TIDMAMYT
RNS Number : 7774H
Amryt Pharma PLC
15 March 2018
15 March 2018
AIM: AMYT
ESM: AYP
AMRYT PHARMA PLC
("Amryt" or the "Company")
Trading Update
&
In-licence Agreement for Novel Gene Therapy Treatment
Amryt, the biopharmaceutical company focused on rare and orphan
diseases, is pleased to provide the following trading update, and
announces that it has concluded terms for an exclusive in-licence
agreement for a novel gene therapy platform, which offers a
potentially exciting treatment for patients with Epidermolysis
bullosa ("EB").
Key Points
-- A very strong year and encouraging start to 2018
-- Revenues for the year ended 31 December 2017 increased to
EUR12.8m (2016: EUR1.48m)
-- Cash balance (unaudited) at 31 December 2017 was EUR20.5
million with EUR10 million undrawn from the EIB facility
-- Major in-licence agreement signed on 14 March 2018 with
University College Dublin for exciting non-viral gene therapy
platform technology, which offers a potential treatment
for patients with EB
- Preliminary data suggests that the treatment could
be potentially disease-modifying for patients with
Recessive Dystrophic Epidermolysis Bullosa ("RDEB"),
a subset of EB
- Amryt intends to conduct various pre-clinical studies
in the coming months and will report initial results
in early Q4 2018. These initial studies will be funded
from Amryt's existing cash resources
-- Revenues from Lojuxta (lomitapide), which treats a rare
life-threatening disorder that causes abnormally high levels
of "bad" cholesterol, increased to EUR11.9m
- This is a growth rate of 65% compared to the annualised
sales of Lojuxta for the period prior to its in-licensing
by Amryt, in December 2016
- Five new distribution agreements signed since November
2017 - significantly broadening potential sales
-- Lead development asset, AP101 (a potential treatment for
rare, genetic, skin condition, EB), continues to make significant
progress:
- Amicus Therapeutics granted Amryt access to the data
from its landmark Phase 3 clinical study in EB
- Insights from these data enable Amryt to refine its
ongoing global Phase III EASE study of AP101 in EB
- with the potential to increase the probability of
success for the study
-- As a result, clinical trial interim analysis is
now expected to be completed in early Q4 2018,
with read out of top-line data in Q2 2019
- Additional market opportunities for AP101 in partial
thickness wound indications are under evaluation.
-- Opportunities include AP101 as a treatment for
Toxic Epidermal Necrolysis Syndrome (TENS)/Stevens-Johnson
Syndrome (SJS), Bullous Pemphigoid, Pemphigus
Vulgaris and grade III/IV radiotherapy and chemotherapy-induced
dermatitis. The scope of the current EMA approval
may offer the opportunity to launch in some of
these indications in Europe.
-- Full year results are expected to be announced in mid-April
2018
Commenting on the Company's progress, Joe Wiley, CEO of Amryt,
said:
"2017 was a very strong year for Amryt and we are encouraged by
the start to 2018, which places us in a good position to be able to
drive further expansion through this year and beyond. We have grown
our Lojuxta business significantly since we in-licensed it in
December 2016, and our recent distribution agreements throughout
Europe and the Middle-East mean that we now are able to reach more
people living with the ultra-rare and life-threatening condition,
HoFH, than ever before. In EB, Amicus Therapeutics generously
provided us with a valuable opportunity to review the data from
their ESSENCE study in EB. Based on these data, Amryt now has the
opportunity to refine its ongoing global Phase III EASE study of
AP101 in EB, with the potential to increase the probability of
success for the study. We are truly grateful to Amicus Therapeutics
for the opportunity, which is a heartening example of collaboration
in our industry in the best interests of patients.
"We have ambitious plans for the remainder of 2018 and we look
forward to announcing a series of agreements in the months to come.
This is a pivotal year for Amryt and our focus continues to be on
ensuring that we are delivering real change for people with rare
diseases across the world. I am proud to say we are delivering on
our promise."
Commenting specifically on the new in-licence agreement, Joe
Wiley, CEO of Amryt, said:
"This is a great opportunity for Amryt to get involved in the
area of gene therapy, which is one of the most exciting and
potentially transformative areas of medicine today. Gene therapy
has come of age in the last number of years and is being applied to
multiple orphan therapeutic areas. The HPAE polymer technology
gives us a potential platform technology, with an initial topical
application in EB, that does not rely on the use of viral vectors
for the delivery of gene therapy. If successful, this platform has
the potential to be broadly applicable in other dermatological
conditions and possibly beyond. In the meantime, Amryt will
continue to seek to in-license further commercial stage assets to
continue to grow our revenues and provide cash resources that will
help support these development assets. Amryt now has in place an
exceptionally strong leadership team with the necessary commercial,
regulatory and medical infrastructure also in place in Europe. Our
strategy is to leverage this capacity to in-license more commercial
stage assets, which we are actively pursuing."
