The EspeRare Foundation, Pierre Fabre to Develop XLHED Treatment

The EspeRare Foundation and the Pierre Fabre group have entered into a license and development collaboration agreement for the development and commercialization of ER-004, a prenatal treatment for XLHED (X-linked Hypohidrotic Ectodermal Dysplasia), a rare, debilitating congenital disease. The next clinical study is expected to start in 2021 and will aim at qualifying and registering what may become the first approved treatment for XLHED by 2026.

According to the terms of the agreement, EspeRare and the Pierre Fabre Group will pool their respective expertise together in order to co-develop ER-004. The Pierre Fabre group will be granted exclusive worldwide rights for the development, manufacturing and commercialization of ER-004.

XLHED is a genetic disorder that affects the structure of the ectoderm, the most exterior part of the three primary germ layers formed during early embryonic life, from which the skin and its appendages are derived. ER-004 is pioneering an in-utero therapy designed to replace the function of endogenous Ectodysplasin A1 (EDA1), a protein key to the normal development of ectodermal structures in the fetus. ER-004 is a recombinant, soluble, and humanized form of EDA1 that is given as a single course treatment and delivered through intra-amniotic injections during the late stage of pregnancy. This approach has already demonstrated a significant potential in humans where it normalized sweat gland function in three patients treated in this fashion by Prof. Holm Schneider at the University Hospital Erlangen in Germany. First results were published in the New England Journal of Medicine1 and in the British Journal of Clinical Pharmacology2 as well as featured in Nature Medicine's Research Highlights3.

"At EspeRare we are thrilled to collaborate with the Pierre Fabre Group to co-develop this very inspiring program. We are confident that joining forces with Pierre Fabre will allow to bring this ground-breaking therapy to XLHED patients in alignment with our patient-centered values," said Caroline Kant, Executive Director of the EspeRare Foundation.

"We are proud to ally with the EspeRare foundation to find a therapeutic solution to a debilitating disease affecting 500 babies worldwide every year. We cannot think of a better way to epitomize our corporate purpose: 'Every time we take care of one single person, we make the world better,'" said Eric Ducournau, CEO of the Pierre Fabre Group.