Ark gets nod from EMEA for early-stage fetal drug

UK-based Ark Therapeutics says that it has received positive feedback from the European Medicines Agency's (EMEA) Gene Therapy Working Party regarding the preclinical toxicology and Phase I trial requirements for EG013 (Marketletter June 23), an adenovirally-mediated VEGF-based product being developed for fetal growth restriction.

The GTWP clarified the indication (severe fetal growth restriction), the method of administration (imaging guided catheter introduced via the femoral vessels in the groin) and commented on a program of in vivo model and in vitro tissue work incorporating biodistribution and germ line integration. The GTWP rapporteur had no major concerns with regard to the Phase I trial proposed by Ark, with neonatal outcome being an efficacy endpoint, said the firm.

Results reported by Ark from the first two trials of EG013 in a preclinical model of placental blood flow have shown that a single treatment with EG013, given directly into the mother's uterine artery, increased blood flow to the placenta by 25%, and that the effect is maintained for 50 days. Preliminary biodistribution results using immuno-histochemical techniques have indicated that there is no transfer of the gene into the fetus in the first days. If confirmed in human studies, a therapy with this magnitude and duration of effect could allow the fetus to grow satisfactorily to a stage where cesarean delivery of a healthy baby could be reliably performed, the firm says. Ark is currently undertaking work to select the optimal VEGF gene variant for EG013 and expects to complete this early in the fourth quarter prior to commencing final preclinical work.