The P-I/II (EXPLORE44) study is assessing SAD & MAD of del-zota (IV) in healthy subjects & patients with DMD mutations amenable to exon 44 skipping; recruitment is completed. Individuals with DMD44 can also join EXPLORE44-OLE extension study 

Initial evaluation was carried out among 25 subjects in 2 dose levels (5 & 10mg/kg). In the 5mg/kg cohort, 10 individuals received 3 doses of del-zota or PBO (Q6W) over 4mos. to assess muscle delivery, exon skipping, dystrophin production & creatine kinase 

Results showed unsurpassed 200 nM PMO delivery in skeletal muscle, 37% increase in exon 44 skipping (up to 66%), a 25% increase in dystrophin production (54% of normal was restored) & >80% reduction in creatine kinase levels to near normal