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DX5057
First oral small molecule inhibitor of DUX4 for facioscapulohumeral muscular dystrophy (FSHD).
What is DX5057?
First oral small molecule inhibitor of DUX4 for facioscapulohumeral muscular dystrophy (FSHD). Shows preclinical efficacy comparable to leading IV-administered gene-targeting therapies with added benefit of ease of use and scalable delivery. In IND-enabling stage, advancing to Phase 1/2a in 2025-2026.
Product typeDrug Discovery & Biotech
Pricing ·
Deployment
APINot available
What teams use DX5057 for
disease-modifying therapy for FSHD via DUX4 inhibition
STAT3 inhibition for inflammatory diseases and cancer
undisclosed oncology targets relevant in sarcomas and prostate cancer
Integrations and access
Platforms, integrations, and language support vary by plan and region. Confirm final requirements with the vendor.
Platforms
IntegrationsAPI and webhooks
Languages
Support
What reviewers say
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Enterprise readiness
Compliance claims are normalized from current vendor documentation and independently reviewed by SOTA2.
Data retention:
Benchmarks and comparisons
Independent benchmarksStructured task results are being verified.
Side-by-side comparisonsComparison workspaces will be available in a later release.
