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Friday - Cantor Fitzgerald reaffirmed its Overweight rating and $96.00 price target for Avidity Biosciences (NASDAQ:RNA) stock, representing significant upside from the current price of $29.08. According to InvestingPro data, analyst targets range from $51 to $96, reflecting strong confidence in the company’s potential. The firm’s analyst, Eric Schmidt, expressed continued confidence in the company after a recent visit with the Avidity team in San Diego during Rare Disease Week. Schmidt noted the company’s strong pipeline, which includes potential first-in-class and best-in-class drugs for myotonic dystrophy, FSHD, and DMD Exon44 patients, with projected peak sales that could reach $5-10 billion.
Avidity Biosciences is advancing its Phase 3 HARBOR trial for myotonic dystrophy, with enrollment expected to be completed by mid-2023. The company’s strong financial position, with a current ratio of 15.73 and more cash than debt on its balance sheet, provides ample runway for trial completion. The trial’s 30-week primary endpoint results are anticipated in the first half of 2026, with the company planning to initially release only top-line data to maintain the integrity of the full 54-week placebo-controlled study.
Additionally, Avidity Biosciences is preparing to publish full Phase 1/2 data from the MARINA trial in a leading medical journal soon. The company believes that recent competitive datasets in DM1 (myotonic dystrophy type 1) support the biological basis and the effect of therapeutic intervention, although they do not show differentiation. Schmidt highlighted Avidity’s lead time to market and the robust design of its global Phase 3 trial as key factors that could set it apart from competitors. Get deeper insights into RNA’s financial health and growth potential with InvestingPro, which offers exclusive analysis and 7 additional ProTips for informed investment decisions.
Avidity Biosciences’ focus on developing treatments for rare diseases has positioned it as a top pick for Cantor Fitzgerald, with the potential to significantly impact the market for these conditions. With a market capitalization of $3.47 billion and revenue growth of 14% in the last twelve months, the company’s strategic approach to clinical trial design and data disclosure is intended to strengthen its position in the competitive landscape.
In other recent news, Avidity Biosciences has garnered attention with H.C. Wainwright initiating coverage on the company with a Buy rating and a price target of $72.00. This decision was influenced by the successful establishment and de-risking of Avidity’s Antibody Oligonucleotide Conjugate (AOC) platform, alongside progress in three clinical programs targeting rare diseases. The analyst highlighted the validation of Avidity’s myotonic dystrophy program through the MARINA and MARINA OLE studies, where the drug Del-desiran showed safety and sustained improvement in clinical endpoints. Additionally, the Phase 3 HARBOR trial is actively enrolling participants and remains on schedule.
In the FSHD program, the drug Del-brax has demonstrated safety and potential for accelerated approval, with significant reductions in DUX-4 related gene expression. The DMD program’s Del-Zota has shown favorable safety and efficacy, with dystrophin expression nearing normal levels, potentially meeting the criteria for accelerated approval for Exon 44 patients. Avidity Biosciences is also expanding its pipeline with two new programs in precision cardiology. Looking ahead, updates are anticipated in 2025 for the FORTITUDE biomarker cohort and regulatory steps for Del-zota.
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