US Market News
1月前
Atrium Therapeutics Reports First Quarter 2026 Financial ResultsMay 14, 2026 4:40 PM
PR Newswire (US) 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 HighlightsLaunched 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 MilestonesSubmit IND application for ATR 1072 in the second half of 2026Initiate Phase 1/2 clinical trial for ATR 1072, subject to regulatory clearanceFile IND application for ATR 1086 in 2027, with IND-enabling studies initiating in 2026First Quarter 2026 Financial ResultsGiven 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 TherapeuticsAtrium 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 TherapeuticsInvestors 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 SyndromePRKAG2 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 CardiomyopathyPhospholamban ("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 StatementsThis 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.Atrium Therapeutics, Inc. Selected Condensed Financial Information(in thousands except share and per share information)(Unaudited)Statements of Operations
Three Months Ended March 31,
2026
2025
Collaboration revenue
$19,635
$1,573
Operating expenses:
Research and development
16,657
6,937
General and administrative
20,258
2,088
Total operating expenses
36,915
9,025
Loss from operations
(17,280)
(7,452)
Other income, net
647
3
Net loss and comprehensive loss
$(16,633)
$(7,449)
Basic and diluted net loss per common share
$(0.97)
$(0.44)
Weighted average common shares outstanding used in the calculation of basic and diluted
net loss per common share
17,105,643
17,105,643
Balance Sheets
March 31,
December 31,
2026
2025
Assets
Current assets:
Cash and cash equivalents
$267,849
$—
Collaboration receivable
15,000
—
Prepaid assets
2,578
1,535
Other current assets
138
1,310
Total current assets
285,565
2,845
Restricted cash
415
—
Property and equipment, net
4,337
2,724
Right-of-use asset
2,043
2,784
Total assets
$292,360
$8,353
Liabilities and Stockholders' Equity/Former Parent Deficit
Current liabilities:
Accounts payable
$807
$4,398
Accrued liabilities
6,983
8,945
Accrued compensation
2,091
3,147
Lease liabilities
2,680
3,672
Deferred revenue, current portion
9,344
21,639
Total current liabilities
21,905
41,801
Deferred revenue, net of current portion
36,351
28,691
Other long-term liabilities
—
574
Total liabilities
58,256
71,066
Commitments and contingencies
Stockholders' equity/ Former Parent's deficit:
Preferred stock, $0.001 par value: 40,000,000 shares authorized; no shares issued and
outstanding
—
—
Common stock, $0.001 par value: 400,000,000 shares authorized; 17,105,643 shares
issued and outstanding as of March 31, 2026, and no shares authorized, issued, or
outstanding as of December 31, 2025
16
—
Additional paid-in capital
221,960
—
Retained earnings
12,128
—
Net Investment from Former Parent
—
(62,713)
Total stockholders' equity/Former Parent's deficit
234,104
(62,713)
Total liabilities and Stockholders' equity/Former Parent's deficit
$292,360
$8,353
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US Market News
4月前
Atrium Therapeutics Launches with Approximately $270 Million to Advance Novel RNA Medicines for Rare Genetic CardiomyopathiesFebruary 27, 2026 9:19 AM
PR Newswire (US)
Spinoff from Novartis AG's acquisition of Avidity Biosciences advances precision cardiology programs using targeted RNA delivery platform Lead candidates ATR 1072 and ATR 1086 expected to enter clinical trials for PRKAG2 syndrome and PLN cardiomyopathy, respectivelySAN DIEGO, Feb. 27, 2026 /PRNewswire/ -- Atrium Therapeutics, Inc. (Nasdaq: RNA) launched today as a newly independent, publicly traded company dedicated to delivering RNA therapeutics directly to the heart to transform care for people living with rare, life-threatening genetic cardiomyopathies. Atrium Therapeutics was established in connection with Avidity Biosciences, Inc.'s acquisition by Novartis AG. The company is led by Kathleen Gallagher, President and Chief Executive Officer (CEO), and begins operations with two precision cardiology candidates, two undisclosed research targets and approximately $270 million in cash and cash equivalents.
