#313 Becker Muscular Dystrophy Clinical Trials with Edgewise Therapeutics
DNA Today: A Genetics Podcast
Deep Dive
What is Becker Muscular Dystrophy (Becker)?
Becker is a genetic, progressive disease causing muscle degeneration and weakness due to dysfunctional dystrophin protein, which protects muscles during contraction. Symptoms typically begin in middle childhood, around age 9, and lead to loss of muscle function over time.
How does Becker Muscular Dystrophy differ from Duchenne Muscular Dystrophy (DMD)?
In Becker, the dystrophin protein is present but dysfunctional, while in DMD, it is entirely absent. Becker is often considered milder, but it still leads to significant muscle weakness and loss of function, with 30-40% of patients requiring a wheelchair by their late 40s.
What role does sevasemten play in treating Becker Muscular Dystrophy?
Sevasemten is an investigational fast myosin inhibitor designed to reduce contraction-induced muscle injury by slightly limiting muscle contraction. This helps protect muscle fibers from damage, potentially stabilizing or improving muscle function.
What are the key findings from Edgewise Therapeutics' earlier trials with sevasemten?
In a two-year open-label study, patients treated with sevasemten showed stabilization of muscle function, as measured by the North Star Ambulatory Assessment. Some patients even improved, and biomarkers of muscle damage decreased, indicating reduced muscle deterioration.
What is the Grand Canyon Study, and who is eligible to participate?
The Grand Canyon Study is a pivotal Phase 2 trial enrolling ambulatory adult men (18-50 years) with Becker Muscular Dystrophy. Participants must have a mutation in the dystrophin gene and a North Star score within a specific range to ensure the study can detect meaningful differences.
What are the inclusion and exclusion criteria for the Grand Canyon Study?
Inclusion criteria include having Becker Muscular Dystrophy, a dystrophin gene mutation, and a North Star score within a specific range. Exclusion criteria include low ejection fractions (below 40%), use of other investigational agents, or poor pulmonary function.
What are the common side effects of sevasemten observed in clinical trials?
The most common side effects reported are dizziness and sleepiness, which typically occur in the first few weeks of treatment and tend to diminish over time.
How does Edgewise Therapeutics support patients participating in their clinical trials?
Edgewise offers a concierge travel service to cover travel costs and other expenses, aiming to make participation cost-neutral for patients. They also provide a small stipend and support for childcare if needed.
Why is it important to have a placebo-controlled study for sevasemten?
A placebo-controlled study is crucial to determine if sevasemten is effective in slowing or reversing muscle deterioration. Without a placebo group, it would be difficult to attribute any improvements to the drug rather than natural disease progression or other factors.
What is the North Star Ambulatory Assessment, and why is it used in the Grand Canyon Study?
The North Star Ambulatory Assessment is a functional test that evaluates 17 daily living activities, such as climbing stairs, running speed, and getting up from a chair. It is used to measure changes in muscle function over time in the Grand Canyon Study.
- Becker MD is a genetic disease affecting dystrophin protein function.
- Symptoms usually appear in the middle childhood years.
- The disease leads to progressive muscle weakness and loss of function, potentially resulting in wheelchair dependence.
Shownotes Transcript
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