DX5057
First oral small molecule inhibitor of DUX4 for facioscapulohumeral muscular dystrophy (FSHD).
What it does
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.
- disease-modifying therapy for FSHD via DUX4 inhibition
- STAT3 inhibition for inflammatory diseases and cancer
- undisclosed oncology targets relevant in sarcomas and prostate cancer
- Category
- Drug discovery and biotech
- Built for
- Healthcare and life sciences
- Users
- patients with facioscapulohumeral muscular dystrophy (FSHD), patients with inflammatory diseases, patients with oncology indications including sarcomas and prostate cancer
- Public API
- Not offered
Not yet disclosed: deployment options, platforms, languages and support channels.
Pricing
Pricing isn't published
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Security and compliance
No security details published yet
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