First patient dosed in pivotal Phase 3 CONnECT clinical trial for US and Japanese markets
Headline data from CONnECT is anticipated in 2024
Diurnal Group plc (AIM: DNL), the specialty pharmaceutical company targeting patient needs in chronic endocrine (hormonal) diseases, announces that the first patient has been dosed in its Phase 3 pivotal clinical trial of hydrocortisone modified-release hard capsules (DNL-0200 – approved in Europe and the UK under the commercial name Efmody®) for adults with congenital adrenal hyperplasia (CAH).
The pivotal Phase 3 randomised, double-blind, active-controlled, clinical trial, known as CONnECT, is evaluating the efficacy, safety and tolerability of modified-release hydrocortisone compared with immediate-release hydrocortisone replacement therapy in participants aged 16-years and over with CAH. The pivotal study for key US and Japan markets worth together in excess of $100m per annum will recruit up to 150 subjects with CAH who will be treated for a period of 52 weeks in centres across Japan, the US, France, and Turkey. The study is anticipated to take up to one year to recruit all patients, with subsequent data readout anticipated in 2024. The study is being conducted under a Special Protocol Assessment (SPA) that has been agreed with the US FDA. The SPA provides agreement that the Phase 3 trial design adequately addresses objectives that would support the regulatory submission for drug approval.
DNL-0200 is a preparation of hydrocortisone that has been specifically designed for patients with diseases of cortisol deficiency such as CAH and is currently approved as the only near physiological cortisol replacement treatment for CAH in Europe and the UK.
Richard Bungay, Interim Chief Executive Officer of Diurnal, commented:
“I am delighted that we have dosed the first patient in this important clinical trial. CONnECT builds on knowledge gained from our European study for DNL-0200, to date the largest ever completed study in CAH, and as the first blinded study, CONnECT should prove to be the definitive evidence for optimal treatment of patients with CAH.”
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Diurnal Group plc
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Richard Bungay, Interim Chief Executive Officer
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Notes to Editors
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 glucocorticoid (steroid) preparations 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 between 1 in 10,000 and 1 in 15,000 individuals globally.
About DNL-0200 (modified-release hydrocortisone)
DNL-0200 is a modified-release oral formulation of hydrocortisone that is designed to match the normal serum levels of the endogenous cortisol circadian rhythm by replicating the normal overnight rise in serum cortisol levels and providing physiological levels upon waking. It is anticipated that providing a near physiological circadian rhythm of cortisol will improve health outcomes in patients with diseases of cortisol deficiency such as CAH and adrenal insufficiency (AI) who are receiving glucocorticoid replacement therapy. DNL-0200 has been extensively studied in 239 human subjects who have taken part in clinical trials in Europe and the US.
DNL-0200 (commercial name Efmody®) was approved by the European Commission in May 2021 and by the UK Medicines and Healthcare Regulatory Agency (MHRA) in July 2021 as a replacement treatment in patients suffering from the genetic condition congenital adrenal hyperplasia (CAH). Efmody ® is commercially available in both Europe and the UK for the treatment of adolescents (greater than 12 years old) and adults with CAH.
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.
Date of Preparation: May 2022 Code: CORP-GLO-0038