AMYT.L Regulatory News (AMYT)

AMRYT PHARMA PLC

("Amryt" or the "Company")

EASE CLINICAL STUDY UPDATE

EASE Study Enrollment Closed; Top Line Data Expected in Late Q3/Early Q4 2020

Amryt, a global, commercial-stage biopharmaceutical company dedicated to commercializing and developing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, today provides the following update with regard to its Global Phase 3 clinical study ("EASE") in Epidermolysis Bullosa ("EB").

EASE Trial Update:

In recent weeks, the COVID-19 pandemic has had a material impact on clinical trials globally, including patient recruitment. Given that the EASE study was already close to full enrollment, Amryt has taken advice from an independent expert and concluded that the statistical impact of further patient recruitment would most likely be negligible. Amryt has therefore decided to close the EASE study to further enrollment.

Shortly after the last patient completes the end of the double-blind treatment period (Day 90), the study data will be cleaned and the database locked. Statistical analyses will then be performed and the Company now anticipates top line data read out in late Q3 / early Q4 2020.

Dr Joe Wiley, CEO of Amryt Pharma, commented: "We are pleased to have completed recruitment into our EASE study. Operating clinical trials in rare diseases is always challenging and we are proud to have conducted the largest ever global study in EB. We are fortunate that our patient recruitment was already near conclusion before the onset of COVID-19, and having taken expert advice that the statistical impact of further patient recruitment would most likely be negligible, we have decided to close enrollment at this point.

I would like to take this opportunity to thank all of our patients, their carers and physicians for their participation in and commitment to the EASE trial. We look forward to announcing top line data from this study in the coming months."

About AP101 and EASE

AP101 is a potential treatment for EB, a rare, chronic and distressing genetic skin disorder that causes the skin layers and internal body linings to separate, affecting infants, children and adults, for which there is currently no approved treatment. It is estimated that the incidence of EB is approximately one in 20,000, which implies that there are as many as 30,000 affected individuals in the US and over 500,000 worldwide.

In March 2017 Amryt commenced EASE, a Phase 3 prospective double-blind randomised placebo controlled efficacy and safety study of AP101 in patients with EB. EASE is the largest ever global Phase 3 study conducted in patients with EB, operating across 55 sites in 27 countries globally. In January 2019, Amryt reported the outcome of an unblinded interim efficacy analysis, at which point an Independent Data Monitoring Committee ("IDMC") recommended continuing the trial.

In July 2018, AP101 was granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration ("FDA"). This means that if a New Drug Application ("NDA") for AP101 is approved, the Directors expect Amryt to be eligible to receive a Rare Pediatric Disease Priority Review Voucher ("PRV") that can be used, sold or transferred.

AP101 was granted Fast Track designation by the FDA in September 2019. The Fast Track programme is designed to accelerate the development and review of products such as AP101, which are intended to treat serious diseases and for which there is an unmet medical need. Fast Track designation enables more frequent communication with the FDA. Amryt intends to request and submit a rolling-NDA in the coming months to the FDA and will request a priority review in the US. Amryt also intends to pursue an accelerated assessment in the EU.

AP101 has been granted Orphan Drug status for the treatment of EB in the EU and the US. Should AP101 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.

Amryt's next scheduled market update will be the release of FY 2019 Audited Results and Q1 Trading Update in early May.

Enquiries:

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 or over 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, AP101 (Oleogel-S10), 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. AP101 has been granted FDA Pediatric Rare 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 Directors to be in excess of $1 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.

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 directors' current beliefs and assumptions and are based on information currently available to management.