FDA Lifts Clinical Hold on Entrada’s Duchenne Muscular Dystrophy Drug ENTR-601-44 After Two Years
The FDA has lifted the clinical hold on Entrada Therapeutics' ENTR-601-44, allowing the initiation of the ELEVATE-44-102 Phase 1b study13.
ENTR-601-44 is designed to treat Duchenne muscular dystrophy (DMD) patients with mutations amenable to exon 44 skipping15.
The ELEVATE-44-102 study will involve approximately 32 non-ambulatory and ambulatory adult DMD patients3.
Dosing in the study will range from 0.16 mg/kg to 1.28 mg/kg, administered every six weeks across four cohorts35.
The global ELEVATE-44 program includes studies in both the U.S. (ELEVATE-44-102) and outside the U.S. (ELEVATE-44-201)15.
This decision follows the recent authorization from the UK's Medicines and Healthcare Products Regulatory Agency for the ELEVATE-44-201 study14.
Entrada plans to initiate study enrollment in the first half of 202635.
The clinical hold removal comes after a two-year wait, as the FDA initially declined to lift the hold in November 202378.
ENTR-601-44 uses Entrada's proprietary Endosomal Escape Vehicle (EEV) technology to improve delivery to muscle tissue16.
Nearly half of those living with Duchenne who are amenable to exon 44 skipping are adults, highlighting the importance of including this population in clinical studies5.
Sources:
1. https://ir.entradatx.com/news-releases/news-release-details/entrada-therapeutics-announces-fda-removal-clinical-hold-entr
3. https://www.stocktitan.net/news/TRDA/entrada-therapeutics-announces-fda-removal-of-clinical-hold-on-entr-l44jfp0s3zfa.html
4. https://www.clinicaltrialsarena.com/news/entrada-trial-dmd-treatment/
5. https://www.globenewswire.com/news-release/2025/02/24/3031005/0/en/entrada-therapeutics-announces-fda-removal-of-clinical-hold-on-entr-601-44.html
6. https://musculardystrophynews.com/news/phase-1-2-trial-exon-44-skipping-therapy-dmd-cleared-uk/
7. https://www.neurologylive.com/view/fda-removes-clinical-hold-duchenne-muscular-dystrophy-agent-entr-601-44