RNA Avidity Biosciences Inc.

29.2
-1.68  -5%
Previous Close 30.88
Open 31.22
52 Week Low 23.06
52 Week High 37.46
Market Cap $1,095,777,625
Shares 37,526,631
Float 32,445,460
Enterprise Value $772,586,875
Volume 121,763
Av. Daily Volume 202,029
Stock charts supplied by TradingView

Latest News

  1. LA JOLLA, Calif., Nov. 10, 2020 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs™), today reported financial results for the quarter ended September 30, 2020 and highlighted recent corporate progress.  

    "During the third quarter, we continued to invest in all aspects of our business and advanced our muscle-disease programs toward the clinic," said Sarah Boyce, President and Chief Executive Officer. "In the near term, we are focused on preparing for the initiation of our first clinical study with AOC 1001 in adults with myotonic dystrophy type 1. We are also advancing our research efforts in new tissues and cell types beyond muscle that we believe we can effectively target with AOCs to create new therapies designed to improve the lives of patients with serious diseases."

    "As we prepare for clinical development with AOC 1001, we maintain a strong financial position with $341 million in cash at quarter-end," said Mike MacLean, Chief Financial Officer. "With our current financial resources, we are well-positioned to meet our goals and advance multiple AOC programs into the clinic in the next two years."

    Third Quarter 2020 and Recent Corporate Highlights

    • Presented at Key Scientific Meetings. Avidity presented at several key virtual scientific meetings, including TIDES USA 2020, the 16th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) 2020, the Next Generation Protein Therapeutics and Bioconjugates Summit 2020, and TIDES Europe 2020. In early December 2020, the company plans to present an overview of its AOC technology and preclinical data at the 3rd Annual Neuromuscular Drug Development Summit.
    • Entered into Collaboration to Study the Natural History of DM1 to Support Lead Program AOC 1001. Avidity entered into a collaboration supporting END-DM1 (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1), a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1 (DM1). This new collaboration supports Avidity's lead program, AOC 1001, in development for the treatment of DM1.  END-DM1 is a non-interventional study designed and run by the Myotonic Dystrophy Clinical Research Network (DMCRN), a network of medical centers. DMCRNaims to support future clinical trials of potential therapies for DM1 through the generation of evidence around endpoint measures and testing methods. 
    • Appointed Key New Hires to Leadership Positions. Avidity welcomed three new team members into leadership positions: Teresa McCarthy, Chief Human Resources Officer, Kelly DiTrapani, Vice President of Medical Affairs, and Monica Zepeda, Vice President of Program and Alliance Management. Avidity strives to achieve a strong diversity climate and celebrates its newest female executives.    

    Financial Results

    • Cash and Cash Equivalents:  Cash and cash equivalents totaled $341.1 million as of September 30, 2020, which includes net proceeds of $274.1 million from the company's IPO in June 2020, compared to cash and cash equivalents of $94.6 million as of December 31, 2019.
    • Collaboration Revenue: Collaboration revenue, including reimbursable expenses, was $1.7 million for the third quarter of 2020 compared with $0.7 million for the third quarter of 2019, and $4.6 million for the first nine months of 2020 compared with $0.9 million for the first nine months of 2019.
    • Research and Development (R&D) Expenses: R&D expenses, including external and internal costs associated with research activities, primarily relate to the progression of the company's research on AOC 1001 and other programs. These expenses were $9.5 million for the third quarter of 2020 compared with $5.1 million for the third quarter of 2019, and $24.0 million for the first nine months of 2020 compared with $8.9 million for the first nine months of 2019. The increases were primarily driven by the advancement of AOC 1001, as well as other programs.
    • 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 $3.8 million for the third quarter of 2020 compared with $0.8 million for the third quarter of 2019, and $8.6 million for the first nine months of 2020 compared with $3.3 million for the first nine months of 2019. The increases were primarily due to higher personnel costs (including noncash stock-based compensation), professional fees and insurance costs related to being a public company, as well as higher patent filing fees.

