Amryt Pharma plc Amryt Announces Positive Results From Phase 3 Trial Of Filsuvez(R) In Epidermolysis Bullosa
29 Outubro 2020 - 4:01AM
UK Regulatory
TIDMAMYT
AMRYT ANNOUNCES POSITIVE RESULTS FROM PHASE 3 TRIAL OF FILSUVEZ(R) IN
EPIDERMOLYSIS BULLOSA
LATE-BREAKING ORAL PRESENTATION AT THE 29(TH) EADV (VIRTUAL) CONGRESS ON
OCTOBER 31
Virtual Analyst & Investor Event | Tuesday November 3 at 0830 EST / 1330
GMT
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DUBLIN, Ireland, and Boston MA, 29 October 2020, Amryt (Nasdaq: AMYT,
AIM: AMYT), a global, commercial-stage biopharmaceutical company
dedicated to developing and commercializing novel therapeutics to treat
patients suffering from serious and life-threatening rare diseases, is
pleased to announce positive results from its pivotal Phase 3 EASE trial
of FILSUVEZ(R) (Oleogel-S10/previously AP101). Further to Amryt's
announcement of September 9, 2020, the Company today releases additional
data which will be presented as a late-breaking oral presentation on
behalf of the trial investigators by Professor Dedee Murrell (Chair,
Department of Dermatology, St George Hospital, UNSW, Sydney, Australia)
at the 29(th) EADV (European Association of Dermatology and Venereology)
Virtual Congress 2020 on October 31, 2020. The data will also be
presented by Amryt at its forthcoming Virtual Analyst and Investor event
on November 3, 2020.
Highlights
-- The primary endpoint of the trial was met with statistical significance
-- The proportion of patients with first complete closure of EB target wound
within 45 days was 41.3% in the Oleogel-S10 group and 28.9% in the
control group (p value=0.013)
-- This translates to a 44% increase in the probability of target wound
closure with Oleogel-S10 compared to the control gel
-- The proportion of Recessive Dystrophic EB ("RDEB") patients with first
complete closure of EB target wound within 45 days was 44.0% in the
Oleogel-S10 group and 26.2% in the control group (nominal p value=0.008)
-- This translates to a 72% increase in the probability of target wound
closure with Oleogel-S10 compared to the control gel in RDEB patients
-- A greater reduction in pain associated with dressing changes was observed
in the Oleogel-S10 treatment group at each time point compared with the
control group. At Day 14, this difference was nominally significant (p
value=0.02)
-- There was a greater reduction in total body wound burden as measured by
EB Disease Activity and Scarring Index ("EBDASI") and total body surface
area of EB partial thickness wounds with Oleogel-S10 although the
differences were not statistically significant
-- Oleogel-S10 had an acceptable safety profile and was well tolerated when
compared with the control gel
Dr Joe Wiley, CEO of Amryt Pharma, commented: "Today's announcement of
the positive data from EASE marks another significant milestone for
Amryt as we seek approval for FILSUVEZ(R). These results also represent
a potentially important advancement for patients and families living
with this rare and distressing disorder. Our existing commercial
business is performing and growing and if FILSUVEZ(R) is approved, we
already have the capacity, infrastructure and resources in place to
commercialize FILSUVEZ(R) and our plans for full launch are well
advanced.
We would like to extend our gratitude to all of the patients, their
families, carers and physicians for their participation in the EASE
trial and we look forward to working with regulatory authorities to
potentially make FILSUVEZ(R) available as the first approved treatment
for EB patients. The entire team at Amryt is very excited by today's
results and the potential to help patients with this very distressing
condition."
Dr Mark Sumeray, Chief Medical Officer of Amryt commented: "It is very
gratifying to see the results from the EASE trial regarding the effect
of FILSUVEZ(R) on the speed of wound healing in such a complex clinical
situation. EASE is the fourth Phase 3 trial to demonstrate a
statistically significant acceleration in healing of partial thickness
wounds and the first in EB. We look forward to progressing our
discussions with the respective regulatory authorities as we work to
bring FILSUVEZ(R) to patients."
Primary Efficacy Endpoint
The proportion of patients with first complete closure of EB target
wound within 45 days was 41.3% in the Oleogel-S10 group and 28.9% in the
control group. This difference reached statistical significance (p
value=0.013). This translates to a 44% increase in the probability of
target wound closure with Oleogel-S10 compared to the control gel.
