RNA Avidity Biosciences Inc.

20.92
+0.5  (+2%)
Previous Close 20.42
Open 20.45
52 Week Low 19.12
52 Week High 37.46
Market Cap $786,911,260
Shares 37,615,261
Float 32,309,848
Enterprise Value $461,140,629
Volume 78,672
Av. Daily Volume 349,536
Stock charts supplied by TradingView

Drug Pipeline

Drug Stage Notes
AOC 1001
Myotonic dystrophy type 1 (DM1)
Phase 1/2
Phase 1/2
Phase 1/2 trial to commence 2H 2021.

Latest News

  1. LA JOLLA, Calif., May 12, 2021 /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 first quarter ended March 31, 2021 and highlighted recent corporate progress.       

    "AOC 1001, the first program from our AOC platform, is advancing toward the clinic and is on track to begin a Phase 1/2 study in adults with DM1 in the second half of this year. Our skeletal muscle pipeline is also progressing as planned while we continue to expand our discovery efforts to other indications," said Sarah Boyce, president and chief executive officer. "We are proud to be at the forefront of RNA innovation by delivering on the promise of our AOC platform and pipeline."

    Mike MacLean, chief financial officer, added, "We are off to a great start this year with a strong financial position of over $300 million at the end of the first quarter. This allows us to progress AOC 1001 while continuing to invest in our other pipeline programs and our AOC platform, which we believe is a sustainable engine with the potential to have a broad impact across both rare and other serious diseases."

    AOC Platform and Pipeline Highlights

    • Advanced Lead Program, AOC 1001 for DM1, and Broad Pipeline for Untreated Rare Muscle Diseases. 



      In April 2021, Avidity presented preclinical data for AOC 1001 for myotonic dystrophy type 1 (DM1) in an oral presentation as part of the Emerging Science Session at the American Academy of Neurology (AAN) 2021 Virtual Annual Meeting. Avidity scientists have demonstrated activity and potency of siRNAs against the dystrophy protein kinase (DMPK) gene in muscle cells derived from patients with DM1. These data demonstrated activity in the nucleus and cytoplasm. The presentation also showed that single doses of AOC 1001 produced 75% reductions in DMPK mRNA expression in preclinical studies that were maintained for months post-dosing. Following regulatory clearance, Avidity remains on track with plans to begin a Phase 1/2 clinical study of AOC 1001 in adults with DM1 in the second half of 2021. 



      Avidity also advanced additional programs in its muscle franchise including a program for facioscapulohumeral muscular dystrophy (FSHD) and three programs for Duchenne muscular dystrophy (DMD). The AOC FSHD program and the lead AOC DMD program targeting Exon 44 are the most advanced. Avidity remains on track with plans to commence clinical trials for both programs in 2022, following additional preparatory preclinical studies and regulatory clearance.

    Upcoming Events

    • Avidity's Virtual Investor & Analyst Event Series – Volume 1.  Recently, Avidity launched its Virtual Investor and Analyst Event Series.  The first virtual event entitled "Engineering Antibody Oligonucleotide Conjugates" will be held on Wednesday, May 19th, 2021 at 8am PT/11am ET



      The virtual event will feature a panel discussion with leading RNA therapeutics key opinion leaders Phillip D. Zamore, Ph.D., Chair, RNA Therapeutics Institute at UMASS and Steven F. Dowdy, Ph.D., Professor of Cellular and Molecular Medicine at UCSD.  To register for the event or for more information, please email .         

    First Quarter 2021 Financial Results

    • Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $307.9 million as of March 31, 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 $2.7 million for the first quarter of 2021 compared with $1.4 million for the first quarter of 2020.
    • 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 muscle programs. These expenses were $20.7 million for the first quarter of 2021 compared with $5.5 million for the first quarter of 2020. The increase was primarily driven by the progression of AOC 1001 toward the clinic, as well as research for 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 $5.9 million for the first quarter of 2021 compared with $2.0 million for the first quarter of 2020. The increase was primarily due to higher personnel costs (including noncash stock-based compensation), professional fees and insurance costs related to being a public company.

    About Avidity Biosciences

    Avidity Biosciences, Inc. is driven to change lives with a new class of therapies called Antibody Oligonucleotide Conjugates (AOCs) that are designed to overcome current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity's proprietary AOC platform combines the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies 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 other muscle programs are focused on the treatment of Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, Pompe disease and muscle atrophy. In addition to its muscle franchise, Avidity has research efforts focused on immune cells, cardiac tissue 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 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 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 skeletal muscle that can be targeted with Avidity's AOC approach; 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 and all of its development programs are in the preclinical or discovery stage; 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; 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; risks related to integration of new management personnel; 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.

