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SAN DIEGO - The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to Avidity Biosciences’ (NASDAQ:RNA) delpacibart zotadirsen (del-zota) for treating Duchenne muscular dystrophy in patients with mutations amenable to exon 44 skipping, the company announced Wednesday. With a market capitalization of $4.04 billion, Avidity maintains a strong financial position, holding more cash than debt on its balance sheet and a healthy current ratio of 16.91, according to InvestingPro data.
Del-zota is designed to deliver phosphorodiamidate morpholino oligomers to skeletal muscle and heart tissue to enable production of near-full length dystrophin by skipping exon 44 of the dystrophin gene.
The drug is currently being evaluated in the Phase 2 EXPLORE44 Open-Label Extension trial. In the completed Phase 1/2 EXPLORE44 trial, del-zota demonstrated statistically significant increases in exon skipping, substantial increases in dystrophin production, and significant reductions in creatine kinase levels to near normal, according to the company.
"Breakthrough Therapy designation further underscores the FDA’s appreciation for the significant potential of del-zota to address the underlying cause of DMD44," said Steve Hughes, chief medical officer at Avidity. Seven analysts have recently revised their earnings expectations upward for the upcoming period, suggesting growing confidence in the company’s development pipeline, as revealed by InvestingPro analysis.
Avidity plans to present topline and functional data from the ongoing Phase 2 trial in the fourth quarter of 2025 and remains on track for a planned Biologics License Application submission by the end of 2025.
Duchenne muscular dystrophy is a rare genetic condition characterized by progressive muscle damage and weakness due to the loss of dystrophin protein, typically beginning at a very young age. It affects approximately one in 3,500 to 5,000 boys worldwide.
Del-zota has previously received Orphan designation from both the FDA and European Medicines Agency, as well as Rare Pediatric Disease and Fast Track designations from the FDA.
The Breakthrough Therapy designation is designed to expedite development and review of drugs intended to treat serious conditions where preliminary clinical evidence indicates substantial improvement over available therapies.
This information is based on a press release statement from Avidity Biosciences. While current InvestingPro analysis indicates the stock is trading near its Fair Value, investors seeking deeper insights can access comprehensive Pro Research Reports covering 1,400+ top stocks, including detailed analysis of Avidity’s financial health, market position, and growth potential. The company’s next earnings report is scheduled for August 12, 2025, which could provide further updates on del-zota’s development progress.
In other recent news, Avidity Biosciences announced that its drug candidate, delpacibart zotadirsen, received Breakthrough Therapy designation from the U.S. Food and Drug Administration. This status is intended to expedite the development of drugs that show significant improvement over existing therapies, particularly for serious conditions like Duchenne muscular dystrophy. The drug is currently undergoing evaluation in a Phase 2 trial. Additionally, Avidity Biosciences has caught the attention of several investment firms. Goldman Sachs resumed coverage with a Buy rating, citing a positive outlook on the company’s platform technology despite potential regulatory uncertainties. Cantor Fitzgerald reiterated its overweight rating, expressing confidence in Avidity’s leadership and their work on rare muscle diseases. Wolfe Research initiated coverage with an outperform rating, noting that while the stock may align with broader biotech trends, specific company catalysts could impact movement. Lastly, Avidity Biosciences held its 2025 Annual Meeting of Stockholders, where three Class II directors were elected to serve until 2028.
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