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SAN DIEGO - Avidity Biosciences, Inc. (NASDAQ:RNA), which has seen its stock surge over 24% year-to-date and maintains a market capitalization of $4.37 billion, announced Monday it has completed enrollment in its global Phase 3 HARBOR clinical trial of delpacibart etedesiran (del-desiran) for myotonic dystrophy type 1 (DM1), with topline data expected in the second quarter of 2026.
The company plans to submit marketing applications for del-desiran in the United States, European Union, and Japan starting in the second half of 2026. If approved, del-desiran could become the first globally approved drug for DM1, a rare, hereditary, progressive neuromuscular disease. According to InvestingPro data, seven analysts have recently revised their earnings expectations upward for the upcoming period, suggesting growing confidence in the company’s development pipeline.
The HARBOR trial enrolled approximately 150 patients aged 16 and older across 40 global sites. Participants receive either del-desiran or placebo every eight weeks in a 1:1 ratio. The primary endpoint measures video hand opening time, which assesses myotonia, a hallmark symptom of DM1.
Del-desiran, an Antibody Oligonucleotide Conjugate (AOC), is designed to address the underlying genetic cause of DM1 by reducing levels of toxic DMPK mRNA. The drug has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA, along with Orphan designation from European and Japanese regulators.
"Completing enrollment in our Phase 3 HARBOR study marks a significant step toward bringing the first potentially approved drug for DM1 to people around the world," said Steve Hughes, Chief Medical Officer at Avidity, in the press release.
The company also plans to share updates from its ongoing MARINA-OLE trial of del-desiran, including long-term efficacy and safety data, in the fourth quarter of 2025.
Currently, there are no approved treatments for DM1, which affects multiple body systems and requires lifelong care. While the company maintains a strong financial position with more cash than debt and a current ratio of 16.91, InvestingPro analysis indicates the stock is trading slightly above its Fair Value. Investors seeking deeper insights can access the comprehensive Pro Research Report, available exclusively to InvestingPro subscribers, which covers detailed financial analysis and growth prospects for RNA among 1,400+ top US stocks.
In other recent news, Avidity Biosciences announced that its drug candidate delpacibart zotadirsen (del-zota) received Breakthrough Therapy designation from the U.S. Food and Drug Administration for treating Duchenne muscular dystrophy in patients with mutations amenable to exon 44 skipping. This designation aims to expedite the development and review of drugs that treat serious conditions and show preliminary evidence of substantial improvement over existing therapies. Currently, del-zota is being assessed in the Phase 2 EXPLORE44 Open-Label Extension trial.
In terms of analyst activity, Goldman Sachs resumed coverage on Avidity Biosciences with a Buy rating and a price target of $55.00, citing a positive outlook on the company’s platform technology. Cantor Fitzgerald maintained its overweight rating and $96.00 price target, expressing confidence in the company’s leadership and their work in developing treatments for rare muscle diseases. Wolfe Research also initiated coverage with an outperform rating and a $55.00 price target, noting that upcoming company-specific catalysts may not significantly move the stock in the short term. These developments reflect a broad interest and confidence in Avidity Biosciences’ ongoing projects and future potential.
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