Avidity is on-track to initiate global Phase 3 HARBOR™ trial of AOC 1001 for DM1 in mid-2024
Company to share first-look at long-term efficacy and safety data from MARINA-OLE™ trial in people living with DM1 at MDA Clinical & Scientific Conference and via webcast on March 4, 2024
Avidity to report data from clinical programs for people living with FSHD and DMD44 later this year
SAN DIEGO, Feb. 28, 2024 /PRNewswire/ — Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today reported financial results for the fourth quarter and year ended December 31, 2023, and highlighted recent corporate progress.
“With proven delivery of RNA to muscle and robust efficacy and long-term safety data from our ongoing clinical development programs, we are revolutionizing the delivery of RNA with our AOC technology,” said Sarah Boyce, president and chief executive officer at Avidity. “2024 is poised to be a transformative year for Avidity as we plan to initiate the global Phase 3 HARBOR™ trial for people living with DM1 and report data from all three of our clinical development programs to treat rare muscle diseases – DM1, FSHD and DMD44. We also plan to advance our cardiology and additional skeletal muscle programs toward clinical development. We know how important these potential new treatments are for the patient community and are working diligently to bring these much-needed therapies to people living with rare diseases.”
“We are pleased to be in a strong financial position, with $595 million at the close of 2023 and a cash runway through 2025,” said Mike MacLean, chief financial officer and chief business officer at Avidity. “We are well positioned to rapidly advance our clinical development programs, strategically progress our pipeline and build the infrastructure needed as part of Avidity’s next phase of growth.”
Clinical Development Programs – Achievements & Updates
AOC 1001
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 living with myotonic dystrophy type 1 (DM1). These data augmented previously presented positive topline data from the Phase 1/2 MARINA® trial in April 2023 showing improvements in myotonia, muscle strength and mobility.
Avidity will share a first-look at long-term efficacy and safety data from the MARINA-OLE™ trial of AOC 1001 in people living with DM1 via poster presentation during the 2024 MDA Clinical & Scientific Conference March 3-6 in Orlando, Florida. The company will also host a live webcast event on March 4 at 8:00 a.m. ET as part of its ongoing investor and analyst event series.
Avidity is on-track to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with DM1 in mid-2024.
AOC 1020
In February 2023, the FDA and the European Medicines Agency (EMA) granted Orphan Designation for AOC 1020 for facioscapulohumeral muscular dystrophy (FSHD), and in January 2023, the FDA granted AOC 1020 Fast Track Designation for FSHD.
In the second quarter of 2024, Avidity is planning to share preliminary data in approximately half of the study participants in the Phase 1/2 FORTITUDE™ trial of AOC 1020 in FSHD.
AOC 1044
In December 2023, Avidity announced positive AOC 1044 data in healthy volunteers showing unprecedented delivery to muscle and up to 1.5% exon skipping from the Phase 1/2 EXPLORE44™ clinical trial for the treatment of Duchenne muscular dystrophy 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. The FDA granted AOC 1044 Fast Track Designation in April 2023 and Rare Pediatric Disease Designation in February 2024 for DMD44.
In the second half of 2024, Avidity is planning to share 5 mg/kg cohort data from the Phase 1/2 EXPLORE44™ trial of people living with DMD44.
Collaboration Announcements
In November 2023, Avidity announced the expansion of a global licensing and research collaboration with Bristol Myers Squibb. The expanded collaboration will focus on the discovery, development and commercialization of up to five cardiovascular targets with $100 million upfront and potential cumulative milestone payments to Avidity of up to $2.2 billion.
Organizational Highlights
Avidity announced in January 2024 the appointment of Eric B. Mosbrooker as Chief Strategy Officer. Mr. Mosbrooker previously served as a member of Avidity’s Board of Directors.
Fourth Quarter and Year-End 2023 Financial Results
Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $595.4 million as of December 31, 2023, which reflects $160.5 million raised in 2023, inclusive of approximately $100 million through our collaboration with Bristol Myers Squibb and $60.5 million through our “at the market offering” program.
Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $2.2 million for the fourth quarter of 2023 compared with $2.8 million for the fourth quarter of 2022, and $9.6 million for the full year 2023 compared with $9.2 million for the full year 2022.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $52.8 million for the fourth quarter of 2023 compared with $45.6 million for the fourth quarter of 2022, and $191.0 million for the full year 2023 compared with $150.4 million for the full year 2022. The …
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