On November 8, 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 and recent highlights for the third quarter ended September 30, 2023 (Press release, Avidity Biosciences, NOV 8, 2023, View Source [SID1234637246]). Avidity ended the third quarter of 2023 with cash, cash equivalents and marketable securities totaling $542.6 million.
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"In early October we announced new positive AOC 1001 data that demonstrated improvement in multiple additional functional endpoints, as well as favorable long-term safety and tolerability, in people living with DM1," said Sarah Boyce, president and chief executive officer. "In addition, we announced the completion of the 12-participant dose escalation from 2 mg/kg to 4 mg/kg as part of the partial clinical hold easement and look forward to finalizing the Phase 3 study design and global regulatory path for AOC 1001. We continue to make significant strides across all three of our clinical development programs, remaining on track to report data this year for AOC 1044 in DMD and in the first half of 2024 for both AOC 1001 in DM1 and AOC 1020 in FSHD".
"With funding into the second half of 2025 and continued execution across our portfolio, we remain very confident in the progress we are making in advancing our clinical development programs for DM1, DMD and FSHD," said Mike MacLean, chief financial officer and chief business officer. "The recently presented positive AOC 1001 data at World Muscle Society (WMS) Congress reinforces our confidence in our AOC platform, which spans multiple therapeutic areas, and our ability to deliver on our vision to profoundly improve people’s lives by revolutionizing the delivery of RNA therapeutics".
Recent Highlights
•In October 2023, Avidity announced new positive AOC 1001 data demonstrating improvement in multiple additional functional endpoints and favorable long-term safety and tolerability in people with DM1 at WMS. The new data demonstrated improvement in additional functional measures including hand grip, muscle strength (Manual Muscle Testing composite score and both upper and lower Quantitative Muscle Testing composite scores) and patient reported outcomes, augmenting previously reported positive data showing improvements in myotonia, muscle strength and mobility.
•Data from the dose escalation of 12 participants from 2 mg/kg to 4 mg/kg of AOC 1001 as part of the easement of the partial clinical hold showed no neurological events and no MRI changes following dosing. Avidity continues to work as quickly as possible to resolve the partial clinical hold. In parallel, the company is finalizing a Phase 3 study design and a global regulatory path for AOC 1001.
•Avidity has initiated dosing participants living with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44).
•In August and October 2023, the FDA and EMA respectively, granted Orphan Designation for AOC 1044 for DMD44.
Upcoming Milestones
•The company continues to advance three distinct rare disease clinical programs with AOC 1001 for DM1, AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and AOC 1044 for the treatment of DMD44. Upcoming milestones include:
◦Data from healthy volunteers in the EXPLORE44 trial planned for Q4 of this year
◦A first look at efficacy data from the MARINA-OLE trial planned for first half of 2024
◦Data from a preliminary assessment in approximately half of participants in the FORTITUDE trial planned for the first half of 2024
Third Quarter 2023 Financial Results
Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $542.6 million as of September 30, 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 Eli Lilly and Company and totaled $2.8 million for the third quarter of 2023 compared with $2.5 million for the third quarter of 2022, and $7.4 million for the first nine months of 2023 compared with $6.5 million for the first nine months 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 $47.7 million for the third quarter of 2023 compared with $37.3 million for the third quarter of 2022, and $138.2 million for the first nine months of 2023 compared with $104.8 million for the first nine months of 2022. The increases were primarily driven by the advancement of AOC 1001, AOC 1020 and AOC 1044, as well as external and internal 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 $13.7 million for the third quarter of 2023 compared with $10.1 million for the third quarter of 2022, and $38.1 million for the first nine months of 2023 compared with $27.3 million for the first nine months of 2022. The increases were primarily due to higher personnel costs and professional fees to support the company’s expanded operations.