Amryt Pharma PLC AP101 trial discussions completed with FDA & EMA (5424Y)
March 06 2017 - 2:00AM
UK Regulatory
TIDMAMYT
RNS Number : 5424Y
Amryt Pharma PLC
06 March 2017
AIM: AMYT, ESM: AYP
Amryt Pharma plc
("Amryt" or the "Company")
Completion of Pivotal Clinical Trial Discussions with FDA and
EMA
Phase 3 trial now ready to commence for
AP101 as a treatment in Epidermolysis Bullosa
Amryt, the pharmaceutical company focused on best-in-class
treatments for rare and orphan diseases, is pleased to announce the
completion of discussions with the Food and Drug Administration
("FDA") and European Medicines Agency ("EMA") regarding the design
of its pivotal phase 3 clinical trial for AP101 as a potential
treatment for Epidermolysis Bullosa ("EB"). EB is a rare genetic
skin disorder that leads to exceptionally fragile skin and children
with the disorder are often referred to as "Butterfly Children".
The global market for a treatment in EB is estimated to be in
excess of EUR 1.3 billion.
With regulatory authority discussions now completed with FDA and
EMA and the design of the clinical trial established, Amryt is on
track to commence the phase 3 trial at the end of March. Amryt has
also agreed to conduct some further non-clinical studies in
parallel with this phase 3 study. INC Research has been appointed
as the contract research organisation for the phase 3 study, and
approximately 30 clinical trial sites in 15 countries have already
been pre-qualified.
Adult and paediatric patients with EB will be enrolled into a
randomised double blind placebo controlled trial. A total of 164
evaluable patients will be treated for a 90 day blinded period. The
proportion of patients with completely healed target wounds within
45 days will be evaluated as the primary endpoint. Secondary
endpoints include the time to achieve wound healing and changes in
pain and pruritus (itch).
An important component of the phase 3 study is an independent
data monitoring committee that will conduct an un-blinded interim
efficacy analysis after 50% enrolment. The potential outcomes of
this interim analysis include continuation of the study unchanged,
discontinuation of the study for futility, or an increase in the
number of patients in the study to preserve adequate statistical
power.
Joe Wiley, Chief Executive Officer of Amryt, commented:
"We are delighted to have reached this critical milestone in the
development of Amryt. Reaching agreement with the regulatory
authorities for our phase 3 study in EB was a precursor to
commencing our pivotal study, which we now expect will enrol its
first patients in the coming weeks. Significantly, we now have
agreement from the regulatory agencies in both the US and in Europe
for a single phase 3 study, which we hope in due course will lead
to approval in EB. There remains substantial unmet need for drugs
which can address the symptoms and we are delighted to be
progressing our potential treatment to this pivotal phase."
Mark Sumeray, Chief Medical Officer of Amryt, commented:
"We are very pleased to have reached an understanding with the
US and EU regulatory authorities on the design of our pivotal phase
3 trial for AP101 as a potential treatment for EB. We believe that
the adaptive approach to study sample size will help to ensure that
we achieve a reliable assessment of the potential benefit of our
new skin healing treatment in this orphan disease."
Enquiries:
c/o KTZ Communications
Amryt Pharma plc +44 (0)20 3178 6378
Joe Wiley, CEO
Rory Nealon, CFO/COO
Shore Capital +44 (0) 20 7408 4090
Nomad and Joint Broker
Bidhi Bhoma, Edward Mansfield
Davy +353 (1) 679 6363
ESM Adviser and Joint Broker
John Frain, Anthony Farrell
Stifel +44 (0) 20 7710 7600
Joint Broker
Jonathan Senior, Ben Maddison
KTZ Communications +44 (0) 20 3178 6378
Katie Tzouliadis, Emma Pearson
About Amryt Pharma plc - see www.amrytpharma.com
Amryt Pharma is a specialty pharmaceutical company focused on
developing and delivering innovative new treatments to help improve
the lives of patients with rare or orphan diseases. The Company is
building a diversified portfolio of commercially attractive,
best-in-class, proprietary new drugs to help address some of these
rare and debilitating illnesses for which there are currently no
available treatments.
It recently acquired an exclusive licence to sell LOJUXTA
(lomitapide), across the EU and other territories including the
Middle East, North Africa, Turkey and Israel. LOJUXTA is used to
treat a rare life-threatening disease called Homozygous Familial
Hypercholesterolemia.
Amryt's product, AP101, received marketing approval for the
treatment of partial-thickness wounds from the European Commission
in January 2016. Amryt intends to develop AP101 as a new treatment
for Epidermolysis Bullosa ("EB"), a rare and distressing genetic
skin disorder affecting young children for which there is currently
no treatment. AP101 has been granted US and EU orphan drug
designation. It has patent grants in the US and in Europe, as well
as other territories including Japan, Canada and Australia. The
global market opportunity for EB is estimated to be in excess of
EUR 1.3 billion.
Amryt's earlier stage product AP102 is focused on developing
novel, next generation somatostatin analogue ("SSA") peptide
medicines for patients with rare neuroendocrine diseases, where
there is a high unmet medical need, including acromegaly and
Cushing's disease. AP102 was recently granted orphan designation in
the US in acromegaly by the FDA.
The Company joined AIM and Dublin's ESM in April 2016 following
the reverse takeover of Fastnet Equity PLC.
This information is provided by RNS
The company news service from the London Stock Exchange
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