TIDMAMYT
RNS Number : 8082Z
Amryt Pharma PLC
15 January 2020
15 January 2020
AIM: AMYT
Euronext Growth: AYP
AMRYT PHARMA PLC
("Amryt" or the "Company")
Amryt to present updates from its Epidermolysis Bullosa ("EB")
research programme at the first World Congress 'EB2020' dedicated
to this rare skin disease
Amryt, an international biopharmaceutical company focused on
developing and delivering innovative new treatments to help improve
the lives of patients with rare and orphan diseases, is pleased to
announce its participation at the inaugural Epidermolysis Bullosa
World Congress ("EB2020"), being held on 19-23 January, 2020 in
London.
Amryt's EB research programme aims to develop transformative
treatments to improve symptoms and potentially offer a cure for
this rare and distressing genetic skin disorder affecting young
children and adults for which there is currently no approved
treatment. The programme includes the largest ever global Phase 3
trial in EB, the EASE trial, which is being conducted at 55 sites
in 27 countries with results expected later this year.
At EB2020, information on an investigational treatment for EB,
Oleogel-S10 (AP101) will be discussed in a dedicated scientific
satellite symposium. In addition, Amryt scientist Dr Lara Cutlar
has been invited by the congress organisers to present a talk on
non-viral gene therapy delivery mechanisms. Both events will take
place on Tuesday 21 January in the main congress hall. Amryt's
Chief Medical Officer - Dr Mark Sumeray - will present on the Amryt
research programme as part of the conference programme on Wednesday
22 January. Amryt is proud to be a platinum sponsor of the
Congress.
Dr Joe Wiley, CEO of Amryt Pharma, commented today:
"Collaboration is central to Amryt's culture and EB2020 presents
an ideal opportunity for us to share our ideas, meet new partners
and galvanise our shared vision to make a real difference for
people living with EB. We have partnered with the EB community for
over three years to highlight the condition and raise funds to
support their research efforts, alongside the significant
investment we have made in EB research at Amryt."
Caroline Collins, EB2020 Congress Director & DEBRA UK's
Director of Research, commented today:
"We are delighted with the support for the first EB World
Congress. This meeting will give guidance to professionals involved
in EB, and drive forward strategies for research, regulatory,
funding, and for the healthcare communities. Collaborating with
industry partners, sponsors such as Amryt, healthcare
professionals, and the EB Community gives us hope that people
living with EB will have a much brighter future."
EB2020 is an independent, global congress bringing together the
world's leading experts in EB research, clinical management, and
the EB Community. The congress is being held in London on January
19-23, 2020. For more information please visit
www.ebworldcongress.org.
ENDS
Enquiries:
Amryt Pharma plc +353 (1) 518 0200
Joe Wiley, CEO
Rory Nealon, CFO/COO
Consilium Strategic Communications +44 (0) 20 3709 5700
Amber Fennell, Matthew Neal, Nicholas
Brown
LifeSci Advisors, LLC +1 (212) 915 2564
Tim McCarthy
About Amryt
Amryt is a biopharmaceutical company focused on developing and
delivering innovative new treatments to help improve the lives of
patients with rare and orphan diseases. Amryt comprises a strong
and growing portfolio of commercial and development assets.
Amryt's commercial business comprises two orphan disease
products.
Juxtapid(R)/ Lojuxta(R) (lomitapide) is approved as an adjunct
to a low-fat diet and other lipid-lowering medicinal products for
adults with the rare cholesterol disorder, Homozygous Familial
Hypercholesterolaemia ("HoFH") in the US, Canada, Columbia,
Argentina and Japan (under the trade name, Juxtapid(R)) and in the
EU (under the trade name, Lojuxta(R)). HoFH is a rare genetic
disorder which impairs the body's ability to remove low density
lipoprotein ("LDL") cholesterol ("bad" cholesterol) from the blood,
typically leading to abnormally high blood LDL cholesterol levels
in the body from before birth - often ten times more than people
without HoFH - and subsequent aggressive and premature
cardiovascular disease.
Myalept(R) / Myalepta(R) (metreleptin) is approved in the US
(under the trade name, Myalept(R)) as an adjunct to diet as
replacement therapy to treat the complications of leptin deficiency
in patients with congenital or acquired generalized lipodystrophy
(GL) and in the EU (under the trade name, Myalepta(R)) for the
treatment of leptin deficiency in patients with congenital or
acquired GL in adults and children two years of age and above and
familial or acquired partial lipodystrophy (PL) in adults and
children 12 or over for whom standard treatments have failed to
achieve adequate metabolic control. Metreleptin is also approved
for lipodystrophy in Japan. Generalised and partial lipodystrophy
are rare disorders characterised by loss or lack of adipose tissue
resulting in the deficiency of the hormone leptin, produced by fat
cells and are associated with severe metabolic abnormalities
including severe insulin resistance, diabetes, hypertriglyceridemia
and fatty liver disease.
Amryt's lead development candidate, AP101 (Oleogel-S10), is a
potential treatment for the cutaneous manifestations of
Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin
disorder affecting young children and adults for which there is
currently no approved treatment. It is currently being studied in a
Phase 3 clinical trial and recently reported that unblinded interim
efficacy data supported continuation of the study with a modest
increase in sample size and unblinded interim safety data allowed
the inclusion of children from as young as 21 days old. AP101 has
been granted FDA Pediatric Rare Disease Designation and has also
received a Fast Track Designation from the FDA. The European and US
market opportunity for EB is estimated by the Directors to be in
excess of $1 billion.
In March 2018, Amryt in-licenced a pre-clinical gene-therapy
platform technology, AP103, which offers a potential treatment for
patients with Recessive Dystrophic Epidermolysis Bullosa, a subset
of EB, and is also potentially relevant to other genetic
disorders.
For more information on Amryt, including products, please visit
www.amrytpharma.com
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END
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