RNA

-- IND clearance for ATR 1072 and launch of Corventis Phase 1/2 trial in PRKAG2 syndrome -- -- Achieved second milestone payment under global cardiovascular collaboration with Bristol Myers Squibb -- SAN DIEGO, Aug. 13, 2026 /PRNewswire/ -- Atrium Therapeutics, Inc. (Nasdaq: RNA) ("Atrium," "Atrium Therapeutics," or the "Company"), a biopharmaceutical company advancing precision cardiology by developing RNA therapeutics targeted to the heart, today reported financial results for the second quarter ended June 30, 2026, and highlighted recent corporate progress including FDA clearance of its Investigational New Drug (IND) application for ATR 1072 and continued achievements under its collaboration with Bristol Myers Squibb (BMS). "Our team continues to execute well, achieving FDA clearance of our IND for ATR 1072 and launching Corventis — Atrium's first Phase 1/2 trial and the first clinical study to evaluate a potential disease-modifying treatment for people living with PRKAG2 syndrome," said Kathleen Gallagher, President and Chief Executive Officer of Atrium Therapeutics. "Atrium's precision approach to genetic cardiomyopathies is part of a burgeoning frontier in medicine. Our experienced team is well-positioned to continue advancing and efficiently expanding our pipeline with urgency on behalf of patients and clinicians." Recent Highlights Received FDA clearance of IND application and Health Canada No Objection Letter for ATR 1072. FDA cleared Atrium's IND application for ATR 1072, allowing the Company to proceed with Corventis, a Phase 1/2 open-label, multicenter clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of ATR 1072 in participants living with PRKAG2 syndrome. Additionally, the Company has received a No Objection Letter from Health Canada enabling the activation of planned Corventis study sites in Canada. The study will enroll approximately 37 participants across two parts: Part A, multiple ascending dose cohorts to characterize safety and support dose selection, and Part B, a single-arm expansion cohort at the recommended Phase 2 dose to further evaluate efficacy trends in cardiac structure and function. ATR 1072 is Atrium's first precision cardiology program to enter the clinic. Initiated clinical site activities for Corventis. Atrium continues to expect the first participant to be enrolled by the end of 2026. Earned a second milestone payment from Bristol Myers Squibb. Atrium achieved a second milestone under its global cardiovascular collaboration with BMS in August, triggering a payment of $15 million which will be accounted for in the third quarter financial statements. Anticipated Upcoming Milestones Enroll first participant in the Corventis Phase 1/2 trial for ATR 1072 by the end of 2026. Report initial trial data from Corventis demonstrating proof of concept in the second half of 2027. File IND application for ATR 1086 in 2027, with IND-enabling studies initiating in 2026. We are also advancing two undisclosed pipeline programs in rare cardiomyopathy targets and expect to select our next development candidate in 2027. Second Quarter 2026 Financial Results Collaboration Revenue: Collaboration revenue was $3.0 million for the second quarter of 2026, primarily related to R&D services under Atrium's research collaboration and license agreement with Bristol Myers Squibb. Research and Development (R&D) Expenses: R&D expenses were $15.3 million for the second quarter of 2026, primarily reflecting clinical trial preparations, IND-enabling activities, and continued development of the Company's overall research capabilities. General and Administrative (G&A) Expenses: G&A expenses were $10.3 million for the second quarter of 2026, driven by employee-related expenses, professional fees, and costs to support the Company's expanded operations. Cash, Cash Equivalents, and Short-term Investments: As of June 30, 2026, Atrium $263.9 million in cash, cash equivalents and short-term investments. The Company believes its current cash resources, inclusive of the receipt of the second milestone payment from had BMS earned in August, are sufficient to fund planned operations through mid-2028. About Atrium Therapeutics Atrium Therapeutics, Inc. (Nasdaq: RNA) is pioneering targeted delivery of ribonucleic acid (RNA) therapeutics to the heart to transform the standard of care for people living with cardiomyopathies. With the U.S. Food and Drug Administration's (FDA) recent clearance of its Investigational New Drug (IND) application for ATR 1072 for PRKAG2 (Protein Kinase AMP-activated non-catalytic subunit Gamma 2) syndrome, Atrium is advancing its first precision cardiology program into the clinic through the Corventis Phase 1/2 clinical trial. The Company's proprietary technology - designed at Avidity Biosciences, Inc. - combines the tissue selectivity of monoclonal antibodies (mAbs) and other targeted delivery ligands with the precision of oligonucleotides and is designed to selectively target the underlying drivers of genetically driven cardiac diseases through targeted, non-viral delivery of small interfering RNA (siRNA). This approach builds upon learnings from demonstrated delivery to skeletal muscle and applies it for efficient delivery to the heart, with the potential to overcome challenges associated with non-specific tissue delivery. Beyond ATR 1072, the Company's pipeline includes ATR 1086 for PLN (phospholamban) cardiomyopathy and two undisclosed research targets in rare cardiomyopathies. For more information about our RNA delivery platform, development pipeline and people, please visit https://atriumtherapeutics.com/ and engage with us on LinkedIn. Availability of Other Information About Atrium Therapeutics Investors and others should note that Atrium Therapeutics communicates with its investors and the public using its website https://atriumtherapeutics.com/, including, but not limited to, Atrium Therapeutics' disclosures, investor presentations and FAQs, Securities and Exchange Commission ("SEC") filings, press releases, public conference call transcripts and webcast transcripts, as well as on LinkedIn. The information that Atrium Therapeutics posts on its website or on LinkedIn could be deemed to be material information. As a result, Atrium Therapeutics encourages investors, the media, and others interested to review the information that it posts there on a regular basis. The contents of Atrium Therapeutics' website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. About PRKAG2 Syndrome PRKAG2 syndrome is a rare, autosomal dominant, early-onset cardiomyopathy caused by mutations in the PRKAG2 gene, which encodes the Gamma 2 regulatory subunit of AMPK. Mutations enhance AMPK activity leading to abnormal glycogen accumulation in heart, thickened heart muscles, electrical conduction problems, and arrhythmias. Based on current scientific literature estimates, there are at least 1,000 – 2,000 people with PRKAG2 syndrome in the U.S. Current management is limited to symptomatic treatment; no approved therapies exist to address the underlying genetic driver of disease. About PLN Cardiomyopathy Phospholamban ("PLN") cardiomyopathy is a rare autosomal dominant, progressive cardiac disease caused by mutations in PLN, a key regulator of sarcoplasmic reticulum Ca2+-ATPase 2a ("SERCA2a") calcium pump. PLN mutations produce protein aggregates that disrupt endoplasmic reticulum processes and lead to dilated, arrhythmogenic, or hypertrophic cardiomyopathies and a significantly increased risk of heart failure and sudden cardiac death. There are 2,000 – 4,000 people with pathogenic PLN variants in the United States. No approved therapies target the underlying molecular cause of the disease. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "may," "could," "would," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding Atrium Therapeutics' ("Atrium's" or "our") future results of operations and financial condition; the Company's expected cash runway and the period over which existing cash, cash equivalents and investments are expected to fund planned operations; research and development plans; anticipated timing, design and conduct of ongoing and planned preclinical studies and clinical trials for product candidates; the expected development, advancement and clinical evaluation of ATR 1072 for the treatment of PRKAG2 syndrome, including the expected timing of initiation, enrollment, dosing and availability of data from Corventis; the disease-modifying potential of ATR 1072 to treat PRKAG2 syndrome; our expectations regarding our RNA delivery platform and ability to generate high-quality cardiology development candidates, the timing and likelihood of regulatory filings and approvals for product candidates; the potential safety and therapeutic benefits of our product candidates; the timing and likelihood of success; plans and objectives of management for future operations; and future results of anticipated product development efforts. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Particular areas where risks or uncertainties could cause Atrium's actual results to be materially different than those expressed in Atrium's forward-looking statements include but are not limited to: the initiation, timing, progress, potential registrational quality, and results of our research and development programs, preclinical studies, any clinical trials, and other regulatory submissions; the potential for clinical trial results to differ from our preclinical studies; our ability to timely enroll a sufficient number of patients in our clinical trials, such as Corventis; the beneficial characteristics, including potential safety, efficacy and therapeutic effects of our product candidates and the potential advantages of our product candidates compared to alternative therapies; the success and capabilities of the RNA delivery platform; the prevalence of certain diseases and conditions we intend to treat and our estimates of the potential market opportunity for our product candidates; the timing of and costs involved in obtaining and maintaining regulatory approval of our current and any future product candidates; our ability to develop our current and future product candidates; the implementation of our strategic plans for our business, product candidates, research programs and technologies; developments related to our competitors and our industry; our competitive position and the success of competing therapies that are or may become available; our ability to maintain our current license agreements and collaborations and identify and enter into future license agreements and collaborations; the expected potential benefits of strategic collaborations with third parties and our ability to attract collaborators in the future; our reliance on third parties for manufacturing and to conduct preclinical studies and clinical trials of our product candidates; our ability to efficiently and cost-effectively conduct our current and future trials; the costs of operating as a public company; the accuracy of our estimates regarding future expenses, future revenue, capital requirements and the need for additional financing; the period over which we estimate our existing cash and cash equivalents will be sufficient to fund our future operating expenses and capital expenditure requirements; and other factors specified under the heading "Risk Factors" in Atrium's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026 filed with the SEC and in other filings and furnishings made by Atrium with the SEC from time to time, which are all available on the SEC's website at www.sec.gov. Atrium is providing the information in this communication as of this date and does not undertake any obligation to update any forward-looking statements contained in this communication as a result of new information, future events or otherwise, except as required by law. View original content to download multimedia:https://www.prnewswire.com/news-releases/atrium-therapeutics-reports-second-quarter-2026-financial-results-302851394.html

