Posted on Monday, December 14, 2020
The EspeRare Foundation and the Pierre Fabre group have announced the conclusion of a licensing and collaboration agreement to develop and market ER-004, a prenatal treatment for XLHED (X-linked hypohidrotic ectodermal dysplasia), a rare and debilitating congenital disease. The next clinical study, which will begin in 2021, aims to validate the efficacy and safety of ER-004 therapy, which could then become the first treatment for XLHED by 2026.
Under the terms of the agreement, the EspeRare Foundation and the Pierre Fabre group will pool their respective know-how to co-develop the ER-004 treatment. Pierre Fabre will benefit from exclusive worldwide rights for the development, manufacture and marketing of the treatment.
XLHED is a genetic disease that affects the structure of the ectoderm, the outermost part of the three germ layers that form during embryonic life and are responsible for the development of the skin and integuments. ER-004 protein is a novel in utero therapy aimed at replacing the function of endogenous ectodysplasin-A1 (EDA1), a protein essential for the normal development of ectodermal structures in the fetus. ER-004 is a recombinant, soluble, humanized form of the EDA1 protein, which is administered by intra-amniotic injections at an advanced stage of fetal development. This approach has already demonstrated significant potential in correcting sweat gland function in three patients subjected to this protocol by Professor Holm Schneider at Erlangen University Hospital in Germany. The first results were published in the New England Journal of Medicine1 and British Journal of Clinical Pharmacology2, as well as in the Research Highlights section of Nature Medicine3.
“The EspeRare Foundation is enthusiastic about the idea of collaborating with the Pierre Fabre group and jointly developing this very promising program. We are convinced that by working together with Pierre Fabre, we will be able to offer an innovative treatment to patients with XLHED and that, while respecting our values centered on the patient’s needs ”, declared Caroline Kant, Executive Director of the EspeRare Foundation.
“We are proud of this alliance with the EspeRare Foundation in order to develop a therapy to treat a particularly debilitating disease which affects 500 infants each year around the world. This project perfectly illustrates the raison d’être of the Pierre Fabre group: “Every time we take care of one person, we make the world a better place,” commented Eric Ducournau, CEO of the Pierre Fabre Group.
EspeRare, a non-profit foundation specializing in finding treatments for rare diseases, and the Pierre Fabre group, a recognized player in the health sector for nearly 60 years, owned by the eponymous foundation recognized as being of public utility , share the same commitment to patients and work to put their know-how at the service of the discovery of innovative therapies capable of transforming their lives. This collaboration agreement is the realization of a common goal: to develop treatments for patients with rare diseases for which there is no therapy.
References :
1. Prenatal Correction of X-Linked Hypohidrotic Ectodermal Dysplasia. Schneider H, Faschingbauer F, Schuepbach-Mallepell S, Körber I, Wohlfart S, Dick A, Wahlbuhl M, Kowalczyk-Quintas C, Vigolo M, Kirby N, Tannert C, Rompel O, Rascher W, Beckmann MW, Schneider P. N Engl J Med 2018; 378: 1604-1610
2. Safety and immunogenicity of Fc-EDA, a recombinant ectodysplasin A1 replacement protein, in human subjects. Körber I, Klein OD, Morhart P, Faschingbauer F, Grange DK, Clarke A, Bodemer C, Maitz S, Huttner K, Kirby N, Durand C, Schneider H. Br J Clin Pharmacol. 2020;86(10):2063-2069
3. In utero correction of a genetic disorder. Stower H. Nature Medicine 2018; 24: 702
Source: Pierre Fabre
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