Genetic Diseases, X-Linked

WVE-007 (INHBE GalNAc-siRNA for obesity) is on track to initiate a clinical trial in 1Q 2025; new preclinical data for WVE-007 demonstrate opportunities for monotherapy, for synergistic use with GLP-1s, and for maintenance to avoid rebound weight gain following cessation of GLP-1s Recent proof-of-mechanism data for WVE-006 unlocked Wave’s best-in-class RNA editing capabilities; three new wholly owned RNA editing programs targeting PNPLA3, LDLR and APOB are first-in-class approaches to address significant unmet needs in cardiometabolic diseases Wave plans to have five GalNAc-RNA editing or GalNAc-siRNA programs in the clinic by 2026 Wave’s clinically validated platform continues to lead on the improvement of intracellular delivery using novel chemistry, including PN backbone chemistry modifications CAMBRIDGE, Mass., Oct. 30, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced updates across its growing pipeline, which were also discussed during the company’s virtual analyst and investor Research Day (link to webcast). Updates included new preclinical data for Wave’s GalNAc-small interfering RNA (siRNA) approach for obesity, WVE-007, which is on track to initiate a clinical trial in the first quarter of 2025. Wave also introduced new, wholly owned GalNAc-RNA editing programs and demonstrated its leadership in oligonucleotide chemistry innovation. “This year, we showed through multiple positive clinical datasets that our leading RNA medicines platform is opening up new possibilities for the treatment of human disease. Next to the clinic will be our WVE-007 program, which reimagines obesity treatment with a mechanism that directly impacts fat cells – particularly visceral fat – and which could be used as monotherapy, add-on to GLP-1s, or for maintenance post-GLP-1 cessation,” said Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences. “Additionally, we are excited to introduce three new programs for mRNA correction and upregulation to treat cardiometabolic diseases with significant unmet needs. These programs have substantial, well-defined patient populations, strong support from human genetics, efficient paths to proof-of-concept in the clinic, and offer the opportunity to meaningfully change health outcomes for millions of people. This truly is an exciting time for Wave and for patients as we unlock new biology and build our high-value and innovative pipeline.” Key Highlights from Today’s Virtual Research Day WVE-007: INHBE GalNAc-siRNA and potential best-in-class obesity therapeutic approaching the clinic WVE-007 is Wave’s GalNAc-siRNA that targets INHBE as a novel approach to treat obesity. The approach is based on human genetics, where individuals who have a protective loss-of-function mutation in the INHBE gene have a healthier cardiometabolic profile, including less unhealthy visceral fat, lower triglycerides, and lower risk of type 2 diabetes and cardiovascular disease.Silencing of INHBE in vivo directly impacts adipocytes (fat cells) and induces lipolysis (fat burning), which is different from GLP-1s’ centrally acting mechanism that involves the digestive system and central nervous system to decrease appetite.New preclinical data in diet induced obese (DIO) mice demonstrate the potential of WVE-007 to be used across the treatment continuum, including as monotherapy, as an add-on to GLP-1s, and for maintenance to prevent weight regain and weight cycling after discontinuing GLP-1s.Wave is on track to initiate a Phase 1 clinical trial for WVE-007 in adults living with overweight or obesity in the first quarter of 2025. The trial is planned to assess safety, tolerability, pharmacokinetics, and Activin E lowering (a biomarker for target engagement), as well as body composition. New GalNAc-RNA editing programs designed to optimally address high-impact cardiometabolic diseases Building on Wave’s recent achievement of the first-ever therapeutic RNA editing with WVE-006 in alpha-1 antitrypsin deficiency, the company today announced three new, wholly owned RNA editing programs that are on track for candidate selection in 2025. Like WVE-006, these programs leverage GalNAc conjugation and have efficient clinical paths to proof-of-concept. These new programs include: PNPLA3 (mRNA correction): Homozygous PNPLA3-I148M patients are at high risk for a variety of liver diseases and there are more than 9 million impacted individuals in the US and Europe. Wave’s RNA editing approach aims to achieve at least 50% correction to restore the heterozygous phenotype with low risk of liver disease, similar to the approach with WVE-006.LDLR (mRNA upregulation) and APOB (mRNA correction): High unmet needs remain among familial hypercholesterolemia (FH) patients, with approximately half of patients not reaching goal LDL-c levels with available therapies. Wave’s AIMers to upregulate LDLR and correct APOB have potential