Shares of Avidity Biosciences Inc. (NASDAQ:RNA) dropped 15% March 30 as the company disclosed details of the serious adverse event (SAE) that prompted a partial clinical hold in September 2023 on the phase I/II trial testing AOC-1001 in adults with myotonic dystrophy type 1 (DM1).
A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”
A serious adverse event (SAE) in one participant has led the U.S. FDA to place a partial clinical hold on Avidity Biosciences Inc.’s lead program. The action is centered on the phase I/II Marina study of AOC-1001, an antibody oligonucleotide conjugate for treating myotonic dystrophy type 1, the most common form of muscular dystrophy in adults.
As Neubase Therapeutics Inc. gears up to unveil preclinical data from its myotonic dystrophy type 1 (DM1) effort during the MDA Clinical & Scientific Conference later this month, investors are watching other programs moving through pipelines – but none of the research, except for that underway by Avidity Biosciences Inc., has entered the clinic.
Although the general markets have bounced around in June and the BioWorld Biopharmaceutical index has dipped almost 5% in the period, the uncertain capital environment has not affected investor appetite for biopharma IPOs.
In a big day of setting up IPOs for launch, the charge is being led by Royalty Pharma, a buyer of biopharmaceutical royalties and an industry funder, which is aiming at a $2 billion offering. That massive number is more than half of the total biotech offerings brought in through May.
Avidity Biosciences Inc. CEO Sarah Boyce told BioWorld it's "a little too soon" to say when the company's lead program in myotonic dystrophy type I might reach the clinic, but the firm's $100 million series C financing will help with that effort and the portfolio in rare muscle diseases, as the antibody-oligonucleotide conjugate (AOC) platform undergoes further development.
