Entrada Therapeutics Announces Updates on ENTR-601-44 in Duchenne Muscular Dystrophy
25 Nov 2023
INDOligonucleotide
– Company completes dosing of first and second cohorts in Phase 1 clinical trial, ENTR-601-44-101 –
– ENTR-601-44 clinical development program remains on track with data readout expected in second half of 2024 –
– U.S. FDA clinical hold on IND application remains in effect –
– Cash runway expected through 2025 –
Entrada Therapeutics, Inc. (Nasdaq: TRDA), a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing intracellular Endosomal Escape Vehicle (EEV™)-therapeutics as a new class of medicines, today announced the completion of dosing for the first and second cohorts of its Phase 1 clinical trial, ENTR-601-44-101. Entrada plans to announce data from ENTR-601-44-101 in the second half of 2024.
“Our strategy has always been to run a single Phase 1 clinical trial for ENTR-601-44 and, notably, that trial is progressing in the United Kingdom. We are pleased to have completed dosing of the first and second cohorts of participants,” said Dipal Doshi, President and Chief Executive Officer at Entrada Therapeutics. “In parallel with the Phase 1 clinical trial, we continue to plan for the global development of ENTR-601-44 which will include clinical trials in patients with Duchenne who are exon 44 skipping amenable.”
The primary objective of Entrada’s double-blind, single ascending dose Phase 1 clinical trial, which is expected to enroll approximately 40 participants, is to evaluate the safety and tolerability of a single dose of ENTR-601-44 in healthy male volunteers. ENTR-601-44-101 will also evaluate pharmacokinetics and target engagement, as measured by exon skipping in the skeletal muscle.
Update on ENTR-601-44 Investigational New Drug (IND) Application
Entrada was previously notified by the FDA that the IND for the Phase 1 clinical trial of ENTR-601-44 had been placed on clinical hold. Despite providing additional information to the FDA, the Company was informed that the Agency declined to lift the clinical hold. Importantly, the information that was submitted to the FDA supported the initiation of the Phase 1 clinical trial in the United Kingdom in September 2023.
“We are disappointed that the U.S. clinical hold has not been lifted, especially given the strength of the data package submitted to the FDA. It’s important to emphasize that the ongoing ENTR-601-44 development program continues to progress, with ENTR-601-44-101 clinical data expected in the second half of 2024. We will re-engage the FDA to discuss next steps in due course,” concluded Mr. Doshi.
About ENTR-601-44
ENTR-601-44, a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), is the lead product candidate within Entrada’s Duchenne muscular dystrophy franchise from its growing pipeline of EEV-therapeutics. Each EEV-PMO therapeutic candidate has an oligonucleotide sequence designed and optimized for the specific subpopulation of interest. ENTR-601-44 is designed to address the underlying cause of Duchenne due to mutated or missing exons in the DMD gene. ENTR-601-44, an investigational therapy for the potential treatment of people living with Duchenne who are exon 44 skipping amenable, is being evaluated for its potential to restore the mRNA reading frame and allow for the translation of dystrophin protein that is slightly shortened but still functional.
About Entrada Therapeutics
Entrada Therapeutics is a clinical-stage biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines, Endosomal Escape Vehicle (EEV™)-therapeutics, to engage intracellular targets that have long been considered inaccessible and undruggable. The Company’s EEV therapeutics are designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues, resulting in an improved therapeutic index. Through its proprietary, highly versatile and modular EEV platform, Entrada is building a robust development portfolio of RNA-, antibody- and enzyme-based programs for the potential treatment of neuromuscular, immunological, ocular and metabolic diseases, among others. The Company’s lead oligonucleotide programs include ENTR-601-44, ENTR-601-45 and ENTR-601-50 for the potential treatment of people living with Duchenne who are exon 44, 45 and 50 skipping amenable, respectively, as well as our partnered candidate ENTR-701 for myotonic dystrophy type 1.
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