AMYT.L Regulatory News (AMYT)

AMRYT PHARMA PLC

("Amryt" or the "Company")

AMRYT SUPPORTS GLOBAL FH AWARENESS DAY - SEPTEMBER 24, 2020

DUBLIN, Ireland, and Boston MA, September 23 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, announces its support today for Global FH Awareness Day 2020 which will take place on Thursday, September 24, 2020.

Dr Joe Wiley, CEO of Amryt Pharma, commented: "All of the Amryt team are pleased to support the great work of the FH Foundation in the US and FH Europe as they seek to raise awareness of FH and HoFH amongst patients, clinicians and the wider community. For our part, we are collaborating with all stakeholders to continue to deliver therapies to patients in desperate need".

Magdalena Daccord, CEO of FH Europe, commented: "FH Awareness Day in year 2020 is not just another awareness campaign aimed at making as much noise as possible. It is an absolute opportunity and a necessity to make common Familial Hypercholesterolemia (FH) and its severe and rare form, Homozygous FH, a public health priority. The Global Call to Action on FH equips us with 9 actionable recommendations to make it happen. The COVID pandemic, on the other hand, highlights the urgency and the gravity of the inherited high cholesterol directly impacting over 30 million people worldwide...starting from birth. #FHaware2020 is the time to come together and make seriously good things happen!".

About FH and HoFH

Familial Hypercholesterolaemia ("FH") is an inherited genetic condition that leads to levels of cholesterol that are much higher than that of the general population.[i] FH is the most common genetic condition leading to early heart disease.[ii] Millions of people around the world do not know that they and their families are at severe risk for early heart disease, heart attacks, and even death. Nine out of ten people born today with familial hypercholesterolemia are undiagnosed. Yet, with early diagnosis and treatment, individuals diagnosed with FH can reduce their risk for heart disease by 80%.[iii] 

Homozygous Familial Hypercholesterolaemia ("HoFH") is a rare and severe form of FH, causing excessive levels of low-density lipoprotein cholesterol ("LDL-C") or 'bad cholesterol' to accumulate in the body from conception. People with HoFH are at a continued risk of progressive atherosclerotic disease and life-threatening cardiac events, for example, heart attack, stroke, major cardiac surgery and premature cardiac death. Untreated, most patients with markedly elevated LDL-C levels develop overt cardiovascular disease before the age of 20 years, and generally do not survive past 30 years.[iv] 

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 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® in EB. The primary endpoint of the trial was met (p-value = 0.013). FILSUVEZ® 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 preclinical gene-therapy platform technology, AP103, which offers a potential treatment for patients with 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.

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.