    Contacts:

    Company:

    Kathleen Gallagher

    (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 March 31,









    2021





    2020



    Collaboration revenue





    $

    2,704





    $

    1,358



    Operating expenses:



















           Research and development







    20,677







    5,544



           General and administrative







    5,884







    1,964



    Total operating expenses







    26,561







    7,508



    Loss from operations







    (23,857)







    (6,150)



    Other income (expense), net







    13







    65



    Net loss





    $

    (23,844)





    $

    (6,085)



    Net loss per share, basic and diluted





    $

    (0.64)





    $

    (2.14)



    Weighted-average shares outstanding, basic and diluted







    37,521







    2,845























    Balance Sheets



    March 31,





    December 31,







    2021





    2020



    Assets

















    Current assets:

















           Cash, cash equivalents and marketable securities



    $

    307,914





    $

    328,141



           Prepaid and other assets





    4,036







    3,537



    Total current assets





    311,950







    331,678



    Property and equipment, net





    2,187







    1,468



    Restricted cash







    251







    251



    Other assets





    364







    501



    Total assets



    $

    314,752





    $

    333,898



    Liabilities and Stockholders' Equity



















    Current liabilities:



















           Accounts payable and other accrued liabilities



    $

    13,157





    $

    10,897



           Deferred revenue, current portion





    4,357







    3,690



    Total current liabilities







    17,514







    14,587



    Lease liabilities, net of current portion







    951







    938



    Deferred revenue, net of current portion







    10,171







    12,150



    Total liabilities







    28,636







    27,675



    Stockholders' equity





    286,116







    306,223



    Total liabilities and stockholders' equity





    $

    314,752





    $

    333,898



     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/avidity-biosciences-reports-first-quarter-2021-financial-results-and-recent-highlights-301289275.html

    SOURCE Avidity Biosciences, Inc.

    View Full Article Hide Full Article
  2. LA JOLLA, Calif., May 3, 2021 /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 it will host a virtual investor and analyst event focused on the company's AOC platform on Wednesday, May 19, 2021, from 8:00 a.m. to 10:00 a.m. PT/11:00 a.m. to 1:00 p.m. ET.  This event is the first in a series of investor and analyst events that the company plans to host in 2021. 

    The May 19th event will feature a panel discussion with Phillip D. Zamore, Ph.D., Chair of the RNA Therapeutics Institute at UMASS, Steven F. Dowdy, Ph.D., Professor of Cellular and Molecular Medicine at UCSD, and Art Levin, Ph.D., Avidity's Chief Scientific Officer.  The Avidity management team and Drs. Zamore and Dowdy will host a question and answer session following the panel. 

    The event will also feature a presentation from Dr. Levin who will provide an overview of the company's AOC platform and the translation of the platform into a pipeline of programs.

    Background on Drs. Zamore and Dowdy:

    Phillip D. Zamore, Ph.D., the Gretchen Stone Cook Professor of Biomedical Sciences, Professor of Biochemistry and Molecular Pharmacology, and Investigator of the Howard Hughes Medical Institute, is Chair of the RNA Therapeutics Institute, which was established at the University of Massachusetts Medical School in 2009. He has a long history in RNA-targeting therapeutics and has been associated with the leading companies in the field. Dr. Zamore received his A.B. and Ph.D. degrees in Biochemistry and Molecular Biology from Harvard University. He then pursued postdoctoral studies at MIT and the Whitehead Institute for Biomedical Research.

    Steven F. Dowdy, Ph.D., is a professor in the Department of Cellular & Molecular Medicine at the University of California San Diego School of Medicine. His research focuses on the delivery of novel therapeutics, especially RNAi therapies, into cells. He has been involved in multiple RNA biotech startups and currently sits on five Scientific Advisory Boards. Dr. Dowdy received his Ph.D. in Molecular Genetics from the University of California Irvine and performed his postdoctoral fellowship at MIT and the Whitehead Institute for Biomedical Research. Dr. Dowdy was a Howard Hughes Medical Institute Investigator for 18 years.

    Video Webcast Information

    A live video webcast of the 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. A replay of the webcast will be archived on Avidity's website following the event.

    About Avidity Biosciences

    Avidity Biosciences, Inc. is driven to change lives with a new class of therapies called Antibody Oligonucleotide Conjugates (AOCs) that are designed to overcome current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity's proprietary AOC platform combines the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies 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 other muscle programs are focused on the treatment of Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, Pompe disease and muscle atrophy. In addition to its muscle franchise, Avidity has research efforts focused on immune, cardiac 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

    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-host-virtual-investor-and-analyst-event-on-may-19-2021-301282130.html

    SOURCE Avidity Biosciences, Inc.

    View Full Article Hide Full Article
  3. LA JOLLA, Calif., April 16, 2021 /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 preclinical data for their lead candidate, AOC 1001 in development for myotonic dystrophy type 1 (DM1), will be presented at the American Academy of Neurology (AAN) 2021 Virtual Annual Meeting being held April 17-22, 2021. 

    The abstract on AOC 1001 was chosen by the AAN Science Committee as one of 11 abstracts to be highlighted in the Emerging Science Session.

    "We are honored to be part of the Emerging Science session at such a prestigious meeting and are grateful to the AAN Science Committee for highlighting AOC 1001. AOC 1001 is targeting DM1, a complex disease with variability from patient to patient and no approved treatments," said Art Levin, Ph.D., Chief Scientific Officer. "Our data presented at AAN show that AOC 1001 is highly potent with demonstrated activity in the nucleus and cytoplasm and that single doses of AOC 1001 produced 75% reductions in DMPK mRNA expression that were maintained for months post-dosing. These results further solidify AOC 1001 as a promising program with the potential to be the first therapy for people living with DM1."

    Details of the oral presentation are below:

    Title: "Optimization of AOC-1001, an antibody-oligonucleotide conjugate targeting the underlying cause of myotonic dystrophy type 1"

    Date: Sunday, April 18, 2021 at 3:45 p.m. ET

    Session: Emerging Science

    Presenter: Barbora Malecova, Ph.D., Associate Director, Biology at Avidity

    Avidity scientists have demonstrated activity and potency of siRNAs against the dystrophy protein kinase (DMPK) gene in muscle cells derived from patients with DM1. These data showed significant reductions in DMPK mRNA levels in the nucleus and in the cytoplasm. Treatment with Avidity's therapeutic agent were able to correct the incorrect splicing in these DM1 cells, moving the splicing signature closer to that of healthy cells and indicating the potential to target the underlying cause of DM1. In addition, Avidity scientists have shown the robust, durable activity of AOC 1001 in vivo in a broad range of muscles of non-human primates including cardiac muscle.  These data demonstrated a 75% reduction in DMPK mRNA levels in muscles following a single dose of AOC 1001.

    Avidity's abstract is available on the AAN meeting website. Following regulatory clearance, the company plans to begin a Phase 1/2 study for AOC 1001 in the second half of 2021.

    About Avidity

    Avidity Biosciences, Inc. is driven to change lives with a new class of therapies called Antibody Oligonucleotide Conjugates (AOCs) that are designed to overcome current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity's proprietary AOC platform combines the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies 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 other muscle programs are focused on the treatment of Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, Pompe disease and muscle atrophy. In addition to its muscle franchise, Avidity has research efforts focused on immune, cardiac 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 current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: Avidity's evolution to a clinical-stage company; Avidity's plans to conduct clinical trials of AOC 1001 in patients with DM1 and the expected timing thereof; 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: the company is early in development efforts and all of its development programs are currently in the preclinical or discovery stage; the company's approach to the discovery and development of product candidates based on the 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 the company's operations from the COVID-19 pandemic; the success of its preclinical studies and clinical trials for its product candidates; the results of preclinical studies and early clinical trials are not necessarily predictive of future results; its 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; 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; risks related to integration of new management personnel; and other risks described in the company's prior press releases and in its 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.

    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-oral-presentation-on-aoc-1001-for-dm1-at-american-academy-of-neurology-aan-2021-virtual-annual-meeting-301270244.html

    SOURCE Avidity Biosciences, Inc.

    View Full Article Hide Full Article
  4. LA JOLLA, Calif., March 25, 2021 /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 management will be participating in the following virtual investor conferences:

    • Guggenheim Healthcare Talks—Genomic Medicines and Rare Disease Day Conference
      Thursday, April 1st, 2021, 6:00 am PDT
      • Panel on Duchenne Muscular Dystrophy (DMD)
      • Participants include:
        • Arthur Levin, PhD, Chief Scientific Officer, Avidity Biosciences
        • Michael Binks, MD, Vice President of Clinical Research, Pfizer
        • Louise Rodino-Klapac, PhD, Chief Scientific Officer, Sarepta Therapeutics
        • Carl Morris, PhD, Chief Scientific Officer…

    LA JOLLA, Calif., March 25, 2021 /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 management will be participating in the following virtual investor conferences:

    • Guggenheim Healthcare Talks—Genomic Medicines and Rare Disease Day Conference

      Thursday, April 1st, 2021, 6:00 am PDT
      • Panel on Duchenne Muscular Dystrophy (DMD)
      • Participants include:
        • Arthur Levin, PhD, Chief Scientific Officer, Avidity Biosciences
        • Michael Binks, MD, Vice President of Clinical Research, Pfizer
        • Louise Rodino-Klapac, PhD, Chief Scientific Officer, Sarepta Therapeutics
        • Carl Morris, PhD, Chief Scientific Officer, Solid Biosciences
        • Sam Wadsworth, Chief Scientific Officer, Ultragenyx Pharmaceutical
    • 2021 Virtual Wells Fargo Biotech Corporate Access Day

      Tuesday, April 6th, 2021
      • Avidity management participation in investor 1x1 meetings
    • 20th Annual Needham Virtual Healthcare Conference

      Monday, April 12th, 2021, 12:45pm PDT
      • Webcast presentation

    Live webcasts of the Guggenheim panel and Needham presentation will be available on the Investors section of Avidity's website and a replay of both events archived on the site.

    About Avidity

    Avidity Biosciences, Inc. is driven to change lives with a new class of therapies called Antibody Oligonucleotide Conjugates (AOCs) that are designed to overcome current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity's proprietary AOC platform combines the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies 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 other muscle programs are focused on the treatment of Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, Pompe disease and muscle atrophy. In addition to its muscle franchise, Avidity has research efforts focused on immune, cardiac 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

    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-participate-in-upcoming-investor-conferences-301256325.html

    SOURCE Avidity Biosciences, Inc.

    View Full Article Hide Full Article
  5. LA JOLLA, Calif., March 15, 2021 /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 fourth quarter and year ended December 31, 2020 and highlighted recent corporate progress.       

    "In 2020, we made significant advances across our AOC pipeline and platform. Our work supports Avidity's evolution to a clinical-stage company as we plan to advance AOC 1001 into the clinic in the second half of this year and progress our FSHD and DMD programs," said Sarah Boyce, President and Chief Executive Officer.  "Our discovery efforts continue to focus on expanding our AOC platform into additional muscle diseases and other tissues as we begin to realize our vision of profoundly improving people's lives by revolutionizing the delivery of RNA treatments."

    "Last year we built a solid foundation for growth, anchored by our successful IPO and strong financial position with $328 million in cash at year-end," said Mike MacLean, Chief Financial Officer. "We are investing in our platform and have assembled an experienced team in RNA therapeutics and rare diseases to deliver on our discovery, clinical and commercial objectives."

    AOC Platform and Pipeline Highlights

    • Advanced Lead Program, AOC 1001 for DM1, and Broad Pipeline for Untreated Rare Muscle Diseases.  Avidity advanced its first-in-class, lead rare disease program, AOC 1001, toward the clinic and entered into a collaboration with Myotonic Dystrophy Clinical Research Network supporting END-DM1, a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1 (DM1).  The company plans to initiate a Phase 1/2 clinical study of AOC 1001 in adults with DM1 in the second half of 2021. 

       

      Avidity also advanced additional programs in its muscle franchise including a program for facioscapulohumeral muscular dystrophy (FSHD) and three programs for Duchenne muscular dystrophy (DMD). The AOC FSHD program and the lead AOC DMD program targeting Exon 44 are the most advanced. In 2022, following additional preparatory preclinical studies and regulatory clearance, Avidity plans to commence clinical trials for both of these programs.

       
    • Demonstrated Preclinical Proof-of-Concept in Skeletal Muscle and Other Tissues; Advancing Beyond Muscle with Discovery Efforts and Partnering.  In preclinical models, Avidity's AOCs demonstrated robust mRNA reductions in skeletal muscle, cardiac muscle, activated B- and T-cells and tumor infiltrating lymphocytes, macrophages and the liver. 

       

      Avidity is advancing beyond muscle with its own discovery efforts and through partnering, as evidenced by its strategic collaborations with Eli Lilly in immunology and MyoKardia, a wholly-owned subsidiary of Bristol Meyers Squibb, in cardiac tissue.

    Organizational Highlights

    • Appointed Experienced and Diverse Team Members to Management and Board of Directors. Avidity has assembled a full management team with deep expertise in the discovery, development and commercialization of RNA therapeutics and rare diseases. Avidity also welcomed Jean Kim and Tamar Thompson to its Board of Directors.    

