RNA Avidity Biosciences Inc.

24.22
-0.01  0%
Previous Close 24.23
Open 23.8
52 Week Low 17.58
52 Week High 36.02
Market Cap $1,135,847,350
Shares 46,897,083
Float 41,591,670
Enterprise Value $724,164,321
Volume 137,339
Av. Daily Volume 242,113
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Drug Pipeline

Drug Stage Notes
AOC 1001 (MARINA)
Myotonic dystrophy type 1 (DM1)
Phase 1/2
Phase 1/2
Phase 1/2 trial first patient dosed, November 4, 2021.

Latest News

  1. LA JOLLA, Calif., Nov. 9, 2021 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today reported financial results for the third quarter and nine months ended September 30, 2021 and highlighted recent corporate progress.

    "This past quarter we initiated our Phase 1/2 MARINATM trial for AOC 1001, which transitions us into a clinical stage company. This trial is important for both AOC 1001 and our platform as it is expected to generate the first proof of concept data on the AOC platform's potential to deliver RNA therapy outside of the liver – a first for the field of RNA conjugates and for Avidity," said Sarah Boyce, president and chief executive officer. "This is also an important milestone for the myotonic dystrophy community who currently have no therapeutic options. We look forward to sharing preliminary data on AOC 1001 in the second half of next year as we work to achieve our ambitious agenda of having AOCs for DM1, DMD and FSHD in the clinic by the end of 2022."

    "Following completion of our successful first follow-on financing in August, we are well funded into 2024 with a cash balance of $413 million at quarter end. Given these significant financial resources, we are well positioned to complete the ongoing MARINA trial for AOC 1001, as well as initiate proof of concept clinical trials in 2022 for AOC 1044, the first of our DMD programs, and for our AOC FSHD program while we continue our investment in expanding our AOC platform," said Mike MacLean, chief financial officer.

    Recent Highlights:

    • Initiated first clinical trial for an AOC – transitioning Avidity into a clinical stage company
      • Initiated the Phase 1/2 MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1)
        • Received clearance from the U.S. Food and Drug Administration (FDA) to proceed with the Phase 1/2 MARINA trial under Avidity's initial new drug application (IND) in July 2021
        • Enrolled the first patient in the MARINA trial in October 2021, marking the first time a person has been dosed with an AOC
      • AOC 1001 was granted Orphan Designation by the FDA and the European Medicines Agency (EMA)
      • AOC 1001 was granted Fast Track Designation by the FDA in October 2021
    • Entered a collaboration with a key physician network for a natural history study in FSHD
      • Avidity is supporting the natural history study called the Motor Outcomes to Validate Evaluations Plus (MOVE+) Study run by the facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial Research Network (FSHD CTRN) to enhance the understanding of how to utilize whole-body MRI and other tools to identify specific biomarkers for FSHD that can accelerate and support future clinical trial design.
    • Completed a follow-on financing, resulting in net proceeds of $155.1 million

    Upcoming Events

    Volume 3 of Avidity's Virtual Investor and Analyst Event Series will be focused on FSHD and will be held on Thursday, December 9, 2021 at 8am PT/11am ET.  To register for the live video webcast, please visit the "Events and Presentations" page in the "Investors" section of Avidity's website. A replay of the webcast will be archived on Avidity's website following the event.

    Art Levin, Ph.D., Avidity's chief scientific officer, and W. Michael Flanagan, Ph.D., Avidity's chief technical officer, will discuss the scientific rationale and potential benefits of leveraging AOCs for FSHD.

    The virtual event will also feature Jeffrey Statland, M.D., professor, Department of Neurology, University of Kansas Medical Center, to discuss the clinical impact of FSHD and the ongoing natural history studies. Dr. Statland is one of the principal investigators in the ongoing MOVE and MOVE+ natural history studies for people living with FSHD.  

    Third Quarter 2021 Financial Results

    • Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $413.0 million as of September 30, 2021, compared to $328.1 million as of December 31, 2020.
    • Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity's partnership with Eli Lilly and Company and totaled $2.2 million for the third quarter of 2021 compared with $1.7 million for the third quarter of 2020, and $7.5 million for the first nine months of 2021 compared with $4.6 million for the first nine months of 2020.
    • Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $24.8 million for the third quarter of 2021 compared with $9.5 million for the third quarter of 2020, and $68.2 million for the first nine months of 2021 compared with $24.0 million for the first nine months of 2020. The increases were primarily driven by the advancement of AOC 1001, AOC 1044 and the AOC FSHD program, as well as costs related to the expansion of the company's overall research capabilities.
    • General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $6.6 million for the third quarter of 2021 compared with $3.8 million for the third quarter of 2020, and $18.8 million for the first nine months of 2021 compared with $8.6 million for the first nine months of 2020. The increases were primarily due to higher personnel costs. The year-to-date increase was also due to higher professional fees and insurance costs.