    About Avidity Biosciences

    Avidity Biosciences, Inc. is pioneering a new class of oligonucleotide-based therapies called AOCs designed to overcome the current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity utilizes its proprietary AOC platform to design, engineer and develop therapeutics that combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies in order to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, and its four other muscle programs are focused on the treatment of muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and Pompe disease. In addition to its muscle franchise, Avidity has research efforts focused on immune and other cell types.

    Avidity is headquartered in La Jolla, CA. For more information about Avidity's science, pipeline and people, please visit www.aviditybiosciences.com and engage with Avidity on LinkedIn.  

    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 to develop a meaningful pipeline of novel AOC therapeutics; the initiation of a clinical trial of AOC 1001 in patients with myotonic dystrophy type 1 and other planned clinical trials, and the timing thereof; the potential to identify new targets beyond the muscle that can be targeted with Avidity's AOC approach; and the broad potential of AOCs to treat serious diseases. 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 all 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 management 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:

    Mike MacLean

    (858) 401-7900

              

    Media and Investors:

    Amy Conrad

    Juniper Point

    (858) 366-3243

     

    Avidity Biosciences, Inc.

    Selected Condensed Financial Information

    (In thousands, except per share data)

    (Unaudited)





















    Statements of Operations





    Three Months Ended



    Nine Months Ended







    September 30,



    September 30,







    2020



    2019



    2020



    2019

    Collaboration revenue





    $        1,746



    $          650



    $        4,645



    $            874

    Operating expenses:



















           Research and development





    9,455



    5,099



    23,983



    8,894

           General and administrative





    3,757



    757



    8,646



    3,265

    Total operating expenses





    13,212



    5,856



    32,629



    12,159

    Loss from operations





    (11,466)



    (5,206)



    (27,984)



    (11,285)

    Other income (expense), net





    27



    (1,462)



    (96)



    (3,048)

    Net loss





    $      (11,439)



    $        (6,668)



    $      (28,080)



    $       (14,333)

    Net loss per share, basic and diluted





    $          (0.31)



    $          (2.43)



    $          (1.72)



    $          (5.32)

    Weighted-average shares outstanding,

       basic and diluted





    37,420



    2,739



    16,361



    2,692





















    Balance Sheets











     September 30, 



     December 31, 













    2020



    2019

    Assets

















    Current assets:

















           Cash and cash equivalents











    $      341,142



    $        94,578

           Prepaid and other assets











    3,155



    1,098

    Total current assets











    344,297



    95,676

    Property and equipment, net











    1,295



    631

    Restricted cash













    124



    Other assets











    417



    600

    Total assets











    $      346,133



    $        96,907

    Liabilities, Convertible Preferred Stock and Stockholders' Equity (Deficit) 

    Current liabilities:



















           Accounts payable and other accrued liabilities











    $          8,460



    $          3,622

           Deferred revenue, current portion











    5,220



    3,840

           Long-term debt, current portion















    2,774

    Total current liabilities













    13,680



    10,236

    Lease liabilities, net of current portion













    689



    393

    Deferred revenue, net of current portion













    11,600



    15,100

    Long-term debt, net of current portion















    1,770

    Other long-term liabilities















    45

    Total liabilities













    25,969



    27,544

    Convertible preferred stock















    134,720

    Stockholders' equity (deficit)











    320,164



    (65,357)

    Total liabilities, convertible preferred stock and

       stockholders' equity (deficit)













    $      346,133



    $        96,907

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/avidity-biosciences-reports-third-quarter-2020-financial-results-and-recent-highlights-301168213.html

    SOURCE Avidity Biosciences, Inc.

    View Full Article Hide Full Article
  2. LA JOLLA, Calif., Nov. 5, 2020 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs™), today announced that Sarah Boyce, President and Chief Executive Officer, will be presenting at the Credit Suisse 29th Annual Virtual Healthcare Conference 2020 on Thursday, November 12th, 2020 at 8:45am PST. The conference is being held in a virtual format.

    A live webcast of the virtual fireside chat will be available on the Company's website at www.aviditybiosciences.com in the Investor Resources section. A replay of the fireside presentation will be archived on the site for one year.

