Diurnal receives Marketing Authorisation for Efmody® in Great Britain from the MHRA


2nd July 2021

Efmody® provides a new treatment option for c.5,000 patients suffering from CAH in GB

GB launch of Efmody® expected in Q1 2022

Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a Marketing Authorisation for Efmody® (hydrocortisone modified-release hard capsules – development name Chronocort®) as treatment of adult and adolescent patients (12 years and older) with the rare condition congenital adrenal hyperplasia (CAH). The Marketing Authorisation for Efmody® from the MHRA applies in Great Britain (England, Wales and Scotland) and follows the recent approval of the product in the European Economic Area (including Northern Ireland)(EEA) by the European Commission as announced on 28 May 2021.

The submission to the MHRA included detailed analysis of data from the Company's Phase 3 study, the largest ever completed interventional clinical trial in CAH involving 122 subjects, and an open-label safety extension study of Efmody®. To facilitate timely commercial availability, Diurnal has already commenced market access activities in Great Britain, with the commercial launch anticipated in Q1 2022. The Company intends to mirror its strategy for Alkindi® (hydrocortisone granules in capsules for opening) by commercialising the product itself in Great Britain as well as other core European markets.

Following feedback from the MHRA and consistent with the approach taken in the EEA, the Company has decided not to pursue Orphan Drug Status for Efmody® in Great Britain as it would likely cause a significant delay to the commercial launch. Whilst Orphan Designation provides market exclusivity for 10 years from launch, the Company currently holds granted patents for Efmody® for the treatment of patients with CAH and believes these patents provide sufficient protection for Efmody® until at least 2033 in Great Britain.

Efmody® is a preparation of hydrocortisone that has been specifically designed for patients with CAH, an orphan condition caused by deficiency of adrenal enzymes, most commonly 21-hydroxylase. 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 5,000 patients in Great Britain.

Professor Wiebke Arlt, Institute of Metabolism and Systems Research, University of Birmingham, commented:

“I am excited to see Efmody® approved in Great Britain as a treatment for patients with CAH, a rare condition where patients and doctors need to face the challenge of deficient cortisol production and adrenal androgen excess. Efmody® has been developed for these patients and I believe this drug approval provides a new option for the treatment of CAH patients, where there is a significant unmet medical need.”

Martin Whitaker, Chief Executive Officer of Diurnal, commented:

“We are pleased to have received approval for Efmody® from the MHRA and look forward to making Efmody® available to CAH patients in Great Britain as the first licensed treatment specifically designed to mimic the physiological circadian rhythm of cortisol. Today’s approval is yet another important step forward for Diurnal as we continue to drive towards becoming the world’s leading specialty endocrinology business.”

This is a business press release containing financial information and/or data for the benefit of shareholders and potential investors. Data are included to allow informed investment decisions.

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

For further information, please visit www.diurnal.co.uk or contact:

Diurnal Group plc +44 (0)20 3727 1000

Martin Whitaker, Chief Executive Officer

Richard Bungay, Chief Financial Officer

Panmure Gordon (UK) Limited (Nominated Adviser and Sole Broker) +44 (0)20 7886 2500

Corporate Finance: Freddy Crossley, Emma Earl

Corporate Broking: Rupert Dearden

FTI Consulting (Media and Investor Relations) +44 (0)20 3727 1000

Simon Conway

Victoria Foster Mitchell

Alex Davis

Notes to Editors

About Congenital Adrenal Hyperplasia

Congenital adrenal hyperplasia (CAH) is a rare 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 glucocorticoid (steroids including hydrocortisone, dexamethasone, prednisolone and prednisone in the US) 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 and Great Britain, with over 400,000 in the rest of the world.

About Efmody® (hydrocortisone modified-release hard capsules)

Efmody® is a preparation of hydrocortisone that has been specifically designed to mimic the circadian rhythm of cortisol when given in a twice-a-day "toothbrush" regimen (administered 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 indication for Efmody® is Congenital Adrenal Hyperplasia (CAH) in adults and adolescents (children older than 12 years of age). Efmody® has been extensively studied in 239 human subjects including 138 CAH patients who have taken part in clinical trials in Europe and the US.

The MHRA and European Commission marketing authorisation approval of Efmody® is based on a Phase 3 study conducted in a total of 122 patients enrolled across 11 clinical sites, including sites in Great Britain, the largest ever interventional clinical trial completed in CAH. The Phase 3 data was supported by detailed analysis of data from an open-label safety extension study for patients completing treatment in the Phase 3 study, which is assessing the impact of treatment with Efmody® over an extended period, with a number of patients on this trial having been treated for over 54 months. Patient retention rates in this study have been high and patients on this trial have, to date, shown sustained benefit from extended Efmody® treatment. Summary of Product Characteristics (SmPC) for UK (Northern Ireland) can be found here.

About Diurnal Group plc

Diurnal Group plc is a European, UK-headquartered, specialty pharmaceutical company dedicated to developing hormone therapeutics to aid lifelong treatment for rare and chronic endocrine conditions, including congenital adrenal hyperplasia, adrenal insufficiency, hypogonadism and hypothyroidism. 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: July 2021               Code: CORP-GB-0137

Diurnal Group plc (Company Number: 09846650) is registered in England & Wales. Registered office: Cardiff Medicentre, Heath Park, Cardiff CF14 4UJ, UK.