    About Avidity Biosciences

    Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCsTM). Avidity's proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity's lead product candidate, AOC 1001, is designed to treat patients with myotonic dystrophy type 1 (DM1). AOC 1001 has commenced clinical testing with the ongoing Phase 1/2 MARINATM trial in adults with DM1. Its advancing and expanding pipeline also includes programs in facioscapulohumeral muscular dystrophy (FSHD), Duchenne Muscular Dystrophy (DMD), muscle atrophy and Pompe disease. The company is planning for AOC 1044, the lead of three programs for the treatment of DMD, and its AOC FSHD program to enter the clinic in 2022.  Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution. Avidity is headquartered in La Jolla, CA. For more information about our science, pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and Twitter.

    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 potential to develop a meaningful pipeline of novel AOC therapeutics; the potential of AOC 1001 in patients with DM1 and the expected timing of preliminary data from the MARINA trial; the progression of clinical programs for AOC 1044 and the company's AOC FSHD program and timing of planned clinical trials; the expected benefits associated with Orphan Designation and Fast Track Designation; the sufficiency of the company's current financial position to fund its development programs, investments in its pipeline and platform, and operations into 2024; the potential to apply Avidity's AOC approach to new targets beyond muscle tissues; and the broad potential of AOCs to treat rare and serious 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 the business, including, without limitation: 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 and completion of clinical trials;  disruption to its operations from the COVID-19 pandemic; risks that the benefits associated with Orphan Designation may not be realized, including that Orphan Designation exclusivity may not effectively protect a product from competition and that such exclusivity may not be maintained; the success of its preclinical studies and clinical trials for the company's product candidates; the results of preclinical studies and early clinical trials are not necessarily predictive of future results; Avidity's dependence on third parties in connection with preclinical testing and product manufacturing; unexpected adverse side effects or inadequate efficacy of its product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; Avidity's dependence on third parties for existing collaborations and Avidity may not realize any benefits from such collaborations; regulatory developments in the United States and foreign countries, including acceptance of INDs and similar foreign regulatory filings and the proposed design of future clinical trials; Avidity could use its available capital resources sooner than it currently expects; and other risks described in prior press releases and in filings with the Securities and Exchange Commission (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 exist 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.

    Company Contact:

    Kathleen Gallagher

    (858) 401-7900

            

    Avidity Biosciences, Inc.

    Selected Condensed Financial Information

    (in thousands, except per share data)

    (unaudited)





















    Statements of Operations





    Three Months Ended September 30, 



    Nine Months Ended September 30, 







    2021



    2020



    2021



    2020

    Collaboration revenue





    $            2,163



    $            1,746



    $            7,474



    $            4,645

    Operating expenses:



















           Research and development





    24,831



    9,455



    68,214



    23,983

           General and administrative





    6,612



    3,757



    18,764



    8,646

    Total operating expenses





    31,443



    13,212



    86,978



    32,629

    Loss from operations





    (29,280)



    (11,466)



    (79,504)



    (27,984)

    Other income (expense), net





    6



    27



    33



    (96)

    Net loss





    $         (29,274)



    $         (11,439)



    $         (79,471)



    $         (28,080)

    Net loss per share, basic and diluted





    $            (0.68)



    $            (0.31)



    $            (2.01)



    $            (1.72)

    Weighted-average shares outstanding,

       basic and diluted





    43,265



    37,420



    39,477



    16,361





















    Balance Sheets











     September 30, 



     December 31, 













    2021



    2020

    Assets



















    Current assets:



















           Cash, cash equivalents and marketable securities







    $        413,029



    $        328,141

           Prepaid and other assets













    8,929



    3,537

    Total current assets













    421,958



    331,678

    Property and equipment, net













    3,220



    1,468

    Restricted cash













    251



    251

    Other assets













    488



    501

    Total assets













    $        425,917



    $        333,898

    Liabilities and Stockholders' Equity



















    Current liabilities:



















           Accounts payable and other accrued liabilities







    $          18,157



    $          10,897

           Deferred revenue, current portion













    4,261



    3,690

    Total current liabilities













    22,418



    14,587

    Lease liabilities, net of current portion













    877



    938

    Deferred revenue, net of current portion













    7,745



    12,150

    Total liabilities













    31,040



    27,675

    Stockholders' equity













    394,877



    306,223

    Total liabilities and stockholders' equity













    $        425,917



    $        333,898

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-reports-third-quarter-2021-financial-results-and-recent-highlights-301418018.html

    SOURCE Avidity Biosciences, Inc.

