Avidity Biosciences reported that the final results of its Phase 1/2 MARINA trial of del‑desiran (del‑pacibart etedesiran) were published in the February 19 issue of the New England Journal of Medicine. The study enrolled 38 adults with myotonic dystrophy type 1 and demonstrated a 40 % mean reduction in DMPK mRNA, along with measurable improvements in myotonia, muscle function, and patient‑reported outcomes.
Safety data from the trial were encouraging, with del‑desiran being well tolerated and most adverse events classified as mild or moderate. Functional assessments showed reductions in missplicing and gains in hand grip strength and mobility, underscoring the therapeutic potential of the antibody‑oligonucleotide conjugate platform in a rare neuromuscular disease.
The publication marks a key clinical milestone for Avidity’s DM1 program and is expected to accelerate the company’s path toward regulatory submission. The company is concurrently conducting the global Phase 3 HARBOR trial, which will evaluate del‑desiran in approximately 150 patients, with topline data anticipated in the second half of 2026. In addition, Avidity is in the process of being acquired by Novartis for roughly $12 billion, a transaction that is expected to provide additional resources for the program.
Investors have reacted positively to the data, noting the robust clinical evidence and the strategic context of the pending acquisition. The strong NEJM publication reinforces confidence in the AOC platform and supports the company’s valuation in light of the Novartis deal.
Lead investigator Dr. Nicholas E. Johnson said, “These final results from the MARINA study further reinforce del‑desiran data reported thus far showing acceptable safety profile and improvements across a range of key functional assessments including myotonia, a hallmark symptom of DM1.”
Overall, the NEJM publication provides the first peer‑reviewed evidence of del‑desiran’s efficacy and safety, positioning Avidity to advance its DM1 program and potentially bring a first‑in‑class therapy to patients while aligning with the company’s broader strategic objectives.
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