Insmed Announces Initiation of Phase II Clinical Trial with IPLEX(TM) in Myotonic Muscular Dystrophy

Promising Results Observed in Ongoing Open Label Trial

07-Nov-2007

Insmed Inc., a biopharmaceutical company focused on the development and approval of drugs for the treatment of metabolic diseases with unmet medical needs, announced the planned initiation of a 24-week, multi-center, randomized, double blind, placebo-controlled Phase II clinical trial with IPLEX(TM) in patients with myotonic muscular dystrophy. The decision to initiate this trial is based on the promising results from an ongoing open-label, dose-escalation trial of IPLEX(TM) in this indication.

Up to 70% of the patients analyzed in an ongoing open-label, dose-escalation trial have reported improvement in one or more of several symptoms commonly associated with Myotonic Muscular Dystrophy, including cognitive function, gastrointestinal function, muscle pain, arm and leg strength, fatigue and endurance. Specific assessment of endurance demonstrated an improved performance in the six minute walk test, a well accepted FDA approval end point. Improvements in endurance were comparable to other drugs approved where this test was used for FDA approval. The six minute walk test is a well accepted, validated, quantitative measure of endurance and has been used as a primary endpoint in pivotal studies of several FDA approved drugs.

The purpose of the Phase II study is to confirm the positive results obtained in the open-label dose escalation trial in a multi-center, randomized, double-blind, placebo-controlled setting. The study will include 60 patients and will be powered to detect a 75 meter difference between IPLEX(TM) and placebo for the change in distance walked during the six minute walk test. The results from this Phase II study will be used to establish the design for Phase III clinical development.

https://www.bionity.com/en/news/73857/insmed-announces-initiation-of-phase-ii-clinical-trial-with-iplex-tm-in-myotonic-muscular-dystrophy.html