US-based biotechnology firm Edimer Pharmaceuticals has completed dosing of the first XLHED-affected neonate in a Phase II clinical trial of the company's ectodysplasin replacement protein, EDI200.
Edimer develops new therapies for the rare genetic disorder X-linked hypohidrotic ectodermal dysplasia (XLHED), an ultra-rare orphan disease of ectoderm development related to a lack of sweat glands, poor temperature control, respiratory problems, and hair and tooth malformations.
The Phase II study is designed to assess the safety, pharmacokinetics, pharmacodynamics and efficacy of EDI200 in XLHED-affected male newborns in the first two weeks of life, with each study subject receiving two doses per week for a total of five doses.
According to the company, affected individuals are at risk for serious and potentially life-threatening hyperthermia and respiratory infections.
Edimer's EDI200 replaces EDA-A1, the protein missing in XLHED and a major regulator of skin and tooth development.
If approved, EDI200 will be the first protein therapeutic to offer a sustained correction of the symptoms of this disorder.
Edimer president and CEO Neil Kirby said the completed dosing of the first patient in the neonate study of EDI200 represents a significant milestone for the company and those affected with XLHED.
"We celebrate the courage of conviction that supported the science at the foundation of EDI200's development and the selfless participation of those involved in the clinical trials who share our goal to create a clinically-significant, life-long health benefit for those affected with XLHED," Kirby said.
The Phase I trial was an open-label, multicentre study that assessed the safety and pharmacokinetics of EDI200.
In the Phase I trial, four adult males and two adult females, were enrolled at two US sites and all have completed the five dose course of EDI200 over two week period.
EDI200 was found to be well-tolerated at the doses to be studied in neonatal subjects and no serious adverse events were reported in the Phase I study.
Previously, EDI200 showed substantial and durable efficacy with notable reduction in mortality and morbidity in the preclinical studies.
Edimer has obtained both FDA and European orphan drug designations and FDA fast-track status for EDI200.