FDA Clears Entrada Therapeutics' Early-Stage For Potential Treatment Of Duchenne Muscular Dystrophy

On Monday, the U.S Food and Drug Administration (FDA) lifted the clinical hold on Entrada Therapeutics, Inc. TRDA ENTR-601-44. The agency issued the hold in December 2022.

In November 2023, despite providing additional information to the FDA, the company was informed that the agency declined to lift the clinical hold.

The agency authorized to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose (MAD) clinical study of ENTR-601-44 for 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.

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“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,” said Dipal Doshi, CEO of Entrada Therapeutics. “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.”

ELEVATE-44-102 is a randomized, double-blind placebo-controlled Phase 1b study evaluating the safety and tolerability of ENTR-601-44 in approximately 32 non-ambulatory and ambulatory adult patients 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 as well as 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 enroll in 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.

Price Action: TRDA stock is up 5.44% at $13.75 during the premarket session on last check Monday.

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