SAN DIEGO, May 14, 2026 /PRNewswire/ -- Atrium Therapeutics, Inc. (Nasdaq: RNA) ("Atrium," "Atrium Therapeutics" or the "Company"), a biopharmaceutical company advancing precision cardiology by developing RNA therapeutics targeted to the heart, today reported financial results for the first quarter ended March 31, 2026, and highlighted recent corporate progress following its launch as a public company. "Our focused pipeline, strong cash balance and experienced team are driving significant momentum as we build a dedicated precision cardiology company," said Kathleen Gallagher, President and Chief Executive Officer of Atrium Therapeutics. "This quarter, we advanced our lead development programs, ATR 1072 and ATR 1086, while establishing the strategic and operational foundation required to pioneer RNA therapeutics in the precision cardiology space. With a clear strategy, a well-characterized RNA delivery platform and a deep commitment to patients, we believe we are well-positioned to advance therapies for people living with genetic cardiomyopathies who have limited or no therapeutic options." Recent Highlights Launched as an independent, publicly traded precision cardiology company. Atrium launched on February 27, 2026 following its separation and spin-off from Avidity Biosciences, Inc. (Avidity) after Novartis AG's acquisition of Avidity. Atrium began trading on the Nasdaq Global Select Market under the ticker symbol "RNA." Earned $15 million milestone payment from Bristol Myers Squibb (BMS). Atrium successfully delivered the first development candidate targeting a cardiology indication under its ongoing collaboration with BMS, triggering a $15 million milestone payment. Under the terms of the agreement, Atrium is eligible to receive up to approximately $1.35 billion in research and development milestone payments, up to approximately $825 million in commercial milestone payments, and tiered royalties up to low double-digits on net sales. Progressed ATR 1072 program toward the clinic. Atrium has conducted the Good Laboratory Practice (GLP) toxicology studies required for the submission of an Investigational New Drug (IND) application for ATR 1072, a potential treatment designed to address the underlying genetic cause of Protein Kinase AMP-activated non-catalytic subunit Gamma 2 (PRKAG2) syndrome. Additionally, the Company has completed initial discussions with both the U.S. Food and Drug Administration (pre-IND meeting) and Health Canada (pre-CTA consultation meeting) regarding its proposed Phase 1/2 clinical trial design. Anticipated Upcoming Milestones Submit IND application for ATR 1072 in the second half of 2026 Initiate Phase 1/2 clinical trial for ATR 1072, subject to regulatory clearance File IND application for ATR 1086 in 2027, with IND-enabling studies initiating in 2026 First Quarter 2026 Financial Results Given the timing of Atrium's spin-off from Avidity, the operating results presented for the first quarter of 2026 are not necessarily indicative of the results for any future periods. Collaboration Revenue: Collaboration revenue was $19.6 million for the first quarter of 2026, primarily related to the achievement of a $15 million development milestone pursuant to the Company's partnership with BMS, with the remainder related to R&D services. Research and Development (R&D) Expenses: R&D expenses were $16.7 million for the first quarter of 2026, primarily reflecting clinical trial preparations, IND-enabling activities, and continued development of the Company's overall research capabilities. General and Administrative (G&A) Expenses: G&A expenses were $20.3 million for the first quarter of 2026, driven by employee-related expenses, professional fees, and costs associated with launching Atrium as a publicly traded company. Cash and Cash Equivalents: As of March 31, 2026, Atrium had $267.8 million in cash and cash equivalents. The Company believes its current cash resources are sufficient to fund planned operations through key clinical proof-of-concept milestones. About Atrium Therapeutics Atrium Therapeutics, Inc. (Nasdaq: RNA) is pioneering targeted delivery of ribonucleic acid (RNA) therapeutics to the heart to transform the standard of care for people living with cardiomyopathies. The Company's proprietary technology - designed at Avidity Biosciences, Inc. - combines the tissue selectivity of monoclonal antibodies (mAbs) and other targeted delivery ligands with the precision of oligonucleotides. Atrium Therapeutics' platform is designed to selectively target the underlying drivers of genetically driven cardiac diseases through targeted, non-viral delivery of small interfering RNA (siRNA). This approach builds upon learnings from demonstrated delivery to the skeletal muscle and applies it for efficient delivery to the heart with the potential to overcome challenges associated with non-specific tissue delivery. The Company's pipeline consists of two precision cardiology candidates, ATR 1072 for PRKAG2 (Protein Kinase AMP-activated non-catalytic subunit Gamma 2) syndrome and ATR 1086 for PLN (phospholamban) cardiomyopathy, and two undisclosed research targets in rare cardiomyopathies. For more information about our RNA delivery platform, development pipeline and people, please visit https://atriumtherapeutics.com/ and engage with us on LinkedIn. Availability of Other Information About Atrium Therapeutics Investors and others should note that Atrium Therapeutics communicates with its investors and the public using its website https://atriumtherapeutics.com/, including, but not limited to, Atrium Therapeutics' disclosures, investor presentations and FAQs, Securities and Exchange Commission ("SEC") filings, press releases, public conference call transcripts and webcast transcripts, as well as on LinkedIn. The information that Atrium Therapeutics posts on its website or on LinkedIn could be deemed to be material information. As a result, Atrium Therapeutics encourages investors, the media and others interested to review the information that it posts there on a regular basis. The contents of Atrium Therapeutics' website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. About PRKAG2 Syndrome PRKAG2 syndrome is a rare, autosomal dominant, early-onset cardiomyopathy caused by mutations in the PRKAG2 gene, which encodes the Gamma 2 regulatory subunit of AMPK. Mutations enhance AMPK activity leading to abnormal glycogen accumulation in heart muscle cells leading to thickened heart muscles, electrical conduction problems, and arrhythmias. There are 1,000 – 2,000 people with PRKAG2 syndrome in the United States. Current management is limited to symptomatic treatment; no approved therapies exist to address the underlying genetic driver of disease. About PLN Cardiomyopathy Phospholamban ("PLN") cardiomyopathy is a rare autosomal dominant, progressive cardiac disease caused by mutations in PLN, a key regulator of sarcoplasmic reticulum Ca2+-ATPase 2a ("SERCA2a") calcium pump. PLN mutations produce protein aggregates that disrupt endoplasmic reticulum processes and lead to dilated, arrhythmogenic, or hypertrophic cardiomyopathies and a significantly increased risk of heart failure and sudden cardiac death. There are 2,000 – 4,000 people with pathogenic PLN variants in the United States. No approved therapies target the underlying molecular cause of the disease. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "may," "could," "would," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding Atrium Therapeutics' ("Atrium's" or "our") future results of operations and financial condition; research and development plans; anticipated timing, design and conduct of ongoing and planned preclinical studies and clinical trials for product candidates; our expectations regarding our RNA delivery platform and ability to generate high-quality cardiology development candidates, the timing and likelihood of regulatory filings and approvals for product candidates; the potential safety and therapeutic benefits of our product candidates; the timing and likelihood of success; plans and objectives of management for future operations; and future results of anticipated product development efforts. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Particular areas where risks or uncertainties could cause Atrium's actual results to be materially different than those expressed in Atrium's forward-looking statements include but are not limited to: the initiation, timing, progress, potential registrational quality, and results of our research and development programs, preclinical studies, any clinical trials, and other regulatory submissions; the beneficial characteristics, including potential safety, efficacy and therapeutic effects of our product candidates and the potential advantages of our product candidates compared to alternative therapies; the success and capabilities of the RNA delivery platform; the prevalence of certain diseases and conditions we intend to treat and our estimates of the potential market opportunity for our product candidates; the timing of and costs involved in obtaining and maintaining regulatory approval of our current and any future product candidates; our ability to develop our current and future product candidates; the implementation of our strategic plans for our business, product candidates, research programs and technologies; developments related to our competitors and our industry; our competitive position and the success of competing therapies that are or may become available; our ability to maintain our current license agreements and collaborations and identify and enter into future license agreements and collaborations; the expected potential benefits of strategic collaborations with third parties and our ability to attract collaborators in the future; our reliance on third parties for manufacturing and to conduct preclinical studies and clinical trials of our product candidates; our ability to efficiently and cost-effectively conduct our current and future clinical trials; the costs of operating as a public company; the accuracy of our estimates regarding future expenses, future revenue, capital requirements and the need for additional financing; the period over which we estimate our existing cash and cash equivalents will be sufficient to fund our future operating expenses and capital expenditure requirements; and other factors specified under the heading "Risk Factors" in Atrium's Registration Statement on Form 10-12B/A, as amended (File No. 001-43008), which was filed with the SEC and became effective on February 26, 2026, most recent Quarterly Report on Form 10-Q filed with the SEC and in other filings and furnishings made by Atrium with the SEC from time to time, which are all available on the SEC's website at www.sec.gov. Atrium is providing the information in this communication as of this date and does not undertake any obligation to update any forward-looking statements contained in this communication as a result of new information, future events or otherwise, except as required by law. View original content to download multimedia:https://www.prnewswire.com/news-releases/atrium-therapeutics-reports-first-quarter-2026-financial-results-302772898.html

Del-desiran effectively delivered siRNA to muscle, resulting in approximately 40% mean reduction in DMPK mRNA and amelioration of missplicing Treatment demonstrated improvements in multiple measures including myotonia, muscle function and strength, mobility and patient-reported outcomes Del-desiran showed acceptable safety and tolerability with most adverse events mild or moderate SAN DIEGO, Feb. 18, 2026 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the final results from the completed Phase 1/2 MARINA® trial of delpacibart etedesiran (del-desiran) in people living with myotonic dystrophy type 1 (DM1) will be published in the February 19 issue of The New England Journal of Medicine (NEJM). The manuscript is titled "An Antibody Oligonucleotide Conjugate for Myotonic Dystrophy Type 1." DM1 is an underrecognized, progressive and often fatal neuromuscular disease with no disease modifying therapies. Del-desiran is an investigational treatment designed to address the underlying genetic root cause of DM1 by reducing total levels of the toxic, DMPK (myotonic dystrophy protein kinase) mRNA. The accumulation of these toxic mRNA sequester key RNA-regulatory proteins that subsequently lead to missplicing of several downstream genes, resulting in the diverse clinical manifestations of disease. The Phase 1/2 MARINA trial was a randomized, double-blind, placebo-controlled study designed to evaluate the safety and tolerability of single and multiple ascending doses of del-desiran administered intravenously in adults with DM1 for six months. Data were assessed from 38 participants who were randomized 3:1 to receive one dose of 1 mg/kg del-desiran, three doses of either 2 mg/kg del-desiran or 4 mg/kg del-desiran (reflected as siRNA dose), or placebo. The primary endpoint of the study was to evaluate the safety and tolerability of del-desiran. Exploratory endpoints were to evaluate the clinical activity of del-desiran across multiple efficacy measures. "These final results from the MARINA study further reinforce del-desiran data reported thus far showing acceptable safety profile and improvements across a range of key functional assessments including myotonia, a hallmark symptom of DM1," said Nicholas E. Johnson, M.D., M.Sci., FAAN, professor and vice chair of research in the Department of Neurology at Virginia Commonwealth University, lead author and primary investigator of the MARINA trial. "DM1 is a progressive disorder that is often fatal, increases in severity from generation to generation, and impacts thousands of people and families in the U.S. alone. There is an urgent need for an approved therapy that can address the underlying genetic cause of this disease, and these del-desiran data, as well as available data from the ongoing OLE study, are very encouraging for the DM1 community." Results from the Phase 1/2 MARINA study of del-desiran published in NEJM demonstrated: Effective delivery of del-desiran (siRNA) to muscle, with a mean ~ 40% reduction in DMPK mRNA across all treated participants. Splicing improvements in a key set of muscle-specific genes following treatment with del-desiran 2 mg/kg and 4 mg/kg. Improvements in exploratory functional measures including: Hand function/myotonia (video hand opening time, or vHOT) Muscle strength (Quantitative Muscle Testing, or QMT total score) Mobility (10-Meter Walk/Run Test, or 10mWRT, and Timed Up and Go test, or TUG) DM1-Activ, a patient-reported outcome that measures activities of daily living (e.g., taking a shower, visiting family or friends, and walking up stairs) Acceptable safety and tolerability with most treatment emergent adverse events (TEAEs) mild or moderate in participants with DM1 and did not result in discontinuation. Two severe, serious AEs occurred in two participants in the 2 mg/kg and 4 mg/kg dose cohorts, with one participant discontinuing the study in the 4 mg/kg cohort. One of these SAEs was deemed drug-related. "We are pleased that The New England Journal of Medicine recognizes the significance of the Phase 1/2 MARINA data. The favorable safety profile, coupled with robust analyses of exploratory endpoints in our del-desiran program reinforce our belief that this investigational therapy may offer a transformational treatment option for people living with DM1 and their families," said Sarah Boyce, President and Chief Executive Officer at Avidity. "We remain focused on advancing the ongoing Phase 3 HARBOR study of del-desiran, which is on track to be the first globally approved drug for DM1." Del-desiran (4 mg/kg) is currently being assessed in the global Phase 3 HARBOR™ study in people living with DM1 who are age 16 and older and in the ongoing HARBOR open-label extension (HARBOR-OLE™) trial with all the participants who completed the Phase 1/2 MARINA trial. The global Phase 3 HARBOR study completed enrollment in July 2025 and a 54-week topline data readout is expected in the second half of 2026. About the Phase 3 HARBOR™ Trial The global Phase 3 HARBOR™ trial is a randomized, placebo-controlled, double blind pivotal study designed to evaluate del-desiran in approximately 150 people (age 16 and older) living with DM1. The trial is being conducted at approximately 40 sites globally. Patients are administered either del-desiran or placebo (1:1) every eight weeks. HARBOR is designed to assess multiple key functional aspects of DM1. The primary endpoint is video hand opening time (vHOT), a measurement of myotonia, which is a hallmark symptom of DM1. Key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. All study participants, regardless of whether they receive active treatment or placebo, have the option to enroll into the ongoing open-label extension trial. For more information about the HARBOR trial, visit http://www.clinicaltrials.gov and search for NCT06411288. For more information about the HARBOR-OLE trial, visit http://www.clinicaltrials.gov and search for (NCT07008469). About the Phase 1/2 MARINA® Trial The MARINA® trial was a randomized, double-blind, placebo-controlled, Phase 1/2 clinical trial that enrolled 38 adults with DM1. The primary objective of this study was to evaluate the safety and tolerability of single and multiple ascending doses of del-desiran (AOC 1001) administered intravenously. The MARINA trial assessed the activity of del-desiran (AOC 1001) across key biomarkers, including reduction of DMPK mRNA in skeletal muscle and amelioration of aberrant alternative splicing. Though the Phase 1/2 trial was not powered to assess functional benefit, it explored the clinical activity of del-desiran (AOC 1001) in multiple measures of muscle function including myotonia, muscle strength, measures of mobility as well as patient reported outcomes and quality of life measures. Patients had the option to enroll in MARINA-OLE™, an open-label extension study, at the end of the post-treatment period. For more information on this study click here or visit http://www.clinicaltrials.gov and search for NCT05027269. About Myotonic Dystrophy Type 1 Myotonic dystrophy type 1 (DM1) is a rare, hereditary (autosomal dominant), progressive neuromuscular disease that shortens life expectancy and requires life-long care caused by a triplet-repeat in the DMPK gene, resulting in a toxic gain of function mRNA. The disease is highly variable with respect to severity, presentation and age of onset, however, all forms of DM1 are associated with high levels of disease burden. DM1 is characterized by multisystemic manifestations including myotonia and progressive muscle weakening and may be underrecognized because it presents heterogeneously across skeletal, cardiac, and smooth muscles, leading to impairment of the cardiovascular, gastrointestinal, respiratory, ocular, and/or endocrine systems. Currently, there are no approved drugs for people living with DM1. About del-desiran Del-desiran, utilizing Avidity's AOC platform technology, is designed to address the underlying genetic cause of DM1 by reducing levels of toxic DMPK mRNA. Del-desiran consists of a proprietary monoclonal antibody that binds to transferrin receptor 1 (TfR1) and is conjugated to a siRNA that targets DMPK mRNA. Del-desiran is currently being assessed in the global Phase 3 HARBOR™ trial and in the ongoing HARBOR-OLE™ trial in people with DM1. Long-term data from the MARINA-OLE trial showed reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT) as a measure of hand function and myotonia, muscle strength and activities of daily living when compared to END-DM1 natural history data. Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). Del-desiran was also the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and X. Forward-Looking Statements Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the meaningfulness of the del-desiran data from the MARINA® study; the potential for del-desiran to offer a transformational treatment option for DM1 and become the first globally approved drug to treat DM1; the status of the clinical study of del-desiran; Avidity's plans to present additional data from the HARBOR™ study and the timing thereof; the characterization of data associated with del-desiran from the MARINA study and the impact of such data on the advancement of del-desiran; the design, goals and status of the MARINA, MARINA-OLE™ and HARBOR studies; Avidity's plans and expectations to advance its clinical programs, and the timing thereof; and Avidity's platform, planned operations and programs. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: analysis of new data may lead to conclusions different from those established in the MARINA trial of del-desiran, and such data may not meet Avidity's or regulators' expectations; unexpected adverse side effects to, or inadequate efficacy of, del-desiran that may delay or limit its development, regulatory approval and/or commercialization; later developments with the FDA and other global regulators that could be inconsistent with the feedback received to date regarding del-desiran and which could delay its currently anticipated timelines; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven; potential delays in the HARBOR study; Avidity's dependence on third parties in connection with clinical testing and product manufacturing; legislative, judicial and regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and subsequent filings with the SEC. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact: Kat Lange (619) 837-5014 [email protected] Media Contact: Kristina Coppola (619) 837-5016 [email protected] View original content to download multimedia:https://www.prnewswire.com/news-releases/the-new-england-journal-of-medicine-publishes-results-from-phase-12-marina-trial-of-delpacibart-etedesiran-del-desiran-for-treatment-of-myotonic-dystrophy-type-1-302692044.html

Announced Novartis agreement to acquire Avidity for total equity value of approximately $12 billion; Avidity expects to separate its early-stage precision cardiology programs into a new public company ("SpinCo") Clear path forward aligned with FDA following pre-BLA meeting for del-zota, with BLA submission planned for 2026 Del-zota one-year data demonstrated sustained muscle protection leading to meaningful improvement and reversal of disease progression across multiple key functional measures Strong balance sheet with ~$1.9 billion in cash, cash equivalents, and marketable securities SAN DIEGO, Nov. 10, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™) to profoundly improve people's lives, today reported financial results for the third quarter ended September 30, 2025, and highlighted recent progress. "In October, we announced that Avidity entered into a definitive merger agreement with Novartis, which we believe maximizes value for our investors, accelerates the global reach of our innovative neuroscience pipeline, and advances even more possibilities for our innovative science," said Sarah Boyce, president and chief executive officer of Avidity. "This important transaction, alongside compelling del-zota data and a successful pre-BLA meeting with the FDA in the third quarter, underscores the remarkable consistency of our AOC platform and the significant potential of del-zota, del-desiran, and del-brax to transform outcomes for people living with serious rare diseases. These achievements are possible because of our incredibly talented Avidity team and the close collaboration of the dedicated patient and clinical communities we serve." Company Announcements, Highlights and Upcoming Milestones Definitive Merger Agreement with Novartis AG In October 2025, Avidity announced it had entered into a definitive merger agreement with Novartis AG ("Novartis") which was unanimously approved by the Boards of Directors of both Avidity and Novartis. The closing of the acquisition will follow the separation of Avidity's early-stage precision cardiology programs into SpinCo, which is expected to be a publicly traded company. SpinCo will be led by Kathleen Gallagher, currently Avidity's chief program officer, as chief executive officer. Sarah Boyce, currently Avidity's chief executive officer, will serve as chair of the board. Novartis will acquire Avidity's programs and pipeline in neuroscience and gain access to its differentiated RNA-targeting delivery platform, which includes three late-stage clinical development programs: delpacibart zotadirsen (del-zota) for the treatment of Duchenne muscular dystrophy (DMD), delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1) and delpacibart braxlosiran (del-brax) for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Expected closing is in the first half of 2026, subject to completion of the separation of SpinCo from Avidity and other customary closing conditions. Delpacibart zotadirsen (del-zota) for the treatment of people living with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44): Clear path forward aligned with FDA following October 2025 pre-BLA meeting. The BLA submission is planned for 2026 for accelerated approval. In September 2025, Avidity shared positive topline and functional del-zota data from EXPLORE44® and EXPLORE44-OLE™ trials demonstrating consistent, clinically meaningful improvements across functional endpoints at approximately one year of treatment. Data demonstrated reversal of disease progression and unprecedented improvement compared to baseline and natural history across multiple functional measures. Del-zota continued to demonstrate a favorable long-term safety and tolerability profile. In July 2025, Avidity announced the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to del-zota. Delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1): In July 2025, Avidity announced completion of enrollment for the Phase 3 HARBOR™ trial, the first global Ph3 trial of del-desiran for the treatment of DM1. Expected publication of data analyses from the completed Phase 1/2 MARINA® trial in the fourth quarter of 2025. 54-week topline data readout from global Phase 3 HARBOR™ study expected in the second half of 2026. Delpacibart braxlosiran (del-brax) for the treatment of facioscapulohumeral muscular dystrophy (FSHD): Topline data from FORTITUDE™ biomarker cohort expected in the second quarter of 2026. Alignment with FDA on accelerated and full approval pathways for del-brax, and initiated global, confirmatory Phase 3 study, FORTITUDE-3™, intended to support global approval strategy for del-brax. Phase 3 FORTITUDE-3™ readout and global regulatory submissions expected in 2028. Collaboration Progress: In the third quarter of 2025, Avidity received collaboration revenue of a $10.0 million clinical development milestone under Avidity's research collaboration and license agreement with Eli Lilly and Company. Third Quarter 2025 Financial Results Cash, cash equivalents and marketable securities totaled approximately $1.9 billion as of September 30, 2025, which reflects net proceeds of $651.4 million from a public offering and $185.5 million from the sale of common stock under the Company's sales agreement. The Company expects that its cash, cash equivalents and marketable securities as of September 30, 2025, will be sufficient to fund its operations to mid-2028. Collaboration revenues of $12.5 million for the third quarter of 2025 and $17.9 million for the first nine months of 2025, primarily relate to a $10.0 million clinical development milestone under Avidity's research collaboration and license agreement with Eli Lilly and Company, as well as additional revenues under Avidity's research collaboration and license agreement with Bristol Myers Squibb. Collaboration revenues of $2.3 million for the third quarter of 2024 and $7.9 million for the first nine months of 2024, primarily relate to revenues under Avidity's research collaboration and license agreement with Bristol Myers Squibb. Research and development expenses for the third quarter of 2025 were $154.9 million, compared to $77.2 million for the same period of 2024. Research and development expenses for the nine months ended September 30, 2025 were $392.6 million, compared to $208.0 million for the same period of 2024. The increases were primarily driven by increased costs associated with the advancement of del-desiran, del-brax and del-zota, higher manufacturing costs, and higher personnel costs. General and administrative expenses for the third quarter of 2025 were $46.3 million, compared to $23.3 million for the same period of 2024. General and administrative expenses for the nine months ended September 30, 2025 were $116.8 million, compared to $57.9 million for the same period of 2024. The increases were primarily due to higher personnel and commercial infrastructure costs to support the company's expanded operations. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare muscle diseases: DM1, DMD and FSHD. Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and X. Additional Information and Where to Find It In connection with the spin-off and the merger (the "Transactions"), Novartis, Avidity and SpinCo intend to file relevant documents with the Securities and Exchange Commission (the "SEC"), including a preliminary and definitive proxy statement to be filed by Avidity. The definitive proxy statement and proxy card will be delivered to the stockholders of Avidity in advance of the special meeting relating to the Transactions. This document is not a substitute for the proxy statement or any other document that may be filed by Avidity with the SEC. AVIDITY'S STOCKHOLDERS ARE URGED TO READ THE DEFINITIVE PROXY STATEMENT IN ITS ENTIRETY WHEN IT BECOMES AVAILABLE AND ANY OTHER DOCUMENTS FILED BY EACH OF NOVARTIS AND AVIDITY WITH THE SEC IN CONNECTION WITH THE TRANSACTIONS OR INCORPORATED BY REFERENCE THEREIN BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTIONS AND THE PARTIES TO THE PROPOSED TRANSACTIONS. Investors and security holders will be able to obtain a free copy of the proxy statement and such other documents containing important information about Novartis and Avidity, once such documents are filed with the SEC, through the website maintained by the SEC at www.sec.gov. Novartis and Avidity make available free of charge at the Novartis website at www.novartis.com/investors/financial-data/sec-filings and Avidity's website at investors.aviditybiosciences.com/sec-filings, respectively, copies of documents they file with, or furnish to, the SEC. The contents of the websites referenced above will not be deemed to be incorporated by reference into the proxy statement. Participants in the Solicitation This communication does not constitute a solicitation of a