Entrada Therapeutics, Inc. (NASDAQ: TRDA) has recently received authorization from the United States Food and Drug Administration (FDA) to initiate the 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. This follows the FDA's removal of the clinical hold on entr-601-44.
The elevate-44-102 study is designed to evaluate 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 study will also assess target engagement as measured by exon skipping and dystrophin production, as well as pharmacokinetics. The dosing will be administered every six weeks, with planned doses across four cohorts anticipated to range from 0.16 mg/kg up to 1.28 mg/kg.
In addition, the company plans to initiate study enrollment in the first half of 2026, enabling a seamless transition into an open-label extension study (OLE) where the safety, efficacy, and tolerability of entr-601-44 will be evaluated over a longer period of time.
Entr-601-44, a proprietary endosomal escape vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), is the lead product candidate within Entrada's DMD franchise. It is designed to address the underlying cause of DMD due to mutated or missing exons in the DMD gene, with the aim of restoring the mRNA reading frame and allowing for the translation of a slightly shortened but still functional dystrophin protein.
Duchenne Muscular Dystrophy (DMD) is a rare disease caused by mutations in the DMD gene, leading to inadequate dystrophin production, which is essential for maintaining the structural integrity and function of muscle cells. Lack of functional dystrophin leads to progressive loss of muscle strength, impacting mobility and causing heart or respiratory complications.
Entrada Therapeutics is a clinical-stage biopharmaceutical company with a focus on developing a new class of medicines that engage intracellular targets previously considered inaccessible. Their endosomal escape vehicle (EEV™)-therapeutics are designed to enable the efficient intracellular delivery of a wide range of therapeutics into various organs and tissues, resulting in an improved therapeutic index. They are advancing a robust development portfolio of RNA-, antibody* and enzyme-based programs for the potential treatment of neuromuscular, ocular, metabolic, and immunological diseases, among others. The market has reacted to these announcements by moving the company's shares -0.9% to a price of $12.93. If you want to know more, read the company's complete 8-K report here.
