Entrada Therapeutics, Inc. (NASDAQ: TRDA) has received authorization from the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) and research ethics committee to initiate the Elevate-44-201, a phase 1/2 multiple ascending dose (MAD) clinical study of Entr-601-44 for the potential treatment of Duchenne Muscular Dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 44 skipping.
The Elevate-44-201 study 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. The study is designed to evaluate safety, pharmacokinetics, pharmacodynamics, exon skipping, and dystrophin production. It is worth noting that the company has submitted regulatory filings for Entr-601-44 in additional geographies including the US and EU, with ongoing regulatory discussions.
Entr-601-44, a proprietary endosomal escape vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), is the lead product candidate within Entrada's DMD franchise and is designed to address the underlying cause of DMD due to mutated or missing exons in the DMD gene.
The company's phase 1 clinical study to evaluate the safety and tolerability of a single dose of Entr-601-44 demonstrated that it was generally well-tolerated in healthy volunteers with no serious adverse events, no drug-related adverse events, and no clinically significant changes or trends noted in vital signs, electrocardiograms, physical exams, or laboratory assessments. The study also demonstrated significant plasma concentration, muscle concentration, and exon skipping.
Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to establish a new class of medicines that engage intracellular targets considered inaccessible. Through its proprietary endosomal escape vehicle (EEV™)-therapeutics, the company is advancing a robust development portfolio of RNA-, antibody-, and enzyme-based programs for potential treatment of neuromuscular, ocular, metabolic, and immunological diseases, among others.
In addition to the UK authorization, Entrada is on track to initiate the Elevate-44-201 study in the second quarter of 2025. This development is significant as it marks a step forward in Entrada's commitment to addressing the unmet medical needs of individuals living with DMD and advancing potential treatment options for this life-shortening disease. Following these announcements, the company's shares moved 0.1%, and are now trading at a price of $13.45. Check out the company's full 8-K submission here.
