Avidity Biosciences, Inc. Stock Price, News & Analysis

Avidity Biosciences has initiated the Phase 1/2 EXPLORE44 clinical trial for AOC 1044, targeting Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping. AOC 1044 is the first antibody oligonucleotide conjugate engineered to deliver phosphorodiamidate morpholino oligomers to treat DMD. This marks a significant step forward as there are currently no approved therapies for DMD44. The trial aims to assess the safety and efficacy of AOC 1044 in healthy volunteers and DMD patients, with enrollment expected for 64 participants.

Avidity Biosciences, Inc. (Nasdaq: RNA) announced it will present five poster presentations at the 27th International Hybrid Annual Congress of the World Muscle Society from October 11-15, 2022, in Halifax, Nova Scotia. Key presentations include the MARINA™ study for myotonic dystrophy type 1 and data on facioscapulohumeral muscular dystrophy (FSHD) and Duchenne muscular dystrophy (DMD). Notable presentations are scheduled for October 14, including a Phase 1/2 trial of AOC 1001 in adults with myotonic dystrophy type 1.

Avidity announced the initiation of the Phase 1/2 FORTITUDE clinical trial for AOC 1020, targeting facioscapulohumeral muscular dystrophy (FSHD), affecting 16,000-38,000 individuals in the U.S. AOC 1020 aims to address the underlying cause of FSHD by targeting the DUX4 gene. This is Avidity's second siRNA AOC to enter clinical development, adding to their portfolio of three rare disease programs, including myotonic dystrophy type 1 and Duchenne muscular dystrophy. The trial will evaluate safety and tolerability, with a preliminary safety assessment expected in Q4 2022.

Avidity Biosciences (Nasdaq: RNA) announced a partial clinical hold by the FDA on new participant enrollment in the Phase 1/2 MARINA™ trial of AOC 1001 for myotonic dystrophy type 1 (DM1) due to a serious adverse event in a participant. Around 40 participants continue in the ongoing MARINA and MARINA-OLE™ trials, but no new enrollments are permitted until the issue is resolved. Avidity received IND clearance for AOC 1020 for facioscapulohumeral muscular dystrophy (FSHD) and AOC 1044 for Duchenne muscular dystrophy (DMD), advancing these programs into clinical trials.

Avidity Biosciences (Nasdaq: RNA) is poised to advance three muscular dystrophy programs into clinical trials by the end of 2022. In conjunction with National Muscular Dystrophy Awareness Month, the company will engage with patient communities and partake in several events, including the MDF Annual Conference and the FSHD Society's Walk and Roll. The lead program, AOC 1001, targets myotonic dystrophy type 1. Avidity's innovative Antibody Oligonucleotide Conjugates (AOCs™) aim to address previously untreatable rare diseases.

Avidity Biosciences (Nasdaq: RNA) announced its participation in two key investor conferences: the 2022 Wells Fargo Healthcare Conference on September 7 at 1:20 pm ET and the Chardan 6th Annual Genetic Medicines Conference on October 4 at 11:30 am ET. Interested parties can access live webcasts and replays via Avidity's 'Events and Presentations' page. The company focuses on innovative RNA therapeutics, specifically Antibody Oligonucleotide Conjugates (AOCs™), with three programs expected in clinical development by the end of 2022, including AOC 1001 for myotonic dystrophy.

Avidity Biosciences (RNA) reported Q2 2022 financial results, announcing progress on its three AOC programs targeting rare diseases: myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne muscular dystrophy (DMD). The company is on track to have these programs in the clinic by year-end. Avidity holds over $400 million in cash, expected to fund operations through 2024. R&D expenses surged to $39.8 million, reflecting advancements in their pipeline, while collaboration revenue saw a decline to $2.2 million, attributed to timing issues.

Avidity Biosciences (Nasdaq: RNA) has initiated the MARINA-OLE study, allowing patients enrolled in the Phase 1/2 MARINA trial to receive ongoing treatment with AOC 1001 for myotonic dystrophy type 1 (DM1). This follows the FDA's Fast Track and Orphan Designations for AOC 1001. The MARINA trial, which aims to evaluate safety and tolerability, is expected to report preliminary data by Q4 2022. DM1, a progressive and potentially fatal neuromuscular disease, currently lacks disease-modifying treatments. AOC 1001 targets DMPK mRNA, addressing the root cause of DM1.

Avidity Biosciences (Nasdaq: RNA) announced at the FSHD IRC in Orlando, Florida, preclinical data demonstrating that AOC 1020 can prevent muscle weakness by reducing DUX4 expression, a critical factor in the progression of Facioscapulohumeral muscular dystrophy (FSHD). With approximately 16,000-38,000 patients in the U.S. lacking approved therapies, AOC 1020 is on track for clinical development by the end of 2022. The data showed robust dose-dependent downregulation of DUX4 and prevention of muscle weakness in mouse models, highlighting the potential of AOC 1020 for treating FSHD.