Summary written by DDC Research Volunteer Omar Sheikh
On September 10, Avidity Biosciences provided an update on the Del-zota treatment for DMD. The company announced data that “demonstrated reversal of disease progression” and improvement compared to baseline from the EXPLORE44 and EXPLORE44-OLE trials.
Significant improvements include:
- 25% of normal dystrophin production
- Greater than 80% reduction of creatine kinase levels
- 50% of participants having normal CK levels after over 16 months of follow-up
- 2.1 second improvement from baseline for the 4-Stair Climb
- 3.2 second improvement from baseline for Time to Rise from Floor
- 1.5 point improvement from baseline for upper limb performance in ambulatory and non-ambulatory participants
A beneficial safety and tolerability profile emerged with little need for hospitalization. There were mild to moderate reactions from the treatment, most commonly diarrhea, fall, backpain, and headache. One participant discontinued from EXPLORE44-OLE after experiencing hypersensitivity.
As a reminder, this treatment attaches a synthetic molecule called ‘oligonucleotide’ to an antibody, a molecule that can follow signals from the muscle and heart tissue. When given to a patient with DMD, this combination skips exon 44 of the dystrophin gene, producing shorter yet potentially active dystrophin protein.
Del-zota has received a host of special designations from the U.S. FDA and is currently on track for submission of the Biologics License Application end of 2025, which facilitates faster approval. This step could position the treatment for clinical trials in Canada. Defeat Duchenne Canada is following up with Avidity Biosciences for more information and monitoring this process closely.
Read the full press release from Avidity Biosciences below.
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