CMC Biologics will manufacture Edimer Pharmaceuticals’ EDI200, a recombinant protein for the treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED). This rare genetic disease has orphan designation in the U.S. and Europe.
CMC Biologics was selected due to its depth of expertise in protein development, analytical development, and its cGMP manufacturing capabilities. "CMC's extensive experience in the GMP manufacture of therapeutic proteins makes them an ideal company to help us move this product forward," remarks Neil Kirby, Ph.D., Edimer CEO.
XLHED is diagnosed on the basis of fine, sparse hair, few and often pointed teeth, and diminished or absent sweat function. There are a number of secondary features of HED that may include a reduction in mucous glands in the pharynx, larynx, trachea, and bronchi, dry eye symptoms, eczema, asthma, and dry mucous membranes in the mouth and nose.
Edimer says that EDI200 is at the clinical stage of investigation. It is a form of ectodysplasin-A1 (EDA-A1), a signaling protein expressed in healthy individuals that is involved in the formation of sweat glands, teeth, hair, and certain glandular structures. This protein is missing in patients with XLHED.
EDI200 has reportedly been shown to substitute for lack of functional EDA-A1 protein in mouse and dog models of XLHED. In a particular species of mouse known as Tabby, EDA-A1 is missing, and they have many of the symptoms associated with XLHED. Treatment of pregnant Tabby mice with EDI200 permanently corrected the appearance of the offspring. Equivalent results were achieved after injecting the protein into Tabby mice shortly after birth. In addition dogs lacking EDA-A1 (and with symptoms quite similar to humans with XLHED) have been treated successfully with EDI200, according to Edimer.