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].

Core Viewpoint - Novartis is reportedly proposing to acquire Avidity Biosciences Inc. to expand its pharmaceutical pipeline, leading to a significant increase of over 24% in Avidity's stock price [1]. Company Overview - Avidity Biosciences, Inc. (NASDAQ: RNA) is a biopharmaceutical company focused on developing a new class of RNA therapies known as Antibody Oligonucleotide Conjugates (AOC™) [3][12]. - The company has received Breakthrough Therapy designation from the FDA for its drug delpacibart zotadirsen (del-zota), aimed at treating Duchenne Muscular Dystrophy (DMD) patients with exon 44 skipping mutations [3][6]. Drug Development and Clinical Trials - Del-zota is currently being evaluated in the Phase 2 EXPLORE44-OLE trial, which is an open-label extension study for DMD patients [4][8]. - The Phase 1/2 EXPLORE44 trial demonstrated significant improvements in biomarkers, including increased dystrophin production and decreased creatine kinase levels, indicating a favorable safety profile [4][11]. - Avidity plans to submit a Biologics License Application (BLA) for del-zota by the end of 2025, with commercial preparations underway for a potential U.S. launch [4][6]. Disease Context - Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle degeneration due to the absence of dystrophin, leading to severe muscle weakness and a significantly reduced lifespan [9]. - DMD primarily affects males, with an incidence of approximately 1 in every 3,500 to 5,000 male births globally [9]. Technology and Innovation - Avidity's AOC platform combines the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, enabling targeted delivery of RNA to muscle tissues [12]. - The company is advancing clinical development projects for three rare neuromuscular diseases: DM1, DMD, and FSHD, while also exploring candidates for rare genetic cardiomyopathies [12].

Core Insights - Avidity Biosciences has completed enrollment in the Phase 3 HARBOR clinical trial for del-desiran, targeting myotonic dystrophy type 1 (DM1), with topline data expected in Q2 2026 [1][3] - The company plans to submit marketing applications for del-desiran in the U.S., EU, and Japan starting in H2 2026, aiming to be the first approved drug for DM1 globally [1][2] - Del-desiran has received multiple designations from the FDA and EMA, including Breakthrough Therapy and Orphan Drug status, indicating its potential significance in treating DM1 [4][7] Company Overview - Avidity Biosciences focuses on developing a new class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs™), which aim to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies [8] - The company is advancing clinical programs for rare muscle diseases, including DM1, Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD) [8] Clinical Trial Details - The HARBOR trial is a randomized, placebo-controlled study involving approximately 150 participants aged 16 and older, assessing the efficacy of del-desiran every eight weeks [5] - Primary and secondary endpoints include video hand opening time (vHOT), muscle strength, and activities of daily living, with an open-label extension trial expected to begin in Q3 2025 [5] Disease Context - Myotonic dystrophy type 1 (DM1) is a rare, hereditary neuromuscular disease characterized by multisystemic manifestations and a significant disease burden, with no approved treatments currently available [6][7]

Core Insights - The report maintains a positive outlook on small molecule drug conjugates (SMDCs) and related companies, highlighting their potential in cancer treatment due to their ability to enhance efficacy while reducing toxicity [1][5][17] - The report emphasizes the clinical advantages of SMDCs, including better tumor penetration, reduced toxicity to normal cells, and easier control over synthesis and costs compared to antibody-drug conjugates (ADCs) [5][11][12] - The report identifies domestic biopharmaceutical companies, particularly Affinivax, as leaders in the SMDC space, showcasing significant advancements in innovative cancer drug development [5][12][17] Industry Overview - The pharmaceutical and biotechnology sector has shown a relative price-to-earnings (P/E) ratio of 51.14 as of July 25, 2025, which is significantly higher than its historical low of 24.38, indicating a premium valuation compared to the broader market [19] - The report notes that the healthcare sector's valuation is 279% higher than the Shanghai Composite Index, reflecting strong investor interest and confidence in the industry [19] - Recent market performance indicates a 1.90% increase in the pharmaceutical and biotechnology sector from July 21 to July 25, 2025, ranking it 16th among 27 sub-industries [26][29] Company Focus - The report suggests monitoring companies involved in the SMDC space, including Affinivax, and those collaborating with them, such as Innovent Biologics and others, which are expected to benefit from the growing interest in innovative cancer therapies [5][17][18] - Specific companies highlighted for their innovative drug development capabilities include Innovent Biologics, Shunyi Pharmaceutical, and others, which are positioned to capitalize on the advancements in SMDC technology [5][18]

Core Insights - Avidity Biosciences has received Breakthrough Therapy designation from the FDA for delpacibart zotadirsen (del-zota) aimed at treating Duchenne muscular dystrophy (DMD) in patients with mutations suitable for exon 44 skipping [1][4][7] - The company is on track for a Biologics License Application (BLA) submission by the end of 2025, with ongoing preparations for a potential U.S. launch of del-zota following FDA approval [5][4] - Del-zota is currently in the Phase 2 EXPLORE44 Open-Label Extension trial, building on positive results from the Phase 1/2 trial that showed significant improvements in biomarkers related to DMD [2][4][10] Company Overview - Avidity Biosciences focuses on developing a new class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs™) [13] - The company aims to address rare neuromuscular diseases, including DMD, myotonic dystrophy type 1 (DM1), and facioscapulohumeral muscular dystrophy (FSHD) [13] - Avidity's proprietary AOC platform allows for targeted delivery of RNA into muscle tissue, which is a significant advancement in the field of RNA therapeutics [13] Product Development - Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to specifically skip exon 44 of the dystrophin gene, facilitating the production of near-full length dystrophin [3][12] - The Phase 1/2 EXPLORE44 trial demonstrated statistically significant increases in exon skipping and dystrophin production, along with a reduction in creatine kinase levels to near normal [4][12] - The ongoing EXPLORE44-OLE study will evaluate the long-term safety and efficacy of del-zota, with a total treatment duration of approximately 24 months [9][10] Regulatory Designations - In addition to Breakthrough Therapy designation, del-zota has received Orphan designation, Rare Pediatric Disease designation, and Fast Track designation from the FDA [7][12] - These designations highlight the drug's potential to significantly improve treatment options for patients with DMD, a condition with a high unmet medical need [11][6]

Core Insights - Avidity Biosciences announced FDA alignment on accelerated and full approval pathways for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD) [1] - The company will present two oral and one poster presentations at the 32nd Annual FSHD Society International Research Congress in Amsterdam on June 12-13, 2025 [1][2] - Key presentations will include topline data from the FORTITUDE trial and characterization of a novel DUX4-regulated circulating biomarker [1][6] Company Overview - Avidity Biosciences focuses on delivering a new class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs™) [4] - The company aims to revolutionize RNA therapy by combining the specificity of monoclonal antibodies with the precision of oligonucleotide therapies [4] - Avidity is advancing clinical development programs for three rare neuromuscular diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD) [4] Upcoming Presentations - Jeffrey M. Statland, M.D. will present topline data from the dose escalation cohorts of the FORTITUDE trial on June 13, 2025 [6] - Stephen Tapscott, M.D., Ph.D. will present findings on a DUX4-regulated circulating biomarker on June 12, 2025 [6] - A poster presentation is also scheduled for June 12, 2025 [6]

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