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Avidity Biosciences Reports Fourth Quarter 2024 Results

Avidity Biosciences, Inc. (Nasdaq: RNA), a leading biopharmaceutical company known for pioneering a new class of RNA therapeutics referred to as Antibody Oligonucleotide Conjugates (AOCs™), recently announced its fourth quarter financial results for 2024, showcasing significant advancements and a robust outlook for the upcoming year.

Clinical Program Successes

The company is making strong progress in its clinical programs, especially in the realm of rare neuromuscular diseases. According to Sarah Boyce, President and CEO of Avidity, the data from their three clinical-stage programs indicate consistent and reproducible outcomes that highlight the efficacy of the AOC platform. This year is projected to be transformative for Avidity as they prepare for what will be their first Biologics License Application (BLA) submission as well as three potential product launches slated to begin in 2026.

In particular, Avidity is focusing efforts on its lead program, delpacibart zotadirsen (del-zota), targeted at Duchenne muscular dystrophy (DMD) and amenable to exon 44 skipping (DMD44). The company has completed enrollment in their Phase 1/2 EXPLORE44 Open-label Extension study, which will provide critical data for the anticipated BLA submission by the end of 2025. Encouragingly, top-line data from this trial will be presented at the 2025 Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, Texas, scheduled for March 19, 2025.

Financial Stability and Growth

Avidity reports a strong financial position, with approximately $1.5 billion in cash, cash equivalents, and marketable securities as of December 31, 2024. This solid financial footing allows the company to expedite the development of its global commercial infrastructure and to further expand its team of industry professionals across various domains. Mike MacLean, CFO of Avidity, expressed confidence in the company’s capability to develop advanced therapeutic options through their innovative AOC technology, extending into new areas such as precision cardiology.

Additionally, Avidity's recent financial report highlighted collaboration revenues of $3.0 million in the fourth quarter of 2024, primarily from its partnership with Bristol Myers Squibb. However, the company did report an increase in research and development expenses to $95.6 million for the fourth quarter, reflecting its commitment to advancing its novel therapeutics.

Future Anticipations

Looking ahead, Avidity is optimistic about approaching critical milestones in its three main neuromuscular programs: del-zota for DMD44, delpacibart etedesiran (del-desiran) for myotonic dystrophy type 1 (DM1), and delpacibart braxlosiran (del-brax) for facioscapulohumeral muscular dystrophy (FSHD). Each therapy is positioned to potentially become the first globally approved treatment for these serious and underserved conditions. Avidity has made substantial progress with del-desiran and del-brax, with their respective trials moving forward as planned. The anticipated approval pathways from the FDA for del-zota indicate a promising trajectory for Avidity's pipeline and the patients who rely on these therapies.

In summary, Avidity Biosciences is well-positioned for a transformational 2025, with strong clinical data, a solid balance sheet, and a clear trajectory toward regulatory submissions and product launches. As they continue to push the boundaries of RNA therapeutic innovation, Avidity remains committed to improving the lives of those affected by rare neuromuscular diseases. The company's advancements could pave the way for substantial innovations in treatment for conditions that have long lacked effective options.