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  2. LA JOLLA, Calif., Nov. 4, 2021 /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 first participants in the Phase 1/2 MARINA trial have been dosed with Avidity's lead AOC product candidate, AOC 1001, marking the first time a person has been dosed with an AOC.

    "This is a first for this new class of drugs and it is a significant milestone for the DM1 community, the Avidity team and the RNA field," said Sarah Boyce, president and CEO of Avidity. "AOCs have the potential to expand the possibilities of how we can treat diseases and our goal is to deliver…

    LA JOLLA, Calif., Nov. 4, 2021 /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 first participants in the Phase 1/2 MARINA trial have been dosed with Avidity's lead AOC product candidate, AOC 1001, marking the first time a person has been dosed with an AOC.

    "This is a first for this new class of drugs and it is a significant milestone for the DM1 community, the Avidity team and the RNA field," said Sarah Boyce, president and CEO of Avidity. "AOCs have the potential to expand the possibilities of how we can treat diseases and our goal is to deliver meaningful drugs to patients as quickly as possible. AOC 1001's MARINA trial will offer a first glimpse of proof-of-concept data for the AOC platform to better inform the development path for DM1 as well as future treatments for other diseases with limited therapeutic options."

    AOCs are designed to combine the proven technology of monoclonal antibodies with the precision and potency of oligonucleotide therapies to access previously untreatable tissue and cell types. AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA). It is designed to address the root cause of DM1 by targeting DMPK, the disease-related mRNA.

    Tanya Stevenson, EdD, MPH, CEO of the Myotonic Dystrophy Foundation in the U.S. stated, "This milestone represents years of research to advance treatment for DM1. Opportunities for the myotonic dystrophy community to participate in research, like the MARINA trial, are critical because the knowledge gained may help lead to earlier diagnosis, improved quality of life and, ultimately, a treatment. The MARINA trial offers much needed advancement in the treatment of DM1 and may also advance therapies for other repeat expansion diseases. We are more hopeful, encouraged and excited than ever before."

    "The AOC platform was developed entirely in-house at Avidity. Our team has created this new technology through years of engineering and following the data to optimize each component of our AOCs," said Art Levin, Ph.D., Avidity's CSO. "This is a turning point for Avidity and the RNA field as AOCs target other cells and tissues that were previously unreachable.  Avidity's AOCs are designed to expand the reach of oligonucleotide therapeutics to treat a broader range of diseases."

    The AOC 1001 Phase 1/2 MARINA trial is enrolling adults with DM1. The first doses in the MARINA trial were administered to patients at Virginia Commonwealth University and University of Rochester Medical Center (NY). For more information on the MARINA trial, including a full list of participating sites, visit www.clinicaltrials.gov.

    About AOC 1001 and the Phase 1/2 MARINATM Trial

    AOC 1001, Avidity's lead product candidate utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. In preclinical studies, AOC 1001 successfully delivered siRNAs to muscle cells, resulting in durable, dose-dependent reductions of DMPK RNA across a broad range of muscles including skeletal, cardiac, and smooth muscles. AOC 1001 has commenced clinical testing with the ongoing Phase 1/2 MARINATM trial in adults with DM1. The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted Orphan Designation for AOC 1001 and the FDA has granted AOC 1001 Fast Track Designation.

    The MARINA trial is a randomized, double-blind, placebo-controlled, Phase 1/2 clinical trial expected to enroll approximately 44 adults with DM1. The primary objective of this study is to evaluate the safety and tolerability of single and multiple ascending doses of AOC 1001 administered intravenously. The MARINA trial will begin to assess the activity of AOC 1001 across key biomarkers, including spliceopathy, an important biomarker for DM1, and knockdown of DMPK mRNA. Though the Phase 1/2 trial is not powered to assess functional benefit, it will explore the clinical activity of AOC 1001 including measures of mobility and muscle strength as well as patient reported outcomes and quality of life measures. Patients will have the option to enroll in an open label extension study at the end of the post-treatment period. In the second half of 2022, Avidity plans to conduct a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the study participants. 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 an underrecognized, progressive and often fatal disease 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 and may cause premature mortality. DM1 primarily affects skeletal and cardiac muscle, however patients can suffer from a constellation of manifestations including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment. Currently, there are no treatments for patients living with DM1.