    About Avidity Biosciences

    Avidity Biosciences, Inc. is pioneering a new class of oligonucleotide-based therapies called AOCs designed to overcome the current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity utilizes its proprietary AOC platform to design, engineer and develop therapeutics that combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies in order to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. Avidity's lead

    product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, and its four other muscle programs are focused on the treatment of muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and Pompe disease. In addition to its muscle franchise, Avidity has research efforts focused on immune and other cell types.

    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.

    Contacts:

    Company:

    Mike MacLean

    (858) 401-7900

    Media and Investors:

    Amy Conrad

    Juniper Point

    (858) 366-3243

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/avidity-biosciences-to-present-at-the-credit-suisse-29th-annual-virtual-healthcare-conference-2020-301167367.html

    SOURCE Avidity Biosciences, Inc.

    View Full Article Hide Full Article
  3. LA JOLLA, Calif., Sept. 28, 2020 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs), today announced that Sarah Boyce, President and Chief Executive Officer, Arthur Levin, Ph.D., Chief Scientific Officer, and Mike MacLean, Chief Financial Officer, will participate in a fireside chat at the Chardan 4th Annual Genetic Medicines Conference 2020 on Monday, October 5th, 2020 at 12:45 pm PDT. In addition, Dr. Levin will participate in a featured panel entitled "Small RNA Therapies: Upcoming Advancements in Delivery and Stabilization Chemistries" on Tuesday, October 6th, 2020 at 12:00 pm PDT. The conference is…

    LA JOLLA, Calif., Sept. 28, 2020 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs), today announced that Sarah Boyce, President and Chief Executive Officer, Arthur Levin, Ph.D., Chief Scientific Officer, and Mike MacLean, Chief Financial Officer, will participate in a fireside chat at the Chardan 4th Annual Genetic Medicines Conference 2020 on Monday, October 5th, 2020 at 12:45 pm PDT. In addition, Dr. Levin will participate in a featured panel entitled "Small RNA Therapies: Upcoming Advancements in Delivery and Stabilization Chemistries" on Tuesday, October 6th, 2020 at 12:00 pm PDT. The conference is being held in a virtual format.

    A live webcast of the virtual fireside chat will be available on the Company's website at www.aviditybiosciences.com in the Investor Resources section. A replay of the fireside chat will be archived on the site for 90 days.

    About Avidity Biosciences

    Avidity Biosciences, Inc. is pioneering a new class of oligonucleotide-based therapies called AOCs designed to overcome the current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity utilizes its proprietary AOC platform to design, engineer and develop therapeutics that combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies in order to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, and its four other muscle programs are focused on the treatment of muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and Pompe disease. In addition to its muscle franchise, Avidity has research efforts focused on immune and other cell types.

    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.

    Contacts:

    Company:

    Mike MacLean

    (858) 401-7900

         

    Media and Investors:

    Amy Conrad

    Juniper Point

    (858) 366-3243

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/avidity-biosciences-to-present-at-the-virtual-chardan-4th-annual-genetic-medicines-conference-2020-301139080.html

    SOURCE Avidity Biosciences, Inc.

    View Full Article Hide Full Article
  4. SAN DIEGO, Sept. 14, 2020 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the company will present at several upcoming virtual scientific meetings.

    (PRNewsfoto/Avidity Biosciences, Inc.)

    TIDES USA 2020 (Sept. 15th - 18th)
    Session Chair
    Date: Wednesday, September 16th, 3:45 PM – 4:30 PM, ET

    16th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) 2020 (Sept. 27th - 30th)
    Title: Recent Progress with Antibody Oligonucleotide Conjugates    
    Date: Monday, September 28th, 12:45 PM – 1:10 PM, ET

    Next Generation Protein Therapeutics & Bioconjugates Summit 2020 (Nov. 2nd - 5th)
    Title: Oligonucleotide Therapeutics…

    SAN DIEGO, Sept. 14, 2020 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the company will present at several upcoming virtual scientific meetings.

    (PRNewsfoto/Avidity Biosciences, Inc.)

