SAN DIEGO - Avidity Biosciences, Inc. (NASDAQ: RNA) has announced the commencement of a biomarker cohort for its Phase 1/2 FORTITUDE trial, which is evaluating delpacibart braxlosiran (del-brax/AOC 1020) in individuals with facioscapulohumeral muscular dystrophy (FSHD). The biopharmaceutical company, specializing in RNA therapeutics, is targeting a potential accelerated approval path with enrollment in the biomarker cohort expected to complete in the first half of 2025, and a functional cohort slated to begin in the same period.
Del-brax is the first investigational treatment aimed at addressing the root cause of FSHD by targeting the DUX4 gene, which is responsible for the disease. FSHD is a rare genetic disorder characterized by progressive muscle weakness, with no approved treatments currently available. The biomarker cohort will assess the impact of a 2 mg/kg dose of del-brax administered every six weeks on DUX4 regulated gene expression and circulating biomarker in patients aged 16-70.
Initial data from the trial has shown promising results, with del-brax demonstrating reductions in DUX4 regulated genes, decreases in novel circulating biomarker and creatine kinase, and trends of functional improvement at the four-month mark, along with favorable safety and tolerability. These findings were presented at the 31st Annual FSHD Society International Research Congress earlier this year.
The FORTITUDE trial is a randomized, placebo-controlled, double-blind study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of del-brax. The trial also includes an open-label extension study, FORTITUDE-OLE, offering long-term evaluation of the drug's safety and tolerability for participants who have completed the FORTITUDE study.
Avidity's del-brax has received Orphan designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), as well as Fast Track designation from the FDA. The company's mission is to profoundly improve lives by delivering new RNA therapeutics, with a focus on rare muscle diseases.
This article is based on a press release statement, presenting the initiation of the biomarker cohort as a key step in Avidity's strategy to potentially accelerate the approval process for del-brax. The company is advancing its clinical studies with urgency to provide a potential new treatment option for individuals living with FSHD.
In other recent news, Avidity Biosciences has seen significant progress in its clinical trials. The U.S. Food and Drug Administration (FDA) lifted the partial clinical hold on Avidity's drug candidate, del-desiran, paving the way for the continuation of the Phase 3 HARBOR trial. This decision followed positive preliminary results from a Phase 1/2 clinical trial for another therapeutic candidate, delpacibart zotadirsen.
Avidity Biosciences also announced a $250 million public offering of common stock, managed by Leerink Partners and TD Cowen, to support its clinical programs and advance its research and development. Goldman Sachs (NYSE:GS) and TD Cowen have reiterated their Buy rating on Avidity's stock, emphasizing the potential of its drugs del-brax and del-desiran, with projected peak sales of $2.7 billion and $4.0 billion, respectively.
Analysts from BofA Securities and Wells Fargo (NYSE:WFC) also maintained positive ratings on Avidity's stock, reflecting the recent developments. These include the FDA granting Breakthrough Therapy designation to Avidity's lead investigational drug, delpacibart etedesiran, for the treatment of myotonic dystrophy type 1. These are the latest developments in the company's journey.
InvestingPro Insights
Avidity Biosciences' (NASDAQ: RNA) recent announcement of the biomarker cohort for its FORTITUDE trial aligns with the company's focus on developing innovative RNA therapeutics. Despite the promising clinical progress, InvestingPro data reveals some financial challenges that investors should consider.
As of the last twelve months ending Q2 2024, Avidity reported revenue of $10.6 million, with a modest growth of 8.15%. However, the company's financials reflect the typical profile of a clinical-stage biopharmaceutical company investing heavily in research and development. This is evident in the negative gross profit of $220.7 million and an operating income margin of -2690.64%.
InvestingPro Tips highlight that Avidity "holds more cash than debt on its balance sheet" and "liquid assets exceed short term obligations," which are positive indicators for a company in the capital-intensive biotech sector. These factors provide financial flexibility as Avidity advances its clinical programs, including the del-brax trial for FSHD.
The market appears to be optimistic about Avidity's potential, with the stock showing a remarkable 775.64% price return over the past year. This aligns with the InvestingPro Tip noting a "high return over the last year." However, investors should be aware that the company is "not profitable over the last twelve months" and "net income is expected to drop this year," reflecting the ongoing investments in clinical development.
For those interested in a deeper analysis, InvestingPro offers 10 additional tips for Avidity Biosciences, providing a more comprehensive view of the company's financial health and market position.
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