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Avidity Biosciences Advances FSHD Therapy Trial From Investing.com

SAN DIEGO – Avidity Biosciences, Inc. (NASDAQ: RNA) announced the initiation of a biomarker cohort for its Phase 1/2 FORTITUDE study. This study is evaluating delpacibart braxlosiran (del-brax/AOC 1020) in patients with fascioscopiculohumeral muscular dystrophy (FSHD). The biopharmaceutical company specializing in RNA therapeutics is aiming for an accelerated approval path. Recruitment for the biomarker cohort is expected to be completed in the first half of 2025, and a functional cohort is planned to start in parallel.

Del-brax is the first treatment approach to address the root cause of FSHD by targeting the disease-causing DUX4 gene. FSHD is a rare hereditary disease characterized by progressive muscle loss and for which there is currently no approved treatment. The biomarker cohort will examine the effect of a dose of 2 mg/kg del-brax given every six weeks on DUX4-regulated gene expression and circulating biomarkers in patients aged 16-70 years.

Initial study data shows promising results: after four months, reductions in the DUX4-regulated gene, decreases in novel circulating biomarkers and creatine kinase, and trends in functional improvement were observed. In addition, del-brax showed a favorable safety and tolerability profile. These findings were presented on January 31 at the FSHD Society International Research Congress earlier this year.

The FORTITUDE study is designed as a randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of del-brax. In addition, the open extension study FORTITUDE-OLE is ongoing, which enables a long-term assessment of the safety and tolerability of the active ingredient in participants who have completed the FORTITUDE study.

Avidity's del-brax has been granted orphan drug designation by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). It also received Fast Track designation from the FDA. The company's goal is to sustainably improve the lives of patients with rare muscle diseases by developing new RNA therapeutics.

This article is based on a press release and represents the initiation of the biomarker cohort as an important milestone in Avidity's strategy to potentially accelerate the approval process for del-brax. The company is aggressively advancing its clinical trials to offer FSHD patients a new treatment option in a timely manner.

In other recent developments, Avidity Biosciences recorded significant progress in its clinical trials. The FDA lifted the partial clinical hold on del-desiran drug candidates, paving the way for the Phase 3 HARBOR trial to continue. This decision followed positive preliminary results from a Phase 1/2 trial for another therapeutic candidate, delpacibart zotadirsen.

Avidity Biosciences also announced a $250 million public offering of common stock led by Leerink Partners and TD Cowen. The funds will be used to support the clinical program and promote research and development. Goldman Sachs and TD Cowen reiterated their buy rating on Avidity shares and underlined the potential of the active ingredients del-brax and del-desiran with forecast peak sales of $2.7 billion and $4.0 billion, respectively.

Analysts at BofA Securities and Wells Fargo also gave Avidity shares positive ratings, reflecting recent developments. This also includes the granting of breakthrough therapy status by the FDA to Avidity's lead compound delpacibart etedesiran for the treatment of myotonic dystrophy type 1. These are the latest developments in Avidity's company history.

Investing Pro Insights

Avidity Biosciences' (NASDAQ: RNA) recent biomarker cohort announcement for the FORTITUDE study underscores the company's focus on innovative RNA therapeutics. Despite promising clinical advances, InvestingPro data reveals some financial challenges that investors should keep in mind.

For the trailing twelve months through the second quarter of 2024, Avidity generated revenue of $10.6 million with moderate growth of 8.15%. The financial metrics reflect the typical profile of a clinical biopharma company that invests heavily in research and development. This is reflected in a negative gross profit of $220.7 million and an operating profit margin of -2,690.64%.

InvestingPro Tips positively highlights that Avidity “holds more cash than debt on its balance sheet” and “cash assets exceed short-term liabilities.” These are favorable indicators for a company in the capital-intensive biotech sector and provide financial flexibility to advance clinical programs such as the del brax trial for FSHD.

The market appears optimistic about Avidity's potential, reflected in an impressive share price return of 775.64% over the past year. This is in line with the InvestingPro tip, which states a “high return in the last year”. However, investors should note that the company

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