Atrium Therapeutics' two lead development candidates are: ATR 1072 for PRKAG2 (Protein Kinase AMP-activated non-catalytic subunit Gamma 2) syndrome and ATR 1086 for PLN (phospholamban) cardiomyopathy. Both conditions are severe, life-threatening, rare autosomal dominant progressive cardiomyopathies with no approved therapies to treat the underlying cause of disease."The launch of Atrium Therapeutics marks an important milestone for people living with genetic cardiomyopathies," said Kathleen Gallagher, President and CEO of Atrium Therapeutics. "Patients and families facing these genetically driven rare cardiomyopathies have few if any options that address the underlying cause. Building on Avidity's pioneering work in targeted RNA delivery, Atrium is positioned to advance precision medicines designed to directly target the biologic drivers of cardiac disease. Atrium has the opportunity to help pave the way for a new era for RNA therapies in precision cardiology.""As Atrium Therapeutics embarks on a new chapter today, I am incredibly proud of the team's commitment to advancing groundbreaking science for people with genetically driven cardiomyopathies," said Sarah Boyce, Chair of Atrium Therapeutics' board of directors and former CEO of Avidity. "Precision cardiology is an area of immense opportunity, and I am confident the Atrium Therapeutics team's experiences in rare disease, drug development and RNA therapeutics and patient-focused approach will urgently move its pipeline forward."Pipeline and Development Milestones ATR 1072 (PRKAG2 syndrome): Investigational New Drug (IND)-enabling studies and CMC manufacturing underway. Atrium expects to file an IND in the second half of 2026.ATR 1086 (PLN cardiomyopathy): Chemistry Manufacturing and Controls (CMC) manufacturing planned to support initiation of IND-enabling preclinical studies in 2026, targeting an IND submission in 2027.Pending supportive Phase 1 trial results, Atrium Therapeutics anticipates advancing both programs into clinical trials, while continuing to expand its additional precision cardiology pipeline and develop its next-generation RNA delivery platform.About Atrium TherapeuticsAtrium 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 delivery to the skeletal muscle and applies it for efficient delivery to the heart overcoming challenges associated with non-specific tissue delivery. The company's pipeline consists of two precision cardiology candidates, ATR 1072 and ATR 1086, 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.About PRKAG2 SyndromePRKAG2 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 US. Current management is limited to symptomatic treatment; no approved therapies exist to address the underlying genetic driver of disease.About PLN CardiomyopathyPLN (phospholamban) cardiomyopathy is a rare autosomal dominant, progressive cardiac disease caused by mutations in PLN, a key regulator of SERCA2a calcium pump. Pathogenic variants 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 US. No approved therapies target the underlying molecular cause of the disease.Forward-Looking StatementsThis 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 Atrium Therapeutics' 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; 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 Therapeutics' actual results to be materially different than those expressed in Atrium Therapeutics' 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 product candidates and any future product candidates that we may identify or develop; 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; anticipated 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 with development, regulatory, manufacturing or commercialization expertise; our reliance on third parties 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; our reliance on third parties for the manufacture of our product candidates; 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 in Atrium Therapeutics' Registration Statement on Form 10, initially publicly filed by Atrium Therapeutics with the Securities and Exchange Commission (the "SEC") on December 10, 2025 and in other filings and furnishings made by Atrium Therapeutics with the SEC from time to time. Atrium Therapeutics 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 and Media Contact:Stephanie Kenney, Chief Corporate Affairs Officer
investors@atrium-tx.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/atrium-therapeutics-launches-with-approximately-270-million-to-advance-novel-rna-medicines-for-rare-genetic-cardiomyopathies-302699662.htmlSOURCE Atrium Therapeutics
Original: Atrium Therapeutics Launches with Approximately $270 Million to Advance Novel RNA Medicines for Rare Genetic Cardiomyopathies
US Market News
4月前
The New England Journal of Medicine Publishes Results from Phase 1/2 MARINA® Trial of Delpacibart Etedesiran (del-desiran) for Treatment of Myotonic Dystrophy Type 1February 18, 2026 5:00 PM
PR Newswire (US)
Del-desiran effectively delivered siRNA to muscle, resulting in approximately 40% mean reduction in DMPK mRNA and amelioration of missplicingTreatment demonstrated improvements in multiple measures including myotonia, muscle function and strength, mobility and patient-reported outcomesDel-desiran showed acceptable safety and tolerability with most adverse events mild or moderateSAN 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
investors@aviditybio.comMedia Contact:
Kristina Coppola
(619) 837-5016
media@aviditybio.com
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.htmlSOURCE Avidity Biosciences, Inc.
