Regeneron Pharmaceuticals has reported positive results from the LUMINA-1 trial of garetosmab (REGN2477) for fibrodysplasia ossificans progressiva (FOP), a very rare bone disease.

FOP leads to abnormal bone formation replacing muscles, tendons and ligaments. The disorder results in skeletal deformities and loss of mobility and can be fatal.

The Phase II, double-blind placebo-controlled trial included 44 patients from North America and Europe.

Primary analysis shows that garetosmab reduced total lesion activity compared to placebo by 25% following 28 weeks of treatment. It was driven by a 90% drop in the number of new lesions compared to placebo.

A 25% relative decrease in bone lesion volume was also noticed in the study.

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 trial utilised F-NaF PET imaging and CT scans to determine the effect of garetosmab on change in heterotopic ossification (HO) among patients.

Regeneron president and chief scientific officer George D Yancopoulos said: “We believe garetosmab may offer important new hope that can potentially transform the course of FOP and look forward to working closely with the FDA and other regulatory authorities to make garetosmab available.”

The company will use detailed results from the LUMINA-1 trial for regulatory submissions. The US-based biotech also plans to undertake a paediatric trial.

Garetosmab is a VelocImmune-derived fully-human monoclonal antibody designed to neutralise Activin A. This Activin A causes abnormal heterotopic bone development in people with FOP.

In 2017, the US Food and Drug Administration (FDA) awarded ‘fast track’ designation to the candidate to prevent HO in patients with FOP.