Financial Performance - The company has incurred significant operating losses of $322.3 million, $212.2 million, and $174.0 million for the years ended December 31, 2024, 2023, and 2022, respectively, with an accumulated deficit of $893.1 million as of December 31, 2024[193]. - The company expects to continue incurring losses for the foreseeable future as it develops its product candidates and seeks regulatory approval[194]. - The company will require substantial additional financing to achieve its goals, with potential capital needs driven by ongoing clinical trials and commercialization efforts[195]. - The company’s future capital requirements will depend on various factors, including the success of its clinical trials and regulatory approvals[198]. - As of December 31, 2024, the company reported federal net operating losses (NOLs) of approximately $249.4 million and state NOLs of approximately $439.3 million[355]. Product Development and Clinical Trials - The company has three product candidates in clinical development, while all other development programs are in preclinical or discovery stages[202]. - The company acknowledges that preclinical and clinical development is lengthy, expensive, and uncertain, with a high historical failure rate for product candidates in the industry[211]. - The company has experienced a partial clinical hold on its Phase 1/2 MARINA clinical trial due to a serious adverse event, which was lifted in October 2024, indicating potential delays in product development timelines[217]. - The company may face challenges in patient enrollment for clinical trials, which could delay development activities and affect the timing of clinical trials[224]. - The timeline for clinical trials is heavily dependent on the ability to recruit a sufficient number of eligible patients, with potential delays if recruitment is inadequate[226]. - The company has not completed any pivotal clinical trials or submitted a Biologics License Application (BLA) for regulatory approval, which may hinder future product commercialization[234]. Regulatory Environment - The company must demonstrate safety, purity, and potency of product candidates to the FDA, which involves conducting well-controlled clinical trials that are expensive and time-consuming[215]. - Regulatory approval processes are extensive and costly, often taking years, and there is no guarantee of approval even after significant investment in clinical development[236]. - The FDA or comparable foreign regulatory authorities can delay or deny approval for various reasons, including negative results from clinical trials or safety concerns[238]. - The company is subject to varying interpretations of clinical data by regulatory authorities, which could affect the approval and commercialization of product candidates[206]. - Regulatory changes, such as the EU Clinical Trials Regulation, may impact the company's development plans and require compliance adjustments[213]. Competition and Market Dynamics - The company faces significant competition in the biopharmaceutical industry, which may adversely affect its ability to develop and commercialize products[191]. - The company faces significant competition from larger pharmaceutical firms and ongoing developments in RNA-targeted therapies[298]. - The total addressable market for the company's product candidates may be smaller than anticipated, affecting revenue potential and business sustainability[306]. - Commercial success of product candidates will depend on market acceptance by physicians, patients, and healthcare payors, which is uncertain[286]. - Factors affecting market acceptance include clinical efficacy, safety, pricing, and reimbursement from third-party payors[287]. Collaborations and Partnerships - In November 2023, the company entered into a collaboration agreement with BMS for the development of up to five cardiovascular targets, with BMS responsible for funding all future clinical development and commercialization activities[273]. - The company has entered into a collaboration agreement with Lilly for the discovery and development of AOCs, with Lilly solely responsible for funding preclinical research and clinical development[271]. - Any unilateral termination of the agreements with Lilly or BMS could prevent the company from receiving research funding and adversely affect public perception of its product candidates[274]. - The company may seek additional collaborations or licenses but may not be successful, potentially relinquishing valuable rights and facing unfavorable terms[276]. Manufacturing and Supply Chain Risks - The company relies on third parties for manufacturing its product candidates, which increases the risk of delays or insufficient quantities[191]. - The company does not own or operate manufacturing facilities and has no plans to develop its own manufacturing capabilities, relying entirely on third-party manufacturers[265]. - The company does not currently have arrangements for redundant supply or a second source for all required raw materials, which could impact manufacturing if issues arise with third-party manufacturers[268]. - The company relies on third parties for preclinical studies and clinical trials, which poses risks if these parties do not meet their contractual obligations[261]. Intellectual Property and Legal Risks - The company faces risks related to intellectual property, as failure to obtain and maintain patent protection could allow competitors to commercialize similar products, adversely affecting its business[359]. - The patent prosecution process is complex and costly, and the company may not be able to secure necessary patent protections in a timely manner, which could harm its financial condition and prospects[362]. - The company may face claims challenging the inventorship of its patents, which could lead to litigation and loss of valuable intellectual property rights[380]. - The company relies on trade secrets and confidentiality agreements to protect its proprietary technology, but enforcement of these agreements can be difficult and costly[381]. Compliance and Regulatory Obligations - Compliance with healthcare laws and regulations is critical, as failure to adhere could negatively impact the company's financial condition[319]. - The company must comply with various federal and state healthcare laws to avoid significant penalties, including exclusion from government-funded healthcare programs[321]. - The company intends to participate in the Medicaid Drug Rebate Program (MDRP) and other governmental pricing programs, which require compliance with reporting and payment obligations[331]. - The company faces increased scrutiny over drug pricing, with potential Congressional inquiries and new regulations aimed at enhancing pricing transparency[326]. Economic and Market Conditions - The U.S. economy experienced significant inflation from 2021 to 2024, impacting costs for commodities, labor, and materials, which could adversely affect the company's financial condition[358]. - Pricing pressures are expected due to managed healthcare trends and governmental cost containment initiatives[297]. - The reimbursement landscape is complex and varies significantly among third-party payors, impacting commercialization efforts[295].

