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EspeRare

Ownership: Private
Development stage: Other

Overview

EspeRare has demonstrated proof of concept in humans for its lead programs. Its most advanced asset, efsudenermin alfa (targeting EDA), has reached Phase 2 clinical trials for X-linked hypohidrotic ectodermal dysplasia. Rimeporide, targeting NHE1 inhibition, was developed through a repositioning strategy and reached Phase 1 for Duchenne muscular dystrophy. The foundation specializes in repositioning or rescuing existing compounds with potential application to rare diseases and has received orphan drug designations from both US and EU regulators.

Therapeutic areas: Rare Diseases
Modalities: Small moleculeFusion proteinProteinEnzyme replacement therapy
Industry: Biotech

Drug pipeline

1 asset

rimeporide

Partnerships

Merck SeronoPharmaceutical companiesBiotechsAcademia/patient organizations

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