Diurnal doses first patient in US Infacort study

The AIM-traded firm said the study supported the planned US registration package for Infacort for the treatment of paediatric adrenal insufficiency.

As it had previously announced, Diurnal would initiate the pivotal Phase III clinical trial in the target paediatric population - 0-16 years of age - following final confirmation on the trial design from the US Food and Drug Administration.

The food matrix compatibility study was described by the company as a single centre, open‑label, randomised, single dose crossover study in 18 healthy adult subjects.

It said the primary objective of the study was to evaluate the bioavailability of Infacort multiparticulate granules administered as sprinkles with soft food or yoghurt, compared with direct administration to the back of the mouth.

The secondary objective was to assess the safety and tolerability of Infacort throughout the study.

AI is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress, Diurnal’s board said.

It had been identified as an orphan disease in the US where there are estimated to be approximately 4,500 sufferers under the age of 16.

Untreated, the disease was associated with “significant” morbidity and increased mortality.

Currently there were no available preparations of hydrocortisone - the synthetic version of cortisol - in the US specifically designed to treat those young patients.

Infacort reportedly had the potential to be the first pharmaceutically-defined dose and consistent formulation of hydrocortisone designed specifically for children.

In the US, Diurnal holds ‘Orphan Drug Designation’ for Infacort in paediatric AI, which afforded the company seven years of market exclusivity after New Drug Application approval.

A “robust” patent portfolio provided protection for Infacort to 2034, the board claimed, which would significantly bolster the company's exclusivity position.

“We are excited to have dosed the first patient in this food matrix compatibility study,” said Diurnal CEO Martin Whitaker.

“This is an important prerequisite to the planned US pivotal Phase III clinical trial for Infacort in the treatment of paediatric adrenal insufficiency, for which we await final confirmation on the study design with the FDA before commencing.

“We believe the US will be an important market for Infacort where it has the potential to address a significant unmet need for children with adrenal insufficiency who are not being treated satisfactorily.”

In Europe, Infacort was currently under regulatory review by the European Medicines Agency or the treatment of paediatric AI, having announced the successful completion of the European pivotal trial in July 2016, with approval anticipated in late 2017.