On May 9, 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 first quarter ended March 31, 2023 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAY 9, 2023, View Source [SID1234631229]). Avidity ended the first quarter of 2023 with cash, cash equivalents and marketable securities totaling $586.3 million.
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"We are pleased with the positive AOC 1001 topline data reported at AAN demonstrating directional functional improvements across multiple functional measurements. We are focused on discussions with the FDA to define a path forward for AOC 1001 as soon as possible," said Sarah Boyce, president and chief executive officer at Avidity. "We have a number of important milestones from our pipeline expected this year including a first look at data from the MARINA-OLE trial and data from healthy volunteers in the Phase 1/2 EXPLORE44 study. We are executing on our clinical and pipeline programs to develop much needed therapies for people living with devastating rare diseases."
"We ended Q1 with $586 million which puts us in a strong financial position with a runway to mid-2025. We continue to invest in our advancing and expanding pipeline in skeletal muscle and cardiology rare disease programs while also expanding the broad utility of our AOC platform through our internal discovery efforts and collaborations," said Mike MacLean, chief financial officer and chief business officer at Avidity
Recent Highlights
•Presented top-line data for MARINA at American Academy of Neurology (AAN) 2023 Annual Meeting in April 2023:
◦The MARINA trial concluded with the 38 participants with myotonic dystrophy type 1 (DM1). The company will continue to dose the participants at both 2 mg/kg and 4 mg/kg of AOC 1001 in the MARINA open-label extension study (MARINA-OLE). AOC 1001 MARINA topline data demonstrated:
▪Directional improvement in multiple functional assessments including measures of myotonia, strength and mobility
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▪Meaningful DMPK reduction and splicing changes in participants treated with AOC 1001 followed by directional improvements in functional measures at 2 mg/kg and 4mg/kg doses of AOC 1001
▪AOC 1001 demonstrated broad splicing improvements in more than a thousand genes impacted by DM1, confirming activity in the nucleus
▪Favorable safety and tolerability profile of AOC 1001 with most adverse events mild or moderate
•The company provided a regulatory update on the AOC 1001 partial clinical hold on new participant enrollment and provided more information on the rare serious adverse event in a single participant that led to the partial clinical hold. Avidity continues to work diligently with the FDA and remains very confident in the benefit/risk profile of AOC 1001.
•Avidity announced that it advanced and expanded its wholly-owned early stage AOC pipeline including adding a rare cardiology program and an additional program in rare skeletal muscle disease. Ongoing collaborations with Eli Lilly and Company (Lilly) and Bristol Myers Squibb (BMS) continue to advance beyond skeletal muscle in immunology and cardiology, respectively.
•The FDA granted AOC 1020 Fast Track Designation in January 2023 and in February 2023, the FDA and the European Medicines Agency (EMA) granted AOC 1020 Orphan Designation.
•The FDA also recently granted AOC 1044 Fast Track Designation in April 2023.
Upcoming Milestones:
•The company continues to advance three distinct rare disease clinical programs: AOC 1001 for DM1, AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping (DMD44). Upcoming milestones include:
◦A first look at data from the MARINA-OLE trial is anticipated in late 2023.
◦Data from healthy volunteers in the EXPLORE44 trial is anticipated in the second half of 2023.
◦Data from a preliminary assessment in approximately half of participants in the FORTITUDE trial is anticipated in the first half of 2024.
First Quarter 2023 Financial Results
Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $586.3 million as of March 31, 2023, compared to $610.7 million as of December 31, 2022.
•Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Lilly and totaled $2.2 million for the first quarter of 2023 compared with $1.8 million for the first quarter of 2022.
•Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $47.8 million for the first quarter of 2023 compared with $27.7 million for the first quarter of 2022. 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 $12.1 million for the first quarter of 2023 compared with $8.6 million for the first quarter of 2022. The increases were primarily due to higher personnel costs and professional fees to support the company’s expanded operations.