Financial Performance - Avidity reported approximately $1.5 billion in cash, cash equivalents, and marketable securities as of December 31, 2024[13]. - Collaboration revenue for Q4 2024 reached $2,973 million, up 35.5% from $2,193 million in Q4 2023[18]. - Total operating expenses increased to $123,963 million in Q4 2024, compared to $68,936 million in Q4 2023, representing an increase of 80%[18]. - Net loss for Q4 2024 was $102,257 million, compared to a net loss of $60,443 million in Q4 2023, indicating a 69.5% increase in losses[18]. - Cash, cash equivalents, and marketable securities rose significantly to $1,501,497 million in 2024 from $595,351 million in 2023, showing a growth of 152.5%[18]. - Total assets increased to $1,563,895 million in 2024, up from $628,555 million in 2023, marking a growth of 149.5%[18]. - Stockholders' equity surged to $1,424,959 million in 2024, compared to $500,764 million in 2023, reflecting a 184.5% increase[18]. - The net loss per share for Q4 2024 was $0.80, slightly up from $0.79 in Q4 2023[18]. - Total current liabilities increased to $98,018 million in 2024 from $80,680 million in 2023, an increase of 21.4%[18]. Research and Development - Research and Development (R&D) expenses increased to $95.6 million for Q4 2024, up from $52.8 million in Q4 2023, and totaled $303.6 million for the year ended 2024 compared to $191.0 million for 2023[13]. - Research and development expenses for the year totaled $303,593 million, up from $190,968 million in 2023, an increase of 59%[18]. - The Phase 1/2 EXPLORE44 trial for del-zota demonstrated an unprecedented unadjusted increase of 25% in delivery to skeletal muscle[4]. - Enrollment in the EXPLORE44 Open-label Extension (OLE) study is complete, supporting the BLA submission anticipated at year-end 2025[3]. - Avidity achieved positive long-term data from the MARINA-OLE study for del-desiran, showing reversal of disease progression across multiple endpoints[10]. - Avidity is preparing for three potential successive product launches for DMD, DM1, and FSHD starting in 2026[1]. - The global Phase 3 HARBOR trial for del-desiran is ongoing and on track for completion in mid-2025[10]. - Avidity is expanding its pipeline into precision cardiology, including candidates for PRKAG2 syndrome and PLN cardiomyopathy[10]. General and Administrative Expenses - General and Administrative (G&A) expenses rose to $28.3 million for Q4 2024, compared to $16.1 million in Q4 2023, and reached $86.2 million for the year ended 2024, up from $54.2 million in 2023[13]. - General and administrative expenses rose to $86,240 million in 2024 from $54,190 million in 2023, representing a 59.3% increase[18]. Future Plans - Avidity plans to submit its first Biologics License Application (BLA) for del-zota by year-end 2025, with the FDA confirming an accelerated approval path[5].

