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Amryt Pharma to Host Key Opinion Leader Call on Epidermolysis Bullosa Disease Landscape

DUBLIN, Ireland, and Boston MA, July 29 2020, Amryt (Nasdaq: AMYT, AIM: AMYT, Euronext Growth: AYP), a global, commercial-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, today announces it will host a Key Opinion Leader (KOL) call on Epidermolysis Bullosa (EB) disease on Monday, August 3, 2020 at 1000 ET (1500 BST).


The call will feature a presentation by KOLs Harper Price, MD (Phoenix Children's Hospital) and Brett Kopelan (debra of America) who will discuss the current disease landscape and unmet medical need in treating patients with EB. Dr. Price and Mr. Kopelan will be available for questions at the conclusion of the event.


To register for the call, please click here.


Harper Price, MD completed her dermatology training at Penn State Milton S. Hershey Medical Center in Hershey, Pennsylvania, and her pediatric dermatology fellowship at New York University Medical Center, New York. She has served at Phoenix Children’s Hospital (PCH) since 2009 where she provides the latest medications and technology available for the specialized treatment of infants, children, adolescents and young adults affected by large nevi.  Shortly after joining PCH, she became the program director of their pediatric dermatology fellowship. She now serves as Associate Chief of the Division of Dermatology. While a student, Dr. Price worked closely with doctors at NYU to review all the major nevus registries in existence at the time and undertook to combine all the data into a single collection. Nevus Outreach invited Dr. Price to attend and assist at the 2010 conference. Based on the strength of her work, she was invited to speak at the 2011 International Expert Meeting for Large Congenital Melanocytic Nevi and Neurocutaneous Melanocytosis in Tübingen, Germany, where she was recruited to serve as Assistant Director of a team of world experts to oversee creation of an all-new world-wide nevus patient data repository under the direction of Dr. Veronica Kinsler at Great Ormond Street Hospital in London. Dr. Price is a regular speaker at national dermatology and pediatric conferences. Her work is published in textbooks and peer-reviewed journals.


Brett Kopelan was initiated into the world of rare diseases ten years ago when his daughter was born with a severe form of Recessive Dystrophic Epidermolysis Bullosa (RDEB). Shortly after her birth, Brett was elected to the board of directors of the Dystrophic Epidermolysis Bullosa Research Association of America (debra of America). Upon returning to New York City after his daughter was the 8th child in the world to undergo a stem cell transplant in hopes of treating her RDEB, he became the Executive Director of debra of America, the only national not-for-profit providing all-inclusive support to the EB community. He currently serves as: President of the board of directors of the Coalition of Skin Diseases; Treasurer on the board of directors of debra international (a consortium of 55 nongovernmental organizations dedicated to curing EB); and, Secretary on a 50-year old financial services firm’s board of directors. Brett also formerly served as both the Chairman of the board of directors, and as the treasurer of the National Organization for Rare Disorders (NORD). Prior to his rare disease work, Brett was an accomplished entrepreneur, starting three different companies raising more than $30 million in venture financing, where he led business development and marketing. He has a graduate degree from Columbia University and an undergraduate degree from New York University.



Amryt Pharma Plc           +353 (1) 518 0200

Joe Wiley, CEO

Rory Nealon, CFO/COO



Consilium Strategic Communications     

+44 (0) 20 3709 5700

Amber Fennell, Matthew Neal, Ashley Tapp



LifeSci Advisors, LLC       +1 (212) 915 2564

Tim McCarthy   


The person making this notification on behalf of Amryt is Rory Nealon, CFO/COO and Company Secretary.


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®/ Lojuxta® (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®) and in the EU (under the trade name Lojuxta®). 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® / Myalepta® (metreleptin) is approved in the US (under the trade name Myalept®) 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®) 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® is a potential treatment for the cutaneous manifestations of Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. FILSUVEZ® has been granted Rare Pediatric Disease Designation and has also received a Fast Track Designation from the FDA. The European and US 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

Forward-Looking Statements

Statements in this announcement with respect to Amryt's business, strategies, timing for completion of and announcing results from the EASE study, the potential impact of closing enrollment in the EASE study, 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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Last Updated: 03-Aug-2020