Avidity Biosciences, Inc. (NASDAQ: RNA) is witnessing a significant stock price rise during the current market session. As of the most recent update, RNA stock has climbed 21.75% to $35.20, driven by encouraging data from its clinical trials.
Positive Data From Phase 1/2 FORTITUDE Trial
Avidity Biosciences (RNA) announced favorable initial data for AOC 1020 from the Phase 1/2 FORTITUDE trial. The data revealed unprecedented and consistent reductions exceeding 50% in DUX4-regulated genes, alongside trends of functional improvement and favorable safety and tolerability in patients with facioscapulohumeral muscular dystrophy (FSHD).
The company plans to expedite the initiation of registrational cohorts within the FORTITUDE study. Furthermore, Avidity announced “delpacibart braxlosiran,” which may be shortened to “del-brax,” as the authorized worldwide nonproprietary name for AOC 1020.
Impact On FSHD Treatment Landscape
Del-brax stands out as the first investigational therapy specifically designed to address the underlying cause of FSHD, a rare hereditary disorder characterized by relentless muscle function loss, significant pain, fatigue, and progressive disability. Currently, there are no approved therapies for FSHD.
The early data indicating consistent reductions in DUX4-regulated genes and functional improvements are highly promising, suggesting that del-brax could potentially alter the disease trajectory for FSHD patients.
Presentation At FSHD Society International Research Congress
The initial data for AOC 1020 will be presented at the 31st Annual FSHD Society International Research Congress, scheduled for June 13-14, 2024, in Denver, Colorado. Avidity Biosciences, recognizing the urgent need for effective treatments for FSHD, is focusing on accelerating its registrational plans. By targeting the root cause of FSHD, del-brax holds the promise of being a first-in-class, best-in-class therapy.
This development marks Avidity Biosciences’ third rare muscle disease program to demonstrate delivery to muscle and target engagement, and its second therapy showing signs of functional improvement in patients with rare neuromuscular diseases. These advancements underscore the potential of Avidity’s AOC platform to revolutionize RNA therapeutics and significantly improve patients’ lives.
