EspeRare and Dermelix partner to tackle rare pediatric genetic diseases

EspeRare is a not-for-profit organization whose model combines pharmaceutical know-how with philanthropic, public and private investments to develop and launch discontinued therapies. One of its company’s products is the DMX-101, a novel in utero protein replacement therapy for the treatment of XLHED, a rare pediatric genetic disease affecting approximately 4/100,000 live male births every year.

There is currently no approved therapy for the treatment of XLHED, thus DMX-101 has the potential to become the first ever in utero administered drug to correct a genetic disease before birth.

To further develop DMX-101, EspeRare has now entered a partnership with Dermelix Biotherapeutics. 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 the 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

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.

Ethical Charter within the license agreement
With Dermelix, EspeRare has won 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.

(Press release)