proxy. Novartis, Avidity and their respective directors, executive officers and certain employees may be deemed to be participants in the solicitation of proxies from the stockholders of Avidity in connection with the Transactions. Information regarding the special interests of these directors and executive officers in the Transactions will be included in the definitive proxy statement referred to above. Security holders may also obtain information regarding the names, affiliations and interests of the Novartis directors and executive officers in the Novartis Annual Report on Form 20-F for the fiscal year ended December 31, 2024, which was filed with the SEC on January 31, 2025. Security holders may obtain information regarding the names, affiliations and interests of Avidity's directors and executive officers in Avidity's definitive proxy statement on Schedule 14A, which was filed with the SEC on April 29, 2025. To the extent the holdings of Avidity's securities by Avidity's directors and executive officers have changed since the amounts set forth in Avidity's definitive proxy statement for its 2025 annual meeting of stockholders, such changes have been reflected in the following Statements of Change in Ownership on Form 4 filed with the SEC: by Eric Mosbrooker, dated October 7, 2025, September 5, 2025 and August 8, 2025; by Steven Hughes, dated October 24, 2025, September 23, 2025, September 17, 2025, August 22, 2025, August 15, 2025 and August 8, 2025; by Teresa McCarthy, dated October 15, 2025, September 17, 2025 and August 15, 2025; by Michael Flanagan, dated September 12, 2025 and June 12, 2025; by Troy Wilson, dated September 9, 2025, August 8, 2025 and June 12, 2025; by Sarah Boyce, dated September 5, 2025 and August 29, 2025; by Kathleen Gallagher, dated September 2, 2025, June 18, 2025, June 4, 2025 and May 2, 2025; by Michael MacLean, dated August 15, 2025; by Arthur Levin, dated August 8, 2025 and June 12, 2025; by John Moriarty, dated August 5, 2025; by Noreen Henig, dated June 12, 2025; by Carsten Boess, dated June 12, 2025; by Edward Kaye, dated June 12, 2025; by Simona Skerjanec, dated June 12, 2025; by Tamar Thompson, dated June 12, 2025; and by Jean Kim, dated June 12, 2025. These documents (when available) may be obtained free of charge from the SEC's website at www.sec.gov, the Novartis website at www.novartis.com/investors/financial-data/sec-filings and Avidity's website at investors.aviditybiosciences.com/sec-filings. The contents of the websites referenced above are not deemed to be incorporated by reference into the proxy statement. No Offer or Solicitation This communication is for informational purposes only and is not intended to and does not constitute, or form part of, an offer, invitation or the solicitation of an offer or invitation to purchase, otherwise acquire, subscribe for, sell or otherwise dispose of any securities, or the solicitation of any vote or approval in any jurisdiction, pursuant to the proposed transaction or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. Forward-Looking Statements This communication contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "may," "could," "would," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding the proposed acquisition of Avidity and Avidity's related spin-off, the expected timetable for completing each of the proposed Transactions, the composition of the assets and liabilities to be held by SpinCo and Avidity following the spin-off, the management team for SpinCo and its cash balance, potential marketing approvals, new indications or labeling for Avidity's product candidates, Avidity's platform and preclinical assets, or potential future revenues from Avidity's product candidates. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that Avidity's investigational products will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time, or that Avidity's approach to the discovery and development of product candidates based on its AOC™ platform will produce any products of commercial value. There can be no guarantee that the conditions to the closing of the Transactions will be satisfied on the expected timetable or at all or that the expected benefits or synergies from the Transactions will be achieved in the expected timeframe, or at all. In particular, expectations regarding Avidity, SpinCo, or the Transactions could be affected by, among other things, the timing of the satisfaction of customary closing conditions, including the receipt of regulatory approvals and the approval of Avidity's stockholders, on acceptable terms or at all; risks and costs related to the implementation of the separation of SpinCo, including the ability to complete the separation in the anticipated timeframe, or at all, and any changes to the configuration of the businesses included in the separation if implemented; the sale of certain of SpinCo's assets pursuant to a third party right of first negotiation; the risk that competing offers or acquisition proposals will be made; the effects of disruption from the Transactions and the impact of the announcement and pendency of the Transactions on Novartis' and/or Avidity's businesses, including their relationships with employees, business partners or governmental entities; the risk that the Transactions may be more expensive to complete than anticipated; the risk that stockholder litigation in connection with the Transactions may result in significant costs of defense, indemnification and liability; a diversion of management's attention from ongoing business operations and opportunities as a result of the Transactions or otherwise; the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; and the risks and factors referred to in Novartis AG's most recent Annual Report on Form 20-F for the year ended December 31, 2024, Avidity's Annual Report on Form 10-K for the year ended December 31, 2024 and Quarterly Reports on Form 10-Q for the quarters ended March 31, 2025 and June 30, 2025, and any subsequent filings made by either party with the SEC, available on the SEC's website at www.sec.gov, Avidity is providing the information in this communication as of this date and does not undertake any obligation to update any forward-looking statements contained in this communication as a result of new information, future events or otherwise, except to the extent required by law. Investor Contact: Kat Lange (619) 837-5014 [email protected] Media Contact: Kristina Coppola (619) 837-5016 [email protected] View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-reports-third-quarter-2025-financial-results-and-recent-highlights-302610417.html

The market expects Avidity Biosciences, Inc. (RNA) to deliver a year-over-year decline in earnings on higher revenues when it reports results for the quarter ended September 2025. This widely-known consensus outlook is important in assessing the company's earnings picture, but a powerful factor that might influence its near-term stock price is how the actual results compare to these estimates. The earnings report might help the stock move higher if these key numbers are better than expectations. On the other hand, if they miss, the stock may move lower. While management's discussion of business conditions on the earnings call will mostly determine the sustainability of the immediate price change and future earnings expectations, it's worth having a handicapping insight into the odds of a positive EPS surprise. This company is expected to post quarterly loss of $1.07 per share in its upcoming report, which represents a year-over-year change of -64.6%. Revenues are expected to be $2.5 million, up 6.8% from the year-ago quarter. The consensus EPS estimate for the quarter has been revised 3.39% higher over the last 30 days to the current level. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period. Investors should keep in mind that an aggregate change may not always reflect the direction of estimate revisions by each of the covering analysts. Price, Consensus and EPS Surprise Estimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. Our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction) -- has this insight at its core. The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier. Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only. A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP. Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell). For Avidity Biosciences, the Most Accurate Estimate is lower than the Zacks Consensus Estimate, suggesting that analysts have recently become bearish on the company's earnings prospects. This has resulted in an Earnings ESP of -7.31%. On the other hand, the stock currently carries a Zacks Rank of #3. So, this combination makes it difficult to conclusively predict that Avidity Biosciences will beat the consensus EPS estimate. Analysts often consider to what extent a company has been able to match consensus estimates in the past while calculating their estimates for its future earnings. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number. For the last reported quarter, it was expected that Avidity Biosciences would post a loss of$0.95 per share when it actually produced a loss of -$1.21, delivering a surprise of -27.37%. Over the last four quarters, the company has beaten consensus EPS estimates two times. An earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss. That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported. Avidity Biosciences doesn't appear a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release. uniQure (QURE), another stock in the Zacks Medical - Biomedical and Genetics industry, is expected to report loss per share of $0.85 for the quarter ended September 2025. This estimate points to a year-over-year change of +6.6%. Revenues for the quarter are expected to be $6.93 million, up 202.6% from the year-ago quarter. Over the last 30 days, the consensus EPS estimate for uniQure has been revised 4.8% up to the current level. Nevertheless, the company now has an Earnings ESP of +22.54%, reflecting a higher Most Accurate Estimate. This Earnings ESP, combined with its Zacks Rank #3 (Hold), suggests that uniQure will most likely beat the consensus EPS estimate. Over the last four quarters, the company surpassed consensus EPS estimates three times. Stay on top of upcoming earnings announcements with the Zacks Earnings Calendar. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Avidity Biosciences, Inc. (RNA) : Free Stock Analysis Report uniQure N.V. (QURE) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