Proportion of patients with first complete closure of EB target wound
within 45 days
Oleogel-S10 Group Control Group
N=104 N=114
----------------- ----------------- -------------
Primary Endpoint 41.3% 28.9%
----------------- ----------------- -------------
Relative Risk 1.44
(95% CI) (1.01,2.05)
----------------- --------------------------------
p value* 0.013
----------------- --------------------------------
*pre-specified adjustment to account for IDMC (Independent Data
Monitoring Committee) interim sample size re-estimation
The difference observed in the proportion of target wounds healed within
45 days between treatment groups for the primary endpoint was driven by
the RDEB subgroup. The proportion of RDEB patients with first complete
closure of EB target wound within 45 days was 44.0% in the Oleogel-S10
group and 26.2% in the control group (nominal p value=0.008). This
translates to a 72% increase in the probability of target wound closure
with Oleogel-S10 compared to the control gel in RDEB patients.
Proportion of patients with first complete closure of target wound
within 45 days by EB subtype
Relative
Subtype Oleogel-S10 Group Control Group Risk p value*
-------- ------------------- ---------------- -------- --------
n Closure Rate n Closure Rate
-------- --- -------------- ------------ -------- --------
RDEB** 91 44.0% 84 26.2% 1.72 0.008
-------- --- -------------- ------------ -------- --------
DDEB** 6 50.0% 14 50.0% 1.10 0.844
-------- --- -------------- ------------ -------- --------
JEB** 11 18.2% 15 26.7% 0.61 0.522
-------- --- -------------- ------------ -------- --------
*Nominal p value
**Recessive Dystrophic EB (RDEB) / Dominant Dystrophic EB (DDEB) /
Junctional EB (JEB)
Secondary Efficacy Endpoints
In wounds that achieved complete closure over the 90 day treatment
period, the mean time to first closure in the Oleogel-S10 group was 37.7
days compared to 44.5 days in the control group. During the entire 90
day double-blind treatment period separation in target wound closure
occurred around Day 30 with the difference narrowing around Day 90. The
proportion of completely closed target wounds within the 90 day
treatment period was 50.5% in the Oleogel-S10 group compared with 43.9%
in the control group (p value=0.296). The difference in the time to
wound healing between the 2 treatment groups over the 90 days was not
statistically significantly different (p value=0.302).
A greater reduction in pain associated with dressing changes was
observed in the Oleogel-S10 treatment group at each time point compared
with the control group. At Day 14, this difference was nominally
significant (p value=0.022). At Day 90, the difference just missed
nominal significance (p value=0.051).
Procedural pain mean change from baseline (Wong-Baker FACES pain rating
scale)
Patients >=4 years of age
Day 14 Day 30 Day 45 Day 60 Day 90
------------------ --------- --------- --------- --------- ---------
n n n n n
------------------ ----- ----- ----- ----- -----
Oleogel-S10 Group 90 -1.44 90 -1.04 84 -0.93 84 -1.29 76 -1.32
------------------ ----- ----- ----- ----- -----
Control Group 95 -0.78 90 -0.27 85 -0.78 86 -0.56 78 -0.18
------------------ ----- ----- ----- ----- -----
Nominal p value 0.022 0.152 0.805 0.095 0.051
------------------ ----- ----- ----- ----- -----
There was a greater reduction in total body wound burden as measured by
EBDASI and total body surface area of EB partial thickness wounds with
Oleogel-S10 although the differences were not statistically significant.
Reduction in total body wound burden (EBDASI)
Day 30 Day 60 Day 90
------------------ ------------------ ------------------ ------------------
Mean change Mean change Mean change
n from baseline n from baseline n from baseline
------------------ -------------- -------------- --------------
Oleogel-S10 Group 99 -2.3 91 -3.1 84 -3.4
------------------ -------------- -------------- --------------
Control Group 99 -2.2 96 -2.0 85 -2.8
------------------ -------------- -------------- --------------
Reduction in total body surface area of EB Partial Thickness Wounds
(Body Surface Area Percentage)
Day 30 Day 60 Day 90
------------------ ------------------ ------------------ ------------------
Mean change Mean change Mean change
n from baseline n from baseline n from baseline
------------------ -------------- -------------- --------------
Oleogel-S10 Group 98 -2.56 92 -2.92 86 -4.32
------------------ -------------- -------------- --------------
Control Group 98 -2.64 96 -1.69 85 -2.53
------------------ -------------- -------------- --------------
Target wound infections occurred infrequently (8 patients in total, with
3 patients in the Oleogel-S10 group and 5 in the control group). Five
patients had a target wound infection reported as an Adverse Event
("AE"). Four of these occurred in the control group of which 3 were
classified as 'moderate' and 1 'severe'. The single target wound
infection reported in the Oleogel-S10 group was classified as 'mild'.
