DNL.L Regulatory News (DNL)

9 January 2018

Diurnal Group plc

("Diurnal" or the "Company")

Diurnal reports positive results from food matrix compatibility study intended to form part of US registration package for Alkindi®

Results show Alkindi® retains bioavailability in adults when administered with food

Dosage shown to be well tolerated

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces positive data from the food matrix compatibility study for Alkindi® (development programme name: Infacort®; hydrocortisone granules in capsules for opening) in healthy adult volunteers. This study supports the planned US registration package for Alkindi® for the treatment of paediatric adrenal insufficiency (AI). The Company has opened an IND for Alkindi® and, following advice from the US Food and Drug Administration (FDA), will be in a position to start the next study in the registration programme, a bioequivalence study in healthy adult volunteers, during H1 2018.

The food matrix compatibility study was a single centre, open‑label, randomised, single dose crossover study in 18 healthy adult subjects (EudraCT NUMBER: 2016-001388-36; ClinicalTrials.gov Identifier: NCT03178214). The primary objective of the study was to evaluate the bioavailability of Alkindi® multi-particulate granules administered as sprinkles onto 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 Alkindi® throughout the study.

The results of the study confirm that the pharmacokinetics of Alkindi® when sprinkled onto soft food or yoghurt are equivalent to Alkindi® administered directly. There were no adverse events and Alkindi® was well tolerated.

Paediatric AI is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. Paediatric 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. Alkindi® has the potential to be the first pharmaceutically defined dose and consistent formulation of hydrocortisone (the synthetic version of cortisol) designed specifically for children.

In the US, Diurnal holds Orphan Drug Designation for Alkindi® for paediatric AI, which affords seven years' market exclusivity post New Drug Application approval. A robust, granted, patent portfolio provides protection for Alkindi® to 2034, which significantly bolsters the Company's exclusivity position.

Martin Whitaker, CEO of Diurnal, commented: 

"We are pleased to announce positive data for this important prerequisite to support the planned Alkindi® registration programme in the US. We believe the US will be an important market for Alkindi® where it has the potential to address a significant unmet need for children with adrenal insufficiency who are not being treated satisfactorily and we continue our discussions with the FDA to finalise additional requirements for the registration package for the product."

In Europe, the Company has received a positive opinion for approval for Alkindi® from the European Medicines Agency and the final decision on the paediatric use market authorisation (PUMA) from the European Commission is anticipated in February 2018. The European launch of Alkindi® is expected to follow in Q2, 2018.

Notes to Editors

About Alkindi®

Alkindi® represents the first preparation of hydrocortisone specifically designed for use in children suffering from paediatric AI. Alkindi® is a patented, oral, immediate-release paediatric formulation of hydrocortisone granules in capsules for opening 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 paediatric 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.

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