Frequently Asked Questions

1. Who is sponsoring the DELIVER clinical trial?

This research trial is being sponsored by Dyne Therapeutics, a clinical stage company based in Waltham, Massachusetts.

2. What is the intent of the DELIVER trial?

The DELIVER clinical trial is assessing an investigational therapeutic called DYNE-251 for the first time in humans. DYNE-251 is designed to target muscle tissue and promote exon skipping, which allows muscle cells to create a shortened, functional dystrophin protein. “Investigational” means DYNE-251 is currently being tested to understand if it can help people with Duchenne. It has not yet been approved for use in treating Duchenne by any governmental regulatory agency.

The DELIVER clinical trial includes boys and adolescents with Duchenne who have a mutation that is amenable to exon 51 skipping. This trial has been designed to help determine whether DYNE-251 is safe, to identify the best dose and dose regimen for DYNE-251 and to assess whether DYNE-251 increases muscle dystrophin production and improves muscle function in affected individuals.

The DELIVER trial consists of a 24-week placebo-controlled period, a 24-week open-label extension (OLE) period and a 96-week long-term extension (LTE) period. A DMD community member who meets all criteria and is accepted into the trial will:

• Receive DYNE-251 or placebo by intravenous (IV) infusion every 4 weeks during the first 24-week placebo-controlled period,
• Receive DYNE-251 by IV infusion every 4 weeks throughout the OLE and LTE trial periods and
• Have his Duchenne and overall health assessed regularly throughout the trial.

3. Who is eligible to participate in the DELIVER trial?

A participant in the DELIVER study must be:

• 4 to 16 years old
• Male with Duchenne and a mutation amenable to exon 51 skipping
• Able to raise his arms over his head
• Currently receiving a steroid medicine

There are additional requirements for participating in the trial. Information on the DELIVER clinical trial is available on the DELIVER trial page at clinicaltrials.gov.

4. Where is the trial being conducted?

Information on current DELIVER clinical trial sites and recruitment status is available on the DELIVER trial page at clinicaltrials.gov. As sites are added to the trial or recruitment status is updated, this information will be reflected on the clinicaltrials.gov page.

5. How long is this clinical trial?

Participation in the trial could last approximately 3 years. However, being in this trial is completely voluntary. Participants can leave at any time. Leaving this clinical trial will have no impact on care received outside the trial.

6. Are there costs for participating?

Clinical trial treatment and all trial tests and procedures are provided at no cost to participants. Participants are reimbursed for approved trial-related travel expenses. Patient support services are available to participants who choose to have trial-related travel arranged for them at no cost.

7. When will you report data from the DELIVER study?

Dyne shared initial data from the DELIVER study in early January 2024. We anticipate reporting additional data in the second half of 2024.