TRADING UPDATE
REVENUE AND CASH
Unaudited revenue for the 12 months to 31 December 2017 was
EUR12.8 million (2016: EUR1.48m), with sales of Lojuxta accounting
for EUR11.9 million of the total. Cash balance (unaudited) at the
end of December 2017 was EUR20.5 million with EUR10 million undrawn
from the EIB facility.
LOJUXTA (LOMITAPIDE)
Lojuxta (lomitapide) is used to treat a rare life-threatening
disease called Homozygous Familial Hypercholesterolaemia ("HoFH")
which impairs the body's ability to remove LDL cholesterol ("bad"
cholesterol) from the blood.
Sales of EUR11.9 million show a 65% growth rate compared to the
annualised sales of Lojuxta for the period prior to in-licencing by
Amryt in December 2016. This growth was underpinned by strong
demand from existing markets within Amryt's licenced territories.
In particular, the Company has experienced positive momentum in the
reimbursement position in certain countries and also an increase in
individual named patients who continue to access funding for
treatment in other countries. Patients are also typically
tolerating and responding well to treatment to enable them to stay
on treatment long term.
Future growth will continue to be driven by existing markets and
also from new territories. Since November 2017, Amryt has agreed
five new distributor relationships, which together cover seventeen
new countries. The Company is actively negotiating national
reimbursement decisions in the UK, France, Spain and Turkey which,
it is hoping will materialise during the course of 2018. If
successful, these market-access decisions will allow Amryt to
provide access for a cohort of HoFH patients in each of these
territories.
AP101
Access to detailed data from Amicus Therapeutics's ESSENCE Phase
3 study in EB
Amicus Therapeutics granted Amryt detailed access to the data
from its landmark ESSENCE trial of SD101 in EB, which read out in
September 2017. Amryt is deeply grateful for this unique
opportunity and would like to publically recognise and thank Amicus
Therapeutics. Based on insights from these data, Amryt management
is now able to refine its protocol for the Company's ongoing global
Phase III (EASE) study of AP101, with the potential to increase the
probability of success for the study.
The Company is currently in the process of amending the protocol
for the EASE study and will discuss any significant changes with
the FDA and the European Medicines Agency ("EMA"). These amendments
include a modest increase in the size of the study from 164 to 192
patients and a restriction on certain wound types, the ultimate
goal of which is to increase the chances of success of the
study.
Based on the analysis of the Amicus Therapeutics data, the
Company will maintain the current primary endpoint which is the
proportion of patients with first complete closure of the target EB
wound treated with AP101 versus placebo within 45 days of
treatment. The exclusion of EB Simplex patients for the EASE study
will help to ensure that patients with likely faster spontaneous
healing rates will not be included in the study and is expected to
increase the likelihood of demonstrating a statistically
significant treatment effect.
Conclusions
These changes will result in a slight delay of the interim
analysis which the Company expects will be complete in early Q4
2018, with read out of top-line data from our AP101 Phase III study
in Q2 2019.
The incremental cost of these changes is expected to be
approximately EUR1 million. The unblinded interim analysis will be
conducted by an independent data-safety-monitoring-board and will
result in three possible outcomes:
-- continue the study with no change to sample size, which would
reflect conditional statistical power of at least 80% or
better;
-- increase the number of patients in the study to maintain an
80% conditional statistical power;
-- or discontinue the study for futility.
The unblinded interim analysis read out potentially represents a
significant milestone for the Company.
Trial sites in the USA
In advance of interim results, Amryt Pharma anticipates filing
an IND with the FDA to allow opening of US trial sites in Q3 2018
based on preliminary data from the study and the non-clinical study
results.
The Company is also pleased to announce that non-clinical
studies, requested by the FDA as part of an IND filing to open
clinical trial sites in the USA, have recently been successfully
completed. No safety signals or concerns were noted from the
preliminary data and the Company is now hopeful that the
combination of these studies, and safety data from patients
enrolled to date in non-US EASE study sites, will enable it to
request an IND to open trial sites in the USA, which it anticipates
will be in Q3 2018.
Exciting future indications for AP101 Asset
Amryt has recently received interest from physicians to study
AP101 in various Partial Thickness Wound ("PTW") indications also
with high unmet medical need. In response to this interest, the
Company is evaluating new life cycle opportunities for AP101. AP101
was approved by the EMA in Europe in January 2016 for the treatment
of PTW in adults. (This approval followed three positive phase III
studies of 280 patients in grade II burns and split thickness skin
graft donor sites).
