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Casimersen

Duchenne Muscular Dystrophy

Explore 56 assets in Duchenne Muscular Dystrophy
Floatz Rating
BB67/100
Confidence
Indicative
v0.2
Sponsor
Kevin Flanigan
Modality
nucleic_acid
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2

Clinical Evidence

Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
60High confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
Completed
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Enrolling By Invitation
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
Terminated
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
Completed
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
Completed

Competitive Position

Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81Moderate confidence
Detailed axis rationale is planned and will be published soon.

Same indication · Duchenne Muscular Dystrophy

AssetSponsorPhaseRating
Casimersen (this asset)Kevin FlaniganP3BB · 67
ViltolarsenNS Pharma, Inc.P4BBB
Delandistrogene MoxeparvovecSarepta Therapeutics, Inc.P3BB
GolodirsenRare Disease Research, LLCP4BB
Aoc 1044Avidity Biosciences, Inc.P3BB
EteplirsenSarepta Therapeutics, Inc.P3BB
PrednisoloneAnne M. ConnollyP4BB
GlutamineAssistance Publique - Hôpitaux de ParisP3BB
VamoroloneSanthera PharmaceuticalsP4BB

+41 more in the Duchenne Muscular Dystrophy cohort

Scientific Foundation

Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR

Planned for methodology v0.2.

Development Feasibility

How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR

Planned for methodology v0.2.

Commercial Opportunity

Commercial prize: addressable population, unmet need, and the value case for the indication.
NR

Planned for methodology v0.2.

IP & Exclusivity

Exclusivity position, covering patent protection and freedom-to-operate runway.
NR

Planned for methodology v0.2.

Manufacturing & Supply

Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR

Planned for methodology v0.2.

Related assets

Citation

Floatz Terminal. Casimersen in Duchenne Muscular Dystrophy. Methodology v0.2.
Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026.
Accessed July 14, 2026.
https://terminal.floatz.ai/assets/casimersen-duchenne-muscular-dystrophy

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