Question-and-Answer SessionMichael MacLeanChief Financial Officer Absolutely. Happy to do that. And first of all, thank you for having us at the conference. It's always wonderful to kind of start out the fall here at the Wells Fargo Conference in Boston. And so look, first, I'd just start with the fact that our vision is to profoundly improve people's lives through the delivery of RNA therapy. And as I look back on the 5 years since we IPO-ed at a time where we had no programs in the clinic. And as we sit h ...

Avidity Biosciences (RNA) FY 2025 Conference September 04, 2025 12:45 PM ET Company ParticipantsKathleen Gallagher - Chief Program OfficerMichael MacLean - CFOConference Call ParticipantsYanan Zhu - Senior Analyst - Biotechnology Equity ResearchYanan ZhuGreat. Thanks, everyone, for being here. My name is Yan Andriu. I'm one of the biotech analysts here at Wells Fargo. It is our great privilege to have the Avidity Biosciences management team here for Fireside Chat. With me on stage, CFO Mike MacLean and Kath ...

Avidity Biosciences (RNA) FY 2025 Conference September 04, 2025 12:45 PM ET Speaker0Great. Thanks, everyone, for being here. My name is Yan Andriu. I'm one of the biotech analysts here at Wells Fargo. It is our great privilege to have the Avidity Biosciences management team here for Fireside Chat. With me on stage, CFO Mike MacLean and Kath Gallagher, Program Manager for the Muscle Disease Programs. Thank you for being here.Speaker2Thank you, Yan.Speaker0Great. I was wondering if, Mike, could you provide an ...

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.

Key Takeaways Avidity shares jumped 36% after reports that Novartis is interested in acquiring the biotech.FT noted talks are in early stages with no guarantee of a deal and other suitors may emerge.Avidity develops RNA therapies for rare muscular diseases and has major pharma partnerships.Shares of Avidity Biosciences (RNA) have soared 36% in the past month, all thanks to a report issued by the Financial Times (FT), which stated that pharma giant Novartis (NVS) is interested in acquiring the company.Per th ...

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].

高盛最新研报以"2Q25EPS更新"为主题，其中重点对Amylyx(AMLX.US)、CG Oncology(CGON.US)、硕 迪生物(GPCR.US)、Ideaya生物科学(IDYA.US)等生物科技公司进行逐一拆解，结论是：运营数据大体 符合预期，投资逻辑无需大改，但个别公司因现金流或临床节点微调目标价。 Amylyx：开启药物商业化尝试 公司二季度运营费用约4,300万美元，略高于市场共识的4,100万美元和高盛原估的4,000万美元，差异主 要来自研发费用，2,700万美元对预期2,200万～2,500万美元区间，销售费用则与市场持平。 投资者仍紧盯减重术后低血糖(PBH)三期临床试验：管理层重申2025年完成入组、2026年上半年读出数 据、2027年有望上市，潜在可及患者约16万人。公司已悄悄启动商业化热身，包括锁定核心内分泌中 心、试水定价，若对标近期罕见内分泌新药，高盛目前"10万美元/年"的定价假设仍有上行空间。 管线方面，进行性核上性麻痹(PSP)二期临床将在三季度揭盲，公司设定了"PSP评分量表延缓≥20%"的 高门槛;Wolfram综合征三期设计正与FDA沟通，今年会有更新;ALS ...

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)

Planned del-zota BLA submission at year end 2025 for DMD44 on track to be Avidity's first BLA submission On track for three potential BLA submissions over a 12-month period Strong balance sheet and cash runway to mid-2027 enabling global commercial launch readiness; first potential commercial launch in U.S. in 2026 SAN DIEGO, August 7, 2025 /PRNewswire/-- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleoti ...