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Tuesday, shares of Avidity Biosciences (NASDAQ:RNA) remained in focus following Citi’s reiteration of a Buy rating and a $70.00 price target. The stock, currently trading at $32.35, has seen a notable 14.37% gain over the past week. According to InvestingPro data, analyst targets range from $48 to $96, with a strong Buy consensus. The endorsement comes after the company reported positive results from its phase 1/2 EXPLORE44 trial for a treatment targeting Duchenne muscular dystrophy exon 44 (DMD44).
The trial evaluated Avidity’s proprietary AOC (antibody oligonucleotide conjugates) del-zota in 24 patients. Findings demonstrated significant exon skipping and dystrophin production, with 37% exon skipping at a 5mg/kg dosage and 43% at 10 mg/kg. Dystrophin production was reported at 25% for the 5mg/kg dosage and 26% for the 10mg/kg dosage.
These results indicate that Avidity Biosciences is on schedule with its plans to file a Biologics License Application (BLA) for accelerated approval of del-zota at the chosen dose of 5mg/kg every six weeks, aiming for the fourth quarter of 2025. The company, with a market capitalization of $4 billion and a strong current ratio of 15.73, has also started preparing for the initial launch of del-zota. InvestingPro analysis shows the company holds more cash than debt on its balance sheet, positioning it well for upcoming developments.
Citi’s analysis suggests that the launch of del-zota could establish a commercial foundation that may expand into larger neuromuscular markets. This includes potential treatments like del-desiran for myotonic dystrophy type 1 (DM1) and del-brax for facioscapulohumeral muscular dystrophy (FSHD).
The firm’s optimism is backed by recent initiation notes and the latest trial data, which bolster confidence in the clinical impact of Avidity’s AOC programs. Citi continues to classify the stock as Buy/High Risk, maintaining a $70 price target. For deeper insights into RNA’s valuation and growth prospects, investors can access comprehensive analysis and 8 additional ProTips through InvestingPro’s detailed research reports, available for over 1,400 US stocks.
In other recent news, Avidity Biosciences announced promising results 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 data revealed statistically significant improvements in dystrophin production and reductions in creatine kinase levels, with favorable safety and tolerability across different dose cohorts. This progress supports Avidity’s plans to submit a Biologics License Application by the end of 2025, with potential U.S. launch plans underway. Analyst firms have responded positively, with H.C. Wainwright maintaining a Buy rating and a $72.00 price target, while BMO Capital Markets initiated coverage with an Outperform rating and a similar price target. Scotiabank (TSX:BNS) also started coverage with a Sector Outperform rating and a $70.00 target, citing the company’s promising therapeutic portfolio. Cantor Fitzgerald reaffirmed its Overweight rating and set a higher price target of $96.00, expressing confidence in Avidity’s strong pipeline and potential market impact. The company’s progress in addressing rare neuromuscular diseases with RNA therapeutics has garnered attention, especially with potential regulatory approvals on the horizon.
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