    TIDES USA 2020 (Sept. 15th - 18th)

    Session Chair

    Date: Wednesday, September 16th, 3:45 PM – 4:30 PM, ET

    16th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) 2020 (Sept. 27th - 30th)

    Title: Recent Progress with Antibody Oligonucleotide Conjugates    

    Date: Monday, September 28th, 12:45 PM – 1:10 PM, ET

    Next Generation Protein Therapeutics & Bioconjugates Summit 2020 (Nov. 2nd - 5th)

    Title: Oligonucleotide Therapeutics: Now on Target with Antibody Conjugates

    Date: Thursday, November 5th, 9:40 AM – 10:00 AM, ET

    TIDES Europe: Oligonucleotide and Peptide Therapeutics (Nov. 11th - 13th)

    Keynote Address: Recent Advances with Antibody-siRNA Conjugates

    Date: Friday, November 13th, 2:45PM3:30 PM, CET

    3rd Annual Neuromuscular Drug Development Summit (Dec. 1st - 3rd) 

    Title: Pioneering a New Class of Oligonucleotide Therapies: Developing an Oligonucleotide Antibody Conjugate for the Treatment of DM1

    Date: Thursday, December 3rd, 10:10 AM – 10:30 AM, ET

    "Presenting at five premier RNA-focused and neuromuscular conferences is an important opportunity to highlight the continued advancement of our novel AOC platform," said Art Levin, Chief Scientific Officer. "We are progressing this research to the clinic as we expect AOC 1001, our first clinical candidate, to enter a Phase 1/2 clinical trial next year in adults with myotonic dystrophy type 1, a serious genetic disease with no available treatment."

    About Avidity Biosciences

    Avidity Biosciences, Inc. is pioneering a new class of oligonucleotide-based therapies called AOCs designed to overcome the current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity utilizes its proprietary AOC platform to design, engineer and develop therapeutics that combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies in order to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, and its four other muscle programs are focused on the treatment of muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and Pompe disease. In addition to its muscle franchise, Avidity has research efforts focused on immune and other cell types. 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.  

    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 to develop a meaningful pipeline of novel AOC therapeutics; the selection of clinical candidates; and the initiation of first-in-human studies with a planned Phase 1/2 trial in adults with myotonic dystrophy type 1. 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 all 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; our dependence on third parties in connection with preclinical testing and product manufacturing; unexpected adverse side effects, off target 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 management 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:

    Mike MacLean

    (858) 401-7900

              

    Media and Investors:

    Amy Conrad

    Juniper Point

    (858) 366-3243

    Cision View original content:http://www.prnewswire.com/news-releases/avidity-biosciences-to-present-at-several-upcoming-virtual-scientific-meetings-301130385.html

    SOURCE Avidity Biosciences, Inc.

    View Full Article Hide Full Article
  5. SAN DIEGO, Sept. 8, 2020 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs™), today announced it entered into a collaboration supporting END-DM1, a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1 (DM1). END-DM1 (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1) is a non-interventional study designed and run by the Myotonic Dystrophy Clinical Research Network (DMCRN), a network of medical centers that aims to support future clinical trials of potential therapies for DM1 through the generation of evidence around…

    SAN DIEGO, Sept. 8, 2020 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs™), today announced it entered into a collaboration supporting END-DM1, a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1 (DM1). END-DM1 (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1) is a non-interventional study designed and run by the Myotonic Dystrophy Clinical Research Network (DMCRN), a network of medical centers that aims to support future clinical trials of potential therapies for DM1 through the generation of evidence around endpoint measures and testing methods.

    "By supporting END-DM1, we strengthen our commitment to patients and physicians to advance the knowledge of the natural progression, burden and premature mortality of this severe genetic disease," said Jae Kim, M.D., Chief Medical Officer of Avidity. "The findings from END-DM1 will help inform the clinical development of our lead program, AOC 1001, and we look forward to initiating a Phase 1/2 clinical trial in 2021 in adults with myotonic dystrophy type 1."

