AMYT.L Regulatory News (AMYT)

5 December 2016

AIM: AMYT

ESM: AYP

Amryt Pharma plc

("Amryt" or the "Company")

Secures Exclusive Licence

For Drug to Treat Rare Disease,

Homozygous Familial Hypercholesterolaemia ("HoFH")

Amryt, a clinical-stage specialty pharmaceutical company focused on best-in-class treatments for rare and orphan diseases, is pleased to announce that it has reached an agreement with Aegerion Pharmaceuticals, Inc. ("Aegerion"), the NASDAQ-listed biopharmaceutical company, for the exclusive rights to sell Aegerion's drug, LOJUXTA (lomitapide), across the European Union ("EU") and other territories (the "Licence Agreement"). The Licence Agreement will add to the Company's growing portfolio of orphan products and is expected to be immediately cash generative for Amryt.

LOJUXTA is used to treat a rare life-threatening disease called Homozygous Familial Hypercholesterolemia ("HoFH") and was approved in the EU in late 2013. It currently generates material sales from the territories to be licensed by Amryt. Aegerion recorded $22.0 million in net product sales of lomitapide capsules in the third quarter of 2016, 15% of which was from prescriptions written outside of the US, including the territories now being licensed to Amryt. Amryt believes that there is significant potential for the drug to become a mainstay treatment for patients with HoFH.

Under the terms of the Licence Agreement, Amryt has the exclusive right to sell LOJUXTA across the European Economic Area ("EEA"), Middle East and North Africa ("MENA"), Turkey and Israel ("Licenced Territories"). In return, Amryt will make royalty payments to Aegerion, paid quarterly, based on a percentage of sales, and once-off commercial milestone payments, subject to achieving certain sales targets. The Licence Agreement has an initial term until 1 January 2024 ("Initial Term"). On expiry of the Initial Term, Amryt may, at its discretion, extend the Licence Agreement for a further five years with the right to extend in further five year periods, subject to certain conditions. Amryt will also take on the ongoing regulatory and post-marketing obligations and commitments in support of LOJUXTA.

Homozygous Familial Hypercholesterolemia is a life threatening disorder that impairs the body's ability to remove LDL cholesterol ("bad" cholesterol) from the blood. This typically results in extremely high blood LDL cholesterol levels leading to aggressive and premature narrowing and blocking of arterial blood vessels manifesting as cardiovascular disease. If left untreated, heart attack or sudden death may occur in childhood or early adulthood.

Current treatment options, including statin drugs, PCSK9 inhibitors and apheresis (a blood filtration technique similar to dialysis), are not adequate to control LDL cholesterol levels in some patients, particularly those with the most severe genetic mutations. HoFH was historically estimated to occur in about one in one million people worldwide although more recent studies suggest it may affect up to 1 in 300,000 people.

Amryt's Chief Medical Officer, Dr. Mark Sumeray, is very familiar with LOJUXTA, having previously led the clinical development and regulatory approval of the drug while Chief Medical Officer at Aegerion. Amryt intends to establish the relatively limited additional commercial, medical and regulatory infrastructure required to support the commercialisation of LOJUXTA across the Licenced Territories. The establishment of this infrastructure will be cash neutral to the Company, supported by sales of LOJUXTA.

Joe Wiley, CEO of Amryt, commented:

"We are delighted to announce this landmark licensing deal. It transforms Amryt into a fully-fledged commercial orphan pharma company and comes only eight months after Amryt's IPO in April.

This agreement with Aegerion Pharmaceuticals gives us an exclusive licence to sell LOJUXTA across the EEA, MENA and other territories for the treatment of a very rare and life-threatening disease - Homozygous Familial Hypercholesterolaemia - or HoFH - where there is significant unmet need.

If left untreated, HoFH may cause heart attacks, strokes or sudden death in childhood or early adulthood. We believe that LOJUXTA, which was approved in the EEA in late 2013, has the potential to become a mainstay treatment in the management of HoFH. Given our in-depth knowledge of the drug we are confident that we can enhance sales of this product going forward.

The Company has made significant strides since flotation. This agreement enables Amryt to generate material cash flows from the commercial sales of a drug which is well known to us. We will also be in a position to leverage the pan-European LOJUXTA commercial, medical and regulatory infrastructure when we launch our lead asset, Episalvan, upon successful completion of the Phase 3 clinical trial in Epidermolysis Bullosa, expected in 2018.

This agreement is tremendously exciting and underlines our clear focus on building our portfolio of medicines to treat rare and orphan diseases, where there is large unmet medical need. We look forward providing a further update in due course."

Mary Szela, CEO of Novelion Therapeutics, Inc, parent company of Aegerion, said:

"We are extremely pleased to enter into this agreement with Amryt. Amryt's unique combination of orphan drug experience, commitment to developing and commercializing transformative rare disease therapies, and management's familiarity with LOJUXTA, give us confidence in their ability to serve current and future adult HoFH patients. We are pleased to have them as a partner."

Enquiries:

About Amryt Pharma plc

www.amrytpharma.com

Amryt Pharma is a specialty pharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare or 'orphan' diseases. The Company is building a diversified portfolio of commercially attractive, best-in-class, proprietary new drugs to help address some of these rare and debilitating illnesses for which there are currently no available treatments.

Amryt's lead product, Episalvan, received marketing approval for the treatment of partial-thickness wounds from the European Commission in January 2016. Amryt intends to develop Episalvan as a new treatment for Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin disorder affecting young children for which there is currently no treatment. Amryt is currently planning a Phase 3 study of Episalvan in EB, which has been granted US and EU orphan drug designation. The market opportunity for EB is estimated to be circa US$1.5 billion.

Amryt's earlier stage product AP102 is focused on developing novel, next generation somatostatin analogue ("SSA") peptide medicines for patients with rare neuroendocrine diseases, where there is a high unmet medical need, including acromegaly and Cushing's disease. AP102 was recently granted orphan designation in the US in acromegaly by the FDA.

The Company was admitted to trading on AIM and Dublin's ESM in April 2016 following the reverse takeover of Fastnet Equity PLC.

About Aegerion Pharmaceuticals, Inc. 

www.aegerion.com

Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for patients with debilitating rare diseases.