    About Avidity Biosciences

    Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCsTM). Avidity's proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity's lead product candidate, AOC 1001, is designed to treat patients with myotonic dystrophy type 1 (DM1). AOC 1001 has commenced clinical testing with the ongoing Phase 1/2 MARINATM trial in adults with DM1. It's advancing and expanding pipeline also includes programs in facioscapulohumeral muscular dystrophy (FSHD), Duchenne Muscular Dystrophy (DMD), muscle atrophy and Pompe disease. The company is planning for AOC 1044, the lead of three programs for the treatment of DMD, and its AOC FSHD program to enter the clinic in 2022.  Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution. Avidity is headquartered in La Jolla, CA. For more information about our science, pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and Twitter.

    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 timing of commencing clinical trials and generating clinical trial data; the potential for the MARINATM study to inform the development path for DM1 as well as future treatments for other diseases; the potential of AOC 1001 to treat patients with DM1; and the broad potential of AOCs to treat rare and serious 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 the business, including, without limitation: 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 and completion of clinical trials;  disruption to its operations from the COVID-19 pandemic; the success of its preclinical studies and clinical trials for the company's product candidates; the results of preclinical studies and early clinical trials are not necessarily predictive of future results; Avidity's dependence on third parties in connection with preclinical testing and product manufacturing; unexpected adverse side effects or inadequate efficacy of its product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; and other risks described in prior press releases and in filings with the Securities and Exchange Commission (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 exist 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.

    Company & Investor Contact:

    Kath Gallagher



    858-401-7900 ext 550

    Media Contact:

    Cherise Adkins

     

    (301) 267-4161

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-announces-first-person-dosed-with-an-antibody-oligonucleotide-conjugate-aoc-301415970.html

    SOURCE Avidity Biosciences, Inc.

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  3. LA JOLLA, Calif., Nov. 2, 2021 /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 Avidity management team will be participating at the following conferences:

    Credit Suisse 30th Annual Healthcare Conference

    November 10th, 2021

    Fireside Chat @ 2:40pm ET

    4th Annual Evercore ISI HealthCONx Conference

    November 30th, 2021

    Fireside Chat @ 10:05am ET

    Live webcasts of each event, as well as an archived replay of the webcasts following each event, will be available on the "Events and Presentations" page in the "Investors" section of Avidity's website at https://aviditybiosciences.investorroom.com/events-and-presentations.

    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 (AOCsTM). Avidity's proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1 (DM1). The FDA has cleared Avidity to proceed with the Phase 1/2 MARINATM trial of AOC 1001 in adults with DM1. Its advancing and expanding pipeline also includes programs in facioscapulohumeral muscular dystrophy (FSHD), Duchenne Muscular Dystrophy (DMD), muscle atrophy and Pompe disease. The company is planning for AOC 1044, the lead of three programs for the treatment of DMD, and its AOC FSHD program to enter the clinic in 2022.  Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution. Avidity is headquartered in La Jolla, CA. For more information about our science, pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and Twitter.

    Company Contact:

    Kath Gallagher



    (858) 401-7900

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-to-participate-in-upcoming-investor-conferences-301413250.html

    SOURCE Avidity Biosciences, Inc.

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  4. LA JOLLA, Calif., Oct. 18, 2021 /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 U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its lead program, AOC 1001, for the treatment of myotonic dystrophy type 1 (DM1).

    Fast Track Designation enables more frequent interactions with the FDA to expedite the development and review process for drugs intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

    "DM1 is an underrecognized, progressive and often fatal disease with no therapeutic options. Fast Track designation for AOC 1001 underscores this unmet need and allows us to expeditiously work with FDA to potentially deliver this first-in-class therapy to people living with DM1 as quickly as possible," said Sarah Boyce, president and CEO of Avidity.

    About AOC 1001 and the Phase 1/2 MARINA Trial

    AOC 1001, Avidity's lead program utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. In preclinical studies, AOC 1001 successfully delivered siRNAs to muscle cells, resulting in durable, dose-dependent reductions of DMPK RNA across a broad range of muscles including skeletal, cardiac, and smooth muscles. In preclinical studies, AOC 1001 had a favorable safety profile that supports advancement into the clinic. The FDA has cleared Avidity to proceed with the Phase 1/2 MARINATM study of AOC 1001 in adults with DM1. FDA and EMA have granted Orphan Designation for AOC 1001 and the FDA has granted AOC 1001 Fast Track Designation.

