DNL.L Regulatory News (DNL)

15 September 2017

Diurnal Group plc

("Diurnal" or the "Company")

Pivotal Phase III data for Diurnal's Infacort® published in Clinical Endocrinology

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces that data from the pivotal Phase III trial for its lead product Infacort® are published in Clinical Endocrinology. 

The paper titled, "Absorption and tolerability of taste-masked hydrocortisone granules in neonates, infants and children under 6 years of age with adrenal insufficiency" is published on an open access basis. An oral presentation of the data by one of the investigators, Dr. Oliver Blankenstein, will take place on Saturday 16 September 2017 at the joint meeting of the European Society of Paediatric Endocrinology and the Pediatric Endocrine Society in Washington DC; the largest global gathering of more than 4,000 Paediatric Endocrinologists from over 100 countries.

Adrenal Insufficiency (AI) in children is currently treated by crushing or compounding adult preparations of hydrocortisone with no regulated dose or consistent formulation for children. Infacort®, Diurnal's most clinically advanced product, is specifically designed to provide the first regulated, consistent preparation of hydrocortisone to ensure efficacy and safety and ease of use for children suffering from AI, including the related disease, Congenital Adrenal Hyperplasia (CAH). Infacort is currently under regulatory review by the European Medicines Agency (EMA) and Diurnal anticipates receiving recommendation for approval in Europe towards the end of 2017. The US is also expected to be a major territory for sales of Infacort® and the Company continues to progress discussions with the US Food and Drug Administration (FDA) regarding the requirements for the registration programme in the US.

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 licensed 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 Congenital Adrenal Hyperplasia

Congenital Adrenal Hyperplasia (CAH) is an orphan condition caused by deficiency of adrenal enzymes, most commonly 21-hydroxylase. This enzyme is required to produce the adrenal steroid hormone, cortisol. The block in the cortisol production pathway causes the over-production of male steroid hormones (androgens), which are precursors to cortisol. The condition is congenital (inherited at birth) and affects both sexes. The cortisol deficiency and over-production of male sex hormones can lead to increased mortality, infertility and severe development defects including ambiguous genitalia, premature (precocious) sexual development and short stature. Sufferers, even if treated, remain at risk of death through an adrenal crisis.

Approximately two thirds of CAH patients are estimated to have poor disease control, leading to elevated androgen levels. The condition is estimated to affect approximately 71,000 patients of all ages in Europe (51,000) and the US (20,000), with approximately 405,000 in the rest of the world.

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 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 Congenital Adrenal Hyperplasia and Adrenal Insufficiency. 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