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Tuesday, Avidity Biosciences shares received a reaffirmed Buy rating and a $72.00 price target from H.C. Wainwright, following the release of promising Del-Zota trial data. The stock has already responded positively, posting a 14.37% gain over the past week, with analyst targets ranging from $48 to $96. According to InvestingPro data, the company appears slightly overvalued at its current price of $33.83. The data indicated a significant increase in dystrophin levels for exon-44 patients, which is seen as a positive indicator for the company’s DM1 and FSHD programs as well.
The Del-Zota trial data, which was shared on Tuesday, showcased a clean safety profile, considered a positive sign for not only Del-Zota but also for Avidity’s other programs. With a strong financial health score of 2.08 (Fair) on InvestingPro and a robust current ratio of 15.73, the company appears well-positioned to advance its clinical programs. The trial results demonstrated a nearly 25% increase in dystrophin levels for exon-44 patients, a notable achievement when compared to the ~1% levels observed in healthy muscle after nearly 3.5 years of eteplirsen treatment, according to a study by Lim et al.
Furthermore, the biomarker data from the trial suggested that the treatment could be advantageous for a broad range of patients, both ambulatory and non-ambulatory, across various age groups. The concentration of 200 nM of PMO with around 40% skipping also underscores the potential of Avidity’s platform, especially for future pipeline programs targeting heart muscles.
Another highlight from the trial was the impressive creatine kinase (CK) data. The results showed dramatic reductions in this biomarker post-treatment, with consistent data across crossover participants and regardless of their ambulatory status. These findings may have positive implications for the drug’s labeling and reimbursement prospects.
The consistency of the CK data was further supported by other muscle-relevant biomarkers such as AST, ALT, and myoglobin, all of which bolster the mechanism of action of the drug. With the complete data package, including clinical data from around 39 patients, expected to be shared at the end of the year, and with the regulatory path aligned with the FDA, Avidity Biosciences is on track for a BLA filing at the end of 2025, with approval anticipated in 2026. The stock has shown strong momentum with a 41.37% return over the past year. For deeper insights into RNA’s financial health, valuation metrics, and growth prospects, access the comprehensive Pro Research Report available exclusively on InvestingPro, along with 8 additional ProTips and extensive financial metrics.
In other recent news, Avidity Biosciences has reported positive topline data from its Phase 1/2 EXPLORE44 trial for del-zota, a potential treatment for Duchenne muscular dystrophy. The trial demonstrated statistically significant improvements in dystrophin production and biomarkers, with favorable safety and tolerability. Avidity plans to submit a Biologics License Application for del-zota by the end of 2025, following the trial’s promising results. The company has also received Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA for this treatment.
In terms of analyst activity, BMO Capital Markets initiated coverage of Avidity Biosciences with an Outperform rating, citing the company’s potential in addressing neuromuscular disorders. Similarly, Scotiabank (TSX:BNS) assigned a Sector Outperform rating, highlighting the potential for Avidity’s therapies to become blockbusters in markets lacking approved drugs. Cantor Fitzgerald maintained an Overweight rating, expressing confidence in Avidity’s pipeline and potential peak sales. H.C. Wainwright also initiated coverage with a Buy rating, emphasizing the de-risking of Avidity’s Antibody Oligonucleotide Conjugate platform.
These developments underscore Avidity’s strategic progress in advancing treatments for rare diseases, with several programs in the pipeline showing promise for regulatory approval. As the company prepares for potential commercialization and regulatory milestones, investor interest remains focused on its innovative therapeutic approaches.
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