    The MARINA trial is a randomized, double-blind, placebo-controlled, Phase 1/2 clinical trial expected to enroll approximately 44 adults with DM1. The primary objective of this study is to evaluate the safety and tolerability of single and multiple ascending doses of AOC 1001 administered intravenously. The MARINA trial will assess the activity of AOC 1001 across key biomarkers, including spliceopathy, a key biomarker for DM1, and knockdown of DMPK mRNA. Though the Phase 1/2 trial is not powered to assess functional benefit, it will explore the clinical activity of AOC 1001 including measures of mobility and muscle strength as well as patient reported outcomes and quality of life measures. Patients will have the option to enroll in an open label extension study at the end of the post-treatment period. In the second half of 2022, Avidity plans to conduct a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the study participants. 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 an underrecognized, progressive and often fatal disease 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 and may cause premature mortality. DM1 primarily affects skeletal and cardiac muscle, however patients can suffer from a constellation of manifestations including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment. Currently, there are no treatments for patients living with DM1.

    About Avidity Biosciences

    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's proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1 (DM1). The FDA has cleared Avidity to proceed with the Phase 1/2 MARINA™ trial of AOC 1001 in adults with DM1. Its advancing and expanding pipeline also includes programs in facioscapulohumeral muscular dystrophy (FSHD), Duchenne Muscular Dystrophy (DMD), muscle atrophy and Pompe disease. The company is planning for AOC 1044, the lead of three programs for the treatment of DMD, and its AOC FSHD program to enter the clinic in 2022.  Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution. Avidity is headquartered in La Jolla, CA. For more information about our science, pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and Twitter.

    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 our current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the potential of AOC 1001 in people with myotonic dystrophy type 1 and the initiation of a clinical trial, and the timing thereof. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of our plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in our business, including, without limitation: we are early in our development efforts and many of our development programs are in the preclinical or discovery stage; our approach to the discovery and development of product candidates based on our AOC platform is unproven, and we do not know whether we will be able to develop any products of commercial value; potential delays in the commencement, enrollment and completion of clinical trials;  disruption to our operations from the COVID-19 pandemic; the success of our preclinical studies and clinical trials for our product candidates; the results of preclinical studies and early clinical trials are not necessarily predictive of future results; our dependence on third parties in connection with preclinical testing and product manufacturing; unexpected adverse side effects or inadequate efficacy of our product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; regulatory developments in the United States and foreign countries, including acceptance of INDs and similar foreign regulatory filings and our proposed design of future clinical trials; risks related to integration of new  personnel; and other risks described in our prior press releases and in our filings with the Securities and Exchange Commission (SEC). Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur or circumstances that exist 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.

    Contacts:

    Company:

    Kathleen Gallagher

    (858) 401-7900

    Media:

    Cherise Adkins

    (301) 267-4161

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/fda-grants-fast-track-designation-to-aoc-1001-for-the-treatment-of-myotonic-dystrophy-type-1-301401621.html

    SOURCE Avidity Biosciences, Inc.

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  5. LA JOLLA, Calif., Aug. 31, 2021 /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 Avidity management team will be participating at the following conferences:

    2021 Wells Fargo Virtual Healthcare Conference

    September 9th, 2021

    11:20am ET – Fireside Chat

    Chardan Virtual 5th Annual Genetic Medicines Conference

    October 5th, 2021 

    12noon ET – Panel titled "RNA-based Targeted Delivery Approaches

    1:30pm ET – Fireside Chat

    Live webcasts of each event, as well as an archived replay of the webcasts following each event, will be available on the "Events and Presentations" page in the "Investors" section of Avidity's website at https://aviditybiosciences.investorroom.com/events-and-presentations.

    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 (AOCsTM). Avidity's proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1 (DM1). The FDA has cleared Avidity to proceed with the Phase 1/2 MARINATM trial of AOC 1001 in adults with DM1. Its advancing and expanding pipeline also includes programs in facioscapulohumeral muscular dystrophy (FSHD), Duchenne Muscular Dystrophy (DMD), muscle atrophy and Pompe disease. The company is planning for AOC 1044, the lead of three programs for the treatment of DMD, and its AOC FSHD program to enter the clinic in 2022.  Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution. Avidity is headquartered in La Jolla, CA. For more information about our science, pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and Twitter.

    Company & Investors Contact:

    Kath Gallagher



    (858) 401-7900

    Media Contact:

    Cherise Adkins



    (301) 267-4161

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-to-participate-in-upcoming-investor-conferences-301364495.html

    SOURCE Avidity Biosciences, Inc.

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