Avidity Biosciences, Inc. Stock Price, News & Analysis

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.

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.