
Executive Summary & Recent Highlights 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 2025123 - 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™ trial15 - 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 2026126 Recent Corporate Highlights 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 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 DMD443 - Avidity remains on track for its first BLA submission for del-zota by year-end 20253 - Topline and functional data from the EXPLORE44-OLE™ trial are planned for presentation in Q4 20255 Delpacibart etedesiran (del-desiran) for 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 20255 - Updates from the MARINA-OLE™ trial, including long-term efficacy and safety data, are expected in Q4 20255 - 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 20265 Delpacibart braxlosiran (del-brax) for 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-brax5 - 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 safety5 - 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 20265 Second Quarter 2025 Financial Results 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 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, 20255 - The company received net proceeds of $185.5 million from an at-the-market offering program after Q2 20255 - Avidity expects its current cash and proceeds from the offering to fund operations to mid-20276 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 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 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 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 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 therapeutics7 - The proprietary AOC platform combines monoclonal antibodies with oligonucleotide therapies for targeted RNA delivery, successfully demonstrating delivery into muscle7 - 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 - Avidity is also advancing two wholly-owned precision cardiology candidates and expanding its pipeline through partnerships in cardiology and immunology7 Forward-Looking Statements 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-looking8 - 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 platform910 - 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 them910 Contacts This section provides contact information for investor and media inquiries related to Avidity Biosciences - Investor Contact: Kat Lange, (619) 837-5014, investors@aviditybio.com11 - Media Contact: Kristina Coppola, (619) 837-5016, media@aviditybio.com11