Entrada Therapeutics has shared its latest quarterly update across all four of its active Duchenne programmes. For families in the UK, there are several relevant developments: UK clinical sites are active or authorised across multiple studies, and initial data from one of those trials has now been released.
At a Glance
- Positive initial data from the first cohort of phase ½ ELEVATE-44-201 study for ambulatory children and young adults living with Duchenne wo are exon 44 skipping amendable.
- Enrolment for the ELEVATE-44-201 and ELEVATE-45-201 studies ongoing
- Updated timing for ELEVATE-50-201 and ELEVATE-51-201 regulatory submissions.
Exon 44 — First Trial Data Published
The ELEVATE-44-201 study, running across UK, involves ambulatory children and young adults who are amenable to exon 44 skipping, has released its first cohort results (8 participants, lowest dose of 6 mg/kg).
- Safety: Well-tolerated, no serious adverse events, normal kidney function throughout
- Function: Treated participants showed a statistically significant improvement in the functional measurement, time to rise (TTR) velocity, earlier than anticipated.
- Dystrophin: An increase of 2.36% above a 4.00% baseline — lower than originally projected, but a juvenile animal study has since produced closely matching results, giving Entrada confidence that higher levels may be seen at increased doses
All participants in the first cohort have now moved into the open-label phase, where they will each receive six further doses. The second cohort, at the higher dose of 12 mg/kg, is now enrolling. Entrada expects to report data from both the first cohort’s open-label period and the second cohort before the end of 2026.
Exon 45 — Enrolling Now, Including UK Sites
The ELEVATE-45-201 study is active across the UK, Netherlands, Belgium, Italy and Spain. The first cohort has begun dosing, with initial data expected mid-2026.
Exon 50 — UK Regulatory Authorisation Granted
The ELEVATE-50-201 study has received authorisation from the MHRA and Research Ethics Committee to begin in the UK. EU applications will follow once data from ongoing studies have been reviewed.
Exon 51 — Regulatory Filing Planned
Entrada plans to submit regulatory filings for a Phase 1/2 study of ENTR-601-51, with authorisation to follow after review of data from the current trials.
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
