Avidity Biosciences reports progress in DMD treatment trial

SAN DIEGO - Avidity Biosciences, Inc. (NASDAQ:RNA), a biotechnology company with a market capitalization of $3.74 billion, has announced positive topline data from its Phase 1/2 EXPLORE44 trial for del-zota, a potential treatment for Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping. The trial showed statistically significant improvements in dystrophin production and other biomarkers, with favorable safety and tolerability across different dose cohorts. InvestingPro data shows the company maintains a strong financial position with more cash than debt on its balance sheet.

The trial involved participants with DMD, a genetic disorder characterized by progressive muscle degeneration. Del-zota, an RNA therapeutic, is designed to skip exon 44 of the dystrophin gene, which could enable the production of a functional dystrophin protein. The study’s results, which were presented at the Muscular Dystrophy Association Clinical & Scientific Conference in Dallas, Texas, highlighted the consistent effects of del-zota at doses of 5 mg/kg every six weeks and 10 mg/kg every eight weeks.

Data from 26 participants in the completed EXPLORE44 trial and 38 in the ongoing Open-Label Extension (OLE) trial, as of January 22, 2025, showed that del-zota led to statistically significant increases in exon skipping and dystrophin production, with reductions in creatine kinase levels to near-normal values. These results were sustained over a year of treatment in the OLE trial.

Based on these findings, Avidity has selected the 5 mg/kg dose for its planned Biologics License Application (BLA) submission at the end of 2025. The company has received Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the U.S. Food and Drug Administration (FDA) for del-zota. Wall Street analysts maintain a bullish outlook, with price targets ranging from $48 to $96, suggesting significant upside potential despite the stock currently trading at $31.11.

Avidity is hosting an investor and analyst webcast event today to discuss the topline data from the trial. The company is also preparing for a potential U.S. launch of del-zota, which could be followed by other neuromuscular programs in its pipeline.

The positive trial data marks a significant milestone for Avidity and the DMD community, offering hope for a new treatment option. The company’s commitment to addressing rare neuromuscular diseases with its RNA therapeutics continues as it progresses towards regulatory submissions. While the company currently shows weak profit margins, its strong liquidity position with a current ratio of 15.73 provides financial flexibility for continued development. For detailed analysis and additional insights, including 7 more exclusive ProTips, check out the comprehensive research report available on InvestingPro. This report is based on a press release statement from Avidity Biosciences.

In other recent news, Avidity Biosciences has been the focus of several analyst firms, highlighting key developments in its clinical programs. BMO Capital Markets initiated coverage with an Outperform rating, citing the company’s potential in treating neuromuscular disorders and setting a price target of $72.00. Scotiabank also started coverage with a Sector Outperform rating and a $70.00 price target, emphasizing the promising portfolio of therapies in registrational studies that could become significant market players. Cantor Fitzgerald reaffirmed its Overweight rating and a $96.00 price target, noting the strong pipeline and potential peak sales of $5-10 billion for its drugs targeting myotonic dystrophy, FSHD, and DMD Exon44 patients.

H.C. Wainwright initiated coverage with a Buy rating and a $72.00 price target, highlighting the successful establishment of Avidity’s Antibody Oligonucleotide Conjugate platform and the progress of its clinical programs. The company’s Phase 3 HARBOR trial for myotonic dystrophy is on track, with primary endpoint results expected in the first half of 2026. Additionally, the FSHD program’s drug, Del-brax, has shown promise for accelerated approval due to its significant reduction in DUX-4 related gene expression. Avidity’s DMD program with Del-Zota has demonstrated favorable safety and efficacy, potentially meeting criteria for accelerated approval for Exon 44 patients. These recent developments underscore the company’s strategic advancements and potential impact in the market for rare diseases.

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