In patients >=14 years of age, both treatment groups experienced an
improvement in itch, however the pattern across the six assessment
domains did not suggest a consistent effect in favour of either
treatment group. In patients between 4 and 13 years of age, there was a
modest improvement in itch in both treatment groups with a small
difference favouring the control group (not statistically significant).
Safety Profile
The incidence of patients with AEs was similar in the treatment groups.
As expected in this patient population, approximately 80% of patients
experienced at least one AE. The majority of these AEs were classed as
mild or moderate in severity. There were 13 patients with severe (grade
3/4) AEs in the Oleogel-S10 group compared to 6 in the control group.
However, this imbalance was mainly due to reports of 'anaemia', a very
common baseline finding in this population, and one that frequently
requires treatment. The incidence of patients with related AEs and
patients with AEs leading to study withdrawal was similar in the 2
treatment groups.
Summary of Adverse Events during 90 day treatment period
Oleogel-S10 Control All Patients
(N=109) (N=114) (N=223)
Adverse Event Category n (%) n (%) n (%)
----------------------------- ----------- --------- ------------
Patients with any adverse
event 89 (81.7) 92 (80.7) 181 (81.2)
----------------------------- ----------- --------- ------------
Mild AEs (grade 1) 46 (42.2) 41 (36.0) 87 (39.0)
----------------------------- ----------- --------- ------------
Moderate AEs (grade 2) 30 (27.5) 45 (39.5) 75 (33.6)
----------------------------- ----------- --------- ------------
Severe AEs (grade 3/4) 13 (11.9) 6 (5.3) 19 (8.5)
----------------------------- ----------- --------- ------------
Any Related AEs 27 (24.8) 26 (22.8) 53 (23.8)
----------------------------- ----------- --------- ------------
Any AE leading to study
withdrawal 3 (2.8) 2 (1.8) 5 (2.2)
----------------------------- ----------- --------- ------------
The most frequently reported AEs (Oleogel-S10 vs Control) were wound
complication (61.5% vs 53.5%), pyrexia (8.3% vs 13.2%), wound infection
(7.3% vs 8.8%), pruritus (7.3% vs 5.3%) and anemia (7.3% vs 3.5%).
Next Steps
Amryt intends to complete the submission of its rolling New Drug
Application ("NDA") to the US Food and Drug Administration ("FDA") and
request priority review for FILSUVEZ(R). FILSUVEZ(R) previously received
Fast Track Designation and Rare Paediatric Disease Designation from the
FDA. This means that if an NDA for FILSUVEZ(R) is approved, the Company
expects to be eligible to apply for a Rare Pediatric Disease Priority
Review Voucher that can be used, sold or transferred. Amryt also
intends to pursue an accelerated assessment in the EU. Regulatory
submissions in the US and the EU are expected to be filed by late Q1
2021.
FILSUVEZ(R) has been granted Orphan Drug status for the treatment of EB
in the EU and the US. Should FILSUVEZ(R) be granted approval, it should
be entitled to Orphan Drug exclusivity for the treatment of EB,
extending seven years in the US and ten years in the EU from the date of
approval in the respective jurisdictions.
About EB
EB is a rare, chronic and distressing genetic skin disorder that causes
the skin layers and internal body linings to separate and affects
infants, children and adults. The global incidence of all EB subtypes is
estimated to be approximately 1 in 20,000, which implies that there are
as many as 30,000 affected individuals in the US and over 500,000
worldwide. There are currently no approved treatments.
About EASE
The EASE trial (
https://www.globenewswire.com/Tracker?data=5A4xGPffoBljedwGsg5PFQSabfUlEWKDUoPicBJ8dUHDqlb5nFqssBiC3o3d8WToHlpHi9gIhOBFxL-GwxuK2SmaKhU-O9Ki00WuAI5RqQGrypepMABcXL2X-XsXLXX1qWE-5hG5cJDkgEX_Qfp_-w==
NCT03068780) is the largest ever global Phase 3 trial conducted in
patients with EB, performed across 58 sites in 28 countries. It
comprises a 3 month double-blind randomised controlled phase followed by
a 24 month open-label, single-arm phase. Patients with dystrophic and
junctional EB target wounds of between 10 and 50cm(2) in size that were
present for > 21 days and < 9 months were randomized in the double-blind
phase to study treatment in a 1:1 ratio and wound dressings applied
according to standard of care. 223 patients were enrolled into the
trial including 156 pediatric patients. Of those that completed the
double-blind phase, 100% entered the open label safety follow up phase.
