TIDMAMYT
Amryt Pharma PLC
25 July 2017
25 July 2017
AIM: AMYT
ESM: AYP
RNS REACH
Amryt Pharma plc
("Amryt" or the "Company")
Newly published long-term study shows highly positive
results
for Lojuxta, the Company's in-licenced drug used in the
treatment of HoFH
74% of study patients achieved target levels of LDL
cholesterol
Amryt Pharma, a biopharmaceutical company focused on innovative
therapies that transform the lives of patients with rare and orphan
diseases, is pleased to announce the publication of a long-term
extension study evaluating the benefits of Lojuxta (lomitapide) in
the treatment of Homozygous Familial Hypercholesterolaemia
("HoFH"), a rare, genetic, life-threatening disease, which impairs
the body's ability to remove LDL cholesterol ("LDL-C" or "bad"
cholesterol) from the blood. Lojuxta is an approved treatment for
adult patients with HoFH, which was in-licenced by Amryt in
December 2016. The study, which followed patients for up to 5.7
years results, showed Lojuxta as being highly effective in lowering
LDL-C levels, with acceptable tolerability and no new safety
signals. Notably, the majority of patients achieved the recommended
LDL-C target level for all adult patients with HoFH.
The study results have been published recently by Circulation,
an international, peer-reviewed journal in a paper entitled,
"Long-Term Efficacy and Safety of the Microsomal Triglyceride
Transfer Protein Inhibitor Lomitapide in Patients With Homozygous
Familial Hypercholesterolemia".
The study was published by Dirk Blom et al and analysed data in
19 HoFH patients who had previously completed the pivotal phase 3
study (Cuchel et al) in lomitapide. The primary end-point of the
study was LDL-C lowering versus the start of the study when
receiving standard therapy, assessed at Week 126 (2.4 years) with
safety being assessed to the study end at a maximum of 5.7 years.
The median treatment duration with lomitapide was 5.1 years (range,
2.1 - 5.7 years). Of the 19 patients, 14 or 74% were able to
achieve the recommended LDL-C target levels on at least one
occasion of less than 100mg/dL and 11 or 58% of patients achieved
the more stringent target of less than 70 mg/dL, recommended if
they have cardiovascular disease.
The efficacy was maintained with a mean reduction in LDL-C of
45.5% versus baseline and no new safety signals were identified in
the 5.7 years of treatment. The most common adverse events reported
were gastrointestinal but the incidence of treatment related
adverse events was lower in the extension study than those observed
in the pivotal phase 3 trial (42.1% versus 84.2%).
Dr Dirk Blom, Department of Lipidology, University of Cape Town,
South Africa, commented:
"This long-term study confirms the efficacy of lomitapide seen
in the pivotal phase 3 trial. Adding lomitapide to other
lipid-lowering therapies is highly effective in sustainably
reducing LDL-C levels with acceptable tolerability and no new
safety signals. A substantial proportion of patients was able to
achieve their recommended LDL-C target levels, something that
before the advent of lomitapide, we thought would be impossible to
achieve in this difficult to treat condition. Some patients were
also able to discontinue or reduce the frequency of lipid apheresis
- a procedure similar to renal dialysis that removes LDL-C directly
from the blood."
Dr Helen Phillips, Head of Medical Affairs at Amryt,
commented:
"These study results are important and we are delighted that
they show such positive results in patients with HoFH, a rare and
potentially life-threatening disease.
"With all new treatments, especially for those for life-long
treatment, is critically important to understand the long-term
safety profile of a drug. The data from this long term study, which
followed patients over a median of 5.1 years, is reassuring since
no new safety signals were seen.
"Patients with HoFH have an extremely high risk of
cardiovascular events and we are pleased that treatment with
Lojuxta, given alongside other lipid-lowering therapies, is so
effective in reducing "bad" cholesterol - LDL-C".
A link to the study publication is here:
http://circ.ahajournals.org/content/136/3/332
Enquiries:
Amryt Pharma plc C/o KTZ Communications
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
(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.
The Company holds an exclusive licence to sell Lojuxta
(lomitapide) for adults, 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, 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 (Episalvan), is a potential
treatment for Epidermolysis Bullosa ("EB"), a rare and distressing
genetic skin disorder affecting young children for which there is
currently no 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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