EspeRare has entered into a partnership with?Dermelix Biotherapeuticsto develop?DMX-101, a novel?in utero?protein replacement therapy for the treatment of X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED), a rare pediatric genetic disease. There is currently no approved therapy for the treatment of?XLHED?and?DMX-101?has the?potential to become the first ever?in uteroadministered drug to correct a genetic disease before birth. During the second half of 2019 and in view of recent discussions with the EU and US regulatory agencies, both partners are set to?start enrolling patients into a clinical trial geared towards marketing approval. With?Dermelix, EspeRare is fortunate to have found a partner that shares common values and patient-centered commitments. Both partners have gone as far as signing an?Ethical Charter within the license agreement, under which both partners commit to continuously and transparently engage with the patient community during the development and commercialization of the treatment. To our knowledge this is a legal first! Under the terms of the agreement, EspeRare remains the sponsor of?DMX-101?pivotal development in Europe, while?Dermelix?will lead activities of?DMX-101?outside of Europe.?Dermelix?will finance the development of the product and will be responsible for its commercialization worldwide. In exchange for exclusive commercial rights, EspeRare is receiving financial returns. Delivering on EspeRare non-profit model, these financial returns are on one hand, shared with past contributors of?DMX-101?development and on the other hand, reinvested into scaling EspeRare?s organization and portfolio of therapeutic programs. Caroline Kant, Founder and CEO of EspeRare, commented: ?Patients with rare diseases so often lack the treatment options they need. This partnership with Dermelix represents an amazing opportunity to bring an innovative therapy to patients and to potentially radically change their lives. And beyond?XLHED, we are also committed to pave the way for other prenatal treatments to correct genetic diseases before birth.?

Presses releases

Please find the full press release in?English?and in?French.

About?XLHED

XLHED?is a severe, chronically debilitating and life-threatening rare disease affecting approximately 4/100,000 live male births every year.?XLHED?is caused by genetic mutations in the EDA gene, a gene that encodes for an important ectodermal developmental protein, EDA. The absence of functional EDA results in abnormal development of the skin, sweat glands, sebaceous glands, hair, oral cavity, and respiratory mucosal glands resulting in serious life-threatening clinical manifestations from birth including hyperthermia, craniofacial anomalies and recurrent respiratory infections that impair quality of life in patients and their families. For further information on?XLHED, please visit this?Website.

About EspeRare

EspeRare is a Swiss not-for-profit organization 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 our?Website.

About?DMX-101

DMX-101?is a fully humanized EDA molecule consisting of the human IgG1 Fc sequence linked to the human EDA TNF binding domain. Preclinically,?DMX-101?has been shown to bind to the receptor EDAR resulting in activation of the NF?B signaling pathway, which triggers the transcription of genes involved in the normal development of multiple tissue types.?DMX-101?is the first and only treatment specifically targeting?XLHED. Administered during the third trimester of pregnancy, it has the potential to become a ?single course? treatment, significantly improving symptoms of the disease throughout patients? lives. This approach has already demonstrated significant potential in a case series of three patients treated?in utero?with?DMX-101?during the third trimester of pregnancy. The treatment normalized sweat gland function and associated thermoregulation, and improvement in dentition and respiratory function were observed. These results were recently published in the?New England Journal of Medicine?and featured in?Nature Medicine?s 2018 Research Highlights. For more information, please visit?here.

About Dermelix Biotherapeutics

Dermelix?is a privately-held, clinical-stage biopharmaceutical company focused on the development of innovative therapies for rare and debilitating dermatologic conditions with high unmet medical need. For more information, please visit?here.