-- First neonate subject with X-linked hypohidrotic ectodermal
dysplasia completes dosing with the novel ectodysplasin replacement
protein EDI200 --
“The completed dosing of the first patient in the neonate study of EDI200 represents a significant milestone for Edimer and those affected with XLHED,” said
“The first research project the NFED funded was in 1989 for the gene identification of X-Linked Hypohidrotic Ectodermal Dysplasia, XLHED,” said
About the Phase 2 Clinical Trial
The Phase 2 clinical trial is designed to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of EDI200 in XLHED-affected male newborns in the first two weeks of life. EDI200 dosing will be initiated between the 2nd and 14th days of life, with each study subject receiving two doses per week for a total of five doses. For additional information on this clinical trial, please visit clinicaltrials.gov, identifier NCT01775462.
Phase 1 Clinical Trial Outcomes
The EDI200 Phase 1 trial was an open-label, multicenter study to evaluate the safety and pharmacokinetics of EDI200. XLHED-affected males and females were enrolled in anticipation of future studies dosing XLHED-affected neonates. Six adult subjects, four males and two females, were enrolled at two U.S. sites and all successfully completed the five dose course of EDI200 over two weeks. EDI200 was generally well tolerated at the doses to be studied in neonatal subjects. No SAE’s were reported and the majority of AEs in this open-label study were mild and transient with full resolution. An independent Data Safety Monitoring Board reviewed all adult safety data and approved the dosing and monitoring protocol for the Phase 2 neonate study. For a developmental disorder such as XLHED, there was no expectation of clinical benefit following EDI200 administration to adult subjects. However, several subjects administered the higher dose of EDI200 demonstrated a short-term improvement in hair growth, dry eye symptoms and lung inflammation. Larger studies and a longer follow-up period will be required to determine if these changes represent an unanticipated and possibly sustainable benefit related to EDI200 treatment.