Original: The New England Journal of Medicine Publishes Results from Phase 1/2 MARINA® Trial of Delpacibart Etedesiran (del-desiran) for Treatment of Myotonic Dystrophy Type 1
US Market News
4月前
Avidity Biosciences Announces Expected Record Date for Spin-OffFebruary 2, 2026 9:44 PM
PR Newswire (US)
SAN DIEGO, Feb. 2, 2026 /PRNewswire/ -- Avidity Biosciences, Inc. ("Avidity") (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 announced that its board of directors has designated the close of business, Eastern Time, on February 12, 2026 as the record date (the "Record Date") for the pro rata distribution of all of the issued and outstanding shares of common stock of Atrium Therapeutics, Inc. ("SpinCo") to holders of Avidity common stock on the Record Date in connection with the previously announced proposed acquisition of Avidity by Novartis AG (the "Merger") and the separation of Avidity's early-stage precision cardiology programs into SpinCo (the "Spin-Off"). Each such holder will receive one share of SpinCo common stock for every ten shares of Avidity common stock held on the Record Date.
Completion of the Merger and of the Spin-Off remain subject to certain closing conditions noted in Avidity's definitive proxy statement filed with the Securities and Exchange Commission on January 30, 2026, including the receipt of Avidity stockholder approval. Accordingly, the Record Date may change based on the closing date of the Merger and the Spin-Off.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.Additional information and Where to Find ItIn connection with the proposed transaction between Avidity and Novartis AG ("Novartis"), Avidity has filed with the Securities and Exchange Commission (the "SEC") a definitive proxy statement on January 30, 2026 (the "Proxy Statement"). Avidity may also file other documents with the SEC regarding the proposed transaction. 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 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 CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTIONS AND THE PARTIES TO THE PROPOSED TRANSACTIONS. Investors and security holders are able to obtain a free copy of the Proxy Statement and such other documents containing important information about Novartis and Avidity 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 Avidity and certain of its respective directors and executive officers may be deemed to be participants in the solicitation of proxies in respect of the proposed transaction. Avidity stockholders may obtain additional information regarding the direct and indirect interests of the participants in the solicitation of proxies in connection with the proposed transaction, including the interests of Avidity directors and executive officers in the transaction, which may be different than those of Avidity stockholders generally, by reading the Proxy Statement and any other relevant documents that are filed or will be filed with the SEC relating to the transaction. You may obtain free copies of these documents using the sources indicated above.No Offer or SolicitationThis 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 StatementsThis 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 Merger and the Spin-Off (collectively, the "Transactions"), the expected timetable for completing each of the proposed Transactions and the related interim steps, 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, June 30, 2025 and September 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.Media Contact:
Kristina Coppola
(619) 837-5016
media@aviditybio.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-announces-expected-record-date-for-spin-off-302677009.htmlSOURCE Avidity Biosciences, Inc.
Original: Avidity Biosciences Announces Expected Record Date for Spin-Off
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