As an early investor in Avidity Biosciences, we are pleased to share their exciting news that skipping exon 44 in the dystrophin gene with Del-zota was associated with functional improvements in multiple measures after one year of treatment.
Previously, Avidity reported topline data from their Phase 1/2 EXPLORE44 clinical study demonstrating a significant increase in dystrophin production–on average 25% of normal levels–and accompanied with sustained decreases in creatine kinase (CK) levels to near normal in patients treated with Del-zota at 5 mg/kg every 6 weeks.
The results presented today from the EXPLORE44-OLE clinical trial build on the earlier results and demonstrate striking improvements in multiple time function tests measured after one year of treatment. Improvements were seen in the 4-stair climb, 10-meter walk/run test, time to rise from floor, and the performance of upper limb (PUL2). The North Star Ambulatory Assessment (NSAA) remained stable. In contrast, declines in all of these measures are seen in matched natural history groups.
Why this matters to the Duchenne community:
With these results, the company is on track to file a Biological License Application with the FDA (using the Accelerated Approval pathway) later this year. We expect Avidity to obtain FDA approval and commercially launch Del-zota to individuals with Duchenne amenable to skipping exon 44 of the dystrophin gene in mid-2026.
Where can I learn more?
CureDuchenne will be hosting a webinar with Avidity on Thursday, September 18, 2925 at 5 pm ET, 2 pm PT. You can register to join the webinar (include link https://us02web.zoom.us/webinar/register/WN_Fv8yJJWpSba8d14TOj3USg#/registration)
The post Avidity reports multiple functional improvements with Del-zota for individuals with Duchenne amenable to skipping exon 44 appeared first on CureDuchenne.