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Sevasemten

Phase 2

Becker Muscular Dystrophy | Small molecule | Rare Disease |Edgewise Therapeutics, Inc.|Last Updated: Jul 31, 2026

Target and mechanism

ModalitySmall molecule

Also known as Sevasemten Dose 1

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials3
Total Enrollment516

FDA Designations

ORPHAN_DRUGRARE_PEDIATRIC_DISEASEFAST_TRACK

Clinical trial landscape

Sevasemten · 4 trials · 2 indications

Phase 2 2Phase 1 2
NCT06066580Open-Label Extension of EDG-5506 in Participants With Becker Muscular DystrophyBecker Muscular Dystrophy
ENROLLING BY_INVITATION260 Analytics
NCT05291091Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)Becker Muscular Dystrophy
ACTIVE NOT_RECRUITING244 Analytics
PHASE2ENROLLING BY_INVITATION
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
Becker Muscular DystrophyUnlock trial analytics
PHASE2ACTIVE NOT_RECRUITING
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
Becker Muscular DystrophyUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of adverse events in those treated with sevasemten
55 Months
Severity of adverse events in those treated with sevasemten
55 Months
Number of adverse events in those treated with sevasemten or placebo
12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

All participants

Severity of adverse events in those treated with sevasemten or placebo
12 months (CANYON Cohorts 1, 2, 4, 5), 18 months (GRAND CANYON Cohort 6)

All participants

Change from Baseline in serum Creatine Kinase
12 Months (CANYON Cohorts 1, 2)

Adult participants

Change from Baseline in the North Star Ambulatory Assessment scale
18 months (GRAND CANYON Cohort 6)

Adult participants

Pharmacokinetic
Up to 46 days of monitoring

The area under the concentration-time curve, from time 0 to the last observed non-zero concentration (AUC0-t) for sevasemten and metabolites administered with and without verapamil

Percentage of Participants Treated With Sevasemten Experiencing AEs
From first dose of study drug to 25 months

An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The numerator of the percentage is the number of participants experiencing at least one AE after first dose of study drug up to 25 months.

Frequency of AEs in Those Treated With Sevasemten
From first dose of study drug to 25 months

An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The endpoint is the cumulative total number of AEs occurring after first dose of study drug up to 25 months among participants who received at least one dose of study drug.

Number of Participants Treated With Sevasemten With AEs by Maximum Severity
From first dose of study drug to 25 months

An AE is any untoward medical occurrence in a patient administered a medicinal product. The AE does not necessarily have to have a causal relationship with the study drug. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. The severity of an AE is graded, according to the study protocol definitions of AE severity/intensity, as "mild", "moderate" or "severe". Participants who reported multiple AEs are counted only once at the highest severity reported.

Secondary Endpoints

Incidence of treatment-emergent abnormal clinical chemistry laboratory test results
54 Months
Incidence of treatment-emergent abnormal hematology laboratory test results
54 Months
Change from Baseline in the protein fast skeletal muscle Troponin I
12 months (CANYON Cohorts 1, 2), 18 months (GRAND CANYON Cohort 6)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
TreatmentEXPERIMENTALDrug: Sevasemten
Adult Cohort 1EXPERIMENTALDrug: Sevasemten Drug: Placebo
Adult Cohort 2EXPERIMENTALDrug: Sevasemten Drug: Placebo
Adult Cohort 6EXPERIMENTALDrug: Sevasemten Drug: Placebo
Adolescent Cohort 4EXPERIMENTALDrug: Sevasemten Drug: Placebo
Adolescent Cohort 5EXPERIMENTALDrug: Sevasemten Drug: Placebo
Treatment A: single dose sevasemtenEXPERIMENTALSingle dose sevasemten administered on Day 1 under fasting conditions
Treatment B: multiple doses of verapamil and single dose of sevasemtenEXPERIMENTALMultiple doses of verapamil with a single dose of sevasemten under fasting conditions
Treatment C: single dose sevasemten and high-fat mealEXPERIMENTALSingle dose sevasemten under high-fat fed conditions

Interventions

NameTypeDescription
SevasemtenDRUGSevasemten is administered orally once per day
Sevasemten 10 mgDRUGSevasemten is administered orally once per day
Sevasemten 5 mgDRUGSevasemten is administered orally once per day
Sevasemten 12.5 mgDRUGSevasemten is administered orally once per day
PlaceboDRUGPlacebo is administered orally once per day
VerapamilDRUGmultiple doses 240mg verapamil
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Eligibility Criteria

Age Range12 Years to 50 Years
SexMALE
Healthy VolunteersNo
Study Sites46

Key Inclusion Criteria: 1\. Males with a diagnosis of BMD and participation in EDG-5506-002 ARCH, EDG-5506-201 CANYON and GRAND CANYON, or EDG-5506-202 DUNE. Participants are eligible if they complete the respective prior study visits as follows: * EDG-5506-002 ARCH: Complete the final study Visit...

Countries:United StatesBelgiumDenmarkFranceGermanyIsraelItalyNetherlandsSpainUnited KingdomAustraliaNew Zealand
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Recent Changes (Last 90 Days)

LOWJul 31, 2026NCT06066580lastUpdatePostDate: changed
LOWJul 31, 2026NCT05291091lastUpdatePostDate: changed
LOWJul 31, 2026NCT06066580lastUpdatePostDate: changed
LOWJul 31, 2026NCT05291091lastUpdatePostDate: changed

Frequently asked questions about Sevasemten

What is Sevasemten used for?

Sevasemten is an investigational small molecule being developed for Duchenne Muscular Dystrophy and Becker Muscular Dystrophy. It is also studied in healthy subjects for drug interaction and food effect evaluations. Sevasemten is in Phase 2 clinical development for these rare neuromuscular diseases.

What does Sevasemten target?

Sevasemten is a small molecule designed to modulate the fast skeletal muscle troponin complex. By targeting this complex, it aims to stabilize the muscle contractile apparatus and reduce muscle damage associated with muscular dystrophies. This mechanism is being evaluated in Duchenne and Becker Muscular Dystrophy.

Who makes Sevasemten?

Sevasemten is being developed by Edgewise Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker EWTX. The company is conducting Phase 2 clinical trials for Sevasemten in Duchenne and Becker Muscular Dystrophy.

What phase is Sevasemten in?

Sevasemten is in Phase 2 clinical development. It is not FDA approved and remains investigational. The drug has received FDA designations including Orphan Drug, Rare Pediatric Disease, and Fast Track for its muscular dystrophy indications.

What clinical trials is Sevasemten in?

Sevasemten is being studied in three active Phase 2 trials: NCT05291091 in Becker Muscular Dystrophy, NCT05540860 in children with Duchenne Muscular Dystrophy, and NCT06100887 in children with Duchenne previously treated with gene therapy. A Phase 1 trial in healthy subjects has been completed.

Is Sevasemten the same as EDG-5506?

Sevasemten is also known as Sevasemten Dose 1 and Sevasemten 10 mg. The clinical trials listed for Sevasemten reference the compound EDG-5506, indicating these names refer to the same investigational drug being developed by Edgewise Therapeutics.