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Investing.com -- Avidity Biosciences, Inc. (NASDAQ:RNA) stock rose 4% on Wednesday after the company 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 (DMD) in patients with mutations amenable to exon 44 skipping.
The designation is designed to expedite the development and review of drugs that treat serious conditions and show preliminary evidence of substantial improvement over existing therapies. Del-zota is currently being assessed in the Phase 2 EXPLORE44 Open-Label Extension trial for people with DMD44.
In the completed Phase 1/2 EXPLORE44 trial, del-zota demonstrated statistically significant increases in exon skipping, substantial increases in dystrophin production, and a significant reduction in creatine kinase levels to near normal, with favorable safety and tolerability profiles.
"Breakthrough Therapy designation further underscores the FDA’s appreciation for the significant potential of del-zota to address the underlying cause of DMD44 and the urgent need to bring innovative treatment options to the DMD community," said Steve Hughes, M.D., chief medical officer at Avidity.
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.
DMD is a rare genetic condition characterized by progressive muscle damage and weakness due to the loss of dystrophin protein. Del-zota is designed to deliver phosphorodiamidate morpholino oligomers to skeletal muscle and heart tissue to enable production of near-full length dystrophin.
The drug has also received Orphan designation from both the FDA and European Medicines Agency, as well as Rare Pediatric Disease and Fast Track designations from the FDA.
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