Avidity Biosciences, Inc. (RNA), Monday announced topline data from the dose escalation cohorts of the delpacibart braxlosiran (Del-brax) Phase 1/2 FORTITUDE program for the treatment of Facioscapulohumeral Muscular Dystrophy or FSHD, a rare hereditary disorder.
Notably, the primary endpoint of the trial is reduction of KHDC1L, a novel DUX4-regulated circulating biomarker.
The randomized, placebo-controlled, double-blind trial demonstrated a consistent improvement of functional mobility, muscle strength, and quality of life, significant reductions in KHDC1L and creatine kinase, and favorable long-term safety and tolerability.
The company expects topline data from the ongoing, fully enrolled del-brax Phase 1/2 FORTITUDE biomarker cohort in the second quarter of 2026.
Also, the company stated that the data supports planned accelerated approval of Biologics License Application in the second half of 2026.
In the pre-market hours, Avidity Biosciences's stock is moving up 4.86 percent, to $38 on the Nasdaq.
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