to be first-in-class treatment approaches for best-in-class effects to reach target LDL-c levels in Heterozygous FH (HeFH) patients. Combined, LDLR and APOB AIMers would address ~1 million HeFH patients in the US and Europe. LDLR upregulation also offers significant expansion opportunities in patients with statin intolerance or prior cardiovascular events (~30 million patients in the US and Europe). Chemistry innovation enhancing intracellular delivery Today, Wave shared data demonstrating how its proprietary chemistry results in marked improvements in five key areas of intracellular delivery: 1) cellular uptake; 2) endosomal release; 3) cellular residency; 4) nuclear uptake; and 5) target engagement.Wave shared how it is using PN variants and modifications to physicochemical properties to achieve best-in-class siRNA silencing and RNA editing in a variety of extrahepatic tissues.Wave also highlighted novel chemistry used in its AIMers, including proprietary N-3-uridine chemistry (N3U), which substantially enhances RNA editing efficiency. This novel chemistry was the subject of a recent Nucleic Acids Research publication. An archived webcast of the event can be accessed by visiting “Investor Events” on the investor relations section of the Wave Life Sciences website: https://ir.wavelifesciences.com/events-publications/events. About Wave Life SciencesWave Life Sciences (Nasdaq: WVE) is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave’s RNA medicines platform, PRISM®, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. Its toolkit of RNA-targeting modalities includes editing, splicing, RNA interference and antisense silencing, providing Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave’s diversified pipeline includes clinical programs in Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington’s disease, as well as a preclinical program in obesity. Driven by the calling to “Reimagine Possible”, Wave is leading the charge toward a world in which human potential is no longer hindered by the burden of disease. Wave is headquartered in Cambridge, MA. For more information on Wave’s science, pipeline and people, please visit www.wavelifesciences.com and follow Wave on X (formerly Twitter) and LinkedIn. Forward-Looking StatementsThis press release contains forward-looking statements concerning our goals, beliefs, expectations, strategies, objectives and plans, and other statements that are not necessarily based on historical facts, including statements regarding the following, among others: the anticipated initiation, site activation, patient recruitment, patient enrollment, dosing, generation and reporting of data and completion of our clinical trials, including interactions with regulators and any potential registration based on these data, and the timing and announcement of such events; regulatory submissions and timing for regulatory feedback; the protocol, design and endpoints of our clinical trials; the future performance and results of our programs in clinical trials; our expectations with respect to how our clinical data successes to date may predict success for our future therapeutic candidates, future clinical data readouts and further validate of our platform; ongoing and future preclinical activities and programs; the potential of our preclinical data to predict the behavior of our compounds in humans; our identification and expected timing of future product candidates and their therapeutic potential; the anticipated benefits of our therapeutic candidates and pipeline compared to our competitors; patient population estimates related to our therapeutic candidates and the potential addressable market that our therapeutics may address; our ability to design compounds using various modalities and the anticipated benefits of that approach; the breadth and versatility of our PRISM drug discovery and development platform; the expected benefits of our stereopure oligonucleotides compared with stereorandom oligonucleotides; the potential benefits of our RNA editing capability, including our AIMers, compared to others; the benefits of RNA medicines generally; the potential for certain of our programs to be best-in-class or first-in-class; the potential benefits that our “edit-verse” may provide us, including identifying new RNA editing targets; our ability to translate genetic insights into high impact medicines; the status and progress of our programs relative to potential competitors; the progress and potential benefits of our collaborations; ;and the anticipated duration of our cash runway and our ability to fund future operations. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual results to differ materially from those indicated by these forward-looking statements as a result of these risks, uncertainties and important factors, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in Wave’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as amended, and in other filings Wave makes with the SEC from time to time. Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances. Investor Contact:Kate Rausch+1 617-949-4827krausch@wavelifesci.com Media Contact:Alicia Suter+1 617-949-4817asuter@wavelifesci.com

