TIDMAMYT
Amryt Pharma PLC
27 April 2017
27 April 2017
AIM: AMYT
ESM: AYP
Amryt Pharma plc
("Amryt" or the "Company")
Study Results of 'Real-World' Experience of Lojuxta
(lomitapide)
Shows Improved Efficacy
Over Clinical Trials of the Drug
Amryt, the pharmaceutical company focused on best-in-class
treatments for rare and orphan diseases, is pleased to announce the
publication of study results which evaluated the benefits of
Lojuxta (lomitapide) in the treatment of Homozygous Familial
Hypercholesterolaemia ("HoFH"), a rare, life-threatening disease,
which impairs the body's ability to remove LDL cholesterol ("bad"
cholesterol) from the blood. The study results confirmed that
'real-world' experience of a cohort of Italian patients show that
Lojuxta is a very powerful cholesterol-lowering agent and, in
particular, achieved greater efficacy than clinical trials of the
drug. Most notably, the study showed that Lojuxta delivered higher
reductions in LDL cholesterol ("LDL-C") than can currently be
achieved with any other therapeutic options. Lojuxta is an MTP
inhibitor approved for adjunctive treatment in adult patients with
HoFH and was in-licenced by Amryt in early December 2016. Amryt's
exclusive licence covers the European Economic Area ("EEA"), Middle
East and North Africa ("MENA"), Turkey and Israel.
The study results have been presented in a paper entitled,
"Efficacy of Lomitapide in the Treatment of Familial Homozygous
Hypercholesterolemia: Results of a Real-World Clinical Experience
in Italy", and published by Advances in Therapy, an international,
peer-reviewed journal.
The study, by Laura D'Erasmo et al, collected and analysed
clinical and biochemical data in 15 HoFH patients who had been
treated for at least six months with Lojuxta in addition to
lipid-lowering therapies in different Lipid Clinics across Italy.
At a mean dose 19 mg/day, (which was substantially lower than that
used in the Phase 3 clinical trials), Lojuxta achieved a maximal
mean reduction of LDL-C of 76.5%, with 30% of the patients
achieving a greater than 90% reduction. Ten of the 15 patients were
receiving apheresis at baseline. Apheresis is an invasive procedure
to remove LDL-C from the blood stream. Treatment with Lojuxta
enabled 80% of the patients to stop apheresis and reduced the
frequency by 40% in the remaining 2 patients. The mean LDL-C prior
to stopping apheresis was just 38.5mg/dL reduced from a baseline
mean of 426 mg/dL. Despite stopping apheresis, the patients
achieved the recommended but stringent LDL-C targets levels, with
60% of patients achieving an LDL-C<100 mg/dL and 46.6% <70
mg/dL. The treatment with lomitapide was associated with some side
effects (namely diarrhoea, nausea and increase in liver
transaminase) but these were moderate in severity and easily
manageable by treating physicians. Notably, no patients had to stop
the treatment due to these side effects.
Prof. Marcello Arca, Sapienza' University of Rome,
commented:
"This real-world experience, has shown Lojuxta to be a very
powerful and well tolerated cholesterol-lowering agent in patients
with HoFH. The reduction in LDL-C that we observed is far higher
than seen in clinical trials and higher than we can achieve with
any other therapeutic option we have for patients with this
difficult to treat and devastating disease. The ability to stop
apheresis and still achieve LDL-C target levels in patients who had
LDL-C levels up to 8 times the recommended level is
remarkable".
Dr Helen Phillips, Head of Medical Affairs at Amryt,
commented:
"We are delighted that these data have been published.
Physicians used to believe that the LDL-C targets recommended by
the European Atherosclerosis Society were impossible to achieve in
HoFH but this is no-longer the case.
"These data are not unique to Italy - we have seen similar
results in patients treated in Spain, Sweden and Netherlands.
Unlike statins, ezetimibe and PCSK-9 inhibitors, Lojuxta works
independently of LDL receptors which have little to no
functionality in HoFH. Through this different mechanism of action,
Lojuxta offers patients with HoFH an ability to reach the LDL-C
levels of normal individuals and stop the incredibly burdensome and
invasive procedure of apheresis. It offers the potential to
transform the lives of patients with HoFH, with the addition of a
capsule a day".
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 the Real-World Clinical Study
The study, "Efficacy of Lomitapide in the Treatment of Familial
Homozygous Hypercholesterolemia: Results of a Real-World Clinical
Experience in Italy" by Laura D'Erasmo et al was published in
Advances in Therapy, DOI: 10.1007/s12325-017-0531-x.
A retrospective analysis was performed of 15 adult patients with
a genetically confirmed diagnosis of HoFH (10 with mutations in
LDLR and 5 in LDLRAP1 gene) who received treatment with lomitapide
post reimbursement in Italy. The analysis included data from up to
3 years of treatment with lomitapide. Prior to receiving
lomitapide, their median LDL-C level was 426.0 +/- 204.0 mg/dL,
despite receiving treatment with statins, ezetimibe and apheresis
(in 10 of the 15 patients). The addition of lomitapide (average
daily dosage of 19 +/- 13.3 mg/day) lowered LDL-C levels a mean
68.2 +/- 24.8% versus baseline and by 76.5 +/- 16.7% at nadir prior
to changes in apheresis schedules. At their last visit, 60% of
patients showed LDL-C<100 mg/dL and 46.6% <70 mg/dL. During
treatment with lomitapide, 8 over 10 patients receiving LA (80%)
stopped this treatment due to the pronounced cholesterol reduction.
Prior stopping apheresis their LDL-C was a mean nadir 38.5 mg/dL
(26.8-151 mg/dL)]. In addition, in the in the 2 remaining patients
on apheresis, they reduced their apheresis requirements by 40%. A
wide range (43-97%) of individual LDL-C reduction was observed, but
this was not related to underlying mutations causing the HoFH.
During follow-up, 53.3% of patients reported at least one
episode of diarrhoea, but none was severe; none had persistent
liver transaminase >5 X ULN or had to stop treatment due to side
effects. During treatment, 6 HoFH patients were also investigated
by liver imaging techniques according to the European label showing
results well within the normal range. At baseline, only 1 patient
out of 5 was referred to have liver steatosis, while at follow-up
there were 2 patients with fatty liver.
In this real word experience, lomitapide was confirmed to be an
effective cholesterol-lowering agent in HoFH with a good safety
profile.
In the Phase 3 clinical trials with lomitapide (published in the
lancet by Cuchel et al) which employed a forced titration schedule,
the mean LDL-C reduction was 50% at a mean dose of 40mg/day.
Ninety-three percent of patients experienced GI adverse effects,
reducing to 74% and 21% patients experienced LFTs > 3 x ULN in
this 78-week study. The real-world evidence therefore supports a
higher level of efficacy at a mean lower dose demonstrating an
improved benefit : risk profile than that observed in the clinical
trials.
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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