Swiss pharma giant Novartis AG NVS reported core earnings per share (excluding one-time charges) of $2.25 in the third quarter, which missed the Zacks Consensus Estimate by a penny. Nonetheless, the figure was up from $2.06 reported a year ago. The year-over-year improvement was driven by growth in sales. Revenues of $13.9 billion climbed 8% from the year-ago reported figure and marginally beat the Zacks Consensus Estimate. On a constant currency basis, sales increased 7%, driven by continued strong performances from Kisqali, Kesimpta, Scemblix, and Pluvicto, which more than offset the adverse impact of generic competition for Promacta, Tasigna, and Entresto in the United States. Core Operating income was up 7% to $5.5 billion. Shares of Novartis have gained 35.9% year to date compared with the industry’s growth of 7.9%. Image Source: Zacks Investment Research All growth rates mentioned below are on a year-over-year basis and at constant exchange rates. With the successful spin-off of the Sandoz business in 2023, Novartis now focuses on four core therapeutic areas — cardiovascular-renal-metabolic, immunology, neuroscience and oncology. Cardiovascular drug Entresto’s sales decreased 1% from the year-ago level to $1.9 billion as sales were impacted by generic competition in the United States. Sales grew outside the country where the drug is approved for heart failure globally as well as hypertension in China and Japan. Novartis is in a litigation with a generic manufacturer and FDA in the United States to protect its Entresto IP and regulatory rights. Entresto’s sales missed the Zacks Consensus Estimate of $2.1 billion and our model estimate of $2 billion. Cosentyx’s sales (psoriasis, spondylitis and arthritis) were down 1% to $1.7 billion as sales growth was negatively impacted by a one-time revenue deduction adjustment in the United States. Excluding this adjustment, Cosentyx sales were up 4%. Cosentyx’s sales missed the Zacks Consensus Estimate of $1.72 billion and our model estimate of $1.73 billion. Kisqali (breast cancer) maintained its stellar performance, with sales surging 68% to $1.33 billion. Sales grew across all regions, driven by exemplary growth in the United States with strong momentum from the recently launched early breast cancer (eBC) indication as well as continued share gains in the metastatic breast cancer indication. Kisqali sales beat the Zacks Consensus Estimate of $1.29 billion and our model estimate of $1.24 billion. Kesimpta (multiple sclerosis) sales totaled $1.2 billion, which surged 44% on increased demand and strong access. The figure beat the Zacks Consensus Estimate of $1.11 billion and our model estimate of $1.1 billion. Sales of Tafinlar + Mekinist were up 1% to $550 million. Jakavi sales were up 4% to $539 million. However, Promacta sales declined 38% to $362 million due to discontinued promotion in most markets and recent generic entry in the United States in the second quarter of 2025. Pluvicto (prostate cancer) raked in sales of $564 million, surging 45% on sustained demand growth in the United States following approval for pre-taxane metastatic castration-resistant prostate cancer (mCRPC) as well as continued expansion in the post-taxane mCRPC setting outside the country. The FDA’s approval for earlier use of the drug before chemotherapy has approximately tripled the eligible patient population. Sales beat the Zacks Consensus Estimate of $533 million and our estimate of $505 million. Ilaris sales amounted to $473 million, which increased 26% year over year, driven by growth in all regions led by the United States, Europe and Japan. Strong performance in the Periodic Fever Syndromes and Still’s disease indications contributed to this growth. Xolair (asthma and allergies) sales grew 3% to $440 million driven by the chronic spontaneous urticaria (CSU) indication, with contributions from Europe and emerging growth markets. Novartis has a collaboration agreement with Roche RHHBY for Xolair. Novartis and Roche co-promote Xolair in the United States. Tasigna (leukemia) sales plummeted 48% to $221 million due to lower demand and increasing competition, including the entry of generics both in the United States and outside the country. Gene-therapy Zolgensma (spinal muscular atrophy) sales of $301 million were down 5% due to a lower incidence of spinal muscular atrophy compared to the prior year. Scemblix sales skyrocketed 95% to $358 million, driven by continued growth in chronic myeloid leukemia indication and strong momentum from the recently launched early-line indication in the United States and Japan. Sales beat the Zacks Consensus Estimate of $348 million and our estimate of $333 million. Cholesterol drug Leqvio sales soared 54% to $308 million on steady growth in demand, with a focus on increasing accounts and patient adoption. The figure, however, missed the Zacks Consensus Estimate of $323 million and our estimate of $327 million. Luthathera (cancer) sales totaled $213 million, up 11%. Sales grew mainly in the United States, Europe and Japan due to increased demand and earlier line adoption. Sales of Lucentis nosedived 42% to $148 million due to generic competition. Fabhalta generated sales of $149 million, driven by continued launch execution across all markets in paroxysmal nocturnal hemoglobinuria and the launch progress in primary IgA nephropathy and C3 glomerulopathy in the United States. Net sales are expected to grow in high single digits. Core operating income is anticipated to grow in the low teens. Novartis announced that it will acquire San Diego-based Avidity Biosciences, Inc. RNA for $12 billion to strengthen its late-stage neuroscience pipeline. As part of the agreement, Avidity will separate its early-stage precision cardiology programs into a new company prior to closing of the acquisition. Avidity is developing RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs) for serious, genetic neuromuscular diseases. The acquisition will bolster neuroscience franchise for Novartis with three late-stage programs in myotonic dystrophy type 1 (DM1), a rare progressive neuromuscular disorder with a poor prognosis, facioscapulohumeral muscular dystrophy (FSHD), a rare hereditary disorder causing relentless loss of muscle function and progressive disability; and Duchenne muscular dystrophy (DMD), a severe, early-onset disease marked by progressive muscle damage and reduced life expectancy. The companies expect the merger to be closed in the first half of 2026. Novartis received FDA approval for remibrutinib, under the brand name Rhapsido, as an oral treatment for adult patients with CSU who remain symptomatic despite H1 antihistamine treatment. The approval makes it the first FDA-approved Bruton’s tyrosine kinase inhibitor (BTKi) for CSU. The phase III NEPTUNUS-1 and -2 studies evaluating ianalumab in adults with active Sjögren’s disease met their primary endpoint, showing statistically significant improvements in disease activity as measured by a reduction in ESSDAI compared to placebo. Novartis plans to submit applications for ianalumab to health authorities globally. Novartis performance in the third quarter was impacted by generic competition for its blockbuster drug Entresto in the United States. Novartis AG price-consensus-eps-surprise-chart | Novartis AG Quote Nevertheless, solid performance of its key drugs such as Kisqali, Kesimpta, Pluvicto and Scemblix maintains momentum for the company. The pipeline progress is also encouraging. Approval of new drugs and label expansion of existing drugs should enable the company to offset the adverse impacts of generic competition for Tasigna, Promacta and Entresto. While organic growth continues to drive business, NVS is focused on strategic acquisitions to strengthen its pipeline. The recently announced RNA acquisition will strengthen its neuroscience franchise with three late-stage programs that address genetic neuromuscular diseases. The acquisition of Tourmaline Bio, a New York-based clinical-stage biopharmaceutical company for $1.4 billion, adds a phase III ready candidate pacibekitug to NVS’ cardiovascular pipeline. In June 2025, Novartis acquired Regulus Therapeutics Inc., a clinical-stage biopharmaceutical company. Novartis entered into a second collaboration agreement with Monte Rosa Therapeutics, in addition to the existing license agreement for VAV1 degraders. Under the new agreement, Novartis will receive an exclusive license to an undisclosed discovery target and options to license two programs from Monte Rosa’s preclinical immunology portfolio. The company also entered into a global licensing and collaboration agreement with Arrowhead Pharmaceuticals for ARO-SNCA, a preclinical-stage siRNA therapy targeting alpha-synuclein for the treatment of synucleinopathies such as Parkinson’s disease. Novartis currently carries a Zacks Rank #3 (Hold). A better-ranked large-cap pharmaceutical stock is Bayer BAYRY, which sports a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here. BAYRY’s 2025 earnings per share (EPS) estimate has increased from $1.32 to $1.37 for 2025 over the past 60 days, while that for 2026 has gone up from $1.36 to $1.44 over the same timeframe. Year to date, shares of Bayer have surged 63.9%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Novartis AG (NVS) : Free Stock Analysis Report Roche Holding AG (RHHBY) : Free Stock Analysis Report Bayer Aktiengesellschaft (BAYRY) : Free Stock Analysis Report Avidity Biosciences, Inc. (RNA) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

