DNL.L Regulatory News (DNL)

16 December 2019

Diurnal Group plc

("Diurnal" or the "Company")

Chronocort® Marketing Authorisation Application Submitted in Europe

Significant opportunity to address unmet patient need, in a market estimated at $250 million

Marketing authorisation anticipated in Q1 2021

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces that, in line with expectations, a Market Authorisation Application (MAA) has been submitted to the European Medicines Agency (EMA) for Chronocort® (modified release hydrocortisone) as a treatment for adult and adolescent patients with the rare condition congenital adrenal hyperplasia (CAH) in Europe.

The MAA submission follows Diurnal's Intent to Submit letter in May 2019, which was tendered following a positive meeting with the EMA in March 2019 and written formal Scientific Advice received in April 2019 confirming the clinical and regulatory pathway for Chronocort® as a treatment for patients with CAH. The pathway is based on detailed analysis of data from the Company's Phase 3 study, the largest ever interventional clinical trial in CAH, and an open-label safety extension study. Marketing authorisation of Chronocort® in Europe is anticipated in Q1 2021.

In parallel with the MAA submission, Diurnal will apply for confirmation of Orphan Drug Status for Chronocort® in CAH, which requires Diurnal to demonstrate significant clinical benefit for the product compared to existing therapies. Should Chronocort® be approved, it will provide the potential for life-long treatment, with patients commencing treatment with Alkindi® (hydrocortisone granules in capsules for opening), the Company's approved paediatric product, transitioning to Chronocort® in adolescence and continuing with Chronocort® treatment into later life.

Martin Whitaker, CEO of Diurnal, commented: 

"The submission of a Marketing Authorisation Application for Chronocort® to the European Medicines Agency is an important step towards the approval of our second product and continues to build our commercial portfolio in endocrinology. There is a significant need for new therapies to improve outcomes for adult patients with CAH, which still results in increased morbidity and mortality worldwide. We believe that Chronocort® together with our paediatric product Alkindi® has the potential to provide new treatment options for patients with CAH"

CAH is an orphan condition caused by a block in cortisol production, an essential adrenal steroid hormone required for healthy life. A lack of cortisol in turn causes the over-production of male steroid hormones (androgens). Cortisol deficiency and over-production of androgens can lead to increased mortality, infertility and issues during sexual development, including ambiguous genitalia, precocious puberty and short stature. Sufferers, even if treated, remain at risk of death through an adrenal crisis. The condition is estimated to affect a total of approximately 41,000 adult patients in Europe, with over 400,000 in the rest of the world.

This announcement contains inside information for the purposes of Article 7 of Regulation (EU) 596/2014 (MAR).

Notes to Editors

About Chronocort®

Chronocort® is a modified release preparation of hydrocortisone that has been specifically designed to mimic the circadian rhythm of cortisol when given in a twice-a-day "toothbrush" regimen (last thing at night before sleep and first thing in the morning on waking) to control androgen excess and chronic fatigue in patients with diseases of cortisol deficiency. The first planned indication for Chronocort® is Congenital Adrenal Hyperplasia (CAH) in adults and adolescents. Chronocort® has been extensively studied in human subjects having completed four Phase I trials, a Phase II trial in 16 CAH patients in the US in 2014, and a Phase III trial in 122 CAH patients in Europe and the US.

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 issues during sexual development including ambiguous genitalia, premature (precocious) sexual development and short stature. Sufferers, even if treated, remain at risk of death through an adrenal crisis.

Current therapy for CAH uses a variety of generic steroids (hydrocortisone, dexamethasone and prednisolone) with no standard treatment regimen. Approximately two-thirds of CAH patients are estimated to have poor disease control, leading to elevated androgen levels. The condition is estimated to affect a total of approximately 41,000 patients in Europe, with over 400,000 in the rest of the world.

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 

Date of Preparation: December 2019 Code: CORP-GB-0046