EDG-5506 for Becker Muscular Dystrophy

(MESA Trial)

Enrolling by invitation at 51 trial locations

Age: Any Age

Sex: Male

Trial Phase: Phase 2

Sponsor: Edgewise Therapeutics, Inc.

Must not be taking: CYP3A4 inhibitors, CYP3A4 inducers, corticosteroids

Disqualifiers: Significant changes, investigational drugs, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to understand the long-term effects of a drug called sevasemten (EDG-5506) on individuals with Becker muscular dystrophy, a condition that gradually weakens muscles. The researchers focus on assessing the drug's safety, its impact on specific health markers, and participants' mobility. The trial is open to males with Becker muscular dystrophy who have previously participated in specific related studies. Participants should not have experienced significant health changes during or after those studies and should not be taking medications that could interfere with the trial. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but you cannot take certain drugs that affect liver enzymes (cytochrome P450 CYP3A4 inhibitors or inducers) or oral corticosteroids for BMD. If you're on these, you might need to stop or switch.

Is there any evidence suggesting that EDG-5506 is likely to be safe for humans?

Research has shown that EDG-5506, also known as sevasemten, is safe for people with Becker muscular dystrophy. In the CANYON trial, both adults and teenagers tolerated the treatment well. Another study found that EDG-5506 was safe, moved through the body effectively, and reduced signs of muscle damage. These findings suggest that EDG-5506 is generally well-tolerated, making it a promising option for those considering participation in a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike current treatments for Becker Muscular Dystrophy, which often focus on managing symptoms, EDG-5506 is an experimental drug that targets the underlying muscle damage. This treatment is unique because it uses a new active ingredient, Sevasemten, which aims to protect muscle fibers from stress. Researchers are excited because this approach could potentially slow down disease progression rather than just alleviating symptoms, offering a more proactive strategy for managing the condition.

What evidence suggests that EDG-5506 might be an effective treatment for Becker muscular dystrophy?

Research shows that EDG-5506, also known as sevasemten, may help treat Becker muscular dystrophy (BMD). Studies have found that EDG-5506 protects muscles by reducing the use of certain muscle fibers during activity, preventing damage. In earlier trials, patients taking EDG-5506 showed a noticeable decrease in signs of muscle damage. These results suggest that EDG-5506 could lower muscle damage and improve physical abilities in people with BMD. Overall, the early findings are promising for managing this condition. ^{1 2 3 5 6}

Who Is on the Research Team?

Joanne M. Donovan, MD, PhD

Principal Investigator

Edgewise Therapeutics, Inc.

Roxana D. Dreghici, MD

Principal Investigator

Edgewise Therapeutics, Inc.

Are You a Good Fit for This Trial?

This trial is for adults and adolescents with Becker muscular dystrophy who have completed certain prior studies (EDG-5506-002 ARCH, EDG-5506-201 CANYON and GRAND CANYON, or EDG-5506-202 DUNE) without significant safety concerns. They must not have taken any investigational drugs other than EDG-5506 recently or oral corticosteroids in the past 6 months.

Inclusion Criteria

You have participated in certain previous studies and completed all the required visits.

For EDG-5506-002 ARCH: Completion of the final study Visit 27 [Month 24]; or, completion of the ET visit prior to Visit 27 [Month 24]

I have completed at least 36 weeks of a specific treatment.

Exclusion Criteria

I have taken oral steroids for bone marrow disease in the last 6 months.

I haven't taken any experimental drugs recently, except EDG-5506.

Any clinically significant changes during or following participation in specific prior studies that would affect the potential safety of the participant to receive EDG-5506

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive long-term dosing of sevasemten to evaluate safety, tolerability, and durability of effect

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• EDG-5506

Trial Overview The study tests the long-term effects of a drug called EDG-5506 on safety, biomarkers, and functional measures in participants with Becker muscular dystrophy. It's an open-label extension meaning everyone knows they are getting the actual drug without any placebo control.

How Is the Trial Designed?

Group I: Treatment Experimental Treatment 1 Intervention

Drug: Sevasemten

Who Is Running the Clinical Trial?

Edgewise Therapeutics, Inc.

Lead Sponsor

Trials

13

Recruited

1,000+

Medpace, Inc.

Industry Sponsor

Trials

98

Recruited

30,400+

Dr. August J. Troendle

Medpace, Inc.