Dermatological conditions under consideration include:
-- Toxic Epidermal Necrolysis Syndrome (TENS)( including Stevens-Johnson
Syndrome (SJS)),
-- Bullous Pemphigoid
-- Pemphigus Vulgaris
-- Grade III/IV radiotherapy and chemotherapy induced dermatitis.
The scope of the current EMA approval for AP101 may offer the
opportunity to launch AP101 in some of these indications in Europe.
Early indications suggest that collectively these indications of
TEN/SJS, radiotherapy and chemotherapy induced dermatitis, and
bullous pemphigoid and pemphigus vulgaris may have a market
potential greater than the EB opportunity which the Company is
currently investigating in its EASE Phase III study.
Management intends to file applications for orphan designation
for some of these new potential orphan indications in the USA,
Europe and Japan, and believes that there is significant scope to
maximise the value of this existing asset through either a global
multi-orphan strategy or via the current EMA marketing approval to
secure long term growth.
Further information on the indications under consideration:
Toxic Epidermal Necrolysis Syndrome (TENS) (including
Stevens-Johnson Syndrome (SJS)) is a rare, acute, serious and
potentially fatal skin reaction in which there is sheet-like skin
and mucosal loss. Amryt has recently agreed to facilitate a
compassionate use protocol in this area, which may generate
valuable data in the coming quarters.
One of the most common effects of radiation or chemotherapy is
acute skin reaction that ranges from a mild rash to severe
ulceration. Approximately 10% of patients treated with radiation
therapy will experience severe skin reaction resulting in grade
III/IV wounds.
Other potential areas of interest for AP101 include Bullous
Pemphigoid and Pemphigus Vulgaris.
IN-LICENCED GENE THERAPY
-- License agreement completed with University College Dublin
to provide non-viral gene therapy platform technology,
which offers a potential treatment for patients with EB,
and with potential applicability across a range of genetic
diseases
-- Unique platform, which uses a polymer based delivery system
instead of a viral vector
-- Initial application of the technology as a topically-applied,
potentially disease-modifying therapy to address the underlying
cause of Recessive Dystrophic Epidermolysis-Bullosa ("RDEB")
Amryt is delighted to announce the exclusive in-licencing of a
new platform technology for gene therapy with potential
applicability across a range of genetic disorders. This technology
has been exclusively in-licenced from University College Dublin
("UCD") and involves the delivery of gene therapy using High
Branched Poly (<BETA>-Amino Ester) ("HPAE") polymer
technology. The initial focus of development efforts to date has
been in the area of EB and preliminary data suggests that the
treatment could be potentially disease-modifying for patients with
Recessive Dystrophic Epidermolysis Bullosa ("RDEB"). Pre-clinical
data in a xenograft model has shown significant levels of collagen
VII in the skin post therapy. Patients with RDEB have a defect in
their gene coding for collagen VII, consequently the replacement of
collagen VII could be transformative for these patients.
Potential competitors working in the area of gene therapy in EB
are working with viral vectors to deliver collagen VII to the cell.
The patented technology which Amryt has exclusively licenced from
UCD involves the use of a novel gene delivery mechanism using HPAE
polymer technology. If successful, this will eliminate the
requirement for viruses as delivery vectors and provides a
potential competitive advantage to Amryt. Recently Krystal Biotech
Inc, a US company completed a successful listing on NASDAQ in
September 2017, with a topical gene therapy for EB using a viral
vector.
Amryt intends to conduct various pre-clinical studies in the
coming months and will report initial results in early Q4 2018.
These initial studies will be funded from the existing cash
resources of Amryt.
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
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
WG Partners +44 (0)20 3705 9321
Nigel Barnes, Nigel Birks, Chris
Lee
KTZ Communications +44 (0) 20 3178 6378
Katie Tzouliadis, Irene Bermont-Penn,
Emma Pearson
About Amryt Pharma plc
(www.amrytpharma.com)
Amryt Pharma is a specialty biopharmaceutical 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.
The Company holds an exclusive licence to sell Lojuxta
(lomitapide) for adults, across the European Economic Area, Middle
East and North Africa, Turkey and Israel. Lojuxta is used to treat
a rare life-threatening disease called Homozygous Familial
Hypercholesterolaemia, which impairs the body's ability to remove
LDL cholesterol ("bad" cholesterol) from the blood. This typically
results in extremely high blood LDL cholesterol levels, leading to
aggressive and premature narrowing and blocking of arterial blood
vessels. If left untreated, heart attack or sudden death may occur
in childhood or early adulthood.
Amryt's lead drug candidate, AP101, is a potential treatment for
Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin
disorder for which there is currently no approved treatment. It is
currently in Phase 3 clinical trials. 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.
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
END
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