Entrada Therapeutics Receives Green Light for Phase 1 Clinical Trial of ENTR-601-44 in the UK to Explore Treatment Potential for Duchenne Muscular Dystrophy
Wednesday, August 02, 2023
Entrada Therapeutics, Inc. (Nasdaq: TRDA) is a biopharmaceutical company with a mission to revolutionize patient lives by introducing intracellular Endosomal Escape Vehicle (EEV™)-therapeutics as a new category of medicines. The company has recently received approval from the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee (REC) to conduct a Phase 1 clinical trial for ENTR-601-44, their lead product candidate in the Duchenne franchise. This trial aims to evaluate the safety, tolerability, pharmacokinetics, and target engagement of ENTR-601-44 in healthy volunteers who have exon 44 skipping amenable mutations, a condition associated with Duchenne muscular dystrophy.
Dipal Doshi, President and Chief Executive Officer of Entrada Therapeutics, expressed enthusiasm for this important milestone, as it addresses a significant unmet medical need in Duchenne muscular dystrophy. The Phase 1 trial is expected to enroll approximately 40 participants, and the dosing of the first participant is anticipated in September of this year. Data from the trial is expected to be available in the second half of 2024.
ENTR-601-44 is a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO) and is the primary product candidate within Entrada's Duchenne franchise. It is designed to treat the underlying cause of Duchenne muscular dystrophy, caused by mutated or missing exons in the DMD gene. ENTR-601-44 aims to restore the mRNA reading frame, allowing for the translation of a slightly shortened but still functional dystrophin protein.
Duchenne muscular dystrophy is a rare genetic disease characterized by progressive muscle degeneration and weakness throughout the body. It results from mutations in the DMD gene, leading to insufficient production of the dystrophin protein, which is essential for muscle cell structure and function. Although current approved therapies for Duchenne target dystrophin production improvement, their clinical benefits are yet to be fully validated.
This milestone, coupled with the extension of the company's cash runway until the end of 2025, positions Entrada Therapeutics to advance its Duchenne franchise while also expanding the potential of its intracellular therapeutics in treating other severe diseases.
