Resolaris improved muscle strength in FSHD trial

Published Date
04/05/2017
Author
Laura Jacobs
Category
Research

aTyr Pharma has released results from its phase 1/2 trial testing Resolaris in young adults with early-onset facioscapulohumeral muscular dystrophy (FSHD).

Inflammation contributes to muscle damage in muscular dystrophies such as FSHD. Resolaris is a protein that aims to reduce inflammation by altering the body’s immune response.

The trial aimed to test the safety and effectiveness of Resolaris in people with FSHD who were diagnosed before 10 years of age. Eight people aged between 16-20 took part in the 12-week study.

The results showed that Resolaris was safe and well-tolerated, including at the highest dose. 63% of participants showed an improvement in muscle strength and 67% reported an improvement in their quality of life.

Although this trial had small numbers of participants, its findings are promising. They also confirm what was observed in a separate phase 1/2 trial, where 50% of adults with FSHD (not early-onset) had improvements in muscle function.

Dr Sanjay Shukla, aTyr Pharma’s Chief Medical Officer, said:

These results are important as they reinforce previous clinical results (in adult FSHD and adult LGMD2B) with Resolaris in a younger patient population, with a potentially more aggressive progression of disease.

Resolaris has Orphan Drug Designation for the treatment of both FSHD and limb girdle muscular dystrophy (LGMD). This special status gives companies certain incentives to help develop and market their rare disease product.

Further information

For more information on the trial results, read aTyr Pharma’s press release and Powerpoint presentation

Read the latest FSHD research news

Find out about FSHD research that we’re funding

Keep in touch