2024 Outcomes Report demonstrates treatment helps clients get well and stay well, regardless of diagnosis or demographics MIAMI, Oct. 30, 2024 /PRNewswire/ -- A new study reveals that over 52% of individuals entering eating disorder treatment began at or above a normal weight, challenging common misconceptions that people must look underweight to require intervention. Although eating disorders are common, many individuals do not receive the help they need. However, data shows that treatment significantly improves recovery outcomes, helping clients stay well for longer. These important insights are part of the largest longitudinal study on eating disorder treatment outcomes, conducted by Monte Nido, one of the nation's leading eating disorder treatment providers. The study reviewed data from 12,000 participants, and results underscore that eating disorders affect people of all shapes and sizes, and that specialized, client-first programs can have lifesaving, long-lasting benefits. Clients Get Well, Stay Well The long-term impact of eating disorder treatment has proven to be an essential component of the healing process. According to the study: 91% of adults and 94% of adolescents restored weight when needed Clients treated by Monte Nido maintained treatment gains 6-months post-discharge: 81% remained significantly improved in ED symptoms 73% remained significantly improved in depression symptoms 71% remained significantly improved in symptoms of anxiety "We believe that recovery is possible for everyone, and our outcomes study demonstrates that treatment can be effective in helping those with eating disorders live the full life they so deserve," Cassie McLean, Chief Executive Officer of Monte Nido. Co-Occurring Diagnoses Are Common The study shows that 85% of all clients had a co-occurring diagnosis of a mood, anxiety or substance use disorder, obsessive compulsive disorder, and/or posttraumatic stress disorder. A co-occurring diagnosis is a significant marker of the complexity of life with an eating disorder. "By addressing both the eating disorder and co-occurring mental health conditions, providers open sustainable pathways for client's healing," McLean emphasized. 45% of adults and 35.4% of adolescents in residential treatment had PTSD These clients demonstrated significantly higher scores on all measures including measures of eating disorder psychopathology, major depression, anxiety disorders and quality of life These clients experience both clinically and statistically significant decreases in their symptoms from admission to discharge Clients who completed cognitive processing therapy (CPT) sessions showed a significant reduction in PCL-5 scores, dropping from an average of 52.6 to 29, which is below the threshold score of 33, meaning clients no longer met criteria for PTSD at the end of treatment. Eating Disorders Don't Discriminate Despite social and cultural expectations about how those with eating disorders appear, they can and do impact anyone, regardless of gender, sexuality, race, ethnicity, age, socio-economic background and size. Nearly 29 million Americans will experience an eating disorder in their lifetime, and very few will get the care they need, largely due to barriers created by discrimination and weight stigma. "This study has shed light on who is living with eating disorders, and that they are far more complex and diverse than our socially conditioned image of those living with an eating disorder," said McLean. "This data will help expand our cultural understanding of individuals that are struggling with a range of eating disorders, and hopefully help remove some of the stigma that so often goes hand-in-hand with a diagnosis." Size & Weight 52% of people entering treatment were at or above "normal" weight at admission according to the NIH body mass index (BMI) calculator Most clients who were diagnosed with other specified feeding or eating disorder (OSFED) have atypical anorexia nervosa Gender & Sexuality 36% of all clients identified as LGBTQIA+ 63% of LGBTQIA+ adults in residential treatment met criteria for PTSD, compared to 45% cisgender heterosexual adults Age 77.6% of adolescents in residential treatment (average age of 15) had a co-occurring diagnosis, such as a mood disorder, anxiety disorder, or PTSD. 38% reported that someone else in their family has struggled with an eating disorder or disordered eating 75% of these adolescents had their families that were either very or extremely involved in treatment as indicated by regular family therapy and consistent communication with the clinical