Evicenter
P&T meetings

Zeleciment rostudirsen (z-rostudirsen)

Duchenne muscular dystrophy

Also known as DYNE-251
Manufacturer
Dyne Therapeutics
Regulatory submission
BLA submitted May 2026
Last updated
78 sources

Clinical evidence

DELIVER

34 evidence topics · 23 sources

Objective
Location and study date
Study design
Eligibility criteria
Treatment
Study outcomes
Rationale for using dystrophin expression as the primary endpoint
Statistical analysis description
Number of participants (Planned and analyzed)
DELIVER: registrational expansion cohort allocation
GroupDirect quotation
Z-rostudirsen“24 participants”
Placebo“8”
Description of analysis sets
DELIVER registrational expansion cohort: six-month functional analysis populations
Analysis populationDirect quotation
Placebo“Placebo: n=18”; “(REC+MAD)”
Z-rostudirsen“Z-rostudirsen: n=21”; “(REC)”
Results
Participant disposition
Study withdrawals caused by treatment-emergent adverse events: all z-rostudirsen doses
Disposition measureQuoted result
Exposed participants across all study periods“N=85”
TEAEs leading to study withdrawal“0”
Baseline characteristics
Baseline characteristics: pooled MAD and REC placebo versus active REC
CharacteristicPooled placeboZ-rostudirsen REC
Age, years, mean (SD)“8.2 (2.5)”“7.8 (3.6)”
Ambulant participants, n (%)“19 (79.2)”“21 (87.5)”
Efficacy results
DELIVER registrational expansion cohort: six-month dystrophin results
MeasureDirect quotation
Mean absolute dystrophin, muscle-content adjusted“5.46% of normal”
Muscle-content-adjusted dystrophin, comparison with baseline“p<0.0001”
Mean absolute dystrophin, unadjusted“2.87% of normal (p < 0.0001)”
DELIVER registrational expansion cohort: six-month functional results
Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results
DELIVER safety: open-label and long-term extensions
Study limitations
Summary

The 32-participant registrational expansion cohort was not powered for statistically significant functional comparisons. The reported functional analysis was post hoc, combined placebo participants from the registrational and multiple-ascending-dose cohorts, and reported nominal p values; the prespecified plan did not include formal hypothesis testing of functional endpoints. Optional late biopsies involved four participants, compared with three baseline samples. Cardiopulmonary findings were compared with natural history and reflected mixed dose exposures, not continuous 20 mg/kg treatment throughout follow-up. Observational associations involving residual dystrophin should not be treated as direct proof of benefit from therapeutically restored dystrophin.