Industry Partner Update: Dyne Therapeutics
Defeat Duchenne Canada is committed to bringing the latest in Duchenne muscular dystrophy research and drug development to you – our Canadian Duchenne community.
Join Ash Dugar, Ph.D., MBA, Chief Medical Affairs Officer, and Molly White, Vice President, Global Head of Patient Advocacy and KOL Engagement, to learn about Dyne’s DELIVER clinical trial, a Phase 1/2 study evaluating DYNE-251 for the treatment of individuals with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping.
Click the button to the left to read their latest press release and register for this webinar below.
DYNE-251 is Dyne’s product candidate being developed for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create a truncated, functional dystrophin protein, with the goal of stopping or reversing disease progression. In preclinical studies with Dyne’s FORCE™ platform, robust and durable exon skipping and dystrophin expression were observed in the mdx mouse model in skeletal and cardiac muscle as well as reduced muscle damage and increased muscle function. In non-human primates, DYNE-251 demonstrated a favorable safety profile and achieved impressive exon skipping, especially in the heart and diaphragm, muscles in people living with DMD that weaken over time leading to mortality.
In addition to DYNE-251, Dyne is building a global DMD franchise with preclinical programs for patients with mutations amenable to skipping other exons, including 53, 45 and 44.
About the DELIVER Trial
DELIVER is a Phase 1/2 global clinical trial evaluating DYNE-251, consisting of a 24-week multiple ascending dose (MAD) randomized placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The trial, which is designed to be registrational, is expected to enroll approximately 46 ambulant and non-ambulant males with DMD who are ages 4 to 16 and have mutations amenable to exon 51 skipping therapy. The primary endpoints are safety, tolerability and change from baseline in dystrophin levels as measured by Western blot. Secondary endpoints include measures of muscle function, exon skipping and pharmacokinetics. Dyne anticipates reporting data from the MAD placebo-controlled portion of the DELIVER trial on safety, tolerability and dystrophin in the second half of 2023. For more information on the DELIVER trial, visit https://www.clinicaltrials.gov/ (NCT05524883).