Amryt Pharma PLC Positive results for gene therapy AP103 (3034M)
07 January 2019 - 6:00PM
UK Regulatory
TIDMAMYT
RNS Number : 3034M
Amryt Pharma PLC
07 January 2019
7 January 2019
AIM: AMYT
ESM: AYP
Amryt Pharma plc
("Amryt" or the "Company")
Positive results for novel non-viral gene therapy AP103
Topical application of AP103 restored production of collagen VII
in pre-clinical models of Epidermolysis Bullosa
Amryt, a revenue generating orphan drug company focused on
acquiring, developing and commercialising products that help
improve the lives of patients where there is a high unmet medical
need, today announces positive results from two pre-clinical
studies which support the development of its novel non-viral gene
therapy, AP103, as a potentially disease-modifying therapy for
patients with Recessive Dystrophic Epidermolysis Bullosa ("RDEB"),
a subset of Epidermolysis Bullosa ("EB").
RDEB is a particularly severe form of EB and is caused by
mutations in a single gene, COL7A1, which codes for the production
of collagen VII, a structural protein vital for the elastic and
structural integrity of the skin. Restoring production of collagen
VII in skin cells could be transformative for these patients,
potentially making their skin less fragile and more resistant to
damage and blistering. As a result, the quality of life for
patients with RDEB could be dramatically improved.
Pre-clinical studies sought to investigate the potential of
AP103 as a topical gene therapy intervention to restore expression
of the COL7A1 gene.
- In vitro tests on RDEB keratinocytes, the main cell type in
the top layer of skin, showed that a single delivery of the human
collagen VII gene, by AP103, restored collagen VII production to
levels exceeding those produced by healthy human keratinocytes
- Topical application of AP103 onto a 3-D matrix of human RDEB
skin restored collagen VII along the basement membrane to levels
similar to those observed post delivery using a viral vector
- AP103 exhibited no evidence of cellular toxicity after repeated administration
AP103 is based on a new gene therapy delivery platform,
in-licensed by Amryt in March 2018, that utilises a non-viral
delivery vector, HPAE (Highly Branched Poly <BETA>-Amino
Ester), designed to deliver the correct collagen VII gene into skin
cells. It is topically applied to the skin.
Joe Wiley, CEO of Amryt Pharma, commented: "These results
further support the potential of our novel gene therapy platform as
a potentially transformative treatment for patients with RDEB,
which is a particularly severe form of EB. If successful, this
platform has potential in other genetic skin conditions and beyond.
Unlike other gene therapies that rely on viral vectors, AP103 is a
novel synthetic delivery technology, which provides a potential
competitive advantage in the gene therapy field. Following the
recently announced grant funding from the Irish Government, we look
forward to progressing this exciting program in 2019. Today's news
also follows our recent encouraging results from the EASE Global
Phase III study in EB. Both developments are significant milestones
for Amryt and our shareholders as we build a global EB franchise
and become a leader in rare and orphan diseases."
- Ends -
Enquiries:
Amryt Pharma plc +353 (1) 518 0200
Joe Wiley, CEO
Rory Nealon, CFO/COO
Shore Capital +44 (0) 20 7408 4090
NOMAD and Joint Broker
Edward Mansfield, Mark Percy, Daniel
Bush
Stifel +44 (0) 20 7710 7600
Joint Broker
Jonathan Senior, Ben Maddison
Davy +353 (1) 679 6363
ESM Adviser and Joint Broker
John Frain, Anthony Farrell
Consilium Strategic Communications +44 (0) 20 3709 5700
Matthew Neal, David Daley, Nicholas
Brown
About Amryt
Amryt is a biopharmaceutical company focused on developing and
delivering innovative new treatments to help improve the lives of
patients with rare or orphan diseases.
Lojuxta is an approved treatment for adult patients with the
rare cholesterol disorder - Homozygous Familial
Hypercholesterolaemia ("HoFH"). This disorder 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 narrowing and blocking of blood vessels,
heart attacks and strokes, even at a very young age if not properly
diagnosed or receiving adequate treatment. Lojuxta is indicated as
an adjunct to a low-fat diet and other lipid-lowering medicinal
products with or without LDL apheresis in adult patients with
HoFH.
Amryt is the marketing authorisation holder and has an exclusive
licence to sell Lojuxta (lomitapide) across the European Economic
Area, Middle East and North Africa, Switzerland, Turkey, Israel,
Russia, the Commonwealth of Independent States and the non-EU
Balkan states.
Amryt's lead drug candidate, AP101, is a potential treatment for
Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin
disorder affecting young children and adults for which there is
currently no treatment. It is currently in Phase III clinical
trials. The European and US market opportunity for EB is estimated
to be in excess of EUR1 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, please visit amrytpharma.com.
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END
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