Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44
Entrada Therapeutics, today announced that the United States Food and Drug Administration (FDA) has lifted the clinical hold on ENTR-601-44 and provided authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose (MAD) clinical study of ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy (DMD) in adult patients with a confirmed mutation in the DMD gene amenable to exon 44 skipping. The global ELEVATE-44 program, which includes the ELEVATE-44-102 study in the U.S. and ELEVATE-44-201 outside of the U.S., will cover a broad population of patients with DMD and support evaluation of ENTR-601-44 in patients with both early and advanced disease.
About the studies:
- ELEVATE-44-102 will take place in the US. It is a randomized, double-blind placebo-controlled Phase 1b study evaluating the safety and tolerability of ENTR-601-44 in approximately 32 adult patients (ambulatory and non-ambulatory) with DMD who are exon 44 skipping amenable. The MAD study is also designed to evaluate target engagement as measured by exon skipping and dystrophin production, and pharmacokinetics. Dosing will be administered every six weeks, with the planned doses across four cohorts anticipated to range from 0.16 mg/kg up to 1.28 mg/kg. Study participants may be eligible to enter an open label extension study (OLE), in which the safety, efficacy and tolerability of ENTR-601-44 will be evaluated over a longer period of time. The Company plans to initiate study enrollment in the first half of 2026, which would enable a seamless transition into an OLE.
- ELEVATE-44-201 will take place outside of the US (received MHRA authorization, filed in EU with discussions ongoing). It is a global, two-part, randomized, double-blind placebo-controlled Phase 1/2 study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in ambulatory patients with DMD who are exon 44 skipping amenable. Part A is a multiple ascending dose study designed to evaluate the safety, pharmacokinetics, and pharmacodynamics, including exon skipping and dystrophin production in approximately 24 patients. Dosing will be administered every six weeks, with the planned doses across three cohorts anticipated to range from 6 mg/kg up to 18 mg/kg. Part B of the study is designed to further evaluate the optimal dose established in Part A for safety and efficacy, including patient reported outcomes and quality of life measures. Study participants may be eligible to enter an open label extension study (OLE), in which the safety, efficacy and tolerability of ENTR-601-44 will be evaluated over a longer period of time. The Company is on track to initiate ELEVATE-44-201 in Q2 2025.
Dipal Doshi, CEO of Entrada Therapeutics stated:
“Given the strength of our safety and target engagement data from our Phase 1 clinical study and the profound unmet need in adults living with Duchenne, we are pleased to have obtained FDA clearance for the ELEVATE-44-102 study. The study will help assess the potential of ENTR-601-44 in both non-ambulatory and ambulatory adult patients who are unfortunately often left out of clinical studies due to the advanced stage of their disease. Nearly half of those living with Duchenne who are amenable to exon 44 skipping are adults. ELEVATE-44-102 will provide clinical experience from this important population for our growing data package in support of what we believe will be a best-in-class therapy.”
Read the full press release here
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