DNL.L Regulatory News (DNL)

15 June 2017

Diurnal Group plc

("Diurnal" or the "Company")

First patient dosed in food matrix compatibility study intended to form part of US Phase III registration package for lead product, Infacort®

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces that it has successfully dosed the first patient in a food matrix compatibility study for Infacort® in healthy volunteers. This study supports the planned US registration package for Infacort® for the treatment of paediatric adrenal insufficiency (AI). As previously announced, Diurnal will 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 (FDA).

The food matrix compatibility study is a single centre, open‑label, randomised, single dose crossover study in 18 healthy adult subjects (EudraCT NUMBER: 2016-001388-36). The primary objective of the study is 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 is 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. AI has 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 is associated with significant morbidity and increased mortality. Currently there are no available preparations of hydrocortisone (the synthetic version of cortisol) in the US specifically designed to treat these young patients. Infacort® has 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 affords seven years' market exclusivity post New Drug Application approval. A robust patent portfolio provides protection for Infacort® to 2034, which significantly bolsters the Company's exclusivity position.

Martin Whitaker, CEO of Diurnal, commented: 

"We are excited to have dosed the first patient in this food matrix compatibility study. 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® is currently under regulatory review in the European Medicines Agency (EMA) for the treatment of paediatric AI having announced the successful completion of the European pivotal trial in July 2016, with approval anticipated in late 2017.

Notes to Editors

About Adrenal Insufficiency

Adrenal Insufficiency (AI) is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. AI has been identified as an orphan disease in the US where there are estimated to be approximately 4,500 sufferers younger than the age of sixteen, with a similar number in Europe. Currently there are no available hydrocortisone preparations in the US or Europe specifically designed to treat these young patients. These children are often administered compounded adult tablets or other unlicensed products. Poor control of disease can result in precocious puberty in young children, virilisation in girls and chronic fatigue leading to a poor quality of life in adulthood resulting in increased morbidity and mortality.

About Infacort®

Infacort® represents the first preparation of hydrocortisone specifically designed for use in children suffering from AI. It is a patented, immediate-release, oral, paediatric formulation of hydrocortisone that allows for age-appropriate dosing in children. This therapeutic approach has the potential to help young patients less than sixteen years of age in the US suffering from diseases due to cortisol deficiency including AI and Congenital Adrenal Hyperplasia (CAH). AI requires life-long treatment and Diurnal's novel approach to product development has the potential to significantly improve these young patients' lives. Diurnal has already submitted for market authorisation in Europe to the European Medicines Agency via the Paediatric Use Marketing Authorisation (PUMA) route and anticipates approval in late 2017.

About Diurnal Group plc

Founded in 2004, Diurnal is a UK-based specialty pharma company developing high quality products for the global market for the life-long treatment of chronic endocrine conditions, including CAH and AI. Its expertise and innovative research activities focus on circadian-based endocrinology to yield novel product candidates in the rare and chronic endocrine disease arena.

For further information about Diurnal, please visit www.diurnal.co.uk