TIDMAMYT
RNS Number : 3998Y
Amryt Pharma PLC
09 September 2020
AMRYT PHARMA PLC
("Amryt" or the "Company")
AMRYT ANNOUNCES POSITIVE TOP LINE RESULTS FROM PIVOTAL PHASE 3
"EASE" TRIAL OF FILSUVEZ(R) IN EPIDERMOLYSIS BULLOSA
- Primary endpoint met with statistical significance (p-value =
0.013)
- FILSUVEZ(R) demonstrated a good safety profile
- EASE is the first Phase 3 trial to demonstrate a statistically
significant increase in speed of wound healing in Epidermolysis
Bullosa
- Rolling US regulatory submissions already underway with FDA
with priority review request planned - EU regulatory submission
planned with request for accelerated assessment
- FILSUVEZ(R), a topical therapeutic gel, has the potential to
be the first treatment approved for Epidermolysis Bullosa
Analyst and investor call today at 08.30 ET/13.30 BST
DUBLIN, Ireland, and Boston MA, 9 September 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 very pleased to announce
positive top line results from its pivotal Phase 3 EASE trial of
FILSUVEZ(R) (previously AP101 /Oleogel-S10) for the treatment of
dystrophic and junctional Epidermolysis Bullosa ("EB"). The primary
endpoint of the trial was met (p-value = 0.013). EASE is the
largest Phase 3 trial ever conducted in EB.
Dr Joe Wiley, CEO of Amryt Pharma, commented: "This positive
outcome of the Phase 3 EASE trial marks another significant
milestone for Amryt as we seek approval for FILSUVEZ (R) and
represents a potentially important advancement for patients and
families living with this rare and distressing disorder. If
approved, we intend to leverage our existing global infrastructure
to commercialize FILSUVEZ (R) .
We are proud to present these positive and encouraging results,
demonstrating that FILSUVEZ(R) could make an important difference
to the lives of patients. 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 make FILSUVEZ(R) available
as the first approved therapeutic treatment for EB patients. All of
the team at Amryt are very excited by today's news and the impact
this may have in our efforts to help patients with this very
distressing condition."
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 EB 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. The
global market opportunity for EB is estimated by the Company to be
in excess of $1.0 billion.
EASE Results
The EASE trial ( 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 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.
The primary endpoint of the trial was to compare the efficacy of
FILSUVEZ(R) versus control gel according to the proportion of
patients with complete closure of the target wound within 45 days
of treatment. The primary endpoint w as achieved with statistical
significance (p-value = 0.013). This represents the first ever
successful Phase 3 top line readout in EB. It is also the fourth
time FILSUVEZ (R) has demonstrated accelerated wound healing in a
Phase 3 trial .
While the key secondary endpoints did not achieve statistical
significance, a number of favourable differences were observed. In
addition, substantial further secondary endpoint data is expected
and will be analysed over the coming weeks.
Next Steps
The Company will now evaluate and analyse the full data set from
the trial and will present results at an upcoming scientific
symposium.
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.
Dr Mark Sumeray, Chief Medical Officer of Amryt commented: "The
conduct of a global clinical trial involving 58 centres in 28
countries in such a rare serious and predominantly pediatric
disease has been a challenging but enormously important
undertaking. It is very gratifying to see the first results from
the trial provide evidence of the effect of FILSUVEZ(R) on the
speed of wound healing in such a complex clinical situation. We
look forward to further data over the next few weeks."
Brett Kopelan, Executive Director of debra of America and
President of Debra International, commented: "The very positive
results of the largest international clinical trial conducted in EB
is an incredibly exciting development and a very important
milestone for the global EB community. I want to thank the entire
team at Amryt for the commitment and fortitude it took to complete
this trial. The proven ability to address the hallmark
manifestation of this devastating disease, chronic wounds that
don't heal, in such an efficacious manner and as a therapy applied
as part of the standard of care will undoubtedly lead to a
meaningful quality of life improvement for patients living with the
"worst disease you've never heard of". As the executive director of
debra of America and as the President of Debra International, but
more importantly as the father of a 13 year-old daughter with
recessive dystrophic EB, I couldn't be more enthused."
Professor Johannes Kern, of Royal Melbourne Hospital and
Principal Investigator of the trial commented: "I have had the
privilege of working with a number of patients and their families
affected by EB which is an incredibly distressing condition for all
of those involved. I am very excited by the possibility that a
treatment such as FILSUVEZ(R) could potentially accelerate wound
healing and alleviate the symptoms for those affected."
The Amryt Management team will host an analyst and investor call
today at 08.30 ET / 13.30 BST to discuss the news and answer
questions.
Dial-in details for the call:
Standard International +44 (0) 203 009
Number 5709
United States (Local) +1 646 787 1226
-------------------
+44 (0) 844 493
United Kingdom (Local) 6766
-------------------
Ireland (Local) + 353 (1) 506 0626
-------------------
Confirmation Code 8769437
-------------------
A playback facility will be available from September 9, 2020 at
1330 ET / 1830 BST - September 16, 2020 at 1330 ET / 1830 BST .
Access details as follows: Confirmation Code: 8769437 | US: + 1 917
677 7532 | UK/International: +44 (0) 3333 00 9785 | Ireland : +353
(1) 553 8777.
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 commercial business comprises two orphan disease
products.
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.
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.
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. 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. The global market opportunity for EB is estimated
by the Company to be in excess of $1.0 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, 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.
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