Avidity Advances Promising DMD Therapy Toward FDA Submission ##

Avidity plans to submit a Biologics License Application (BLA) to the FDA by the end of 2025 for accelerated approval of del-zota17
Del-zota is designed to treat DMD patients with mutations amenable to exon 44 skipping (DMD44)15

In clinical trials, del-zota demonstrated:

25% increase in dystrophin production57
37% increase in exon 44 skipping5

80% reduction in creatine kinase levels57
Del-zota has received FDA orphan drug, rare pediatric disease, and fast track designations517
Topline data from the EXPLORE44 trial expected in Q1 20251
If approved, del-zota would be the first treatment targeting exon 44 skipping in DMD13
Analysts predict potential U.S. peak sales of $315 million11

This progress positions Avidity as a strong contender in developing next-generation therapies for DMD, with del-zota potentially setting a new standard for exon skipping treatments in this rare disease.

Sources:

1. https://www.parentprojectmd.org/avidity-biosciences-announces-plans-to-file-biologics-license-application-for-delpacibart-zotadirsen-aoc-1044/

5. https://www.parentprojectmd.org/avidity-biosciences-announces-positive-data-from-phase-1-2-explore44-trial-of-aoc-1044-in-individuals-amenable-to-exon-44-skipping/

7. https://www.prnewswire.com/news-releases/avidity-biosciences-announces-positive-topline-del-zota-data-demonstrating-consistent-statistically-significant-improvements-in-dystrophin-exon-skipping-and-creatine-kinase-in-people-living-with-duchenne-muscular-dystrophy-amena-302402470.html

11. https://www.fiercebiotech.com/biotech/analysts-dig-details-aviditys-dmd-win-revealing-nuances-strong-data

13. https://www.ajmc.com/view/avidity-biosciences-gains-fda-orphan-drug-designationfor-patients-with-dmd

17. https://www.neurologylive.com/view/fda-grants-rare-pediatric-disease-designation-dmd-agent-aoc-1044-mutations-amenable-exon-44-skipping