Avidity Biosciences, Inc. (NASDAQ: RNA) is engaged as a biopharmaceutical company, which is focused on developing a new class of RNA therapies called Antibody Oligonucleotide Conjugates (AOCs). Shares of the biopharma company are surging 57% through afternoon trading on Wednesday, December 14, 2022. Over the past three months, Avidity Biosciences has seen average daily volume of 620,570 shares. However, volume of 33.1 million shares or dollar volume of around $571.64 million, has already exchanged hands through afternoon trading.
Shares of Avidity Biosciences are rallying after the company announces positive results from its Phase 1/2 MARINA trial of AOC 1001, the company’s leading clinical program using its AOC platform to treat myotonic dystrophy type 1 (DM1). DM1 is an underrecognized neuromuscular disease that can be fatal and has no approved therapies.
The trial was a major breakthrough because it demonstrated for the first time the ability to successfully target delivery of RNA into a muscle, which is a major advancement in the field of RNA therapeutics. Furthermore, the successful delivery of siRNA into muscle also serves as a proof-of-concept for the company’s AOC platform.
The clinical trial was randomized, double-blind and placebo-controlled, which was the first in-human data to determine safety and tolerability in 38 enrolled patients and key biomarkers in 19 participants. The initial assessment included biomarker data six weeks after dosing. Enrolled patients received a single dose of 1mg/kg AOC 1001, two doses of 2mg/kg AOC 1001 or a placebo.
Patients treated with AOC 1001 demonstrated meaningful DMPK reduction in all participants treated with the clinical treatment. The clinical data found an average reduction of 45% in DMPK after only one dose of 1 mg/kg or two doses of 2 mg/kg of AOC 1001. Furthermore, the data showed a splicing improvement of 31% in muscle-specific genes. The strong splicing activity shows AOC 1001 is working in the nucleus.
Safety and tolerability showed that there were only mild or moderate adverse events. Aside from a single participant who experienced adverse event from the 4 mg/kg treatment group.. Furthermore, early signs of clinical activity demonstrated improvement in myotonia in some patients.
“We are very pleased with this early data set of AOC 1001 from the MARINA trial. We have demonstrated the cascade of delivery to muscle, DMPK reduction and splicing improvements with AOC 1001 and are seeing early signs of clinical activity with improvement in myotonia, just weeks after only one or two doses of AOC 1001,” said Sarah Boyce, president and chief executive officer at Avidity. “AOC 1001 has the potential to deliver on the promise of the AOC platform and significantly impact the underlying disease mechanism of DM1, a devastating disease where there are currently no approved therapies. We look forward to sharing top-line data from the MARINA trial in 2023 and advancing our other clinical programs for the treatment of DMD and FSHD.”
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