In short
ENTR-601-44 is an investigational nucleic acid-based therapy being studied for Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping. It is given as an intravenous infusion. Two authorised trials are currently listed in the EU Clinical Trials Information System, together planning to enrol up to 121 participants across Phase 1 and Phase 2.
Key points
- ENTR-601-44 is an investigational antisense oligonucleotide therapy for Duchenne muscular dystrophy amenable to exon 44 skipping.
- It is given as an intravenous infusion and has no marketing authorisation in the product records.
- A Phase 1 randomised, placebo-controlled trial (ELEVATE-44) is authorised to enrol 41 participants.
- A separate Phase 2 open-label extension study (ELEVATE-LTE) is authorised to enrol 80 participants.
- Both trials measure safety and tolerability, including treatment-emergent adverse events and changes in vital signs, laboratory results, ECG parameters, and physical examination findings.
- The developer listed in the trial records is Entrada Therapeutics, Inc.
What is ENTR-601-44?
ENTR-601-44 is an investigational substance with no marketing authorisation in the product records. It is a nucleic acid-based therapy, meaning it is built from nucleic acids rather than being a small chemical molecule or a protein. The substance is supplied by Entrada Therapeutics, Inc. and appears in the EU trial registry under the product name ENTR-601-44. A peer-reviewed article describes exon 44 skipping therapies as antisense oligonucleotide (ASO) treatments, a class of RNA-based therapy being explored for Duchenne muscular dystrophy.
How does ENTR-601-44 work?
ENTR-601-44 is being investigated for Duchenne muscular dystrophy amenable to exon 44 skipping. In DMD, mutations in the DMD gene disrupt the production of dystrophin, a protein that helps keep muscle cells intact. Antisense oligonucleotide therapies can skip over frame-disrupting exons in the DMD gene and partially restore dystrophin production. Exon 44 skipping is one such approach, targeting the specific exon 44 region of the gene. The trial records describe ENTR-601-44 as part of an endosomal escape vehicle phosphorodiamidate morpholino oligomer platform, a delivery technology designed to help the oligonucleotide reach its target.
Which conditions is ENTR-601-44 studied for?
- Duchenne muscular dystrophy (DMD) — a severe inherited muscle-wasting disorder associated with significant morbidity and mortality. Both authorised trials of ENTR-601-44 enrol participants with DMD amenable to exon 44 skipping.
How is ENTR-601-44 given?
ENTR-601-44 is formulated as a solution for infusion and is given by intravenous infusion. In the Phase 2 extension study, the planned dose of ENTR-601-44 is 18 mg/kg given intravenously.
What do the clinical trials show?
Two authorised trials of ENTR-601-44 are listed in the EU Clinical Trials Information System. The first, ELEVATE-44, is a Phase 1 randomised, double-blind, placebo-controlled study in participants with DMD amenable to exon 44 skipping. It is planned to enrol 41 participants and compares ENTR-601-44 with a sodium chloride placebo infusion. The second, ELEVATE-LTE, is a Phase 2 open-label long-term extension study in DMD amenable to exon skipping, with a planned enrolment of 80 participants. It evaluates ENTR-601-44 at 18 mg/kg and a related product, ENTR-601-45, at 20 mg/kg.
Both trials measure safety and tolerability as their primary focus. The outcomes include the incidence and severity of treatment-emergent adverse events (TEAEs), changes in vital sign measurements, changes in clinical laboratory results, changes in electrocardiogram (ECG) parameters, and changes in physical examination findings.
What side effects are known?
The peer-reviewed article on exon 44 skipping therapies notes that current DMD treatments are only palliative and that there is a need for more treatment options, but it does not report specific side effects for ENTR-601-44. The authorised trials are designed to measure safety and tolerability, including treatment-emergent adverse events.