Entrada Therapeutics has shared encouraging news from its ELEVATE-44-201 clinical study. An independent Data Monitoring Committee (DMC) has completed its review of the first group of participants and given the green light to progress to a higher dose — an encouraging step forward in clinical research for those in the Duchenne community who are exon 44 skipping amenable.
Eight participants completed their dosing in the first cohort (group) of the double-blind, placebo-controlled portion of ELEVATE-44-201. An independent Data Monitoring Committee (DMC) – a group of external safety experts – reviewed all available safety and pharmacokinetic data (looking at how the drug moves through the body) from these participants.
Following that review, the DMC has recommended that the study now move forward with a second cohort at a higher dose of 12 mg/kg, up from 6 mg/kg in the first cohort. This is a planned multiple ascending dose (MAD) portion, and positive step in the study’s progress.
Establishing that ENTR-601-44 is safe at 6 mg/kg is a clear milestone for the program. We expect our Cohort 1 data at 6 mg/kg to show double-digit dystrophin production when we disclose the data in the second quarter. As we dose escalate to 12 mg/kg, we believe we will achieve best-in-class dystrophin restoration with the readout of our Cohort 2 data later this year.
Natarajan Sethuraman PhD, President of Research and Development, Entrada Therapeutics
ELEVATE-44-201: Study at a Glance
- Drug: ENTR-601-44
- Who it is for: Ambulatory patients with Duchenne who are exon 44 skipping amenable
- Study type: Phase 1/2, randomised, double-blind, placebo-controlled
- Countries: UK, Belgium, Italy and Spain
- Cohort 1 dose: 6 mg/kg
- Cohort 2 dose: 12 mg/kg (approved for initiation)
Progress on Each Cohort
Cohort 1 – 6 mg/kg
- Progress: All eight participants have completed dosing of the MAD portion of the study for Cohort 1, and participants have transitioned to the 6-dose open label, Phase 2 portion of the study.
- Safety: The independent DMC reviewed all available data and recommended the study continue — supporting a move to the higher dose.
- Data readout: Expected in Q2 2026 (April–June).
Cohort 2 – 12 mg/kg
- Status: Dosing is now recommended to initiate following the DMC’s review.
- Goal: Entrada believes this higher dose will achieve best-in-class dystrophin restoration.
- Data readout: Expected by end of 2026.
Cohort 3 – up to 18 mg/kg
- Status: Planned to follow after Cohort 2.
- Data readout: To be confirmed.
As we move forward and ELEVATE-44-201 study progresses, we will ensure our community remains informed about key updates and milestones.
If you have any questions about the information presented in this article, please reach out to us at support@actionduchenne.org.
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