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CLINIGEN & DIURNAL PARTNER FOR EUROPEAN MA PROGRAM.

Clinigen Group plc's Idis Managed Access (MA) division and Diurnal Group plc have partnered to launch a Managed Access program in Europe for Infacort to treat pediatric patients with adrenal insufficiency (AI) and Chronocort to treat patients with congenital adrenal hyperplasia (CAH). CAH is the most common inherited hormone disorder affecting both men and women. It is caused by genetic mutations of the enzymes in the adrenal gland that synthesize steroid hormones. Patients with CAH have a deficiency of the stress hormone, cortisol. Their body's response to this is for the adrenal glands to produce more of the male steroid hormones (androgens), which, in excess levels, are harmful to patients. Cortisol deficiency and over-production of androgens can lead to increased mortality, infertility and severe development defects, including ambiguous genitalia, precocious puberty and short stature. 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 51,000 patients in Europe.

Infacort has been specifically designed for use in children suffering from AI and has the potential to help young patients less than six years of age with cortisol deficiency including AI and CAH. Diurnal submitted a Pediatric Use Marketing Authorization (PUMA) application for Infacort to the European Medicines Agency (EMA) in late 2016.

Chronocort has orphan drug designation in Europe and the US for the treatment of CAH and AI in adult patients. Chronocort is currently in Phase III clinical development and therefore not yet commercially available.

Run by Idis MA, the Managed Access programwill enable physicians in the European Union to access Infacort and Chronocort as unlicensed medicines on an individual named patient basis for patients who have no other treatment options, ahead of European approval and commercial launch.

Healthcare Professionals can obtain details about the Infacort and ChronocortManaged Access program by calling +44 (0) 1283 495 010, or emailing customer.services@clinigengroup.com

About Adrenal Insufficiency

Adrenal Insufficiency (AI) is a condition characteried by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress.AI has been identified as a rare disease in Europe where there are estimatedto be approximately 4,000 sufferers younger than the age of six.Currently there are no licensed hydrocortisone preparations in Europe specifically designed to treat these young patients.These children are often administered compounded adult tabletsor other unlicensed products.Poor control of disease can result in precocious puberty in young children, virilization 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 usually caused by deficiencyof the enzyme 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 in Europe (51,000) and the US (20,000), with approximately 405,000 in the rest of the world.

Current therapy for CAH uses a combination of generic steroids (hydrocortisone, dexamethasone and prednisolone) and, at best, these adequately treat approximately one third of CAH patients. Other therapies being developed are at an early stage of development and not expected to receive approval in the short-term.

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, pediatric formulation of hydrocortisone that allows for age- appropriate dosing in children. This therapeutic approach has the potential to help young patients less than six year of age suffering from diseases due to cortisol deficiency including adrenal insufficiency and congenital adrenal hyperplasia. 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 authorization to the European Medicines Agency via the Pediatric Use Marketing Authorization (PUMA) route in late 2016.

About Chronocort

Chronocort is a modified release hydrocortisone preparation that has been designed to mimic the natural 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). Chronocort has been granted orphan drug designations in Europe and the US in the treatment of Congenital Adrenal Hyperplasia (CAH) and Adrenal Insufficiency (AI). The first planned indication for Chronocort is CAH. Chronocort has completed three Phase I trials in 2011, 2012 and 2015 (food effects study) and a Phase II trial in CAH patients in 2014, and is currently in Phase III trials in Europe.

About Clinigen Group

Clinigen Group plc (AIM: CLIN) is a global pharmaceutical and services company with a unique combination of businesses focused on providing access to medicines. The Group consists of five synergistic businesses focused in three areas of global medicine supply; clinical trial, unlicensed and licensed medicines.

Clinigen Clinical Trial Services is a provider of management and supply of commercial medicines for clinical trials. The Group is also the trusted global leader in ethically sourcing and supplying unlicensed medicines to hospital pharmacists and physicians for patients with a high unmet need, through three of its divisions: Idis Managed Access runs early access programs for innovative new medicines. Idis Global Access and Link Healthcare work directly with healthcare professionals to enable compliant access to unlicensed medicines on a global basis and niche essential licensed and generic medicines across Australasia, Africa and Asia (AAA region). Clinigen Specialty Pharmaceuticals acquires global rights, revitalizes and markets its own portfolio of niche hospital medicines. For more information, visit www.clinigengroup.com.

About Diurnal

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.

For more information, visit www.diurnal.co.uk.
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Publication:Worldwide Biotech
Date:May 1, 2017
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