The investors in Avidity Biosciences, Inc.'s (NASDAQ:RNA) will be rubbing their hands together with glee today, after the share price leapt 29% to US$46.29 in the week following its quarterly results. Revenues came in 140% better than analyst models expected, at US$3.8m, although statutory losses ballooned 24% to US$1.21, which is much worse than what was forecast. This is an important time for investors, as they can track a company's performance in its report, look at what experts are forecasting for next year, and see if there has been any change to expectations for the business. We've gathered the most recent statutory forecasts to see whether the analysts have changed their earnings models, following these results. This technology could replace computers: discover the 20 stocks are working to make quantum computing a reality. Following the recent earnings report, the consensus from 13 analysts covering Avidity Biosciences is for revenues of US$10.1m in 2025. This implies a perceptible 6.3% decline in revenue compared to the last 12 months. Per-share losses are expected to explode, reaching US$4.65 per share. Before this earnings announcement, the analysts had been modelling revenues of US$7.27m and losses of US$3.98 per share in 2025. Ergo, there's been a clear change in sentiment, with the analysts lifting this year's revenue estimates, while at the same time increasing their loss per share numbers to reflect the cost of achieving this growth. View our latest analysis for Avidity Biosciences There was no major change to the consensus price target of US$67.00, with growing revenues seemingly enough to offset the concern of growing losses. The consensus price target is just an average of individual analyst targets, so - it could be handy to see how wide the range of underlying estimates is. Currently, the most bullish analyst values Avidity Biosciences at US$96.00 per share, while the most bearish prices it at US$50.00. This is a fairly broad spread of estimates, suggesting that analysts are forecasting a wide range of possible outcomes for the business. Another way we can view these estimates is in the context of the bigger picture, such as how the forecasts stack up against past performance, and whether forecasts are more or less bullish relative to other companies in the industry. These estimates imply that revenue is expected to slow, with a forecast annualised decline of 12% by the end of 2025. This indicates a significant reduction from annual growth of 9.0% over the last five years. Compare this with our data, which suggests that other companies in the same industry are, in aggregate, expected to see their revenue grow 19% per year. It's pretty clear that Avidity Biosciences' revenues are expected to perform substantially worse than the wider industry. The most important thing to note is the forecast of increased losses next year, suggesting all may not be well at Avidity Biosciences. Fortunately, they also upgraded their revenue estimates, although our data indicates it is expected to perform worse than the wider industry. The consensus price target held steady at US$67.00, with the latest estimates not enough to have an impact on their price targets. With that said, the long-term trajectory of the company's earnings is a lot more important than next year. At Simply Wall St, we have a full range of analyst estimates for Avidity Biosciences going out to 2027, and you can see them free on our platform here.. Before you take the next step you should know about the 2 warning signs for Avidity Biosciences that we have uncovered. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email editorial-team (at) simplywallst.com. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.

Aligned with FDA on del-brax accelerated and full approval pathways for FSHD, reported positive topline data from Phase 1/2 FORTITUDE™ trial, and initiated global confirmatory Phase 3 study 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, Aug. 7, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™) to profoundly improve people's lives, today reported financial results for the second quarter ended June 30, 2025 and highlighted recent progress. "Avidity continues to deliver on its leadership in RNA therapeutics as we prepare for three potential BLA submissions in a 12-month period with strong clinical data, regulatory progress, and operational execution," said Sarah Boyce, president and chief executive officer of Avidity. "Our programs in FSHD, DMD44 and DM1 each made meaningful advances during the second quarter of 2025, and we are encouraged by the consistent and reproducible data across these late-stage neuromuscular programs. We are keenly aware of the challenges facing patients and families living with these conditions and are preparing for potential commercialization with great thoughtfulness and urgency." "Avidity continues to operate from a position of financial strength," said Mike MacLean, chief financial officer at Avidity. "Avidity's recent topline data for del-brax continues to demonstrate the consistency of the platform. As we move quickly toward potentially three successive launches starting in 2026, we continue to make meaningful progress in building our global infrastructure." Company Highlights Delpacibart zotadirsen (del-zota) for the treatment of people living with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44): In July 2025, Avidity announced the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to del-zota Remain on track for planned BLA submission at year end 2025, which will be Avidity's first BLA Plan to present topline and functional data from the ongoing EXPLORE44-OLE™ trial in the fourth quarter of 2025 Delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1): In July 2025, Avidity announced completion of enrollment in the ongoing Phase 3 HARBOR™ trial On track to share updates from the ongoing MARINA-OLE™ trial including long-term 4 mg/kg efficacy and safety data in the fourth quarter 2025 Expected publication of data analyses from the completed Phase 1/2 MARINA® trial (2025) Topline data readout from HARBOR™ study anticipated in the second quarter of 2026 Marketing application submissions for del-desiran including in U.S., E.U. and Japan anticipated to start in the second half of 2026 Delpacibart braxlosiran (del-brax) for the treatment of facioscapulohumeral muscular dystrophy (FSHD): In June 2025, Avidity announced multiple milestone updates for del-brax including: Alignment with FDA on accelerated and full approval pathways for del-brax; Positive topline Phase 1/2 FORTITUDE™ data from the dose escalation cohorts, demonstrating consistent improvement compared to placebo on multiple functional and quality of life measures, rapid and significant reductions in levels of KHDC1L or cDUX, a DUX4-regulated biomarker, and creatine kinase, a biomarker of muscle damage, and favorable long-term safety and tolerability; Initiated global, confirmatory FORTITUDE-3™ (formerly known as FORWARD™) study of del-brax 2 mg/kg every six weeks, intended to support Avidity's global approval strategy for del-brax Topline data from FORTITUDE biomarker cohort anticipated in the second quarter of 2026 Planned BLA submission for accelerated approval in the second half of 2026, using data from the ongoing, fully enrolled FORTITUDE biomarker cohort Second Quarter 2025 Financial Results Cash, cash equivalents and marketable securities totaled approximately $1.2 billion as of June 30, 2025 Following the end of the second quarter of 2025, the Company received net proceeds of $185.5 million from the sale of stock through its at-the-market offering program The Company expects that its cash, cash equivalents and marketable securities as of June 30, 2025, together with the net proceeds from the at-the-market offering, will be sufficient to fund its operations to mid-2027 Collaboration revenues were $3.8 million for the second quarter of 2025, compared to $2.0 million for the same period of 2024, and primarily relate to the recognition of revenues under Avidity's research collaboration and license partnership with Bristol Myers Squibb. Collaboration revenues were $5.4 million for the six months ended June 30, 2025, compared to $5.6 million for the same period of 2024, and primarily relate to Avidity's research collaboration and license partnership with Bristol Myers Squibb Research and development expenses for the second quarter of 2025 were $138.1 million, compared to $63.9 million for the same period of 2024. Research and development expenses for the six months ended June 30, 2025 were $237.6 million, compared to $130.8 million for the same period of 2024. The increases were primarily driven by the advancement of del-desiran, del-brax and del-zota, as well as internal and external costs related to the expansion of the company's overall research capabilities General and administrative expenses for the second quarter of 2025 were $36.9 million, compared to $20.7 million for the same period of 2024. General and administrative expenses for the six months ended June 30, 2025 were $70.5 million, compared to $34.6 million for the same period of 2024. The increases were primarily due to higher personnel and commercial infrastructure costs to support the company's expanded operations About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and X. Forward-Looking Statements Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: Avidity's plans for three potential product launches; Avidity's plans for BLA submissions for each of its product candidates and the timing thereof; the status of Avidity's ongoing clinical trials and cohorts therein, including but not limited to initiation, enrollment, design and goals; the ability for any of Avidity's product candidates to achieve accelerated approval; plans to present additional data, analyses and other updates from Avidity's ongoing clinical programs and the timing thereof; planned marketing applications for del-desiran in the U.S., European Union and Japan, and the timing thereof; the regulatory status of each of Avidity's clinical programs; Avidity's plans to become a global commercial organization and the status of its commercialization efforts; the characterization of data associated with Avidity's product candidates, the conclusions drawn therefrom, the reproducibility of such data, the impact of such data on the advancement of Avidity's product candidates and their abilities to treat their respective disease indication; Avidity's platform, planned operations and programs; and Avidity's cash position and runway. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: the data and results produced in Avidity's ongoing clinical trials as of the most recent respective cutoff dates may not be indicative of final results, may not support BLA submissions or accelerated approvals, may not be satisfactory to the FDA and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; even if approved, Avidity may not be able to execute any successful product launches; Avidity's efforts to build a global commercial organization may be unsuccessful; unexpected adverse side effects to, or inadequate efficacy of, Avidity's product candidates that may delay or limit their development, regulatory approval and/or commercialization; later developments with the FDA and other global regulators that could be inconsistent with the feedback received to date; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of clinical trials; Avidity's dependence on third parties in connection with clinical testing and product manufacturing; legislative, judicial and regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and subsequent filings with the SEC. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact: Kat Lange (619) 837-5014 [email protected] Media Contact: Kristina Coppola (619) 837-5016 [email protected] View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-reports-second-quarter-2025-financial-results-and-recent-highlights-302524597.html SOURCE Avidity Biosciences, Inc.

