Entrada Therapeutics has released its latest quarterly update on its Duchenne clinical trial programmes. Below, we’ve summarised what’s new for each exon-skipping group, in plain terms, and linked the original news article here.
At a Glance
- Exon 44 (ELEVATE-44-201): Second cohort enrolment is complete, participants have transitioned to the open-label phase, and the long-term extension protocol has been accepted by UK and European authorities.
- Exon 45 (ELEVATE-45-201): First cohort dosing is finished, an independent committee has recommended a dose escalation to 10 mg/kg for the actively enrolling second cohort, and long-term extension approvals are in place.
- Exon 44 Adult Study (ELEVATE-44-102): Plans are underway to re-engage with the FDA regarding dose increases and study design refinement.
- Exon 50 & 51 Updates: Regulatory applications for Exon 50 are planned for the EU, and CTA-enabling studies for Exon 51 have been successfully completed.
Exon 44 (ELEVATE-44-201)
Enrolment for the second group of participants is now complete, and everyone from the first group has moved into the next phase of the trial at a higher dose (12mg/kg). Regulators in the UK and Europe have approved a plan allowing participants to keep receiving treatment for longer after the main trial ends to support ongoing data collection.
What’s next: Results from the first group are expected by the end of 2026, with results from the second group following in early 2027. These results will guide whether a third, higher-dose group begins.
There’s also a related non-ambulatory and ambulatory adults’ study (ELEVATE-44-102) in the US, where Entrada is planning to discuss increasing the dose and adjusting the study design with the FDA.
Exon 45 (ELEVATE-45-201)
This trial is running across the UK, Netherlands, Belgium, Italy, and Spain. Everyone in the first group has finished their initial dosing and moved to the next phase of the trial. An independent safety panel has approved a higher dose for the second group, who are now being recruited. As with exon 44, participants will be able to continue treatment for longer after the trial ends.
What’s next: Results from the first group are expected in October 2026, with results from the second group in the first half of 2027.
Exon 50 and Exon 51
These programmes are earlier in the process. For exon 50, initial approval has been granted in the UK, and Entrada plans to apply for approval across the EU later in 2026. For exon 51 (the largest group of eligible patients), the necessary approvals to begin trials have been completed, with international applications to follow.
Exons and Exon Skipping
Genes are made up of smaller sections called exons, which work together to give the body instructions for producing important proteins. In Duchenne muscular dystrophy (DMD), one or more exons contain an error, meaning the body cannot produce a functional dystrophin protein. Exon skipping is a therapeutic approach that works by teaching the body to skip over the faulty section, so that a shorter but still functional version of the dystrophin protein can be made. Because different people with Duchenne have errors in different exons, treatments are developed to target specific ones which is why you will see trials referred to as “exon 44 skipping amenable” or “exon 51 skipping amenable.” This simply means the treatment being tested is designed for people whose DMD is caused by an error in that particular exon.
Questions or Concerns?
If any of the information in this article has raised questions or concerns, please do not hesitate to get in touch. Action Duchenne is here to support you and your family every step of the way. Email us: Support@actionduchenne.org