Core Insights - Avidity Biosciences is positioned to lead in rare neuromuscular diseases with significant milestones expected in 2025, including the first BLA submission for its AOC platform [1][2] - The company has a strong financial position with approximately $1.5 billion in cash as of the end of 2024, enabling continued development and commercialization efforts [2][14] - Avidity is preparing for three potential product launches for Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), and facioscapulohumeral muscular dystrophy (FSHD) starting in 2026 [1][4] Clinical Programs - Avidity's del-zota for DMD44 is on track for a BLA submission by the end of 2025, with positive initial data showing a 25% increase in near full-length dystrophin production [4][10] - The EXPLORE44-OLE study has completed enrollment, and data from both the EXPLORE44 and EXPLORE44-OLE studies will support the BLA submission [4][3] - Del-desiran for DM1 is progressing with the completion of enrollment in the Phase 3 HARBOR trial expected by mid-2025, and the FDA has granted breakthrough therapy designation for this candidate [4][10] Financial Performance - For the fourth quarter of 2024, Avidity reported a net loss of $102.3 million, compared to a net loss of $60.4 million in the fourth quarter of 2023 [14][16] - Research and development expenses increased to $95.6 million in Q4 2024 from $52.8 million in Q4 2023, driven by advancements in clinical programs [14][16] - Collaboration revenues for Q4 2024 were $3.0 million, up from $2.2 million in Q4 2023, primarily from partnerships with Bristol Myers Squibb and Eli Lilly [14][16] Strategic Initiatives - Avidity is expanding its global commercial infrastructure and team to support upcoming product launches and enhance its AOC technology [2][4] - The company is also broadening its pipeline into precision cardiology, with two new candidates for PRKAG2 syndrome and PLN cardiomyopathy [10][11] - Avidity's proprietary AOC platform aims to revolutionize RNA therapeutics by combining the specificity of monoclonal antibodies with oligonucleotide therapies [11]

Accelerated approval path confirmed for del-zota; Biologics License Application (BLA) submission planned for year end 2025   On track to complete enrollment in del-desiran HARBORTM Phase 3 and potential registrational del-brax biomarker cohort as well as initiate global pivotal trial for del-brax by mid-2025    Eric Mosbrooker expands role to Chief Commercial Officer; Charles Calderaro III, Chief Technical Officer, and Kat Lange, Chief Business Officer, join as company prepares for global commercialization ...

Shares of Avidity Biosciences (RNA) rose more than 12% on Wednesday after the company announced that it is expanding its current RNA-based pipeline of rare muscle disorders to explore a new therapeutic area — precision cardiology.Avidity Enters the Cardio Space With Two New DrugsThe company has decided to advance two new wholly-owned pipeline drugs, AOC 1086 and AOC 1072, targeting two rare genetic cardiomyopathies, PLN cardiomyopathy and PRKAG2 syndrome, respectively.There is currently no FDA-approved ther ...

Avidity Biosciences, Inc. (RNA) came out with a quarterly loss of $0.65 per share versus the Zacks Consensus Estimate of a loss of $0.79. This compares to loss of $0.71 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 17.72%. A quarter ago, it was expected that this company would post a loss of $0.76 per share when it actually produced a loss of $0.65, delivering a surprise of 14.47%.Over the last four quarters, the company has ...

