The EspeRare Foundation and Pierre Fabre join forces to develop and market a pioneering treatment for XLHED, a dermatologic-related rare genetic disease that requires prenatal therapeutic intervention

PR87212

GENEVA and CASTRES, France, Dec. 14, 2020 /PRNewswire=KYODO JBN/ --

The EspeRare Foundation and the Pierre Fabre group announced today that they

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," stated 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,'" commented Eric

Ducournau, CEO of the Pierre Fabre Group.

EspeRare, a non-profit organization committed to finding treatments for rare

diseases, and the Pierre Fabre Group, a major stakeholder in health care for

nearly 60 years, owned by the Pierre Fabre Foundation, both share a common

engagement for patients and are committed to dedicate their expertise to

discover innovative therapeutic solutions that can transform patients' lives.

This collaboration agreement materializes EspeRare's and Pierre Fabre's

objective to pave the way for treatment supporting underserved patients

affected by rare diseases.

References:

1.  Prenatal Correction of X-Linked Hypohidrotic Ectodermal Dysplasia

[https://pubmed.ncbi.nlm.nih.gov/29694819/].  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 [https://pubmed.ncbi.nlm.nih.gov/32250462/].

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

[https://pubmed.ncbi.nlm.nih.gov/29875458/]. Stower H. Nature Medicine 2018; 24: 702

About XLHED

XLHED is a severe, chronically debilitating and rare disease affecting

approximately 4/100,000 live male births every year. XLHED is caused by

mutations in EDA, a gene that encodes an important developmental signaling

protein, EDA1. The absence of functional EDA1 in the ectoderm results in

abnormal development of the skin, sweat glands, sebaceous glands, hair, oral

cavity, and respiratory mucosal glands resulting, as early as from birth, in serious  

clinical manifestations including hyperthermia, craniofacial anomalies and recurrent

respiratory infections that impair quality of life in patients and their families.

For more information, please visit www.omim.org

About THE ESPERARE FOUNDATION

EspeRare is a Swiss non-profit organization founded in 2013 that is committed

to improve the lives of children with life-threatening rare diseases. EspeRare

addresses the unmet medical needs of these children by uncovering the potential

of existing treatments. EspeRare's innovative model combines pharmaceutical

know-how with philanthropic, public and private investments to develop and

bring to life these discontinued therapies. With its unique patient-centered

approach to drug development, EspeRare engages the patient community at each

step of the process, with the intent of giving children and their families fair

access to these therapies and a new hope for the future.

For more information, please visit: www.esperare.org

About PIERRE FABRE

Pierre Fabre is the 2nd largest private French pharmaceutical group and 2nd

largest dermo-cosmetics laboratory in the world. Its portfolio is made of

medical franchises (oncology, dermatology, pharmaceutical care) and leading

dermo-cosmetic brands (Eau Thermale Avène, Ducray, A-Derma, Klorane, René

Furterer, Pierre Fabre Oral Care…).  In 2019, Pierre Fabre generated revenues

of €2.4 billion, two-thirds of which came from international sales.

Headquartered in the South-West of France, the Group employs some 10,400 people

worldwide, owns subsidiaries or offices in 45 countries, and distributes its

products in over 130 countries.

86% of the Pierre Fabre group is held by the Pierre Fabre Foundation, a

government-recognized public-interest foundation, while a smaller share is

owned by its employees via an employee stock ownership plan.

In 2019, Ecocert Environment assessed the Group's CSR approach in accordance

with the ISO 26000 sustainable development standard and awarded it the Ecocert

26000 "Excellence" rating.

For more information, please visit: www.pierre-fabre.com

Media Contacts:

EspeRare Foundation

foundation@esperare.org;

Pierre Fabre        

Anne Kerveillant, Communication Manager    

anne.kerveillant@pierre-fabre.com

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SOURCE : Pierre Fabre and The EspeRare Foundation