On February 28, 2023 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the fourth quarter and year ended December 31, 2022 and highlighted recent corporate progress (Press release, Avidity Biosciences, FEB 28, 2023, View Source [SID1234627889]).

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"In 2022, we executed on our goal of advancing three rare disease programs into clinical development and demonstrated the first-ever successful targeted delivery of RNA into muscle, a revolutionary advancement for the field of RNA therapeutics," said Sarah Boyce, president and chief executive officer at Avidity. "Building on our AOC proof of platform data, 2023 will be another important year for Avidity. We look forward to Phase 1/2 MARINA and MARINA-OLE data as well as results from healthy volunteers in the Phase 1/2 EXPLORE44 trial while continuing to expand the broad utility of our AOC platform."

"We are in a strong financial position with more than $600 million at year-end reflecting approximately $224M in net proceeds from our successful financing in December. We are well funded into mid-2025 as we advance our clinical development programs for AOC 1001, AOC 1044 and AOC 1020, as well as expand our AOC platform and pipeline programs including our recently announced internal programs in skeletal muscle and cardiology" said Mike MacLean, chief financial officer and chief business officer at Avidity.
Clinical Development Programs – Achievements & Updates
AOC 1001
•Positive interim data from the preliminary assessment of AOC 1001 in the Phase 1/2 MARINA study demonstrated first-ever successful targeted delivery of RNA to skeletal muscle, DMPK reduction, splicing improvements and early signs of clinical activity with improvement in myotonia in some participants.
•Top-line data from the MARINA trial is anticipated in 2023 and preliminary data from the MARINA-OLE trial is anticipated in late 2023.
•The company plans to give an update on the AOC 1001 partial clinical hold at the end of the first quarter in 2023.

AOC 1044
•The FDA cleared Avidity to proceed with the Phase 1/2 EXPLORE44 clinical trial of AOC 1044 for the treatment of DMD mutations amenable to exon 44 skipping (DMD44). AOC 1044 is the first of multiple AOCs being developed for DMD and is the first AOC PMO to advance into the clinic.
◦The EXPLORE44 trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial to evaluate AOC 1044 in approximately 40 healthy volunteers and 24 participants with DMD44, ages seven to 27 years old.

•Data from healthy volunteers in the EXPLORE44 trial is anticipated in the second half of 2023.

AOC 1020
•The FDA cleared Avidity to proceed with the Phase 1/2 FORTITUDE clinical trial of AOC 1020 in adults with facioscapulohumeral muscular dystrophy (FSHD).
◦The FORTITUDE trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate AOC 1020 in approximately 70 adult participants with FSHD.
◦The FDA granted AOC 1020 Fast Track Designation in January 2023.
◦The FDA and the European Medicines Agency (EMA) granted AOC 1020 Orphan Designation in February 2023.
•Data from a preliminary assessment in approximately half of participants in the FORTITUDE trial is anticipated in the first half of 2024.

Pipeline Advancements
•Avidity announced that it advanced and expanded its wholly-owned early stage AOC pipeline including adding a rare cardiac program and an additional program in rare skeletal muscle disease.

Organizational Highlights
•The company announced in February that Arthur A. Levin, Ph.D., joined its board of directors and transitioned to distinguished scientist and strategic leader at Avidity.
•W. Michael Flanagan, Ph.D. was promoted in February to chief scientific and technical officer.
Fourth Quarter and Year-End 2022 Financial Results
•Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $610.7 million as of December 31, 2022, which reflects $344.6 million raised in 2022, inclusive of $223.8 million of net proceeds from our follow-on financing. In addition, subsequent to December 31, 2022, we have raised $22.3 million through our "at the market" program.

•Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company (Lilly) and totaled $2.7 million for the fourth quarter of 2022 compared with $1.9 million for the fourth quarter of 2021, and $9.2 million for the full year 2022 compared with $9.3 million for the full year 2021.
•Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $45.6 million for the fourth quarter of 2022 compared with $33.0 million for the fourth quarter of 2021, and $150.4 million for the full year 2022 compared with $101.2 million for the full year 2021. The increases were primarily driven by the advancement of AOC 1001,

AOC 1020 and AOC 1044, as well as internal and external costs related to the expansion of the company’s overall research capabilities.
•General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $10.4 million for the fourth quarter of 2022 compared with $7.4 million for the fourth quarter of 2021, and $37.7 million for the full year 2022 compared with $26.2 million for the full year 2021. The increases were primarily due to higher personnel costs and professional fees to support the company’s expanded operations.