Avidity Biosciences Q1 2024 Financial Results and Highlights

Avidity Biosciences, Inc., a biopharmaceutical company specializing in RNA therapeutics, has announced significant advancements and financial results for the first quarter of 2024. The company, known for its Antibody Oligonucleotide Conjugates (AOCs™), is preparing to initiate the global Phase 3 HARBOR™ trial for del-desiran (AOC 1001) aimed at treating myotonic dystrophy type 1 (DM1) within this quarter. This follows new long-term data from the MARINA-OLE™ study, which demonstrated a reversal of disease progression in DM1 patients across multiple metrics, including video hand opening time (vHOT), muscle strength, and daily activities.

Sarah Boyce, Avidity's president and CEO, emphasized the company's dedication to advancing their AOC platform, stating that the positive results from the MARINA-OLE study have paved the way for regulatory agreement on including vHOT as the primary endpoint in the HARBOR trial. Additionally, Avidity plans to report preliminary data from the FORTITUDE™ trial for facioscapulohumeral muscular dystrophy (FSHD) and 5 mg/kg cohort data from the EXPLORE44™ trial for Duchenne muscular dystrophy (DMD44) later this year.

Financially, Avidity ended the first quarter of 2024 with a robust cash position of $915 million, bolstered by an oversubscribed equity raise of $400 million. This financial strength is expected to support the company’s operations into late 2026, according to Mike MacLean, CFO and chief business officer at Avidity. The first quarter's revenue from collaborations, primarily with Bristol Myers Squibb and Eli Lilly, reached $3.5 million, up from $2.2 million in the same period the previous year.

Avidity also received Breakthrough Therapy designation from the FDA for del-desiran in treating DM1, accelerating the initiation of the global Phase 3 HARBOR study to the second quarter of 2024. This study will focus on vHOT as the primary endpoint and will include key secondary endpoints like muscle strength and daily living activities. Additionally, the 4 mg/kg data from the MARINA-OLE study showed consistent and durable improvements in DM1 patients, reinforcing the efficacy and safety of del-desiran.

Research and development expenses increased to $66.8 million in the first quarter of 2024 from $47.8 million in the previous year, driven by advancements in del-desiran, AOC 1020, and AOC 1044, and the expansion of research capabilities. General and administrative expenses also rose to $13.9 million from $12.1 million, mainly due to higher personnel costs.

Avidity’s pipeline extends beyond muscle diseases, including programs in cardiology and immunology through internal discovery efforts and key partnerships. The company remains at the forefront of RNA therapeutics, demonstrating the first-ever successful targeted delivery of RNA into muscle, and continues to lead with clinical development programs for rare muscle diseases.

Upcoming milestones for Avidity include the initiation of the global Phase 3 HARBOR trial, sharing preliminary data from the FORTITUDE trial in the second quarter, and releasing cohort data from the EXPLORE44 trial in the second half of 2024. These steps are crucial for the continued development and potential commercialization of their AOC therapies, aimed at profoundly improving the lives of those affected by severe muscle diseases.