SAN DIEGO – Avidity Biosciences, Inc. (NASDAQ:RNA), a biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold on its investigational drug, delpacibart etedesiran.
The FDA's decision is based on the company's submission of additional information requested by the agency. Avidity Biosciences has not disclosed the nature of the FDA's initial concerns or the additional information provided. However, the lifting of the hold signifies that the FDA is satisfied with the company's response and that the drug is considered safe to proceed to the next stages of clinical testing.
The announcement is a significant step for Avidity Biosciences, which focuses on the development of a new class of therapeutics based on its proprietary Antibody Oligonucleotide Conjugate (AOC™) platform. Delpacibart etedesiran is part of the company's pipeline of drugs designed to treat diseases with unmet medical needs.
The news may be of interest to investors and the pharmaceutical industry, as clinical holds can delay drug development and result in increased costs. Resuming clinical trials could potentially lead to advancements in treatment options for patients and financial implications for the company.
Avidity Biosciences is headquartered in San Diego, California, and operates under the jurisdiction of Delaware. The company was previously known as Avidity Biosciences LLC and Avidity NanoMedicines LLC before its name changes in 2016 and 2014, respectively.
This update is based on a press release statement filed with the Securities and Exchange Commission on Thursday. The company's stock is listed on The Nasdaq Global Market under the ticker symbol RNA.
In other recent news, Avidity Biosciences has been making significant strides with its leading drug candidates. Goldman Sachs initiated coverage on the biotech company, highlighting the potential of its drugs del-brax and del-desiran, which are being developed for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1 respectively. The investment firm projected peak sales of $2.7 billion for del-brax and $4.0 billion for del-desiran, with success probabilities estimated at 60% and 75% respectively.
Avidity Biosciences also announced a $250 million public offering of common stock, intending to support its clinical programs and advance its research and development. Leerink Partners and TD Cowen will act as joint bookrunning managers for the offering.
In addition, the company reported positive preliminary results from a Phase 1/2 clinical trial for delpacibart zotadirsen, a potential treatment for Duchenne muscular dystrophy. The trial showed significant increases in dystrophin production and exon 44 skipping, along with a substantial reduction in creatine kinase levels.
Analysts from BofA Securities and Wells Fargo have shown interest in these developments, raising their price targets and maintaining positive ratings on Avidity's stock. Lastly, the U.S. Food and Drug Administration granted Breakthrough Therapy designation to Avidity's lead investigational drug, delpacibart etedesiran, for the treatment of myotonic dystrophy type 1. These are the recent developments in the company's journey.
InvestingPro Insights
The lifting of the FDA's partial clinical hold on Avidity Biosciences' investigational drug is reflected in the company's recent market performance. According to InvestingPro data, RNA has seen a significant 76.88% price total return over the past six months, with a strong 26.63% return in the last three months alone. This positive momentum has brought the stock to trade at 95.37% of its 52-week high, indicating investor optimism surrounding the company's prospects.
InvestingPro Tips highlight that 6 analysts have revised their earnings upwards for the upcoming period, suggesting growing confidence in Avidity's near-term financial performance. Additionally, the company holds more cash than debt on its balance sheet, which could provide financial flexibility as it resumes clinical trials for delpacibart etedesiran.
However, it is important to note that Avidity Biosciences is not currently profitable, with a negative P/E ratio of -21.86 for the last twelve months as of Q2 2024. The company's revenue for the same period stands at $10.6 million, with a concerning gross profit margin of -2082.31%. These figures underscore the early-stage nature of the company and the importance of successful clinical trials for its future.
For investors seeking a more comprehensive analysis, InvestingPro offers 14 additional tips for RNA, providing a deeper understanding of the company's financial health and market position.
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