Chief Executive Officer since 1992

MD from the University of Maryland, School of Medicine; MBA from Boston University

Dr. Reinilde Heyrman

Medpace, Inc.

Chief Medical Officer since 2017

MD

Published Research Related to This Trial

In a phase 3 study involving 131 children aged 4 to under 8 years with Duchenne muscular dystrophy, edasalonexent was generally well-tolerated and had a manageable safety profile, with most side effects being mild gastrointestinal issues like diarrhea.

While edasalonexent did not show statistically significant improvements in overall muscle function compared to placebo, younger patients (6 years or younger) demonstrated more promising results, suggesting that early treatment may help slow disease progression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial. Finkel, RS., McDonald, CM., Lee Sweeney, H., et al.[2021]

In a phase 3 study involving 79 patients aged 7-16 with Duchenne muscular dystrophy, eteplirsen treatment for 96 weeks resulted in a significant increase in dystrophin production (7-fold) and exon skipping (18.7-fold), indicating its efficacy in addressing the underlying cause of the disease.

The study also demonstrated a favorable safety profile, with most adverse events being mild to moderate and unrelated to the treatment, while showing a notable slowing of disease progression compared to untreated controls.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial. McDonald, CM., Shieh, PB., Abdel-Hamid, HZ., et al.[2022]

The bmx mouse model, created using CRISPR/Cas9 technology, successfully mimics Becker muscular dystrophy (BMD) by expressing a common mutation in the dystrophin gene, allowing for the study of muscle and heart dysfunction associated with the disease.

This model shows significant muscle weakness and heart dysfunction compared to wild-type mice, with increased muscle damage and inflammation, making it a valuable tool for understanding BMD and testing potential therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The X-linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45-47. Heier, CR., McCormack, NM., Tully, CB., et al.[2023]

Edasalonexent (CAT-1004) is a promising treatment for Duchenne muscular dystrophy (DMD) that works by inhibiting the NF-κB pathway, which is linked to inflammation and muscle degeneration, showing significant effects in reducing NF-κB activity in a study with 79 adult participants.

The treatment was found to be safe and well tolerated, with mild side effects like diarrhea and headache, indicating its potential for therapeutic use in DMD and other diseases related to NF-κB activation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Novel NF-κB Inhibitor, Edasalonexent (CAT-1004), in Development as a Disease-Modifying Treatment for Patients With Duchenne Muscular Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics in Adult Subjects. Donovan, JM., Zimmer, M., Offman, E., et al.[2018]

Citations

1. edgewisetx.com edgewisetx.com/wp-content/uploads/2024/08/WMS_Symposium_Donovan_ARCH_6_mos_FINAL_SLIDES_for_pdf.pdf

EDG-5506 pharmacokinetics (PK) of EDG-5506 in adults with Becker muscular dystrophy. (BMD). —12 patients enrolled, ambulatory with BMD, including all from Phase 1 study.

2. institut-myologie.org institut-myologie.org/en/2025/07/17/edg-5506-sevasemten-stabilises-functional-scores-in-beckers-myopathy/

EDG-5506 (sevasemten) stabilises functional scores in ... EDG-5506 limits the recruitment of fast muscle fibres during muscular effort, protecting the muscle from possible mechanical damage.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05291091

NCT05291091 | Phase 2 Study of EDG-5506 in Becker ... A phase of research to describe clinical trials occurring after FDA has approved a drug for marketing. They include postmarket requirement and commitment ...

4. parentprojectmd.org parentprojectmd.org/https-investors-edgewisetx-com-news-news-details-2022-edgewise-therapeutics-announces-positive-2-month-interim-results-from-the-arch-open-label-study-of-edg-5506-in-adults-with-becker-muscular-dystr/

Edgewise Announces Positive 2-Month Interim Results ... Treatment with EDG-5506 led to a significant decrease in key biomarkers of muscle damage when assessed by laboratory assays. Importantly, ...

5. neurologylive.com neurologylive.com/view/therapeutic-potential-edg-5506-treating-becker-muscular-dystrophy-joanne-donovan

Therapeutic Potential of EDG-5506 in Treating Becker ... The results so far have been highly encouraging, with reductions in muscle damage biomarkers and physical improvements seen in patients." Becker ...

6. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0960896622004424

P.124 EDG-5506 targets fast skeletal myosin and reduces ... EDG-5506 demonstrated favorable safety and PK profiles at dose levels that resulted in robust reductions in muscle damage biomarkers in adults with BMD. By ...