The trial will assess ENTR-601-44 to treat DMD. Credit: Ines Nepo / Shutterstock.

Entrada Therapeutics has concluded the first and second cohorts dosing for the ENTR-601-44-101 Phase I clinical study of its investigational therapy ENTR-601-44 to treat Duchenne muscular dystrophy (DMD).

DMD is a serious form of muscular dystrophy that mainly impacts boys. Weakness in the muscles typically starts around the age of four and progresses rapidly.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

ENTR-601-44 is an Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer (PMO).

ENTR-601-44-101 is a single ascending dose and double-blind study, which is anticipated to enrol around 40 subjects.

Assessing the tolerability and safety of a single ENTR-601-44 dose in healthy male volunteers is the primary objective of the Phase I study.

See Also:

Furthermore, the trial will assess pharmacokinetics and target engagement, as measured by exon skipping in the skeletal muscle.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

The company intends to reveal data from this trial in the second half of next year.

Entrada Therapeutics president and CEO Dipal Doshi said: “Our strategy has always been to run a single Phase I clinical trial for ENTR-601-44 and, notably, that trial is progressing in the UK.

“We are pleased to have completed dosing of the first and second cohorts of participants. In parallel with the Phase I 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.”

Entrada received prior notification from the US Food and Drug Administration (FDA) about the Phase I clinical trial of ENTR-601-44 being placed on hold.

Even after providing more details to the FDA, the company was informed that the agency decided not to remove the clinical hold.

The information submitted backed the start of the Phase I study in the UK in September.

During that month, Entrada dosed the first subject in the trial to assess ENTR-601-44.