Virtual Analyst & Investor Event
Amryt will host a virtual Analyst and Investor Event on Tuesday,
November 3 2020 from 0830 EST (1330 GMT) - 1030 EST (1530 GMT) to
present data from the EASE trial. Amryt will address the following
topics:
-- EASE Phase 3 trial data
-- Regulatory agency engagement and timelines
-- Commercialization and launch plans
Amryt management will be joined at the event by Professor Jemima
Mellerio (Consultant Dermatologist & Honorary Professor of Paediatric
Dermatology, Chief St John's Institute of Dermatology, Guy's and St
Thomas' Hospital, London) who will discuss the EASE data. The group
will also be joined by Brett Kopelan (Executive Director, debra of
America and President, DEBRA International) and Jimmy Fearon (CEO, DEBRA
Ireland and VP, DEBRA International) who will be available to answer
questions from participants.
You may
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Enquiries
Amryt Pharma plc +353 (1) 518 0200
Joe Wiley, CEO
Rory Nealon, CFO/COO
LifeSci Advisors, LLC +1 (212) 915 2564
Tim McCarthy
Consilium Strategic Communications +44 (0) 20 3709 5700
Amber Fennell, Matthew Neal, Ashley Tapp
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 lead development candidate, FILSUVEZ(R) is a potential treatment
for the cutaneous manifestations of EB, a rare and distressing genetic
skin disorder affecting young children and adults for which there is
currently no approved treatment. In September 2020, Amryt reported
positive top line results from its pivotal global phase 3 trial of
FILSUVEZ(R) in EB. FILSUVEZ(R) has been granted Rare Pediatric Disease
Designation and has also received a Fast Track Designation from the U.S.
Food and Drug Administration.
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 years of age and above 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.
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.
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.
This announcement contains inside information for the purposes of
article 7 of the Market Abuse Regulation (EU) 596/2014.
The person making this notification on behalf of Amryt is Rory Nealon,
CFO/COO and Company Secretary.
Financial Advisors
Shore Capital (Edward Mansfield, Daniel Bush, John More) are NOMAD and
Joint Broker to Amryt in the UK. Stifel (Ben Maddison) are Joint Broker
to the company in the UK. Davy (John Frain, Daragh O'Reilly) act as
Joint Broker to the company.
Forward-Looking Statements
Statements in this announcement with respect to Amryt's business,
strategies, timing for completion of and announcing results from the
EASE trial, the potential impact of closing enrollment in the EASE trial,
as well as other statements that are not historical facts are
forward-looking statements involving risks and uncertainties which could
cause the actual results to differ materially from such statements.
Statements containing the words "expect", "anticipate", "intends",
"plan", "estimate", "aim", "forecast", "project" and similar expressions
(or their negative) identify certain of these forward-looking
statements. The forward-looking statements in this announcement are
based on numerous assumptions and Amryt's present and future business
strategies and the environment in which Amryt expects to operate in the
future. Forward-looking statements involve inherent known and unknown
risks, uncertainties and contingencies because they relate to events and
depend on circumstances that may or may not occur in the future and may
cause the actual results, performance or achievements to be materially
different from those expressed or implied by such forward-looking
statements. These statements are not guarantees of future performance or
the ability to identify and consummate investments. Many of these risks
and uncertainties relate to factors that are beyond each of Amryt's
ability to control or estimate precisely, such as future market
conditions, the course of the COVID-19 pandemic, currency fluctuations,
the behaviour of other market participants, the outcome of clinical
trials, the actions of regulators and other factors such as Amryt's
ability to obtain financing, changes in the political, social and
regulatory framework in which Amryt operates or in economic,
technological or consumer trends or conditions. Past performance should
not be taken as an indication or guarantee of future results, and no
representation or warranty, express or implied, is made regarding future
performance. No person is under any obligation to update or keep current
the information contained in this announcement or to provide the
recipient of it with access to any additional relevant information that
may arise in connection with it. Such forward-looking statements reflect
the Company's current beliefs and assumptions and are based on
information currently available to management.
(END) Dow Jones Newswires
October 29, 2020 03:01 ET (07:01 GMT)
Copyright (c) 2020 Dow Jones & Company, Inc.
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