Entrada Therapeutics has shared several key milestones regarding their ELEVATE clinical trial programs. These studies are designed to evaluate the safety and effectiveness of their investigational exon skipping treatments, which aim to help the body “skip” specific errors in the dystrophin gene. We are provided a summary of the ELEVATE clinical trial plans and progress below, alongside the attached community update from Entrada for more details.
Exon 44 Skipping (ENTR-601-44)
There are currently two primary studies focusing on the exon 44 community:
- ELEVATE-44-201 (Phase 1/2): This global study includes sites in the UK, Belgium, Italy, and Spain.
- Progress: The first group (cohort) of participants has finished the double-blind portion of the study and is moving into the open label phase, where all participants will receive the actual study drug rather than a placebo.
- Safety: An independent Data Monitoring Committee reviewed the initial data and recommended the study continue without any changes.
- Timeline: Data from the first group is expected between April and June 2026.
- ELEVATE-44-102 (Phase 1b): This study is specifically for ambulatory and non-ambulatory adults in the U.S. and is on track to begin in the first half of 2026.
Exon 45 Skipping (ENTR-601-45)
The ELEVATE-45-201 study is a Phase 1/2 trial taking place in the UK, Netherlands, Belgium, Italy, and Spain.
- Status: The first participant has been dosed.
- Timeline: Initial data from the first group of participants is expected in mid-2026.
Exon 50 & 51 Skipping
- Exon 50 (ENTR-601-50): The UK’s The Medicines and Healthcare products Regulatory Agency (MHRA) and the Research Ethics committee has authorised the ELEVATE-50-201 study to begin in the UK. Entrada plans to start this study by the end of 2026.
- Exon 51 (ENTR-601-51): The company remains on track to submit global regulatory filings for a Phase 1/2 study later this year.
Access and Inclusivity
When considering whether to enrol on a suitable clinical trial, families are often presented with a number of financially and logistical restrictions that can act as a barrier to participation.
Entrada are keen to help reduces these barriers, and improve inclusive and accessible clinical studies to access by ensuring participants of all backgrounds feel supported throughout the entire process. This includes covering reasonable study-related expenses, such as travel, accommodation and meals in accordance with local regulations, booking assistance for travel
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