team Data Shines a Light on ARFID and BED Findings also emphasize the commonality of two lesser-known eating disorders, avoidant/restrictive food intake disorder (ARFID) and binge eating disorder (BED) within the client population. This data is important as certain disorders, like BED, are often misunderstood and misdiagnosed due to an overreliance on BMI as a measure of health. This misconception can lead to inadequate treatment, and even unhelpful guidance from providers. Adults, with an average admission age of 32, had binge eating disorder for 20 years before receiving treatment at Monte Nido, and 14 years before seeking any form of treatment. Clients with BED got better on all measures in all levels of care ARFID, which is characterized by "selective behaviors" or disinterest in food, has historically been difficult to diagnose, explaining why it's sometimes considered a "new" eating disorder. This year, Monte Nido developed the first measuring tool of its kind to help inform ARFID treatment, the Food Flexibility Scale™, which illustrates the levels of food flexibility that clients achieve through treatment. By using the novel food flexibility scale that measures progress across various degrees of variety, volume, and exposure to different food settings, the study indicates significant growth over the course of treatment. Of clients treated for ARFID, 92% of clients reached adequate nutritional intake levels by the end of treatment. Flexible Treatment Modalities Increase Accessibility The study also highlights the importance of flexible treatment options in improving accessibility, particularly through expanded virtual programs. These options allow clients to receive care despite geographic, scheduling, or familial barriers. "Access to flexible, innovative treatment options, especially virtual programs, is a game-changer for clients who might otherwise forgo the care they need," said Molly Perlman, MD, MPH, CEDS-C, Chief Medical Officer of Monte Nido. "By removing obstacles, virtual treatment is transforming how and when individuals seek help, making care more adaptable to real-life demands." Regardless of being in-person or online, data shows that less-intensive but highly structured remote treatment facilitates meaningful recovery among clients. 88% of clients discharged from virtual treatment described their experience as good to excellent Clients discharging from virtual programs with Monte Nido rated their experience with program staff- including clinical directors, primary therapists, dieticians, recovery coaches, their psychiatric and medical providers as excellent on average For more information about Monte Nido or to read the complete study, click here. To inquire about treatment at one of the Monte Nido programs, please visit . About the Monte Nido 2024 Outcomes Report The 2024 Outcomes Report is based on analysis of outcomes from Monte Nido programs nationwide, spanning all levels of care and more than 12,000 clients. Clients who consented to participate in the research study completed a series of clinical questionnaires at select intervals: at admission, upon transfer to a new level of care, and upon discharge from a program. In addition, follow-up data was obtained from consenting clients at specified time points after discharge. About Monte Nido Monte Nido is a national mental health provider committed to eradicating eating disorders by offering industry-leading care to adults and adolescents of all genders in a healing environment. Our clinically comprehensive, research-backed treatment in inpatient, residential, and day and virtual treatment programs show exceptional outcomes. Our clients get well and stay well six months after treatment. Founded in 1996, our expert staff of eating disorder professionals deliver compassionate care, breakthrough outcomes, and long-term recovery for our clients. Monte Nido operates 50 programs and in 28 states virtually across our family of programs: Monte Nido, Monte Nido Walden, Monte Nido Rosewood, and Monte Nido Clementine. Contact: Hannah Miller L.C. Williams & Associates 312-565-3900 [email protected] SOURCE Monte Nido WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Biomarker cohort for delpacibart braxlosiran (del-brax), the first potential therapy to target DUX4, is measuring changes in DUX4 regulated biomarkers; del-brax 2 mg/kg will be administered every six weeks Enrollment in the del-brax biomarker cohort expected to be completed in 1H 2025; on track to initiate del-brax functional cohort in 1H 2025In previously reported initial data, del-brax 2 mg/kg every six weeks showed unprecedented and consistent reductions of DUX4 regulated genes, significant decreases in novel circulating biomarker and creatine kinase, and trends of functional improvement at the four-month timepoint SAN DIEGO, Oct. 30, 2024 /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 the