Financial Performance - The company reported net losses of $212.2 million and $174.0 million for the years ended December 31, 2023 and 2022, respectively, and $220.0 million for the nine months ended September 30, 2024, with an accumulated deficit of $790.8 million as of September 30, 2024[68]. - Revenue for the three months ended September 30, 2024, decreased by $0.5 million to $2.336 million compared to $2.818 million in the same period of 2023, while revenue for the nine months increased by $0.6 million to $7.924 million from $7.367 million[78][79]. - Other income increased by $11.5 million for the three months ended September 30, 2024, totaling $17.736 million, and by $20.8 million for the nine months to $37.901 million[78][83]. Cash and Financing - The company has approximately $1.6 billion in cash, cash equivalents, and marketable securities as of September 30, 2024, expected to fund operations for at least 12 months[68]. - The company raised $379.8 million from a private placement of 15,224,773 shares at $16.50 per share on March 4, 2024, and $432.8 million from a public offering of 12,132,500 shares at $38.00 per share on June 17, 2024[84][85]. - Net cash provided by financing activities for the nine months ended September 30, 2024, was $1.2 billion, a substantial increase from $62.0 million in the same period in 2023, mainly from common stock sales[92]. Research and Development - Delpacibart zotadirsen (del-zota) demonstrated a mean increase of 37% in exon 44 skipping and a greater than 80% reduction of creatine kinase compared to baseline in the EXPLORE44 trial[64]. - Delpacibart etedesiran (del-desiran) is currently in Phase 3 development with the ongoing global HARBOR™ trial, with enrollment on track[63]. - The company is advancing additional exon-skipping candidates from its DMD franchise, with exon 45 currently in IND-enabling studies[65]. - The company is pursuing a potential accelerated approval path for del-brax, with enrollment in the biomarker cohort expected to be completed in the first half of 2025[66]. - The company expects research and development expenses to continue increasing as it advances preclinical and clinical development programs[75][76]. Expenses - Research and development expenses increased by $29.5 million for the three months ended September 30, 2024, totaling $77.197 million, compared to $47.714 million in the same period of 2023, and increased by $69.8 million for the nine months to $207.968 million from $138.151 million[78][80][81]. - General and administrative expenses rose by $9.5 million for the three months ended September 30, 2024, reaching $23.273 million, compared to $13.729 million in the same period of 2023, and increased by $19.8 million for the nine months to $57.902 million from $38.071 million[78][82]. - The company expects to incur significant commercialization expenses related to product sales, marketing, manufacturing, and distribution if regulatory approval is obtained for any product candidates[86]. Capital Requirements and Market Risks - Future capital requirements are uncertain and will depend on various factors, including clinical trial costs, regulatory review outcomes, and market acceptance of approved products[86]. - The company faces uncertainty in the timelines and costs associated with research and development activities, which can vary significantly for each product candidate[75]. - As of September 30, 2024, there have been no material changes in market risk from previous disclosures[97]. - The company may seek additional capital due to favorable market conditions or strategic considerations, even if current funds are deemed sufficient[86]. Operational Activities - The company has not generated any revenue from product sales since inception and plans to finance cash needs through equity offerings and collaborations[68]. - The company has incurred operating losses in each year since inception, with expectations for substantial increases in expenses and operating losses as clinical trials progress[68]. - Net cash used in operating activities for the nine months ended September 30, 2024, was $201.0 million, an increase of $65.4 million compared to $135.6 million for the same period in 2023[90]. - Net cash used in investing activities for the nine months ended September 30, 2024, was $790.0 million, significantly higher than $181.1 million for the same period in 2023, primarily due to $1.1 billion in marketable securities purchases[91]. - The company entered into a sublease agreement in April 2024, with total future lease commitments of approximately $72.6 million[95]. - The company does not expect to generate revenue from product sales until successful development and regulatory approval of product candidates, which may take several years[86].

Exhibit 99.1 Avidity Biosciences Reports Third Quarter 2024 Financial Results and Recent Highlights Enrollment in global Phase 3 HARBOR study for del-desiran in DM1 is on track TM Avidity initiated biomarker cohort for del-brax FORTITUDE study for FSHD; pursuing a potential accelerated approval path for del-brax TM TM Reported positive del-zota data from Phase 1/2 EXPLORE44 trial for DMD44 Avidity to provide a first look at precision cardiology candidates and a glimpse at next-generation technology innovati ...

Enrollment in global Phase 3 HARBOR™ study for del-desiran in DM1 is on trackAvidity initiated biomarker cohort for del-brax FORTITUDE™ study for FSHD; pursuing a potential accelerated approval path for del-braxReported positive del-zota data from Phase 1/2 EXPLORE44™ trial for DMD44Avidity to provide a first look at precision cardiology candidates and a glimpse at next-generation technology innovations via webcast event November 12, 2024SAN DIEGO, Nov. 7, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nas ...

Avidity Biosciences, Inc. (RNA) is expected to deliver a year-over-year decline in earnings on higher revenues when it reports results for the quarter ended September 2024. This widely-known consensus outlook gives a good sense of the company's earnings picture, but how the actual results compare to these estimates is a powerful factor that could impact its near-term stock price.The earnings report might help the stock move higher if these key numbers are better than expectations. On the other hand, if they ...