Centronuclear Myopathies (CNM) is a life-threatening disease that that affects skeletal muscles from birth. Credit: Jensflorian.

Drug development company Dynacure has dosed the first patient in the Phase I/II Unite-CNM study to evaluate investigational antisense medicine DYN101.

DYN101 is intended to modulate the expression of dynamin 2 (DNM2) for the treatment of Myotubular and Centronuclear Myopathies (CNM).

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

CNMs, which affect skeletal muscles from birth, are rare, life-threatening disorders. Around 5,000 patients are affected by the disease in the EU, US, Japan and Australia.

The multi-centre, ascending dose study, Unite-CNM (DYN101-C101), aims to examine the tolerability, safety, pharmacokinetics and preliminary efficacy of DYN101 in around 18 patients, above the age of 16 years, with X-linked (XLCNM) or autosomal dominant CNM (ADCNM).

The study will primarily focus on detecting an optimal dose of the drug. It will also access different domains of efficacy in an exploratory analysis involving muscular function, respiratory function and muscle strength.

Once Unite-CNM study is completed, the company will analyse a potentially registration-directed Phase II/III study on all age groups.

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.

Dynacure is developing DYN101 in collaboration with Ionis Pharmaceuticals, a company known for its RNA-targeted drug discovery.

Preclinical efficacy of the medicine was observed in multiple mouse models of the disease.

Dynacure chief medical officer Chris Freitag said: “We are committed to providing CNM patients and their families with a therapeutic solution to treat this devastating, progressive muscle disease. Today, we are pleased to have made progress toward this goal with the first dose of DYN101 in a patient in our Phase I/II ‘Unite-CNM’ study.”