initiation of a biomarker cohort in the Phase 1/2 FORTITUDE™ trial of delpacibart braxlosiran (del-brax/AOC 1020) in people living with facioscapulohumeral muscular dystrophy (FSHD). Avidity is pursuing a potential accelerated approval path for del-brax and expects enrollment in the biomarker cohort to be completed in the first half of 2025. Avidity also remains on track to initiate a functional cohort in the first half of 2025. In addition, enrollment of the FORTITUDE Open-label Extension study (OLE) is ongoing. Continue Reading (PRNewsfoto/Avidity Biosciences, Inc.) Del-brax is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the disease-causing gene, double homeobox 4 (DUX4). Currently, there are no approved therapies for the treatment of FSHD, a rare, hereditary disorder marked by life-long, relentless loss of muscle function, significant pain, fatigue, and progressive disability. "The initiation of the biomarker cohort marks a key step in our strategy to pursue a potential accelerated approval path for del-brax, the first potential treatment to directly target the root cause of FSHD," said Steve Hughes, M.D., chief medical officer at Avidity. "We are very pleased with the del-brax 2 mg/kg data which showed unprecedented and consistent reductions of DUX4-regulated genes, significant decreases in novel circulating biomarker and creatine kinase, and trends of functional improvement with favorable safety and tolerability at the four-month timepoint. We are advancing our clinical studies for del-brax as quickly as possible as we understand the urgency to bring a potential new treatment to people living with FSHD who have no treatment options." The biomarker cohort in the FORTITUDE trial will assess the impact of del-brax 2 mg/kg every six weeks in people living with FSHD, ages 16-70. The primary endpoints of the study are changes in DUX4 regulated gene expression and DUX4 regulated circulating biomarker. For people living with FSHD it is important to maintain suppression of DUX4 at all times as aberrant gene expression is toxic to the muscles. Favorable safety and tolerability, as well as decreases in circulating biomarker and creatine kinase levels were similar for patients treated with 2mg/kg or 4mg/kg of del-brax. Due to this similarity, Avidity selected 2 mg/kg of del-brax to be administered every six weeks, designed to ensure continuous suppression of DUX4 for the biomarker and functional cohorts. In June of this year, Avidity reported positive initial del-brax 2 mg/kg data at four months from the Phase 1/2 FORTITUDE trial demonstrating unprecedented and consistent reductions of greater than 50% in DUX4 regulated genes, mean reductions of 25% or greater in novel circulating biomarker and creatine kinase, trends of functional improvement, and favorable safety and tolerability in people living with FSHD at the 31st Annual FSHD Society International Research Congress. About the Phase 1/2 FORTITUDE™ trial The FORTITUDE™ trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate single and multiple doses of delpacibart braxlosiran or del-brax (AOC 1020) in approximately 100 participants with facioscapulohumeral muscular dystrophy (FSHD). FORTITUDE will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of del-brax administered intravenously. Activity of del-brax will be assessed using key biomarkers, including DUX4-regulated muscle and circulating biomarkers and magnetic resonance imaging (MRI) measures of muscle volume and composition. Though the Phase 1/2 trial is not statistically powered to assess functional benefit, it will explore the clinical activity of del-brax including measures of mobility and muscle strength as well as patient reported outcomes and quality of life measures. Participants will have the option to enroll in FORTITUDE-OLE, an open-label extension study once participation in the FORTITUDE study is complete. For more information about the FORTITUDE trial, visit the FORTITUDE study website or visit and search for NCT05747924. About the Phase 2 FORTITUDE-OLE™ trial FORTITUDE-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of delpacibart braxlosiran or del-brax (AOC 1020) in participants with facioscapulohumeral muscular dystrophy (FSHD) who were previously enrolled in the FORTITUDE Phase 1/2 trial. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-brax in participants that enrolled in the randomized, placebo-controlled, Phase 1/2 FORTITUDE clinical trial. Participants who enroll in the FORTITUDE-OLE study will receive del-brax regardless of whether they received active treatment or placebo in the FORTITUDE study. The total duration of active treatment with del-brax in the FORTITUDE-OLE is approximately 24 months. Avidity may extend active treatment beyond 24 months at a future timepoint. For more information on the FORTITUDE-OLE study click here or visit and search for NCT06547216. About Del-brax (AOC 1020) Del-brax (AOC 1020) is designed to treat the underlying cause of FSHD, which is caused by the abnormal expression of a gene called double homeobox 4 or DUX4. The abnormal expression of DUX4 protein leads to changes in gene expression in muscle cells that are associated with the life-long, progressive loss of muscle function in patients with FSHD. Del-brax aims to reduce the expression of DUX4 mRNA and DUX4 protein in muscles in people with FSHD. Del-brax consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA. Avidity reported positive initial del-brax 2 mg/kg data at four months from the Phase 1/2 FORTITUDE trial demonstrating unprecedented and consistent reductions of greater than 50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability in people living with FSHD. Del-brax is currently in Phase 1/2 development as part of the FORTITUDE™ trial in individuals with FSHD. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Orphan designation for del-brax and the FDA has granted del-brax Fast Track designation. About Facioscapulohumeral Muscular Dystrophy (FSHD) Facioscapulohumeral muscular dystrophy (FSHD) is a rare, progressive, and variable hereditary muscle-weakening condition marked by life-long, relentless loss of muscle function, significant pain, fatigue, and progressive disability. It is characterized by progressive and often asymmetric skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk and progresses to weakness in muscles in the lower body. FSHD is an autosomal dominant disease caused by the aberrant expression of the DUX4 (double homeobox 4) gene in the skeletal muscle, which activates genes that are toxic to muscle cells and leads to a series of downstream events that result in skeletal muscle wasting and compromised muscle function. Skeletal muscle weakness results in physical limitations throughout the whole body, including an inability to lift arms for more than a few seconds, loss of ability to show facial expressions and serious speech impediments. These symptoms cause many people affected by FSHD to become dependent on the use of a wheelchair for mobility. Currently, there are no approved treatments for people living with FSHD. 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 broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through internal discovery efforts and key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit 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 primary endpoints, design, goals and other details related to the FORTITUDE™ trial and each of its cohorts; the status and clinical development of del-brax, including any potential accelerated approval; Avidity's plans for the FORTITUDE-OLE™ study and the timing thereof; the characterization of safety, tolerability and functional data associated with del-brax from the Phase 1/2 FORTITUDE trial; the impact of such data on the advancement of del-brax; the plans and timing of adding a functional cohort for the FORTITUDE trial and the biomarker and functional cohorts potentially serving as the basis for registration; the potential of Avidity's product candidates to treat rare diseases and Avidity's efforts to bring them to people suffering from applicable diseases; and the potential of AOCs to target a range of different cells and tissues beyond the liver, and to treat cardiac and immunological diseases. 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 those beyond its control, including, without limitation: preliminary results of a clinical trial are not necessarily indicative of final results and additional participant data related to del-brax that continues to become available may be inconsistent with the data produced as of the date hereof, and further analysis of existing data and analysis of new data may lead to conclusions different from those established as of the data cutoff; 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, or may result in clinical holds which may not be timely lifted, recalls or product liability claims; Avidity's current and planned cohorts in the FORTITUDE trial may not support the registration of del-brax; Avidity is early in its development efforts; Avidity's approach to the discovery and development of product candidates based on its AOC platform is unproven, and the company does not know whether it will be able to develop 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 preclinical and clinical testing and product manufacturing; regulatory developments in the United States and foreign countries; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed with the Securities and Exchange Commission (SEC) on February 28, 2024, and in 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: Michael MacLean (619) 837-5014 [email protected] Media Contact: Navjot Rai (619) 837-5016 [email protected] SOURCE Avidity Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED