RNA Avidity Biosciences Inc.

19.65
+0.62  (+3%)
Previous Close 19.03
Open 21.37
52 Week Low 18.85
52 Week High 37.46
Market Cap $739,139,879
Shares 37,615,261
Float 32,309,848
Enterprise Value $419,387,689
Volume 2,099,223
Av. Daily Volume 215,930
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Drug Stage Catalyst Date
AOC 1001 (MARINA)
Myotonic dystrophy type 1 (DM1)
Phase 1/2
Phase 1/2
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Latest News

  1. LA JOLLA, Calif., Aug. 3, 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 the pricing of an underwritten public offering of 8,000,000 shares of its common stock at a price to the public of $18.00 per share. All of the shares to be sold in the offering are to be sold by Avidity. The gross proceeds to Avidity from the offering, before deducting the underwriting discounts and commissions and other offering expenses, are expected to be $144.0 million. The offering is expected to close on or about August 6, 2021, subject to the satisfaction of customary closing conditions. In addition…

    LA JOLLA, Calif., Aug. 3, 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 the pricing of an underwritten public offering of 8,000,000 shares of its common stock at a price to the public of $18.00 per share. All of the shares to be sold in the offering are to be sold by Avidity. The gross proceeds to Avidity from the offering, before deducting the underwriting discounts and commissions and other offering expenses, are expected to be $144.0 million. The offering is expected to close on or about August 6, 2021, subject to the satisfaction of customary closing conditions. In addition, Avidity has granted the underwriters a 30-day option to purchase up to an additional 1,200,000 shares of common stock.

    Avidity intends to use the net proceeds from this offering, together with its existing cash, cash equivalents and marketable securities: to complete its Phase 1/2 MARINA trial for AOC 1001; to advance AOC 1044 and its AOC FSHD program into clinical development; to further advance its AOC platform in and beyond its muscle franchise; and towards working capital and other general corporate purposes.

    Cowen, SVB Leerink, Evercore ISI and Wells Fargo Securities are acting as joint bookrunning managers for the offering.

    The securities described above are being offered by Avidity pursuant to a shelf registration statement that became automatically effective upon filing with the Securities and Exchange Commission (SEC). A preliminary prospectus supplement and accompanying prospectus relating to this offering were filed with the SEC and a final prospectus supplement relating to the offering will be filed with the SEC. The offering may be made only by means of a prospectus supplement and accompanying prospectus. When available, copies of the final prospectus supplement and the accompanying prospectus relating to this offering may be obtained from: Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by email at or by telephone at (833) 297-2926; from SVB Leerink LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, or by telephone at (800) 808-7525, ext. 6105, or by email at ; from Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, New York 10055, by telephone at (888) 474-0200, or by email at ; or from Wells Fargo Securities, LLC, Attention: Equity Syndicate Department, 500 West 33rd Street, New York, NY 10001, or by telephone at (800) 326-5897, or by email at . Electronic copies of the final prospectus supplement and accompanying prospectus will also be available on the website of the SEC at http://www.sec.gov.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

    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 myotonic dystrophy type 1 (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.

    Forward Looking Statements

    Avidity cautions you 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 Avidity's expectations of market conditions and the satisfaction of customary closing conditions related to the public offering, the expected closing of the offering and the anticipated use of proceeds therefrom. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties associated with market conditions and the satisfaction of customary closing conditions related to the proposed public offering, as well as risks and uncertainties inherent in Avidity's business, including those described in the company's prior press releases and the company's filings with the Securities and Exchange Commission (SEC), including under the heading "Risk Factors" in the company's Annual Report on Form 10-K and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Avidity 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:

    Kathleen Gallagher

    (858) 401-7900

            

    Amy Conrad

    Juniper Point

    (858) 366-3243

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-inc-announces-pricing-of-public-offering-of-common-stock-301347817.html

    SOURCE Avidity Biosciences, Inc.

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  2. LA JOLLA, Calif., Aug. 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 it intends to offer and sell, subject to market and other conditions, $100 million of shares of its common stock in an underwritten public offering. In addition, Avidity intends to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of common stock offered in the public offering. All of the shares to be sold in the offering are to be sold by Avidity. There can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the…

    LA JOLLA, Calif., Aug. 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 it intends to offer and sell, subject to market and other conditions, $100 million of shares of its common stock in an underwritten public offering. In addition, Avidity intends to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of common stock offered in the public offering. All of the shares to be sold in the offering are to be sold by Avidity. There can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    Avidity intends to use the net proceeds from this offering, together with its existing cash, cash equivalents and marketable securities: to complete its Phase 1/2 MARINA trial for AOC 1001; to advance AOC 1044 and its AOC FSHD program into clinical development; to further advance its AOC platform in and beyond its muscle franchise; and towards working capital and other general corporate purposes.

    Cowen, SVB Leerink, Evercore ISI and Wells Fargo Securities are acting as joint bookrunning managers for the offering.

    The securities described above are being offered by Avidity pursuant to a shelf registration statement that became automatically effective upon filing with the Securities and Exchange Commission (SEC). A preliminary prospectus supplement and accompanying prospectus relating to this offering will be filed with the SEC. When available, copies of the preliminary prospectus supplement and the accompanying prospectus relating to this offering may be obtained from: Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by email at or by telephone at (833) 297-2926; from SVB Leerink LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, or by telephone at (800) 808-7525, ext. 6105, or by email at ; from Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, New York 10055, by telephone at (888) 474-0200, or by email at ; or from Wells Fargo Securities, LLC, Attention: Equity Syndicate Department, 500 West 33rd Street, New York, NY 10001, or by telephone at (800) 326-5897, or by email at . Electronic copies of the preliminary prospectus supplement and accompanying prospectus will also be available on the website of the SEC at http://www.sec.gov.

    This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.

    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 myotonic dystrophy type 1 (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.

    Forward Looking Statements

    Avidity cautions you 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 Avidity's expectations of the completion, timing and size of the proposed public offering and the anticipated use of proceeds therefrom. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties associated with market conditions and the satisfaction of customary closing conditions related to the proposed public offering, as well as risks and uncertainties inherent in Avidity's business, including those described in the company's prior press releases and the company's filings with the Securities and Exchange Commission (SEC), including under the heading "Risk Factors" in the company's Annual Report on Form 10-K and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Avidity 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:

    Kathleen Gallagher

    (858) 401-7900

            

    Amy Conrad

    Juniper Point

    (858) 366-3243

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-inc-announces-proposed-public-offering-of-common-stock-301346363.html

    SOURCE Avidity Biosciences, Inc.

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  3. LA JOLLA, Calif., Aug. 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 U.S. Food and Drug Administration (FDA) cleared the company to proceed with the Phase 1/2 MARINA clinical trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1).

    AOC 1001, Avidity's lead program utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of DMPK, the disease-related mRNA. AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA) that targets DMPK mRNA.

    "The FDA clearance to proceed with our Phase 1/2 clinical trial for AOC 1001 is a significant milestone for Avidity as we move into the clinic with our first AOC engineered to deliver to skeletal muscle," said Sarah Boyce, president and chief executive officer. "We are grateful to Dr. Johnson for joining us on today's call to discuss the current treatment of people living with DM1 and the important research that he and the Myotonic Dystrophy Clinical Research Network are conducting to further understand this progressive disease. We are continuing to engage with the DM1 patient, advocate and physician community as we actively work to get the trial up and running."

    The MARINA Phase 1/2 Trial of AOC 1001 in Adults with DM1 and Recent Highlights

    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, the disease-related mRNA responsible for DM1. 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.

    Recently, the FDA also granted Orphan Drug Designation to AOC 1001 for the treatment of DM1. The FDA grants Orphan Drug Designation to novel drugs that seek to treat a rare disease or condition and, if the drug is approved for the designated orphan indication, provides 7 years of market exclusivity, along with certain financial incentives, including tax credits, opportunities for grant funding towards clinical trial costs and FDA user-fee waivers.

    Today's Video Webcast Information

    The company is hosting Volume 2 of their virtual investor and analyst series today August 2, 2021 beginning at 8:30 am ET to further discuss the AOC 1001 program. The event is a live video webcast and can be accessed here or from 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.

