On March 1, 2022 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, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAR 1, 2022, View Source [SID1234609281]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2021 was a pivotal year for Avidity, the field of RNA therapeutics and for the DM1 community as the first patient was dosed with an AOC as part of the AOC 1001 MARINA trial," said Sarah Boyce, president and chief executive officer. "This milestone transitioned us into a clinical-stage biopharmaceutical company and we continue to make great strides with our advancing and expanding pipeline and AOC platform technology. By the end of 2022, we plan to have three programs in three different rare diseases in the clinic with AOC 1001, AOC 1020 and AOC 1044."

"We are in a strong financial position with $406 million at year-end bolstered through approximately $175 million raised in 2021, inclusive of $155 million of net proceeds from our first follow on financing. We are well funded into 2024 which we expect will allow us to complete the MARINA trial, advance the AOC 1044 and AOC 1020 programs into clinical development and to continue to invest in expanding our pipeline and the AOC platform in muscle and beyond," said Mike MacLean, chief financial officer.

2021 Key Highlights

AOC 1001 Achievements

The Company initiated the Phase 1/2 MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1).
The FDA and the European Medicines Agency (EMA) granted AOC 1001 Orphan Designation and the FDA granted AOC 1001 Fast Track Designation.
The MARINA trial is on track for a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the trial participants in Q4 2022.
Pipeline Advancements

AOC 1044 was named as the clinical development candidate for the Duchenne Muscular Dystrophy (DMD) program targeting Exon 44. AOC 1044 is in IND-enabling studies and is expected to enter the clinic by the end of 2022.
AOC 1020 was named as the clinical development candidate for the facioscapulohumeral muscular dystrophy (FSHD) program. AOC 1020 is in IND-enabling studies and is expected to enter the clinic by the end of 2022.
Avidity also entered a collaboration with the FSHD Clinical Trial Research Network (FSHD CTRN) to support a natural history study called the Motor Outcomes to Validate Evaluations Plus (MOVE+) Study to enhance the understanding of how to utilize whole-body MRI and other tools to identify specific biomarkers for FSHD that can potentially accelerate and support future clinical trial design.
Organizational Highlights

Avidity recently appointed Steve Hughes, M.D. as chief medical officer. Dr. Hughes brings over 20 years of experience in the biotechnology industry and has extensive experience in RNA-based treatments and rare diseases.
Fourth Quarter and Year-End 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $405.5 million as of December 31, 2021, which reflects $174.7 million raised in 2021, inclusive of $155.1 million of net proceeds from our first follow on financing.

Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $1.9 million for the fourth quarter of 2021 compared with $2.1 million for the fourth quarter of 2020, and $9.3 million for the full year 2021 compared with $6.8 million for the full year 2020.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $33.0 million for the fourth quarter of 2021 compared with $13.6 million for the fourth quarter of 2020, and $101.2 million for the full year 2021 compared with $37.6 million for the full year 2020. The increases were primarily driven by the advancement of AOC 1001, AOC 1020 and AOC 1044, as well as costs related to the expansion of the company’s overall research capabilities.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $7.4 million for the fourth quarter of 2021 compared with $4.8 million for the fourth quarter of 2020, and $26.2 million for the full year 2021 compared with $13.5 million for the full year 2020. The increases were primarily due to higher personnel costs. The full year increase was also due to higher professional fees and insurance costs.