Avidity’s Del-zota Shows Promise in Duchenne Muscular Dystrophy Treatment

Avidity Biosciences reported positive topline data from the Phase 1/2 EXPLORE44 trial of del-zota in DMD patients amenable to exon 44 skipping (DMD44)1 4.

Del-zota demonstrated a statistically significant 40% increase in exon 44 skipping1 4.

The treatment achieved a 25% increase in dystrophin production, with total dystrophin restored up to 58% of normal levels1 4.

Creatine kinase levels, a marker of muscle damage, were reduced by over 80% compared to baseline, reaching near-normal levels1 4.

Del-zota showed a favorable safety and tolerability profile across dose cohorts, addressing previous safety concerns1 4 18.

Avidity plans to submit a Biologics License Application (BLA) for accelerated approval of del-zota at 5 mg/kg every six weeks by the end of 20251 5.

The drug has received FDA's Orphan, Rare Pediatric Disease, and Fast Track designations, as well as EMA's Orphan designation8.

Del-zota is part of Avidity's Antibody Oligonucleotide Conjugates (AOC) platform, designed to deliver RNA therapeutics to specific tissues10.

The positive results validate Avidity's AOC technology and have implications for their broader pipeline in neuromuscular disorders4.

Analysts view these results favorably, with projections estimating global sales of over $12 million by the end of 2027, potentially rising to $33 million by 20308.