Entrada Therapeutics has received authorization from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee (REC) for a Phase 1 clinical trial of its Duchenne medication, ENTR-601-44. This experimental drug aims to treat individuals with Duchenne muscular dystrophy who have exon 44 skipping amenable mutations.
Dipal Doshi, President and CEO of Entrada Therapeutics, expressed excitement about the approval and the potential impact of ENTR-601-44 on addressing the significant medical needs of patients with this form of muscular dystrophy. He also highlighted the extension of their cash runway through 2025, positioning the company to advance their Duchenne franchise and explore new possibilities for their intracellular therapeutics in treating serious diseases.
The Phase 1 clinical trial will focus on evaluating the safety of a single dose of ENTR-601-44 in healthy volunteers, with a target enrollment of around 40 participants. The trial will also assess the drug’s tolerability, pharmacokinetics, and target engagement by measuring exon skipping in the skeletal muscle. The first participant is expected to be dosed in September of this year, and data from the trial are anticipated to be available in the second half of 2024.
ENTR-601-44 is a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), which represents the leading candidate within Entrada’s Duchenne franchise. This therapeutic approach aims to address the underlying cause of Duchenne muscular dystrophy caused by mutated or missing exons in the DMD gene. By restoring the mRNA reading frame, ENTR-601-44 has the potential to enable 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 is caused by mutations in the DMD gene that lead to insufficient production of dystrophin, a vital protein for the structural integrity and function of muscle cells. Current approved therapies for Duchenne focus on improving dystrophin production, but their clinical benefits remain unconfirmed.
Entrada Therapeutics is a biopharmaceutical company dedicated to transforming the lives of patients by developing a new class of medicines known as Endosomal Escape Vehicle (EEV™)-therapeutics. These therapeutics aim to target intracellular sites that were previously considered inaccessible and undruggable. Through its highly versatile and modular EEV platform, Entrada is building a robust pipeline of RNA-, antibody-, and enzyme-based programs for various diseases, including neuromuscular, immunological, ocular, and metabolic conditions.
Entrada’s commitment to advancing its Duchenne franchise extends beyond ENTR-601-44, with plans for other lead candidates such as ENTR-601-45 for individuals who are exon 45 skipping amenable. Additionally, the company is collaborating on ENTR-701, which targets myotonic dystrophy type 1 (DM1).
While the journey towards an effective treatment for Duchenne muscular dystrophy continues, Entrada Therapeutics’ progress in gaining UK authorization for its Phase 1 clinical trial marks a significant step forward. By addressing the unmet medical needs of patients with exon skipping amenable mutations, the company’s innovative approach holds promise for potentially transforming the lives of those affected by this devastating disease.