    The management team will be joined by Dr. Nicholas E. Johnson, MD, MSCI, FAAN. Dr. Johnson is one of the principal investigators in END-DM1, an ongoing natural history study being run by the Myotonic Dystrophy Clinical Research Network (DMCRN) and will be the lead investigator in the Phase 1/2 trial for AOC 1001. Dr. Johnson is an associate professor, division chief of neuromuscular, and vice chair of research in the department of neurology at Virginia Commonwealth University.

    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 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.

    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. 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 a 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 MARINA study of AOC 1001 in adults with DM1. FDA has granted Orphan Drug Designation for AOC 1001 for the treatment of 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 myotonic dystrophy type 1 (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 initiation of a clinical trial of AOC 1001 in patients with DM1; the potential benefits associated with Orphan Drug Designation for approved drugs; plans for its other programs to enter the clinic and the timing thereof; and the potential to broaden the reach of AOCs beyond skeletal muscle tissues. 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; risks that the benefits associated with Orphan Drug Designation may not be realized, including that Orphan Drug 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; 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

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-receives-ind-clearance-from-fda-to-proceed-with-the-phase-12-marina-trial-of-aoc-1001-in-adults-with-myotonic-dystrophy-dm1-301345542.html

    SOURCE Avidity Biosciences, Inc.

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  4. LA JOLLA, Calif., May 19, 2021 /PRNewswire/ -- Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs™), announced that new preclinical data on its lead AOC program, AOC 1001, and key programmatic updates will be shared at a virtual investor and analyst event today.  The event at 8:00am PT is titled "Engineering AOCs" and is the first in a series of investor and analyst events that the company plans to host in 2021.

    Avidity Pipeline Updates

    • AOC 1001 for Myotonic Dystrophy Type 1 (DM1) demonstrates safety profile that supports advancement to clinic.  During today's virtual investor and analyst event, Avidity will share comprehensive results from its non-human primate (NHP) toxicology studies (both Good Laboratory Practice, or GLP, and non-GLP) that support the clinical evaluation of AOC 1001. Results from the AOC 1001 GLP toxicology study show a favorable safety profile that supports advancement into the clinic.
    • AOC 1044 named as clinical development candidate for Duchenne Muscular Dystrophy (DMD) program targeting Exon 44. AOC 1044 is the first of three programs for DMD and is now entering IND-enabling studies.  Avidity remains on track with plans to commence clinical trials for AOC 1044 and its AOC FSHD program for facioscapulohumeral muscular dystrophy (FSHD) in 2022, following additional preparatory preclinical studies and regulatory clearance.

    "This event is the first in a series highlighting the great work being done by the team at Avidity. Our AOC platform is built upon years of engineering and rigorously following the data. It has the potential to revolutionize the delivery of oligonucleotide therapeutics to multiple cell and tissue types to profoundly impact the lives of people suffering from serious diseases. We look forward to sharing data today from our first AOC candidate, AOC 1001, which remains on track to enter the clinic in the second half of this year," said Sarah Boyce, president and CEO of Avidity. "We are also grateful to have Dr. Zamore and Dr. Dowdy joining us for a panel discussion on the important role for RNA therapeutics in treating diseases that are currently underserved."

    Today's Video Webcast Information

    A live video webcast of the event will be available today, May 19, 2021 beginning at 8:00 am PT / 11:00am ET 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 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 and on 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 initiation of a clinical trial of AOC 1001 in patients with myotonic dystrophy type 1,  the initiation of a clinical trial of AOC 1044 in patients with Duchenne Muscular Dystrophy (DMD), the initiation of a clinical trial of AOC FSHD in patients with facioscapulohumeral muscular dystrophy  (FSHD), 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:

    Kathleen Gallagher

    (858) 401-7900

            

    Amy Conrad

    Juniper Point

    (858) 366-3243

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/avidity-biosciences-announces-updates-on-the-pipeline-and-platform-at-virtual-investor-and-analyst-event-301294582.html

    SOURCE Avidity Biosciences, Inc.

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  5. 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



     

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    SOURCE Avidity Biosciences, Inc.

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