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Wednesday, June 12, 2024

Avidity: > 50% Reduction in DUX4 Regulated Genes, Functional Improvement in Facioscapulohumeral Muscular Dystrophy

 Avidity plans to accelerate the initiation of registrational cohorts in FORTITUDE trial

Delpacibart braxlosiran (AOC 1020), the first investigational therapy to target the underlying cause of FSHD, provided greater than 50% mean reduction across multiple DUX4 regulated gene panels (2 mg/kg at four months)

Trends of functional improvement including muscle strength, reachable workspace, and positive trends in patient and clinician reported outcomes demonstrated in people treated with delpacibart braxlosiran 2 mg/kg at four months

Delpacibart braxlosiran data demonstrate favorable safety and tolerability with all adverse events mild or moderate

Volume 9 of virtual investor and analyst series today, Wednesday, June 12 at 8:00 a.m. ET

Video Webcast Information
The company is hosting Volume 9 of its investor and analyst event series on June 12, 2024, beginning at 8:00 a.m. ET to discuss the initial data from the FORTITUDE trial of del-brax in people living with FSHD. The virtual event will be available via a live video webcast and can be accessed here or from the "Events and Presentations" page in the "Investors" section of Avidity's website. A replay of the webcast will be archived on Avidity's website following the event.

The management team will be joined by Jeffrey M. Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE™ trial investigator. Dr. Statland is one of the principal investigators in the ReSolve study, an ongoing natural history study being led by the FSHD Clinical Trial Research Network (CTRN).

https://www.prnewswire.com/news-releases/avidity-announces-unprecedented-aoc-1020-data-from-phase-12-fortitude-trial-demonstrating-greater-than-50-percent-reduction-in-dux4-regulated-genes-and-trends-of-functional-improvement-in-people-living-with-facioscapulohumeral-m-302170099.html

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