    END-DM1 is designed to evaluate the full spectrum of disease severity and serve as the foundation for therapeutic development in DM1. END-DM1 plans to enroll approximately 650 people with DM1 aged 18-70 across nine DMCRN study sites in the U.S. and five sites in Europe. In addition to Avidity, supporters of END-DM1 include the U.S. Food & Drug Administration (FDA), the Myotonic Dystrophy Foundation, the Wyck Foundation, the Muscular Dystrophy Association, and other biotechnology companies.

    "We are thrilled to have robust support for END-DM1 from leading industry sponsors, such as Avidity," said Nicholas Johnson, M.D., a co-principal investigator for END-DM1 at VCU Health in Richmond, Virginia. "This partnership allows for us to share the collective learnings about the disease and level of unmet need for patients living with DM1 to optimize therapeutic strategies to further accelerate the development of promising new therapeutics, like AOC 1001."

    For more information about END-DM1, including study requirements and a list of participating sites, please visit www.myotonic.org.        

    About Myotonic Dystrophy Type 1 and AOC 1001

    Myotonic dystrophy type 1 (DM1) is an underrecognized, progressive and often fatal disease caused by a triplet-repeat on the DMPK gene, resulting in a toxic gain of function. 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 (GI) complications, and cognitive and behavioral impairment. Currently, there are no disease-modifying treatments for patients living with DM1.

    AOC 1001, Avidity's lead program utilizing its AOC platform, is potentially the first disease-modifying treatment for DM1. AOC 1001 is designed to address the underlying cause of DM1 by reducing levels of mutant DMPK mRNA. AOC 1001 consists of a proprietary mAb that binds to transferrin receptor 1 (TfR1) conjugated with a siRNA. In preclinical studies, AOC 1001 successfully delivered siRNAs to muscle cells, resulting in a durable, dose-dependent reductions of DMPK RNA across a broad range of muscles including skeletal, cardiac, and smooth muscles. Avidity expects to submit an investigational new drug application for AOC 1001 in 2021 and plans to initiate a Phase 1/2 clinical trial in DM1 patients by the end of 2021.

    For more information about AOC 1001, please visit www.aviditybiosciences.com/programs.

    About Avidity Biosciences

    Avidity Biosciences, Inc. is pioneering a new class of oligonucleotide-based therapies called AOCs designed to overcome the current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity utilizes its proprietary AOC platform to design, engineer and develop therapeutics that combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies in order to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, and its four other muscle programs are focused on the treatment of muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and Pompe disease. In addition to its muscle franchise, Avidity has research efforts focused on immune and other cell types.  

    About VCU and VCU Health

    Virginia Commonwealth University is a major, urban public research university with national and international rankings in sponsored research. Located in downtown Richmond, VCU enrolls more than 30,000 students in 233 degree and certificate programs in the arts, sciences and humanities. Twenty-two of the programs are unique in Virginia, many of them crossing the disciplines of VCU's 11 schools and three colleges. The VCU Health brand represents the VCU health sciences academic programs, the VCU Massey Cancer Center and the VCU Health System, which comprises VCU Medical Center (the only academic medical center in the region), Community Memorial Hospital, Children's Hospital of Richmond at VCU, MCV Physicians and Virginia Premier Health Plan. For more, please visit www.vcu.edu and vcuhealth.org.

    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 planned enrollment of END-DM1 and the study's ability to inform clinical development plans for AOC 1001; the potential to develop a meaningful pipeline of novel AOC therapeutics; the selection of clinical candidates; the initiation of first-in-human studies; the anticipated timing of filing an IND for the treatment of DM1; and the broad potential of AOCs to treat serious diseases. 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 all 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 management 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:

    Mike MacLean

    (858) 401-7900

              

    Media and Investors:

    Amy Conrad

    Juniper Point

    (858) 366-3243

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/avidity-biosciences-enters-into-collaboration-to-study-the-natural-history-of-myotonic-dystrophy-type-1-301124715.html

    SOURCE Avidity Biosciences, Inc.

    View Full Article Hide Full Article
View All Avidity Biosciences Inc. News