    Fourth Quarter and Year-End 2020 Financial Results

    • Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $328.1 million as of December 31, 2020, which includes net proceeds of $274.1 million from the company's IPO in June 2020, compared to $94.6 million as of December 31, 2019.

       
    • Collaboration Revenue: Collaboration revenue solely related to our partnership with Lilly, including reimbursable expenses, was $2.1 million for the fourth quarter of 2020 compared with $1.4 million for the fourth quarter of 2019, and $6.8 million for the full year 2020 compared with $2.3 million for the full year 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 muscle programs. These expenses were $13.6 million for the fourth quarter of 2020 compared with $5.6 million for the fourth quarter of 2019, and $37.6 million for the full year 2020 compared with $14.5 million for the full year 2019. The increases were primarily driven by the progression of AOC 1001 toward the clinic, 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 $4.8 million for the fourth quarter of 2020 compared with $1.8 million for the fourth quarter of 2019, and $13.5 million for the full year 2020 compared with $5.1 million for the full year 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 driven to change lives with a new class of therapies called Antibody Oligonucleotide Conjugates (AOCs) that are designed to overcome current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity's proprietary AOC platform combines the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies 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 other muscle programs are focused on the treatment of Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, Pompe disease and muscle atrophy. In addition to its muscle franchise, Avidity has research efforts focused on immune, cardiac 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 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; Avidity's evolution to a clinical-stage company; Avidity's plans to initiate a Phase 1/2 clinical trial of AOC 1001 in patients with DM1 and the expected timing thereof; Avidity's plans to submit a regulatory filing and commence a clinical trial of its AOC FSHD program and the expected timing thereof; Avidity's plans to submit a regulatory filing and commence a clinical trial of its AOC DMD program and the expected timing thereof; Avidity's plans to expand its AOC platform into additional muscle diseases and other tissues; 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 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: the company is early in development efforts and all of its development programs are in the preclinical or discovery stage; the company's approach to the discovery and development of product candidates based on the 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 the company's operations from the COVID-19 pandemic; the success of its preclinical studies and clinical trials for its product candidates; the results of preclinical studies and early clinical trials are not necessarily predictive of future results; the company may not be able to show utility of AOCs in cardiac or other tissue and may not realize any benefits from its collaborations; its 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; 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; risks related to integration of new management personnel; and other risks described in the company's prior press releases and in its 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.

    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



    Twelve Months Ended







    December 31,



    December 31,







    2020



    2019



    2020



    2019

    Collaboration revenue





    $          2,142



    $          1,445



    $          6,787



    $          2,319

    Operating expenses:



















           Research and development





    13,619



    5,645



    37,602



    14,539

           General and administrative





    4,816



    1,847



    13,462



    5,112

    Total operating expenses





    18,435



    7,492



    51,064



    19,651

    Loss from operations





    (16,293)



    (6,047)



    (44,277)



    (17,332)

    Other income (expense), net





    18



    (4,354)



    (78)



    (7,402)

    Net loss





    $       (16,275)



    $       (10,401)



    $       (44,355)



    $       (24,734)

    Net loss per share, basic and diluted





    $          (0.43)



    $          (3.75)



    $          (2.05)



    $          (9.12)

    Weighted-average shares outstanding,

       basic and diluted





    37,455



    2,776



    21,663



    2,713





















    Balance Sheets











     December 31, 













    2020



    2019

    Assets

















    Current assets:

















           Cash, cash equivalents and marketable securities











    $      328,141



    $        94,578

           Prepaid and other assets











    3,537



    1,098

    Total current assets











    331,678



    95,676

    Property and equipment, net











    1,468



    631

    Restricted cash













    251



    Other assets











    501



    600

    Total assets











    $      333,898



    $        96,907

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

    Current liabilities:



















           Accounts payable and other accrued liabilities











    $        10,897



    $          3,622

           Deferred revenue, current portion











    3,690



    3,840

           Long-term debt, current portion















    2,774

    Total current liabilities













    14,587



    10,236

    Lease liabilities, net of current portion













    938



    393

    Deferred revenue, net of current portion













    12,150



    15,100

    Long-term debt, net of current portion















    1,770

    Other long-term liabilities















    45

    Total liabilities













    27,675



    27,544

    Convertible preferred stock















    134,720

    Stockholders' equity (deficit)











    306,223



    (65,357)

    Total liabilities, convertible preferred stock and

       stockholders' equity (deficit)













    $      333,898



    $        96,907

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/avidity-biosciences-reports-fourth-quarter-and-year-end-2020-financial-results-and-recent-highlights-301246523.html

    SOURCE Avidity Biosciences, Inc.

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