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KER-065

Phase 2

Duchenne Muscular Dystrophy | Small molecule | Neurology |Keros Therapeutics, Inc.|Last Updated: Aug 10, 2026

Target and mechanism

Molecular targetmyostatin/activin A
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment36

FDA Designations

ORPHAN_DRUG

Clinical trial landscape

KER-065 · 1 trial · 1 indication

Phase 2 1
NCT07704099Safety and Efficacy of KER-065 in Participants With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy
NOT YET_RECRUITING36 Analytics
PHASE2NOT YET_RECRUITING
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular DystrophyUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of participants with treatment-emergent adverse events (TEAEs and serious adverse events (SAEs)
Up to approximately 14 months

To evaluate the safety and tolerability of KER-065 in ambulatory and nonambulatory participants with DMD

Secondary Endpoints

KER-065 serum concentration by visit, as appropriate
Up to Week 52
Number and proportion of participants with treatment-emergent ADA (antidrug antibody) by visit
Up to Week 52
Change from baseline by visit in bone mineral density (BMD), fat mass, and lean body mass, as measured by dual- energy X-ray absorptiometry (DXA)
Week 12, Week 24 and Week 48
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort A1 (Late Ambulatory)EXPERIMENTALParticipants will receive stable corticosteroid (CS) along with KER-065.
Cohort A2 (Late Ambulatory)EXPERIMENTALParticipants will receive stable CS, exon skipper along with KER-065.
Cohort N1 (Early Nonambulatory)EXPERIMENTALParticipants will receive stable CS along with KER-065.

Interventions

NameTypeDescription
KER-065DRUGKER-065 will be administered subcutaneously (SC)
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Eligibility Criteria

Age Range9 Years to N/A
SexMALE
Healthy VolunteersNo

Key Inclusion Criteria: * Diagnosis of DMD, defined as the presence of phenotypic features at screening consistent with DMD AND documented mutation in the dystrophin gene consistent with the diagnosis of DMD using a clinically validated genetic test. * Receiving a stable regimen of systemic CS (inc...

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Recent Changes (Last 90 Days)

LOWAug 11, 2026NCT07704099lastUpdatePostDate: changed
LOWAug 11, 2026NCT07704099lastUpdatePostDate: changed
LOWAug 11, 2026NCT07704099lastUpdatePostDate: changed
LOWJul 15, 2026NCT07704099NEW_TRIAL: changed
LOWJul 15, 2026NCT07704099NEW_TRIAL: changed

Frequently asked questions about KER-065

What is KER-065 used for in Duchenne Muscular Dystrophy?

KER-065 is an investigational small molecule being developed for the treatment of Duchenne Muscular Dystrophy (DMD). It is currently in Phase 2 clinical development and has not yet been approved by regulatory authorities. The drug is designed to address the underlying muscle degeneration associated with this condition.

What does KER-065 target?

KER-065 targets myostatin and activin A, two proteins that regulate muscle growth. By inhibiting these targets, the drug aims to promote muscle mass and strength in patients with Duchenne Muscular Dystrophy. This mechanism is being evaluated in clinical trials to assess its potential therapeutic benefit.

Who is developing KER-065?

KER-065 is being developed by Keros Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol KROS. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug for Duchenne Muscular Dystrophy.

What phase is KER-065 in?

KER-065 is in Phase 2 clinical development. It is an investigational drug, meaning it has not received FDA approval and is still being studied in clinical trials. The drug has been granted Orphan Drug designation by the FDA, which provides incentives to support its development for Duchenne Muscular Dystrophy.

What clinical trials is KER-065 in?

KER-065 is being evaluated in a Phase 2 clinical trial with the identifier NCT07704099, titled 'Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy.' This trial is not yet recruiting and plans to enroll 36 male participants aged 9 years and older. The study is controlled but not randomized or double-blinded.