TIDMDNL
RNS Number : 2791M
Diurnal Group PLC
12 October 2016
12 October 2016
Diurnal Group plc
("Diurnal" or the "Company")
Diurnal announces full results from positive European
Infacort(R) Phase III study
As previously disclosed, primary endpoint met with high
statistical significance
Results also show Infacort(R) achieved comparable cortisol
levels in children with adrenal insufficiency to those of healthy
children
95% of parents/carers preferred Infacort(R) to current
treatment
European market authorisation anticipated in late 2017
Diurnal Group plc (AIM: DNL), the specialty pharmaceutical
company targeting patient needs in chronic endocrine (hormonal)
diseases, is pleased to announce full results from the Phase III
European study for Infacort(R) in paediatric Adrenal Insufficiency
(AI). Positive headline data from this pivotal study were published
on 11 July 2016 and today's release follows a comprehensive
analysis of the data.
The Phase III trial was designed in agreement with the European
Medicines Agency (EMA) and conducted in a total of 24 neonates and
children under the age of six years who require replacement therapy
for AI due to Congenital Adrenal Hyperplasia (CAH), primary adrenal
failure or hypopituitarism.
Comprehensive analysis of the results confirms that the study
met its primary endpoint, demonstrating a statistically significant
(p<0.0001) increase in cortisol values following administration
of Infacort(R) compared to pre-dose values. The median 60--minute
cortisol level achieved after Infacort(R) treatment was 535 nmol/L,
which is comparable to the mean peak morning cortisol of healthy
children.
AI is currently treated by unlicensed preparations of
hydrocortisone with no regulated dose or consistent formulation.
Infacort(R) is designed to provide the first regulated, consistent
preparation of hydrocortisone to ensure efficacy and safety and
ease of use for young children. In this respect, Infacort(R) was
well tolerated with no adverse events reported. As part of the
trial, a series of surveys were completed by parents/carers, the
results of which showed that 82% of parents/carers agreed that
their child found swallowing Infacort(R) easy, 95% of
parents/carers preferred Infacort(R) to the usual, unlicensed
preparations, and 95% of parents/carers would be happy to give
their child Infacort(R) in the future. Furthermore, in the cohort
of babies aged 0-28 days, 100% of parents/carers preferred
Infacort(R) to the usual, unlicensed hydrocortisone
preparations.
Prof Heiko Krude of Charité-Universitätsmedizin Berlin, one of
the leading paediatric endocrine hospitals in Germany, and
co-investigator in the trial, commented:
"This carefully controlled study clearly demonstrates, for the
first time, the value of a standard dose of hydrocortisone in
controlling cortisol levels in children with adrenal
insufficiency."
Dr Oliver Blankenstein of Charité-Universitätsmedizin Berlin and
co-investigator in the trial, added:
"These important data suggest that Infacort(R) could be an
effective and safe cortisol replacement treatment able to address a
significant unmet need for children with adrenal insufficiency.
Infacort(R) also has the potential to increase compliance through
the product's taste-masking excipients specifically designed to
eliminate the bitter taste of hydrocortisone."
The EMA previously approved a Paediatric Investigation Plan
(PIP) for Infacort(R) , setting out the regulatory pathway to
market authorisation via the Paediatric Use Marketing Authorisation
(PUMA) route. Diurnal is on track to submit this regulatory dossier
to the EMA around the end of 2016. If approved, Infacort(R) has the
potential to be the first licensed treatment in Europe for AI
(including CAH) specifically designed for use in children. Diurnal
anticipates market authorisation in late 2017.
Martin Whitaker, CEO of Diurnal, commented:
"The successful completion of the pivotal trial programme for
Infacort(R) in Europe is another important step towards realising
our vision of improving the lives of patients with adrenal
insufficiency and CAH, and building Diurnal into a leading global
endocrinology company. We look forward to filing the drug for
approval with the EMA in the coming weeks and anticipate approval
in late 2017."
Overview of Infacort(R) pivotal trial design
The pivotal phase III trial was an open-label study of
Infacort(R) in neonates and children less than 6 years of age with
adrenal insufficiency carried out at Charité-Universitätsmedizin,
Berlin Germany and consisted of three cohorts that were carried out
in series:
- Cohort 1: 12 patients with adrenal insufficiency aged 2 - 6 years of age
- Cohort 2: 6 patients with adrenal insufficiency aged 1 month to 2 years of age (infants)
- Cohort 3: 6 patients with adrenal insufficiency aged 0 to 1 month of age (neonates)
The primary objective of the study was to measure peak and
four-hour cortisol levels after administration of a single dose of
Infacort(R) . The secondary objective was safety.
Please further information, please visit www.diurnal.co.uk or
contact:
Diurnal Group plc +44 (0)20 3727 1000
Martin Whitaker, CEO
Ian Ardill, CFO
Numis Securities Ltd +44 (0)20 7260 1000
Nominated Adviser: Michael
Meade, Freddie Barnfield,
Paul Gillam
Corporate Broking: James
Black
FTI Consulting + 44 (0)20 3727 1000
Simon Conway
Victoria Foster Mitchell
About Adrenal Insufficiency
Adrenal Insufficiency (AI) is a condition characterised 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 estimated to 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 tablets or other
unlicensed products. Poor control of disease can result in
precocious puberty in young children, virilisation in girls and
chronic fatigue leading to a poor quality of life in adulthood
resulting in increased morbidity and mortality.
About Infacort(R)
Infacort(R) represents the first preparation of hydrocortisone
specifically designed for use in children suffering from AI. It is
a patented, immediate-release, oral, paediatric 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. The European Medicines Agency
has already approved a Paediatric Investigation Plan
(EMEA-001283-PIP01-12) for Infacort(R) , which sets out the
regulatory pathway to market authorisation via the Paediatric Use
Marketing Authorisation (PUMA) route.
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.
This information is provided by RNS
The company news service from the London Stock Exchange
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