Avidity Biosciences, Inc. Stock Price, News & Analysis

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.

Avidity Biosciences (Nasdaq: RNA) announced its participation in the 29th Annual FSHD Society International Research Congress in Orlando, Florida, taking place on June 16-17, 2022. The company will present an oral presentation on AOC 1020, an Antibody Oligonucleotide Conjugate (AOC) for treating Facioscapulohumeral Muscular Dystrophy (FSHD) on June 17 at 9:50 AM ET. Additionally, a poster presentation will discuss a retrospective data analysis of inpatient admissions for FSHD patients, scheduled for June 17 from 1:00 PM to 3:00 PM ET.

Avidity Biosciences, Inc. (Nasdaq: RNA) has announced its first-quarter financial results for 2022, reporting a cash balance of $397 million. Collaboration revenue for the quarter was $1.8 million, a decline from $2.7 million in Q1 2021. R&D expenses increased to $27.7 million from $20.7 million, while G&A expenses rose to $8.6 million. The company is advancing its AOC 1001 MARINA trial and plans to initiate additional programs in the clinic by year-end.

Avidity Biosciences, Inc. (Nasdaq: RNA) has announced the expansion of Michael MacLean's role to Chief Financial and Chief Business Officer. His leadership has been crucial in guiding the company through its transition to a publicly-traded, clinical-stage biopharmaceutical firm. MacLean will oversee finance, business development, supply chain, and investor relations while advancing the company's innovative RNA therapeutic platform, Antibody Oligonucleotide Conjugates (AOCs). Avidity aims to initiate three rare disease programs in clinical trials by the end of 2022.

Avidity Biosciences, Inc. (Nasdaq: RNA) will present at the American Academy of Neurology 2022 Annual Meeting in Seattle, Washington, from April 2-7, 2022. Key oral presentations include findings on Antibody-Oligonucleotide Conjugates (AOCs) for treating Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1. Poster presentations will cover topics such as the patient journey in Facioscapulohumeral Muscular Dystrophy. Avidity aims to advance its clinical programs, with three in development by the end of 2022, focusing on RNA therapeutics to address previously untreatable diseases.

Avidity Biosciences, Inc. (Nasdaq: RNA) announced its participation at the 2022 MDA Clinical & Scientific Conference in Nashville, Tennessee, from March 13-16, 2022. The company will present an oral presentation on DUX4 siRNA Optimization for FSHD Treatment on March 16 and multiple poster presentations discussing real-world data analyses on patients with Facioscapulohumeral Muscular Dystrophy and Myotonic Dystrophy. Avidity is advancing three clinical programs, including its lead candidate AOC 1001 for Myotonic Dystrophy Type 1.

Avidity Biosciences (RNA) reported its fourth quarter and full year 2021 financial results on March 1, 2022, highlighting significant advances in RNA therapeutics. The company ended 2021 with $406 million in cash, bolstered by $175 million raised during the year. Avidity's lead candidate, AOC 1001, is undergoing clinical trials for myotonic dystrophy type 1, with additional programs (AOC 1020 and AOC 1044) expected to enter clinical development in 2022. Collaboration revenue increased to $9.3 million for 2021, up from $6.8 million in 2020.