SAN DIEGO – Avidity Biosciences Inc. (NASDAQ:RNA), a biopharmaceutical company, reported positive preliminary results from a Phase 1/2 clinical trial for delpacibart zotadirsen (AOC 1044), a potential treatment for Duchenne muscular dystrophy (DMD).
The trial, named EXPLORE44™, demonstrated significant increases in dystrophin production and exon 44 skipping, along with a substantial reduction in creatine kinase levels, a marker of muscle damage, in patients with DMD mutations amenable to exon 44 skipping.
Delpacibart zotadirsen, also known as del-zota, is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to promote dystrophin production by skipping exon 44 of the dystrophin gene. The study showed a 37% increase in exon 44 skipping and up to 66% skipping with a 5 mg/kg dose at four months.
Additionally, it restored total dystrophin up to 54% of normal and reduced creatine kinase levels to near normal with greater than an 80% reduction compared to baseline after three doses of 5 mg/kg.
The data also indicated favorable safety and tolerability of the drug, with most treatment-emergent adverse events being mild or moderate. The trial involved 25 participants across two dose levels, with 10 participants in the 5 mg/kg cohort assessed for muscle delivery, exon skipping, dystrophin production, and creatine kinase levels.
Del-zota has been granted Orphan designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), as well as Rare Pediatric and Fast Track designations by the FDA. Avidity’s President and CEO, Sarah Boyce, expressed optimism about expediting the regulatory path for del-zota based on these findings.
DMD is a genetic disorder characterized by progressive muscle degeneration due to the absence of dystrophin, a protein that protects muscle cells. It predominantly affects boys, with an incidence of one in 3,500 to 5,000 male births globally.
The company is also hosting a virtual investor and analyst event today to discuss the trial data. These findings are based on a press release statement from Avidity Biosciences.
In other recent news, Avidity Biosciences has been the center of attention due to promising developments in its clinical programs and corporate structure.
The biopharmaceutical company announced positive early-stage results from its FSHD treatment study, leading to an updated price target from BofA Securities. The firm raised its expectation from $40.00 to $45.00 while maintaining a Buy rating on Avidity’s stock.
Avidity also announced plans for a $300 million public offering of its common stock, aiming to fund further development of its clinical programs and research.
In addition, Wells Fargo reaffirmed its Overweight rating on Avidity Biosciences, maintaining a steady price target of $50.00. Cantor Fitzgerald also initiated coverage on Avidity Biosciences with an Overweight rating, indicating potential for outperformance.
The company also received Breakthrough Therapy designation from the U.S. Food and Drug Administration for its lead investigational drug, delpacibart etedesiran, for the treatment of myotonic dystrophy type 1.
Furthermore, Avidity Biosciences expanded its board of directors with the appointment of Simona Skerjanec, a pharmaceutical industry veteran. These are the recent developments in the company’s journey.
InvestingPro Insights
Avidity Biosciences Inc. (NASDAQ:RNA) has been capturing the attention of the biopharmaceutical industry with its promising clinical trial results for delpacibart zotadirsen, aimed at treating Duchenne muscular dystrophy. As the company progresses, investors and analysts are keeping a keen eye on its financial health and market performance. According to InvestingPro data, Avidity Biosciences holds a market capitalization of $4.45 billion, indicating significant investor confidence in its potential. Despite the company’s innovative strides in drug development, it’s important to note that it has a negative gross profit margin of -1832.24% for the last twelve months as of Q1 2024, underscoring challenges in profitability that are also echoed by analysts who do not anticipate the company to be profitable this year.
On a more positive note, Avidity Biosciences has demonstrated a strong return over the last three months, with a 54.5% price total return, and an even more impressive six-month price uptick of 213.63%. This market performance is a testament to the optimism surrounding their clinical advancements. Additionally, an InvestingPro Tip highlights that the company holds more cash than debt on its balance sheet, which is a solid indicator of financial stability and could provide the necessary resources to support ongoing research and development efforts.
For those interested in a deeper analysis, InvestingPro offers a wealth of additional tips—currently listing 11 for Avidity Biosciences—that provide further insights into the company’s financial metrics and market potential. These can be found by exploring the detailed reports available on InvestingPro’s dedicated page for Avidity Biosciences at https://www.investing.com/pro/RNA.
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