On track to deliver key regulatory updates for del-brax and topline data from dose escalation cohorts in the FORTITUDE™ trial in the second quarter Positive topline del-zota data further supports first BLA submission at year end 2025 – continues to highlight reproducibility and consistency across three late-stage clinical trials for DMD44, DM1 and FSHD Executing on global commercial infrastructure development and on track with preparations for first potential commercial launch in U.S. in 2026 Strong balance sheet supports execution across three late-stage clinical programs and commercial launch preparations with cash runway into mid-2027 SAN DIEGO, May 8, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™) to profoundly improve people's lives, today reported financial results for the first quarter ended March 31, 2025, highlighting recent progress. "We continue to build on the consistent and reproducible data readouts from our platform across all three of our clinical programs for DMD44, DM1 and FSHD, and are executing on our transition to become a global commercial organization. In March 2025, we shared positive topline EXPLORE44® Phase 1/2 del-zota data demonstrating remarkable and consistent improvements across multiple measures including a substantial increase in dystrophin production and reduction in creatine kinase levels to near normal, which will support our planned BLA submission at year end 2025 and reinforces del-zota's potential to become a groundbreaking treatment for people living with DMD44," said Sarah Boyce, president and chief executive officer at Avidity. "We look forward to delivering on multiple milestones this year and remain on track to share several key regulatory updates for del-brax in the second quarter that include a potential accelerated approval path in U.S., alignment on the design of the global Phase 3 trial and initiation of the trial. We are also planning to share topline del-brax data from the FORTITUDE™ dose escalation cohorts in the second quarter." "2025 is a pivotal year for Avidity as we build out our global commercial infrastructure to support three potential product launches for del-zota, del-desiran and del-brax. With a strong balance sheet and a cash balance of approximately $1.4 billion at the end of the first quarter, we are well positioned to execute across all of our late-stage clinical trials as we begin to set the stage for our planned first commercial launch in 2026," said Mike MacLean, chief financial officer at Avidity. Company Highlights Delpacibart zotadirsen (del-zota) for the treatment of DMD44: In March 2025, Avidity announced positive topline data from the completed Phase 1/2 EXPLORE44® trial for people living with Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44) demonstrating statistically significant increases in exon skipping, a substantial increase in dystrophin production, a significant reduction in creatine kinase levels to near normal and consistent favorable safety and tolerability results across both dose cohorts. Based on the consistent data between the 5 mg/kg every six weeks and the 10 mg/kg every eight weeks groups across all parameters, Avidity has selected the dose of 5 mg/kg every six weeks of del-zota for the Biologics License Application (BLA) submission and future clinical studies In February 2025, Avidity announced completion of enrollment for the ongoing EXPLORE44-OLE™ study for del-zota Remain on track for planned BLA submission at year end 2025, which will be Avidity's first BLA Plan to present topline and functional data from the ongoing EXPLORE44-OLE trial in the fourth quarter of 2025 Delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1): In April 2025, Avidity announced the Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug designation (ODD) for del-desiran Completion of enrollment for the ongoing Phase 3 HARBOR™ trial is on track for mid-2025 Plan to provide an update from the ongoing MARINA-OLE™ trial including long-term 4 mg/kg and safety data in the fourth quarter 2025 Publication of data analyses from the completed Phase 1/2 MARINA® trial (2025) Planned marketing application submissions starting in 2026, including in the U.S., European Union and Japan Delpacibart braxlosiran (del-brax) for the treatment of facioscapulohumeral muscular dystrophy (FSHD): In March 2025, Avidity announced completion of enrollment for the FORTITUDE™ biomarker cohort for del-brax with 51 total participants enrolled – biomarker cohort supports pathway to potential accelerated approval for del-brax. Enrollment was completed in the first quarter of 2025, ahead of original second quarter guidance. In the second quarter, Avidity plans to share multiple updates from the del-brax program: Regulatory alignment on a potential accelerated approval path in the U.S.; Regulatory alignment on the design of the global Phase 3 trial as well as initiation of the trial; and Topline data from the dose escalation cohorts in the FORTITUDE trial. First Quarter 2025 Financial Results Cash, cash equivalents and marketable securities totaled approximately $1.4 billion as of March 31, 2025. Collaboration revenues were $1.6 million for the first quarter of 2025, compared to $3.5 million for the same period of 2024, and primarily relate to Avidity's research collaboration and license partnership with Bristol Myers Squibb. The decrease was primarily due to the recognition of revenues under Avidity's research collaboration and license partnership with Eli Lilly and Company in the prior year for which there were no revenues recognized in the current year. Research and development expenses for the first quarter of 2025 were $99.5 million, compared to $66.8 million for the same period of 2024. The increase was primarily driven by the advancement of del-desiran, del-brax and del-zota, as well as internal and external costs related to the expansion of the company's overall research capabilities. General and administrative expenses for the first quarter of 2025 were $33.6 million, compared to $13.9 million for the same period of 2024. The increase was primarily due to higher personnel costs to support the company's expanded operations. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare neuromuscular diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and X. Forward-Looking Statements Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: Avidity's plans for three potential product launches; Avidity's plans for a BLA submission for del-zota and the timing thereof; the status of Avidity's ongoing clinical trials and cohorts therein, including but not limited to initiation, enrollment, design and goals; the ability for any of Avidity's product candidates to achieve accelerated approval; the presentation of additional data, analyses and other updates from Avidity's ongoing clinical programs and the timing thereof; planned marketing applications for del-desiran in the U.S., European Union and Japan, and the timing thereof; actual or prospective regulatory alignment related to Avidity's clinical programs; Avidity's plans to become a global commercial organization and the status of its commercialization efforts; the characterization of data associated with del-zota, the conclusions drawn therefrom, the reproducibility of such data, the impact of such data on the advancement of del-zota and its ability to treat DMD44; Avidity's platform, planned operations and programs; and Avidity's cash position and runway. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: the data and results produced in Avidity's ongoing clinical trials as of the most recent respective cutoff dates may not be indicative of final results, may not support BLA submissions or accelerated approvals, may not be satisfactory to the FDA and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; even if approved, Avidity may not be able to execute any successful product launches; Avidity's efforts to build a global commercial organization may be unsuccessful; unexpected adverse side effects to, or inadequate efficacy of, Avidity's product candidates that may delay or limit their development, regulatory approval and/or commercialization; later developments with the FDA and other global regulators that could be inconsistent with the feedback received to date; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of preclinical studies or clinical trials; Avidity's dependence on third parties in connection with clinical testing and product manufacturing; legislative, judicial and regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and subsequent filings with the SEC. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact: Kat Lange (619) 837-5014 [email protected] Media Contact: (619) 837-5016 [email protected] View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-reports-first-quarter-2025-financial-results-and-recent-highlights-302449121.html SOURCE Avidity Biosciences, Inc.