in **H2 2026**[5](index=5&type=chunk) [Delpacibart braxlosiran (del-brax) for FSHD](index=2&type=section&id=Delpacibart%20braxlosiran%20(del-brax)%20for%20the%20treatment%20of%20facioscapulohumeral%20muscular%20dystrophy%20(FSHD)) Delpacibart braxlosiran for FSHD received FDA alignment on accelerated and full approval pathways, with a confirmatory Phase 3 study initiated and a BLA submission planned for H2 2026 - In June 2025, Avidity announced **FDA alignment on accelerated and full approval pathways** for del-brax[5](index=5&type=chunk) - **Positive topline Phase 1/2 FORTITUDE™ data** showed consistent improvement in functional and quality of life measures, significant reductions in biomarkers (KHDC1L/cDUX, creatine kinase), and favorable safety[5](index=5&type=chunk) - A global, confirmatory Phase 3 FORTITUDE-3™ study has been initiated to support global approval, with a planned BLA submission for accelerated approval in **H2 2026**[5](index=5&type=chunk) [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) Avidity Biosciences reported its financial performance for Q2 2025, highlighting a strong cash position, increased collaboration revenues, and rising operating expenses due to advanced clinical programs [Financial Highlights](index=2&type=section&id=Financial%20Highlights) Avidity Biosciences reported a strong cash position of approximately $1.2 billion as of June 30, 2025, with an extended cash runway to mid-2027, collaboration revenues increased year-over-year for Q2 2025, while R&D and G&A expenses significantly rose due to advanced clinical programs and expanded operations - Cash, cash equivalents, and marketable securities totaled approximately **$1.2 billion** as of June 30, 2025[5](index=5&type=chunk) - The company received net proceeds of **$185.5 million** from an at-the-market offering program after Q2 2025[5](index=5&type=chunk) - Avidity expects its current cash and proceeds from the offering to fund operations to **mid-2027**[6](index=6&type=chunk) Collaboration Revenues (in thousands) | Period | 2025 | 2024 | Change (YoY) | Primary Driver | | :--------------------- | :----- | :----- | :----------- | :------------- | | Three Months Ended June 30 | $3,847 | $2,045 | +88.1% | Bristol Myers Squibb partnership | | Six Months Ended June 30 | $5,420 | $5,588 | -3.0% | Bristol Myers Squibb partnership | Operating Expenses (in thousands) | Expense Type | Period | 2025 | 2024 | Change (YoY) | Primary Driver | | :------------- | :--------------------- | :------- | :------- | :----------- | :------------- | | R&D Expenses | Three Months Ended June 30 | $138,125 | $63,940 | +116.0% | Advancement of del-desiran, del-brax, del-zota, and research capabilities expansion | | R&D Expenses | Six Months Ended June 30 | $237,615 | $130,772 | +81.7% | Advancement of del-desiran, del-brax, del-zota, and research capabilities expansion | | G&A Expenses | Three Months Ended June 30 | $36,864 | $20,731 | +77.8% | Higher personnel and commercial infrastructure costs | | G&A Expenses | Six Months Ended June 30 | $70,464 | $34,629 | +103.5% | Higher personnel and commercial infrastructure costs | [Selected Condensed Consolidated Financial Information](index=6&type=section&id=Selected%20Condensed%20Consolidated%20Financial%20Information) This section provides the unaudited condensed consolidated financial statements, including the Statements of Operations for the three and six months ended June 30, 2025 and 2024, and the Balance Sheets as of June 30, 2025, and December 31, 2024 [Statements of Operations](index=6&type=section&id=Statements%20of%20Operations) This section presents the unaudited condensed consolidated statements of operations for the three and six months ended June 30, 2025 and 2024 Statements of Operations (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Collaboration revenue | $3,847 | $2,045 | $5,420 | $5,588 | | Research and development expenses | $138,125 | $63,940 | $237,615 | $130,772 | | General and administrative expenses | $36,864 | $20,731 | $70,464 | $34,629 | | Total operating expenses | $174,989 | $84,671 | $308,079 | $165,401 | | Loss from operations | $(171,142) | $(82,626) | $(302,659) | $(159,813) | | Other income, net | $13,827 | $11,833 | $29,571 | $20,165 | | Net loss | $(157,315) | $(70,793) | $(273,088) | $(139,648) | | Net loss per share, basic and diluted | $(1.21) | $(0.65) | $(2.11) | $(1.44) | | Weighted-average shares outstanding | 129,622 | 106,928 | 129,428 | 97,070 | [Balance Sheets](index=6&type=section&id=Balance%20Sheets) This section provides the unaudited condensed consolidated balance sheets as of June 30, 2025, and December 31, 2024 Balance Sheets (in thousands) | Asset/Liability/Equity | June 30, 2025 | December 31, 2024 | | :---------------------------------- | :-------------- | :---------------- | | Cash, cash equivalents and marketable securities | $1,183,144 | $1,501,497 | | Total current assets | $1,250,560 | $1,542,290 | | Total assets | $1,368,926 | $1,563,895 | | Total current liabilities | $135,061 | $98,018 | | Total liabilities | $176,262 | $138,936 | | Stockholders' equity | $1,192,664 | $1,424,959 | | Total liabilities and stockholders' equity | $1,368,926 | $1,563,895 | [About Avidity](index=4&type=section&id=About%20Avidity) Avidity Biosciences is a biopharmaceutical company focused on developing a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), with their proprietary AOC platform aiming to deliver RNA therapies to previously unreachable targets and diseases, currently focusing on three rare muscle diseases (DM1, DMD, FSHD) and advancing programs in precision cardiology and immunology - Avidity's mission is to improve lives by delivering **Antibody Oligonucleotide Conjugates (AOCs™)**, a new class of RNA therapeutics[7](index=7&type=chunk) - The proprietary **AOC platform** combines monoclonal antibodies with oligonucleotide therapies for **targeted RNA delivery**, successfully demonstrating **delivery into muscle**[7](index=7&type=chunk) - The company has clinical development programs for **three rare muscle diseases**: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD)[7](index=7&type=chunk) - Avidity is also advancing **two wholly-owned precision cardiology candidates** and **expanding its pipeline** through partnerships in cardiology and immunology[7](index=7&type=chunk) [Forward-Looking Statements](index=4&type=section&id=Forward-Looking%20Statements) This section serves as a cautionary statement, highlighting that all forward-looking statements in the press release are based on current beliefs and expectations and are subject to inherent risks and uncertainties, with actual results potentially differing materially due to factors such as clinical trial outcomes, regulatory approvals, commercialization challenges, and the unproven nature of the AOC platform - Statements regarding future plans, BLA submissions, clinical trial status, data readouts, regulatory approvals, commercialization efforts, and financial runway are **forward-looking**[8](index=8&type=chunk) - **Actual results may differ** due to **risks** including, but not limited to, clinical trial data not supporting approvals, unsuccessful product launches, unexpected adverse side effects, regulatory inconsistencies, and the unproven nature of the AOC platform[9](index=9&type=chunk)[10](index=10&type=chunk) - Readers are cautioned **not to place undue reliance** on these statements, which speak only as of the date of the release, and the company undertakes no obligation to update them[9](index=9&type=chunk)[10](index=10&type=chunk) [Contacts](index=5&type=section&id=Investor%20Contact) This section provides contact information for investor and media inquiries related to Avidity Biosciences - **Investor Contact**: Kat Lange, (619) 837-5014, investors@aviditybio.com[11](index=11&type=chunk) - **Media Contact**: Kristina Coppola, (619) 837-5016, media@aviditybio.com[11](index=11&type=chunk)

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].